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STANDARD THERAPIES Allogeneic Autologous

BLOOD CANCERSLEUKAEMIAAcute Biphenotypic Leukaemia xAcute Lymphoblastic Leukaemia xAcute Myelogenous Leukaemia xAcute Undifferentiated Leukaemia xChronic Myelogenous Leukaemia xChronic Lymphocytic Leukaemia xJuvenile Myelomonocytic Leukaemia xJuvenile Chronic Myelogenous

Leukaemia x

MYELOPROLIFERATIVE NEOPLASMSAcute Myelofi brosis xAgnogenic Myeloid Metaplasia xEssential Thrombocythemia xPolycythemia Vera x

MYELODYSPLASTIC SYNDROMESRefractory Anaemia xRefractory Anaemia with

Excess Blasts xRefractory Anaemia with Excess

Blasts in Transformation xRefractory Anaemia with

Ringed Sideroblasts (Sideroblastic Anaemia) x

Chronic Myelomonocytic Leukaemia x

OTHER BLOOD CANCERSMultiple Myeloma x xPlasma Cell Leukaemia x xWaldenstrom’s Macroglobulinemia x xSOLID TUMORSHodgkin Lymphoma x xNeuroblastoma xNon Hodgkin Lymphoma

(Burkitt’s Lymphoma) xRetinoblastoma xMedulloblastoma x


NON MALIGNANT BLOOD DISORDERSANAEMIAS (DEFICIENCIES OR MALFORMATIONS OF RED CELLS)Aplastic Anaemia xCongenital Dyserythropoietic Anaemia xFanconi’s Anaemia xParoxysmal Nocturnal Hemoglobinuria x

HEREDITARY BONE MARROW FAILURE SYNDROMESDiamond Blackfan Syndrome xDyskeratosis Congenita xPearson’s Syndrome xShwachman Diamond Syndrome x

INHERITED RED CELL ABNORMALITIESPure Red Cell Aplasia xSickle Cell Anaemia xBeta Thalassemia Major / Cooley’s Anaemia x

INHERITED PLATELET ABNORMALITIESCongenital Amegakaryocytosis Thrombocytopenia xGlanzmann’s Thrombasthenia xIMMUNE DISORDERSSEVERE COMBINED IMMUNE DEFICIENCY (SCID)Bare Lymphocyte Syndrome xOmenn Syndrome xReticular Dysgenesis xNeutrophil Actin Defi ciency xSCID with Adenosine Deaminase

Defi ciency (ADA SCID) xSCID which is X linked xSCID with absence of T & B Cells xSCID with absence of T Cells,

Normal B Cells x

Diseases Treated with Stem Cells and its Potential ApplicationsDiseases Treated with Stem CellsThe following is a list of some of the diseases that have been treated with Haematopoietic Stem Cell.

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NEUTROPENIASKostmann Syndrome

(Infantile Genetic Agranulocytosis) xMyelokathexis x

PHAGOCYTE DISORDERSChediak Higashi Syndrome xChronic Granulomatous Disease x

INHERITED DISORDERS OF THE IMMUNE SYSTEM & OTHER ORGANSCartilage Hair Hypoplasia xErythropoietic Porphyria xSystemic Mastocytosis x

OTHER INHERITED IMMUNE SYSTEM DISORDERSCommon Variable Immunodefi ciency xDiGeorge Syndrome xHemophagocytic Lymphohistiocytosis xIKK Gamma Defi ciency

(NEMO Defi ciency) xIPEX Syndrome xLeukocyte Adhesion Defi ciency xWiskott Aldrich Syndrome xLymphoproliferative Disorders xX linked Lymphoproliferative Disease

(Duncan’s Syndrome) xAtaxia-Telangiectasia xMETABOLIC DISORDERSLEUKODYSTROPHY DISORDERSAdrenoleukodystrophy xKrabbe Disease

(Globoid Cell Leukodystrophy) xMetachromatic leukodystrophy xPelizaeus-Merzbacher Disease x


LYSOSOMAL STORAGE DISEASESNiemann Pick Disease xSandhoff Disease xWolman Disease x

MUCOPOLYSACCHARIDOSIS (MPS) STORAGE DISEASESHunter Syndrome xHurler Syndrome xMaroteaux Lamy Syndrome xMucolipidosis II (I-cell Disease) xMorquio Syndrome xSanfi lippo Syndrome xScheie Syndrome xSly Syndrome (beta glucuronidase

defi ciency) x

OTHER METABOLIC DISORDERSLesch–Nyhan Syndrome xOsteopetrosis xHermansky-Pudlak Syndrome x

Storing cord blood does not guarantee that the cells will provide a cure or be applicable in every situation. The use of stem cells will ultimately be determined by the treating physician.

References:· Diseases treated page. Parent’s Guide to Cord Blood Foundation website. https://parentsguidecordblood.org/en/diseases. Accessed October 25, 2018.

· Bizzetto R, Bonfi m C, Rocha V, et al. Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia. Haematologica. 2011; 96(1):134-141.

· Picard C, J-L Casanova, Puel A, Infectious Diseases in Patients with IRAK-4, MyD88, NEMO, or IκBα Defi ciency. Clin Microbiol Rev. 2011; 24(3):490-497.

· Rao A, Kamani N, Filipovich A, et al. Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Blood. 2007; 109(1):383-385.


CLINICAL TRIALS Allogeneic AutologousAcute Myocardial Infarction(Heart Attack)

x

Acute Respiratory Distress Syndrome (ARDS)

x

Aging Frailty xAlopecia Areata xAlpha-Mannosidosis xAlzheimer’s Disease xAmyotrophic Lateral Sclerosis xAnkylosing Spondylitis xAutism x xBronchoPulmonary Dysplasia (BPD) (lung disorder due to premature birth)

x

Cardiomyopathy xCartilage Injury xCerebral Palsy x xCleft Palate Repair xCritical Limb Ischemia xCrohn’s Disease xDiabetes, Type 1 x xDiabetes, Type 2 x xDiabetic Foot Ulcer x Diabetic Peripheral Neuropathy xDuchenne Muscular Dystrophy xEczema (Atopic Dermatitis) xEncephalopathy xEpidermolysis Bullosa xErectile Dysfunction xEwing Sarcoma x xEye Diseases xFistula xGaucher’s Disease xGlobal Developmental Delay x xGraft-versus-Host Disease (GvHD) xHearing Loss (acquired sensorineural)

x

Clinical TrialsWith advancements in stem cell* research, the potential use of stem cells continues to grow. Clinical trials are one of the key driving forces behind medical breakthroughs, and they represent hope for families with conditions that currently have no known cure.

Depending on the disease, stem cell* treatments that are currently undergoing clinical trials may slow the progression of the disease or unlock new medical options that are currently unavailable.

There are also diseases where stem cell* treatments may help effect a cure, but further research is needed to determine the best candidate patients for stem cell therapy, optimum stem cell dosage and method of cell delivery, etc.

Patients with diffi cult to treat or currently “incurable” diseases, such as AIDS and certain types of cancer, may want to pursue participation in clinical research trials if standard therapies are not effective.

Below are some of the diseases that are currently undergoing clinical trials. For the full list, please visit ClinicalTrials.gov.

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*Stem cells mentioned here comprises of other cell lines such as Mesenchymal stem cells. The clinical trials listed above may be using other lines of stem cells, and not only haematopoietic stem cells.References:• Therapies in Clinical Trials Page. Parent’s Guide to Cord Blood Foundation website. https://parentsguidecordblood.org/en/diseases#trial. Accessed October 25, 2018.• Clinical trial registry page. ClinicalTrials.gov website. http://clinicaltrials.gov. Accessed October 25, 2018.

CLINICAL TRIALS Allogeneic AutologousHeart Failure xHereditary Ataxia xHIV xHuntington’s Disease xHypoplastic Left Heart Syndrome xIntraventricular Hemorrhage x xIschemic Heart Disease x xLiver Cirrhosis xLiver Failure xLupus xLysosomal Storage Diseases xMetabolic Syndrome xMultiple Sclerosis xNon-Union Fractures xOsteoarthritis xOsteochondral Lesion xOvarian Cancer x xParkinson’s Disease xPeripheral Arterial Disease (PAD) xPeyronie’s Disease xPremature Ovarian Failure xPsoriasis xRheumatoid Arthritis xSevere Combined Immunodefi ciency xSpinal Cord Injury xSpinal Fusion Surgery xSpinal Muscular Atrophy xStroke x xSurgery for Congenital Heart Defects xSystemic Sclerosis xTay-Sachs Disease xTesticular Tumour xTraumatic Brain Injury x xUlcerative Colitis xUterine Scars xWounds x


Media : The Straits Times

Date : 31 May 2018

Associate Professor William Hwang with Ms Judith Chew, the fi rst patient in Asia to undergo a stem cell transplant using the Nicord cord blood expansion technique.

ST PHOTO: DESMOND WEE

New cord blood transplant treatment holds promise for blood cancer patients

A new treatment technique involving cord blood transplants could offer patients with diseases such as blood cancers a lower risk of infection and higher likelihood of surviving the transplant, if the current trial proves successful.

The technique involves culturing and growing cells from a single unit of cord blood in the laboratory over three weeks to produce more stem cells.

More than one-third of patients worldwide die from cord blood transplants that contain a low number of stem cells.

The new technique, called the Nicord cord blood expansion technique, has shown promise with a mortality rate of less than 10 per cent, said Associate Professor William Hwang, a senior consultant at the Singapore General Hospital’s (SGH) Department of Haematology.

For conventional cord blood transplants which contain a high number of stem cells, the current mortality rate is also less than 10 per cent.

Prof Hwang said long-term survival using the new technique has not been defi nitively proven.

The trial is being conducted by Gamida Cell, a biotechnology company which develops cellular and immune therapeutics, and Duke University Medical Centre in the United States.

Besides increasing the number of cells, the second phase of the clinical trial showed a reduction in the average number of total infections during the fi rst 100 days after transplant.

It was 3.7 per patient for the new technique compared to 4.9 per patient for the standard cord blood transplant group.

In addition, during the fi rst 100 days after transplant, the new technique reduced the average number of hospitalisation days to 30 from 50.


These fi gures are based on 36 patients from Singapore, Europe and the US who took part in the trial’s second phase from 2013 to 2017.

The third phase started in November 2016 and is expected to be completed in December 2019, with 120 patients being recruited globally.

Prof Hwang said a patient’s suitability to receive the new treatment depends on his ability to wait for the cells to be grown.

“If the patient has a disease that cannot wait, for example, a previous transplant failed and the patient has a low blood count for a long time, I might say, just use the (conventional) cord blood transplant,” he added.

But for diseases such as acute leukaemia where the disease is in remission and can be maintained in that state, then the new treatment technique is recommended.

Other alternatives like donated bone marrow and peripheral blood are currently the preferred treatment for patients with blood diseases and bone marrow failure because they have a faster recovery period of around 14 and 17 days respectively.

In comparison, patients need around 25 days to recover from conventional cord blood transplants.

But using donated bone marrow and peripheral blood require exact matches, which are hard to fi nd.

More than 80 stem cell transplants are performed at SGH every year on average, out of which four are cord blood transplants.

Ms Judith Chew, 53, is the fi rst patient in Asia to receive a cord blood transplant using the new technique.

The business consultant suffers from acute myeloid leukaemia, a cancer of the blood and bone marrow, and was diagnosed in October 2016. She had to undergo chemotherapy to lower her white blood cell count before the operation in May last year.

“The entire process was really very manageable for me, and I’m most happy with the recovery process,” said Ms Chew, who only needed a month’s stay at SGH.

Correction note: The article was edited for clarity.


The team, comprising (from left) Dr Sudipto Bari, Associate Professor William Hwang, Professor Christina Chai, Dr Zhong Qixing and Associate Professor Gigi Chiu. ST PHOTO: JONATHAN CHOO

Singapore researchers fi nd new breakthrough in search for cure for blood cancers

The search for a cure for blood cancers has received a boost with new research by Singapore scientists shedding light on how stem cells can be more effectively used to treat diseases such as leukaemia.

The fi ve researchers are from the National Cancer Centre Singapore, the National University of Singapore (NUS), Duke-NUS Medical School, and the Singapore General Hospital (SGH).

At a media briefi ng on Monday afternoon (May 21), the team said it discovered a laboratory-synthesised chemical substance that can be used to increase the number of stem cells harvested from umbilical cords.

It said this will help overcome a current challenge of cell levels being too low to help patients recover quickly.

This need for a quick recovery is to minimise the risk of

bacterial, fungi or viral infections, said Associate Professor William Hwang, medical director of the National Cancer Centre Singapore, one of the researchers involved.

Associated health care costs can also be reduced when a patient’s recovery time is shortened, he added.

More than 200 patients in Singapore undergo such stem cell treatments every year for diseases such as blood cancers.

DEMYSTIFYING STEM CELLSIn general, stem cells are “elastic” cells. They are capable of regenerating and differentiating into various cell types in a person’s body. For example, stem cells can be induced to become blood, bone and tendons, among other things.

This potential of stem cells, which can last the lifetime of a patient, to form new cells that can replace degenerated


Date : 21 May 2018


ones has made stem cell therapy the subject of intense medical research.

But there are many different types of stem cells in the body, some more “elastic” than others. When stem cells differentiate, they become progenitor cells, which are more specialised and have a shorter lifespan.

Such intermediary cells include those found in the bone marrow or peripheral blood. They are considered haematopoietic (blood-forming) progenitor cells.

These haematopoietic progenitor cells are slightly more specifi c, in that they regenerate to form cells that constitute blood - red blood cells, platelets, and cells of the immune system. They also last for a shorter period of time.

When it comes to treating blood cancers, stem cells and blood-forming progenitor cells are both important.

HOW THE THERAPY WORKSTo help restore healthy bone marrow in patients with leukaemia, for example, stem cells and blood-forming progenitor cells are injected into the patient.

After entering the bloodstream, the stem cells travel to the bone marrow and start to make new blood cells. This helps stimulate new bone marrow growth and restores the immune system.

These cells are usually harvested from either the umbilical cord, peripheral blood (bloodstream) or bone marrow.

But there are challenges. Because harvesting bone marrow stem cells involves an invasive procedure, there are few donors. As for peripheral blood, the cells collected usually have more immune cells. These cells could in turn attack the patient and cause side effects.

Umbilical cord, however, is promising as it contains the highest number of undifferentiated stem cells. But their numbers are usually not high enough for rapid recovery in adult patients.

This results in the longest recovery time of more than 25 days in a patient who has undergone umbilical cord blood transplant, compared to the 14 days of recovery time from a peripheral blood transplant, and an 18-day recovery period from bone marrow transplant.

THE DISCOVERYThe researchers found a way to increase the numbers of such blood-forming stem cells from umbilical cord blood using a laboratory-synthesised substance called C7, synthesised in the NUS Department of Pharmacy.

They found that this substance is able to expand the number of blood-forming stem cells in the umbilical cord through cell culture.

Said Prof Hwang, who is also a senior consultant with the department of haematology at SGH: “There are patients who are unable to fi nd a fully matched bone marrow or peripheral blood stem cells. For these patients, umbilical cord blood is the only source of grafts.”

But under present conditions, low cell numbers from treatments using umbilical cord blood result in long recovery periods.

“Expanded umbilical cord blood (using C7) would be a life-saving option,” he said.

The researchers said trials involving mice were encouraging, as it showed that the stem cells increased in numbers over time, and did not die.

Clinical trials are expected to start in mid-2019. With support from industrial partners, Prof Hwang said he is optimistic that stem cell treatment could be available to patients within the next fi ve years.


Media : 优1周 – UWeekly

Date : 16 July 2016

Interviewee: Mr Tan (36-year old, IT Professional, father of 6-year-old son, Tan Ee Han)

My child was born with cerebral palsy

Six years ago, when Ee Han was 3 months old, we realised that his neck was too soft and couldn’t hold his own head. We quickly took him to KKH for a check-up. After further checks at the hospital, doctors concluded that Ee Han had growth development delay and quadriplegic cerebral palsy. The ENT doctor also pointed out that he had severe hearing loss in both ears. Upon hearing these, we were devastated. It was as though our world had come to an end.

The doctor told us that Ee Han would not be able to do normal, everyday things like an ordinary person. For example, to carry out simple tasks such as using a straw to drink or clap his hands, Ee Han would have to undergo many expensive therapy sessions.

INFUSION OF CORD BLOOD STEM CELLSAfter several years, while at an outdoor course organised by the “Early Intervention Programme for Infants and Children”, I learnt that a child with cerebral palsy had experienced improvement in his condition. The child’s mother revealed her son’s condition had improved after receiving a cord blood stem cell infusion.

I remembered that we made the decision to collect and store Ee Han’s cord blood with Cordlife Singapore at birth. I wasn’t aware that cord blood could help children with cerebral palsy to improve their cognitive and motor skills.

After medical evaluation and approval from the Ministry of Health, the cord blood transplant using Ee Han’s own cord blood was carried out by Dr Keith Goh in November 2014 and September 2015. Following the transplant, Ee Han did not experience any side effects or allergies. Since then, his movement, interaction skills and temperament has improved. The most unforgettable moment after the infusion was when Ee Han “fought” with his niece, as it was his fi rst time interacting with another child.

Ee Han still needs to undergo a variety of therapies such as speech, language, hearing and feeding therapy. We hope that these treatments will help to further improve his condition.


Media : 优1周 – UWeekly

Date : 16 July 2016

ASK THE DOCTOR – Dr Keith Goh

Q: IS CORD BLOOD TRANSPLANT BENEFICIAL FOR CEREBRAL PALSY PATIENTS? WHAT ARE THE RISKS INVOLVED?A: Umbilical cord blood contains stem cells that can aid in the recovery of neurological conditions. As the cord blood comes from the patients themselves (autologous), there is very low risk of rejection or tissue mismatch. Over the past decade, research has shown that these stem cells are safe and do not pose any medical issues.

Q: WHAT IS THE PROCESS OF THE TRANSPLANT?A: The process is similar to a blood transfusion. Cord blood is intravenously infused into the patient. The whole process takes about 30-45 minutes. The patient will then be put under observation for four to fi ve hours. If everything is fi ne, they can be discharged.

Q: WHAT CONDITIONS CAN CORD BLOOD TREAT?A: There are over 70 treatable diseases using cord blood stem cells and some common conditions include leukaemia and lymphoma, infected bone marrows, certain genetic diseases and metabolic disorders. Some conditions such as juvenile diabetes, spinal injury and autism, are still undergoing clinical trial.

Q: FROM YOUR OBSERVATION, HOW IS EE HAN’S PROGRESS LIKE, AFTER UNDERGOING TWO CORD BLOOD INFUSIONS? A: I have noticed that there is an improvement in his motor skills; he has more strength now and has better control of his limbs. He is also able to take a few assisted steps and is now more responsive with better cognitive ability.


Media : Today

Date : 22 June 2016

Could umbilical cord blood provide hope for children with autism?By Eveline Gan

A cord-blood bank in Singapore, where parents can store their newborns’ cord blood for private use to help cell regeneration in a range of potential diseases and disorders down the track.

TODAY FILEPHOTO

A new treatment using cord blood stem cells to treat autism could offer hope to families of children living with the neuro-developmental disorder, which currently has no known cure.

Run by the Sutter Institute of Medical Research in the United States, the US Food and Drug Administration-approved clinical trial explored the use of stem cells from umbilical cord blood to improve language and behaviour in children with autism.

Affecting an estimated 1 per cent of the population in Singapore, autism is characterised by diffi culties in communicating, using language effectively and forming relationships with other people.

The fi rst-of-its-kind, placebo-controlled study included 30 children with autism, ranging in age from two to seven, who received an infusion of their cord blood stored at birth. Afterwards, the participants were regularly tested for a year using a variety of psychological and cognitive assessment tools.

In town last month for the International Society for Cellular Therapy 2016 annual meeting, the trial’s principal investigator, Dr Michael Chez, told TODAY that 60 per cent of parents of children who participated in the study said they saw moderate to signifi cant improvements, especially in language and social skills.


Could umbilical cord blood provide hope for children with autism? (cont’d)

Previous studies suggest that some children with autism have dysfunctional immune systems that may be damaging or delaying the development of the nervous system, according to Dr Chez, director of Paediatric Neurology at the Sutter Institute.

“The results of this study indicate that cord blood stem cells may offer ways to modulate or repair the immune systems of these patients with autism, and in doing so, improve language and some behaviour in some children. More work is needed to prove this but for a small placebo-controlled pilot study, this is a very good outcome,” he said.

The researchers are looking to defi ne groups of children who responded better to the procedure. Dr Chez also believes that any positive effects seen after the treatment would probably not wear off over time.

“When children with autism gain a new skill, it’s like making a new connection (in the brain). The brain shouldn’t deteriorate unless it is a degenerative disease,” he said.

Dr Cherie Daly, medical affairs consultant for Cordlife, one of the three private cord blood banks in Singapore, said there are plans to bring the autism-stem cell clinical trial to Singapore. Dr Chez said the parties are “still in the process of getting the necessary approvals from (the Health Sciences Authority) to start the trial in Singapore”.

Dr Li Ming Ming, laboratory director of Cordlife, added: “If we are able to bring this therapy into Singapore, it will defi nitely be benefi cial for patients and their families.”

But other experts said there is still insuffi cient evidence to support autism-stem cell therapy.

Autism is a complex neurological disorder whose exact cause is currently not known, said Dr Serena Tung, consultant at National University Hospital’s Child Development Unit. Research shows it has a genetic basis, and is also associated with some medical conditions such as Fragile X syndrome, an inherited condition that causes a range of developmental problems.

“At present, the main form of evidence-based intervention for autism spectrum disorder is early interventional therapy. To date, there is insuffi cient clinical evidence to support the use of stem cell therapy in this group of individuals,” said Dr Tung.

Dr William Hwang, medical director of the public Singapore Cord Blood Bank (SCBB), added that it is too early to comment on whether the use of patients’ own cord blood can successfully treat autism as the trials are still ongoing. “Further trials are also needed to see if bone marrow and other cell sources, as well as unrelated donor cord blood, could be just as useful in treating autism,” he said.

CORD BLOOD’S POTENTIAL

Autism is not the fi rst neurological condition that scientists have attempted to treat using stem cells from umbilical cord blood, which have the potential to replicate other blood cells including platelets, red and white blood cells. In Singapore, 10 children with cerebral palsy have been treated with infusions using their own cord blood, according to consultant neurosurgeon Keith Goh of International Neuro Associates.

Ongoing clinical trials overseas for other neurological disorders include Alzheimer’s disease, Huntington’s disease, amyotrophic lateral sclerosis and Parkinson’s disease, according to Dr Li of Cordlife.

Stem cells from cord blood have been used to treat other diseases, including certain cancers, blood diseases and metabolic disorders. Studies have shown stem cell therapy holds great promise of making the untreatable diseases treatable, said Dr Li.

Umbilical cord blood is routinely discarded as part of medical waste. But in the past fi ve years, about 20 per cent of parents in Singapore have banked their newborns’ cord blood for private use, according to Dr Daly. The proportion is among the highest worldwide.


In general, private banks here charge more than about S$200 a year to store the cord blood for about 21 years, excluding an enrolment fee. For some parents, the cost is a small price to pay as privately-banked cord blood can be tapped for individual or family use in the future.

Having access to it may provide a child or his/her family an additional treatment option when needed, said Dr Teo Cheng Peng from Parkway Cancer Centre at Gleneagles Hospital and medical director for StemCord, a private cord blood bank.

But SCBB’s Dr Hwang said research has shown that the chance of a donor — without known medical conditions — ever benefi ting from his or her own cord blood is “an extremely low probability”. “It is estimated that the chance

of such use is somewhere between one in 2,000 and one in 200,000. In most instances, using a patient’s own blood stem cells for treatment may not be the best choice.”

In line with recommendations from major international medical societies including the American Medical Association, American Academy of Pediatrics and World Marrow Donor Association, the SCBB encourages mothers to donate their cord blood to public banks as it increases the chances of fi nding a suitable match for patients who are in need of life-saving transplants.

As of May 31, the SCBB has facilitated 191 cord-blood transplants in Singapore and around the world.

Cord-Blood storage popular in Singapore

Umbilical cord blood is routinely discarded as part of medical waste. But in the past fi ve years, about 20 per cent of parents in Singapore have banked their newborns’ cord blood for private use. The proportion is among the highest worldwide.



Date : 20 June 2016

Birthing new skin with umbilical cord stem cells

Dr Phan holding his proprietary mixture of proteins and sugars that keeps umbilical cord tissue alive. He discovered the use of stem cells in umbilical cord lining in making cells for the skin, cornea, bone and other body parts.

ST PHOTO: LAU FOOK KONG

By Jalelah Abu Baker

DOCTOR’S FASCINATION WITH WOUND HEALING TURNS INTO A $700M BIOTECH COMPANY

Wounds may cause some to look away, but stem cell researcher Phan Toan Thang sees “magic” in them.

“I like wounds. I see the wound healing. It’s something amazing,” he said. The chief scientist’s passion for healing wounds led him to discover a way to create skin cells quickly and in large quantities by using the lining from umbilical cords.

Rather than using the limited available amount of a patient’s skin to help heal a burn, the cells from the umbilical cord lining can provide an unlimited reserve of unprogrammed cells to create new skin and bone, and even other body parts, such as the cornea in the eye.

Such methods could potentially heal wounds from burns or diseases such as diabetes.

Cellresearch Corp, a biotech company he founded with group chief executive Gavin Tan and fellow plastic surgeon and skin researcher Ivor Lim in 2002, now owns 39 patents worldwide. They include patents for extracting stem cells from umbilical cord lining, banking and cultivating them, and for therapeutic applications. The company is now worth $700 million.

Work is under way for the technology to be approved by the globally recognised the United States Federal Drug Administration (FDA), he told The Straits Times at his workplace, a 3,000 sq ft lab in Ayer Rajah Crescent. The cells are being manufactured in Denver and the first clinical trial on patients is expected to start early next year.

“Non-healing wounds are a big medical burden everywhere. They are associated with diabetes, stroke,heart problems and ageing,” he said, explaining the importance of the technology, which can make it affordable for patients to get skin grafts.


QUIET DETERMINATION

Back then, I lived in the lab. I turned off my phone and my wife used to go crazy.

Back then, no one cared about skin. They were focusing on cancer and heart problems. We were just quietly doing it.

STEM CELL RESEARCHER, PHAN TOAN THANG

They were focusing on cancer and heart problems. We were just quietly doing it.They were focusing on cancer and heart problems. We were just quietly doing it.

STEM CELL RESEARCHER, PHAN TOAN THANG

Back then, I lived in the lab. I turned off my phone and my wife used to go crazy.

Dr Por Yong Chen, vice-chairman of the board of the Chapter of Plastic, Reconstructive & Aesthetic Surgeons, said FDA approval will put Singapore on the map.

“Wound healing is a big market, especially because of diabetics,” he said. “This is a signifi cant breakthrough.I think a lot of people are going to try to do the same thing.”

Dr Phan has come a long way since he started his career here as a scientist and researcher in the burns unit in Singapore General Hospital.

He came here from Vietnam in 1997 and is now a Singaporean.

In 2002, the same year Cellresearch Corp was founded, Dr Phan went to the Institute for Stem Cell Biology and Regenerative Medicine, at Stanford University in the US, which he was collaborating with.

In the two years there, he discovered that researchers were trying to separate stem cells from the placenta for liver repair.

When he returned to Singapore in 2004, he decided to experiment with the placenta and umbilical cord. At the time, stem cells found in umbilical cord blood were already being used to help cure patients of diseases such as leukaemia, and other blood cancers and disorders. He has CEO Mr Tan to thank for getting the ball rolling.

It was Mr Tan who gave him two bottles to experiment with, one holding a placenta and one with an umbilical cord, after his wife gave birth. He tried the placenta fi rst, and realised quickly that that the blood it came with made it too contaminated to work with. He took a break in his lab, and had a “eureka” moment, which eventually changed his life.

He said: “I turned my eye to the next bottle, which had the umbilical cord fl oating in there. It was very clean and there was no blood contamination - white in colour, fl oating beautifully in the bottle with the preservative medium.”

He failed several times to perfect the medium, a proprietary combination of sugars and proteins that keep the tissue in the umbilical cord alive, and decontaminating it at the right level.

“Back then, I lived in the lab. I turned off my phone and my wife used to go crazy,” the father of three laughed. He would even rush to the lab if he had a sudden idea on how to make it work.

“Back then, no one cared about skin. They were focusing on cancer and heart problems. We were just quietly doing it,” he said.

HARD WORK

There were many challenges in moving forward to perfect a product, and to make it inexpensive enough for everyone to use. It’s not easy. But luckily, we managed to do it.

DR PHAN


Dr Phan started his career as a plastic surgeon dealing with wounds in an army hospital in Vietnam in 1991 after graduating with a degree in medicine from the Military Medical University in Hanoi.

“I had no time, no money to fool around. So I just focused on studying. Worked very hard, learnt Russian, learnt English,” he added.

In 1995, he went on a fellowship to Oxford University in Britain after a dermatology professor from there who was visiting the army hospital offered him the chance. It was a move that eventually led him to his life in Singapore.

While at Oxford, he met a Singaporean who put him in touch with the head of plastic surgery at the burns unit at Singapore General Hospital (SGH) then, Professor Lee Seng Teik. Prof Lee offered Dr Phan a job on his team in 1997 and he was tasked with healing wounds and creating skin cells.

“We had to take the skin cells from the patient’s unburnt area and expand them in the lab, and put it back on them,” he said.

Sometimes the cell did not do well and needed time to expand.

“It wasn’t scalable. The whole process was very expensive. We only could do it for some patients - it was very diffi cult to sustain,” he said.

About a year later, he met co-founder Dr Lim, who joined him as a fellow registrar. Together, they focused on understanding scar formation.

In 2001 and 2002, Dr Phan won awards for identifying a function in surface skin that causes scarring.

At the time, research was being done using stem cells from embryos, which raised many red fl ags. By focusing on the umbilical cord, Dr Phan sidestepped the touchy topic.

Dr Phan, who is also an associate professor at the department of surgery at the National University of Singapore’s Yong Loo Lin School of Medicine, achieved his goal using umbilical cord - something considered medical waste. But it can provide a whopping six billion stem cells to expand into skin, bone, the cornea and other parts.

Despite his achievements and a company worth $700 million, the devout Catholic is humble and puts some aspects of his discovery down to chance and luck.

He said: “There were many challenges in moving forward to perfect a product, and to make it inexpensive enough for everyone to use. It’s not easy. But luckily, we managed to do it.”

Birthing new skin with umbilical cord stem cells (cont’d)



Date : 17 June 2016

Boy cured of rare illness after two stem cell graftsDoctors in Singapore have cured a baby of Omenn syndrome, the fi rst known case of the extremely rare disease here. Two-year-old Donovan Tay is now thriving, after a rocky start in life. The Straits Times talks to the people who made this possible.

Two-year-old Donovan developed weepy, fl aky skin when he was two months old. His skin became so infl amed that it turned black. After numerous tests, doctors discovered he had Omenn syndrome, a very rare disease and a severe form of immunodefi ciency. After two stem cell transplants, he is now on the road to recovery. With him in the picture are his parents, Mr Tay (left) and Ms Lee, and Dr Soh, who diagnosed his condition.

PHOTO: MATTHIAS HO FOR THE STRAITS TIMES

By Chang Ai-Lien

It took nine months before Ms Jace Lee saw her baby smile.

What had been an uneventful pregnancy turned into a nightmare for new parents Ms Lee and her husband, Mr Keith Tay, when Donovan was just two months old.

His nappy rash spread across his entire body, leaving him red, fl aky and very itchy. “Every inch of his body was infl amed and we became really worried when his ears and neck got sticky and smelly,” said Ms Lee, 34, a housewife.

Doctors initially thought he had developed an infection due to severe eczema and allergies, and started him on an intensive skin care regime. This included moisturising his body every two hours, and bathing him in a chlorine bath to help ease the symptoms.

“He was scratching non-stop by rubbing his head and body against the mattress,” said Ms Lee. “We had to put his hands in silk mittens and put gauze all over him or his skin would become really weepy and bleed from the scratching.”

Even a simple diaper change took 45 minutes because it involved changing all his dressings.

“He didn’t have time to play and his hands were bound all the time,” she added.

Then Donovan started vomiting, and did so for a month despite medication and constant care. But he stopped scratching, and his parents thought he was fi nally on the mend.


A visit to the doctor soon set them straight. Donovan had developed a dangerously high level of sodium in the blood, a consequence of proteins not playing their part in stopping water from leaking through the skin.

After several weeks at the National University Hospital Paediatric intensive care unit, doctors managed to bring his sodium levels down, but he remained small and very thin.

It was during his hospital stint that he came under the care of Dr Soh Jian Yi, a consultant at the NUH Division of Paediatric Allergy, Immunology and Rheumatology, and a key member of the medical army who would eventually save his life.

Faced with his small patient’s unexplained fever, unusually severe eczema and surprising abnormal blood test results, Dr Soh turned medical detective to try to diagnose Donovan’s mysterious illness.

His fi rst clue: The baby had no detectable antibodies in his blood - the body’s weapon against bacteria and viruses. Even a cold could have killed him.

“Every disease has its own time line,” explained Dr Soh. “There tends to be a very specifi c sequence in which the features of each disease occur.”

Dr Soh recalled his readings some years earlier on rare diseases, one of which resembled Donovan’s situation. He pored over the medical literature and consulted local and international experts. The consensus: It all pointed to Omenn syndrome, a condition so rare that it has never before been reported in Singapore.

It is caused by mutations in genes that are critical to the immune system, which protects the body from infections. If not treated, Omenn syndrome is fatal.

Said Dr Lee Bee Wah, a visiting consultant at the NUH Department of Paediatrics: “To our knowledge, he is the fi rst case that has been diagnosed in Singapore.”

Added Dr Soh: “It’s very, very rare; it would be easier to strike the lottery. But looking at all of the clues in Donovan, nothing else fi tted the entire picture.”

The only treatment was a transplant of blood-forming or cord blood stem cells.

Noted Dr Tan Poh Lin, a senior consultant with the hospital’s Division of Paediatric Haematology and Oncology: “Without a transplant, it would have just been supportive care till death, which would be any time, such as when the most severe infection struck.”

But Donovan was in such bad shape that he could not have survived the procedure then.

Said Dr Soh: “It was diffi cult but we didn’t have a choice.”

There were more hurdles to come. On New Year’s Eve in 2014, Donovan collapsed, his heart stopped and his organs began to shut down. Doctors revived him 18 minutes later. His parents kept vigil at his bedside.

“We kept on getting ‘the talk’ from the doctors,” said Ms Lee. “Be prepared, be prepared, they said. But on the third day, he opened his eyes.”

At the time, he was on dialysis as his kidneys had failed, on a respirator to help him breathe and on 20 different types of medication. His skin was so damaged and infl amed that it had turned a black hue.

“There were IV (intravenous) plugs everywhere. He was connected to a whole ‘garden’ of syringes and tubes,” said Mr Tay, 34, a regional risk manager with a Japanese trading house.

Although he was still very sick, doctors went ahead with the transplant of his father’s stem cells in January last year after discussing it with his parents, as they felt it was his best chance of survival.

But, said Mr Tay, “after 14 days, his own cells came back and killed Daddy’s cells”.

His father’s cells did, however, buy him some time, by rescuing him from severe infections and allowing for a more intensive preparation for his body to receive a second stem cell graft.


The next month, after chemotherapy again to kill off Donovan’s own defective immune system, Ms Lee was the next donor. This time, the transplant was a success.

Said Mr Tay: “Visually there wasn’t any improvement at fi rst. But then his skin stopped peeling for the fi rst time, and slowly he began to need less medication and less support. Slowly he moved from intubator to gas mask to a tube to breathe. I would like to think that with my transplant, I cleared the way for Mummy’s cells.”

Added Ms Lee: “He was still so weak, we had not seen him move for such a long time. But we celebrated all the little victories, such as one fewer antibiotic. When he peed, we jumped because it meant his kidneys were working.”

As Donovan’s blood tests began to show more normal results, his parents allowed hope to creep in.

He was still “a bag of bones”, without the strength to lift his arms. And they were uncertain if he had suffered brain damage.

“The heartache was to see my baby not being able to move,” said Ms Lee, breaking down for the fi rst time during the interview.

“But his eyes were very expressive. We believed that he was in there somewhere. We kept believing he was in there.”

Gradually, their boy was able to turn his head and move his arms. And, best of all, he began to smile and laugh. Two months ago, he no longer had to be tube-fed and could fi nally eat on his own.

These days, there is no stopping Donovan, whose skin is now smooth and itch-free. At the family’s MacPherson fl at, he is always on the go, and interested in everything.

His overjoyed parents reiterated how grateful they are to the doctors, nurses, surgeons and therapists for saving Donovan, be it from the ICU, renal, oncology, immunology or infectious diseases teams.

Said Ms Lee: “For a very long time, he was on the edge. We cannot express how happy we are now seeing him grow and learn, just like any normal child. Our hope for him is one that every parent wants, which is for Donovan to be happy and healthy.”

Boy cured of rare illness after two stem cell grafts (cont’d)


Media : Parent’s Guide to Cord Blood

Date : September 2015

Interview with Matt Farrow, Recipient of World’s 1st Cord Blood Transplant

Matthew Farrow, then a 5 year old boy with Fanconi anemia, received the world’s fi rst cord blood transplant on 6 Oct. 1988. The pioneering medical event was an international effort: Matthew came from North Carolina USA, his donor was his newborn baby sister, the American scientist who stored the cord blood was Dr. Hal Broxmeyer, and the transplant was performed at the French Hôpital Saint-Louis in Paris, where his physician was Dr. Eliane Gluckman. Matt is now 32 years old, married, and a father.

Matt, why do you do public outreach?

My goal is to share my story to help increase awareness and understanding of cord blood. There are so many people who don’t really know the importance of it.

From your own experiences talking to patients and their families, what do you think are the key things that they should know about cord blood transplants?

I mostly talk to families with Fanconi Anemia (FA) because that is my diagnosis. My little sister’s cord blood cured me from bone marrow failure caused by FA and that allowed me to grow up and become an adult. I encourage families of young kids who have gone through transplants, and show them that patients can grow up, have a family, and live a normal life.

Matt, you now work for CORD:USE, a hybrid public/private cord blood bank that is directed by some of the leading doctors in cord blood transplantation. Can you tell us a bit about your work?


It is an honor to be able to work with a company that is led by the pioneers in this fi eld. These doctors played a huge part in my life. I work as a patient advocate and I am able to go to public events and hospitals to share from my patient perspective the importance of cord blood. When I am not traveling, I work in the corporate offi ce where I assist with the day to day operations of the cord blood bank. I do a lot of inventory management and warehouse upkeep. I do all of the ordering, for example I order all the supplies that go into the cord blood collection kits, both for collecting public donations and for family banking.

As a spokesperson for cord blood awareness, you have traveled to medical conferences, testifi ed to politicians, and appeared at public events like NASCAR races. What was the most exciting experience you have had as a spokesperson?

I love doing these events, but my most exciting experience would have to be meeting kids that have had a cord blood transplant, like Rachel who inspired the name of the blog I just started, The Mr Matt. Rachel Lynn Smith was a patient of Dr. Kurtzberg’s who had a cord blood transplant for Blackfan-Diamond Anemia. Rachel’s parents formed a Foundation in her memory called COW Houses. Children like Rachel inspire me to keep going and the people that I meet touch my heart.

Even though you are a celebrity in the cord blood community, the day to day reality is that you are a regular guy trying to make ends meet and raise a family. Do you have any advice for transplant survivors trying to fi nd a “new normal”?

I’ve been very blessed that I havn’t had long-term side effects from my cord blood transplant. Since I was so young when I had my transplant, living with that is all I ever knew. I was just a kid that wanted to play and get dirty and be like the other kids. After my transplant it took me a while to learn that it was OK to get dirty. So my advice would have to be that it is going to take time to adjust and to not dwell on the bad parts of the process. It is easy to ask “Oh, why me?” and I am sad that many of my friends have passed away. It took me a much longer time to realize that I had to focus on the future and really start living. G-d kept me here for a reason.

Matt, you appeared in a movie, Celebrating a Second Chance at Life - The history of Blood and Marrow Transplantation, that was produced by the patient support organization BMTinfonet. The movie featured some footage of you as a 5 year old in the hospital in Paris (minute 15). What do you tell your own son Elijah about those experiences?

Elijah will be twelve this November. We talk all the time about what I had to go through, how the transplant process was different from the way it is now, and I share with him little stories of the things that I can remember. For example I had to stay inside a plastic bubble and I could not have contact with anyone. I had to be sick by myself when all I wanted was to be held by my mom. When my parents came into the room they had to be completely gowned up. Even today, patients are in rooms with positive air pressure instead of being inside bubbles, but parents still have to gown up and wear gloves and there is always a barrier when they touch their children.

Elijah is very proud of me and very proud to share who I am and how many lives have been saved because of his dad’s experience as the fi rst patient.

Many people still are not very aware or informed about cord blood stem cells and banking options. What do you think the cord blood community should do about this?

I think there should be a stronger message educating the public on the truth about cord blood and cord blood stem cells. I feel there is too much negative energy in the media about embryonic stem cells and the public is not hearing enough about cord blood stem cells. This leaves an impression in people’s minds that stem cells are a negative thing. I was in a fast food restaurant one day and I overheard one customer tell another that cord blood banking is all a scam, so I went over and introduced myself. We need to get out more positive messages about cord blood stem cells.

I wish we had a TV presence in the cord blood fi eld. We should have public service commercials about cord blood banking.

I think a lot of doctors don’t explain cord blood options to expectant parents as well as they could, so better education for doctors would be helpful.


Singapore scientists see way to help Parkinson’s patients


Date : 7 August 2015

Scientists in Singapore have potentially found a way to repair damaged brain cells in sufferers of Parkinson’s disease by using stem cells from cord lining - spelling a possible breakthrough in combating the progressive neurological condition for which there is no effective treatment.

The team from the National University of Singapore (NUS) and the National Neuroscience Institute (NNI) used a technique developed by Nobel laureate Shinya Yamanaka to turn mature cells into “induced pluripotent stem cells” capable of turning into any cell the body needs.

It successfully used stem cells derived from the lining of the umbilical cord to generate new dopamine- producing nerve cells in mice.

If successful in humans, it could be the fi rst treatment to target the root cause of the disease, which affl icts three out of every 1,000 people aged over 50 here - costing each patient an estimated $11,000 in medical bills per year.

People affl icted with Parkinson’s disease progressively lose control of their movements due to a disruption in the brain’s production of dopamine neurons, which send signals to other nerve cells.

If these neurons can be re-generated, it could possibly reverse the effects of the disease.

Associate Professor Lim Kah Leong of the NNI and NUS Yong Loo Lin School of Medicine, one of the lead scientists of the study, said an advantage of using cord lining cells is that they are potentially immuno-privileged - that is, the chances of rejection in a recipient are low.

They are also purer then cells taken from the patient.

“If you were to convert cells from a 60- or 70-year-old individual, it is likely the cells will accumulate a lot of gene mutations induced by the environment,” he explained. Such cells might be less ideal genetically for use in the clinical setting.

Earlier attempts to treat the disease saw patients in the West transplanted with dopamine-producing cells from aborted foetuses, but this was discontinued due to ethical issues and side effects, he pointed out.

Cord lining cells - which come from a baby’s umbilical cord which is usually discarded - are abundant and safe, with no reports of tumour formation or adverse effects so far.The team has collaborated with home-grown biomedical fi rm CellResearch Corporation, and is looking at the next stage of animal trials.

By Samantha Boh

Neurons grown from “induced pluripotent stem cells” derived from the lining of the umbilical cord. PHOTO: COURTESY OF LIM KAH LEONG


Six Singapore companies make it to Forbes Asia’s ‘Best Under a Billion’ listBy Mindy Tan

Media : The Business Times

Date : 25 June 2015

SIX Singapore companies have made it to Forbes Asia’s “Best Under a Billion” list this year, three more than in 2014.

The six companies are Cordlife Group, Hock Lian Seng, Pacifi c Century Regional Developments, Q&M Dental Group, Silverlake Axis and Wee Hur.

The “Best Under A Billion” list honours 200 leading public companies in the Asia-Pacifi c region with annual revenue of between US$5 million and US$1 billion, having positive net income and having been publicly traded for at least a year. From a universe of 17,000 companies, the candidates are screened on sales growth and earnings growth in the past 12 months and over three years, and for the strongest fi ve-year return on equity. Of the 200 companies, 123 are new to the list.

This year, China and Hong Kong topped the list with a total of 84 companies, one down from last year. The second largest group of companies on the list came from Taiwan - this year, fi ve more companies made it to the list, bringing the total to 36.

Elsewhere in the region, South Korea is third on the list with 17 companies, up from 12 last year. India and Malaysia both have 11 companies on the list while Australia has nine companies. This year, Japan dropped out of the top fi ve countries, with only eight companies compared to 15 last year.


Media : The New Age Parents

Date : May 2015

Autism is a life-long developmental disorder that affects 1 in every 167 people in Singapore. With over 200 children diagnosed annually, there is a strong need to design an effective treatment option to enhance disease management and help patient improve their overall quality of life.

Cord blood that remains in the umbilical cord and placenta following the birth of a newborn is a rich source of hematopoietic stem cells, which have proven useful in the treatment of over 80 diseases including certain cancers and blood disorders. Compared to stem cells obtained from other sources such as bone marrow, cord blood contains the youngest cells that can be safely collected, presenting a potentially viable option in treating autism in children.

Cordlife has partnered with autism and epilepsy treatment pioneer Dr. Michael G. Chez from Sutter Medical Group, in the

continued study to treat child autism with the patient’s own cord blood stem cells.

We spoke to Dr. Chez for more updates on his research and the early signs of autism.

TNAP: When do early signs appear?

Autism is a brain-based developmental disorder that affects one’s communication, socialisation and engagement in developmentally-appropriate behaviours. As a spectrum disorder there is a wide range of symptoms that can be grouped in many different ways. Signs of autism appear early in development with most children showing symptoms by 12 to 18 months of age, while autism diagnosis in children largely occurs by the age of two years, with characteristic features of autism onset being a delay in language and social skills development, and repetitive behavioural patterns.

New Cord Blood Research: Treating Autism in Children


TNAP: What are the developmental “red fl ags” to watch for?

Development “red fl ags” of autism include but are not limited to diffi culty with verbal and non-verbal communication, avoiding or resisting eye contact, obsessive interests and behaviours. Approximately 40% of children with autism fi nd diffi culty speaking, and about 25% – 30% of children with autism speak several words at 12 to 18 months of age but soon lose them. Others might speak later in childhood.

TNAP: What are signs of late onset autism?

The signs of late-onset or regressive autism are the same as early-onset autism, however the timing is different. Early-onset autism is categorized as children who show signs within 12-18 months of age, whereas late-onset autism appears in children who develop normally then between one to two years of age have diffi culties with their speech capabilities, behaviour and social interactions.

TNAP: What are some of the common misconceptions/ myths about Autism?

Common misconceptions and myths about autism include the following:

• That the appearance of autism is relatively new, when in reality the earliest case was documented in 1799.

• Patients on the autism spectrum prefer isolation, when in fact most children and adults often want social interaction but lack the social development skills.

• That autism is not genetically inherited, however more families are experiencing multiple members with autism spectrum disorders.

• Symptoms of autism get worse with age, but in reality autism is not a degenerative disability and children and adults with autism should continuously improve with the right treatments and behavioural medications.

TNAP: Autism is a life-long developmental disorder, and is currently without a cure. However, personalised therapies and behavioural mediations can bring about signifi cant improvement. What are some of the therapies and behavioural mediations used to improve Autism?

Autism is a life-long developmental disorder that is currently without a cure. At the moment, personalised therapies and behavioural medications can bring about signifi cant improvement.

In Singapore, 1 in 167 people are affected with autism and with 216 new cases of children with Autism being diagnosed annually, the need for better treatment is increasing. Treatment for autism is a fairly new fi eld for the medical community, and is a growing area of interest for geneticists and doctors alike.

With a rising global need for advancing autism treatment and a personal research interest in childhood autism, my working partnership with Cordlife – the pioneer in Singapore operating the largest network of quality cord blood banks in Asia – will enable me to advance my research with physicians both in Singapore and across Asia to help improve the quality of life in patients with autism.

TNAP: Leading medical specialists around the world are also researching the use of umbilical cord blood stem cells for autism treatment in children. What are the new breakthroughs in this research?

I’m currently researching the use of autologous (own self) umbilical cord blood stem cells for autism treatment in children. This undergoing study examines 31 children aged two to seven who are separated into groups based on the type and degree of autism they suffer. I’ve been working with them for about two and a half years so far and I’m hoping to release fi ndings in the next six to nine months.


TNAP: How does the use of umbilical cord blood cells help in the treatment for Autism in children?

As immune dysregulation has been proposed as a possible basis of autism, an effective treatment for autism may involve modulation of the immune system, resulting in improved language and behaviour outcomes. Because cord blood stem cells theoretically have the potential to re-programme or supplement unusual functions of the immune and central nervous systems, and possibly stimulate innate stem cells, cord blood is an attractive candidate for investigation in treating autism in children.

TNAP: Any words of encouragement to parents with children with Autism?

While it can be a tough period when your child is fi rst diagnosed with autism, you can fi nd comfort in the fact that there are treatment options available. For one, umbilical cord blood stem cell therapy has been of rising interest to families who have children with autism. Because cord blood stem cells are a ready source of genetically-related stem cells for someone else like a sibling in the family, there is an exponential increase in having a matched unit available for future medical use if needed.

Also, I am currently in discussion with some of the top physicians in their respective fi elds in Singapore to explore the opportunities that are present here for autism treatment. Thanks to the advancement of medical science, you can always fi nd hope in the options available to improving the quality of life for your child.

These questions were answered by Dr Michael G. Chez, a leading Neurologist and Director of Pediatric Neurology and Pediatric Epilepsy for both Sutter Medical Group and Sutter Neuroscience Institute. He is actively involved in the advancement of treatment of childhood autism, epilepsy, learning or degenerative diseases, and muscular dystrophy and spina bfi da. With over 20 years of experience in the use of drug and immunological therapies for refractory autism and epilepsy, Dr Chez has been working with Cordlife on the possibility of bringing the experimental treatment of autism to Singapore. This is the fi rst of such initiatives in Singapore.

New Cord Blood Research: Treating Autism in Children (cont’d)


Media : Duke Medicine

Date : 18 June 2014

$15 Million Award to Go Toward Exploring New Treatments for Autism, Other Brain DisordersBy Duke Medicine News and Communications

DURHAM, N.C. -- Duke Medicine has been awarded $15 million to support an innovative research program that explores the use of umbilical cord blood cells to treat autism, stroke, cerebral palsy and related brain disorders.

The award from The Marcus Foundation, an Atlanta-based philanthropic organization, will fund the fi rst two years of a planned fi ve-year, $41 million project by Joanne Kurtzberg, M.D., chief scientifi c and medical offi cer of Duke’s Robertson Cell and Translational Therapy Program, and Geraldine Dawson, Ph.D., director of the Duke Center for Autism Diagnosis and Treatment.

“I am excited about this unprecedented opportunity,” said Victor Dzau, M.D., chancellor for health affairs and president and CEO of Duke University Health System.

“Joanne Kurtzberg has done groundbreaking work on cord blood transplantation at Duke, and Geri Dawson brings an enormous wealth of knowledge and experience of autism,” Dzau said. “Together they will explore innovative approaches to treating these challenging brain disorders. This research holds the promise of truly transformational discovery, and we are deeply grateful to The Marcus Foundation for making it possible.”

Kurtzberg and Dawson hope to develop cell-based therapies that can potentially restore brain function in people with the disorders, for which there currently are no cures. If successful, the study could identify therapies for further evaluation in clinical trials to potentially decrease disabilities and improve the quality of life for millions of children and adults.

“Duke University and Health System greatly appreciate the vision of The Marcus Foundation,” said Duke President Richard H. Brodhead. “This gift will enable the expertise of Duke’s medical researchers to be focused on new approaches and treatments, with the goal of bettering the lives of millions.”

The project will consist of a series of clinical trials using umbilical cord blood cells to treat a total of 390 children and adults with autism, 100 children with cerebral palsy and 90 adults with stroke. Based on previous research, Kurtzberg and Dawson hypothesize that cord blood may promote repair of dysfunctional or damaged areas of the brain.

“Funding for this type of research is very scarce, so the only way we can truly make progress is with support from private philanthropic organizations like The Marcus Foundation,” said Nancy Andrews, M.D., Ph.D., dean of Duke University School of Medicine. “With the foundation’s help, we hope to give untold numbers of people with autism and related disorders hope for a better outcome.”

There are approximately 2 million people in the United States with autism spectrum disorder, a group of conditions affecting social communication and behavior. Stroke kills an average of nearly 130,000 in the U.S. every year, while cerebral palsy currently affects an estimated 764,000 children and young adults.


$15 Million Award to Go Toward Exploring New Treatments for Autism, Other Brain Disorders (cont’d)

The initial phase of the program -- a preliminary trial involving 20 pediatric subjects with autism using their own banked cord blood -- is already under way. It will conclude with Phase II trials using donated cord blood in children with autism and cerebral palsy and adults with stroke.

“The whole program has enormous potential,” said Kurtzberg, who is also director of the Pediatric Blood and Marrow Transplant Program and the Carolinas Cord Blood Bank. “Autism, stroke and cerebral palsy are all neurologic conditions that impair function and quality of life for these children and adults. If we can make that better, it will have a huge personal and societal impact.”

Cord blood cells are collected without risk to the mother or baby from the placenta, which is otherwise discarded as medical waste after a baby is born. After collection, the cells can be frozen and stored for future use in blood stem cell transplantation or cellular therapies.

Kurtzberg’s previous research has shown that cord blood cells can reduce infl ammation and signal normal cells to repair damage in areas of the brain affected by inherited pediatric brain diseases. A recently published Duke study indicates that treating children with their own cord blood cells can have similarly benefi cial results in cases of hypoxic ischemic encephalopathy.

Building on those research results, the new study will try to determine whether cord blood cells will have the same effect in cases of autism and stroke. Using methods developed by Dawson, the study will examine whether the therapy not only improves behavioral outcomes in children and adults with autism, but also reshapes the patterns of brain activity.

Kurtzberg and Dawson also will explore the key question of whether the benefi cial effects of cord blood can be achieved by treating patients with donated cord blood rather than only with their own banked blood cells. If that is found to be true, it could make cord blood treatment available to the largest numbers of patients in need.

“We all wanted to have a treatment that, if it’s effective, would be accessible to everyone,” said Dawson, who was the founding director of the University of Washington Autism Center and then chief science offi cer at Autism Speaks before joining the Duke faculty in August 2013.

Established by Bernie Marcus, the co-founder of The Home Depot, The Marcus Foundation has a long-established interest in autism, stroke, cerebral palsy and other neurological conditions, and in stem cell research. It focuses on biomedical research projects that are close to clinical application, that may bring novel therapies to bear on disorders without existing treatments, and that are unlikely to receive funding from traditional sources such as the National Institutes of Health.

Marcus also opened the Marcus Autism Center in Atlanta in 1991 and donated the funds to start the Autism Speaks advocacy organization in 2005.

Families interested in enrolling a child in the study should contact Duke at [email protected] or (844) 800-CORD or (844) 800-2673.


Stem-cell hope for spinal patients as big test nears A leading researcher into severe spinal cord injuries says trials for stem-cell therapy show groundbreaking results in helping immobile patients to walk again.

After progress in a second round of tests using stem cells to regrow nerve fi bers, the China Spinal Cord Injury Network, set up in Hong Kong, has applied for regulatory approval in the mainland for a third and fi nal phase, which could start in the autumn.

“This will convince the doctors of the world that they do not need to tell patients `you will never walk again,”’ said US-based doctor Wise Young, the alliance’s chief executive.

“Hong Kong is going to be way ahead of all countries if the spinal cord injury trial turns out to be positive.

“That means Hong Kong will be the center for stem-cell therapies.”

He said 15 of 20 patients in Kunming who received umbilical cord blood cell transplants and intensive therapy were on average able to walk with minimal assistance seven years after suffering complete spinal cord injury.

“It’s the fi rst time in human history that we can see the regeneration of the spinal cord,” Young added.

The treatment involves injecting umbilical cord blood mononuclear cells into patients’ spines to help regenerate nerves, while lithium is used to promote growth of nerve fi bers.

Each component of the combination therapy will be tested in the third phase, which Young said will involve 120 patients in the mainland and 120 across India, Norway and the United States.

“If the phase-three trial is successful, we should have achieved worldwide regulatory approval by the beginning to the middle of 2015,” Young added.

The non-profi t alliance claims to be the world’s largest clinical trial network for such therapies.

Young also said that the mainland is investing heavily in stem cell research, but the technology remains highly controversial in the United States.

That is because of the anti-abortion camp’s concerns about cells that are derived from human embryos.

The late Hong Kong quadriplegic Tang Siu-pun — better known as Ah Bun — appeared with Young during the network’s launch in 2005 to help raise US$50 million (HK$389 million) for drug trials. AFP and STAFF REPORTER

Media : The Standard

Date : 27 June 2013


At the age of nine, Alessandra Samson, apart from being the smallest in her Grade 3 (equivalent to Primary 3) class at United World College, looks like any other child.

Her mother worries she will never catch up with her classmates — with good reason. Alessandra was diagnosed with acute lymphoblastic leukaemia when she was just seven months old.

By the age of two, she had undergone several rounds of chemotherapy and two cord blood transplants. Cord blood, which is blood that remains in the umbilical cord and placenta after birth, contains stem cells which can develop into a variety of cells.

After the gruelling treatment, during which she suffered side effects, such as vomiting, nausea and mouth sores, she went into remission and has remained so since.


Date : 15 June 2013

Growing up in cancer’s shadowBy Ng Wan Ching

Mrs Lourdes Samson is concerned that the radiation from chemotherapy may have affected the physical development of her daughter, Alessandra Samson.

PHOTO: DIOS VINCOY JR FOR THE STRAITS TIMES

But doctors are still keeping a close watch on Ally — as she is known fondly at the National University Hospital (NUH) — especially on her rate of physical growth. Doctors will be doing more tests to check if she is growing at her “genetic potential”.

Ally is among a growing pool of paediatric patients here who have beaten their cancer, but are now at risk of what doctors call late effects of cancer treatment, such as chemotherapy, years down the road. These include low levels of sex hormones (which can lead to delayed puberty), hypothyroidism (low levels of the thyroid hormone, which is important for growth and intelligence), poor bone health and cataracts (clouded eye lenses).

NUH handled 130 new paediatric cancer cases last year, compared with 100 cases fi ve years ago, while KK Women’s and Children’s Hospital (KKH) now sees about 100 to 120 a year, an increase from about 70 cases a year about fi ve years ago.


The most common childhood cancer here is leukaemia, followed by brain tumour, lymphoma (cancer of white blood cells, usually in the lymph nodes), germ cell tumour (cancer of cells in the ovaries or testes), sarcoma (soft tissue cancer), neuroblastoma (nerve cancer), Wilms’ tumour (kidney cancer) and retinoblastoma (eye cancer).

Overseas studies show around 70 to 80 per cent of children and adolescents diagnosed with cancer will become long-term survivors. An estimated 60 per cent of such survivors will develop chronic treatment-related health conditions years or decades after completing treatment. These are known as late effects.

Their general health is also likely to be marginally poorer than that of their peers. For example, their risk of heart attacks would be higher. The causes are multi-factorial, including overprotective parents who prevent them from exercising, which is key in preventing heart disease. As the number of survivors is expected to increase because of better therapy and higher cure rates, there is a critical need to create the awareness of these late effects, said Dr Tan Poh Lin, senior consultant at the division of paediatric haematology-oncology at NUH.

FOCUS ON LATE EFFECTS

The growing number of childhood cancer survivors led NUH to start a single clinic session for them — involving various specialists, such as endocrinologists, heart and lung specialists, eye specialists and dental surgeons - three months ago.

This makes the late effects service — started in 2000 on an ad-hoc basis and expanded in 2009 by involving more specialists — more convenient for patients as they get to see all the specialists in a single session now.

“It is important for a patient to be monitored and followed up by his oncologist, who will order comprehensive health checks, based on his treatment, type of cancer, age, gender and other factors,” Dr Tan added. Patients can also lower their risk by cultivating good diet and lifestyle habits, for example, eating right, avoiding smoking and limiting the intake of alcohol, she said.

KKH does not have a separate service for late effects but its doctors continue to see patients and monitor them for any late effects of cancer treatment, said Associate Professor Tan Ah Moy, senior consultant at the haematology/oncology service in the department of paediatric subspecialties.

“Late effects are expected and so our care does not stop with the cessation of treatment,” she said.

Doctors continue to monitor the height and weight of patients, and screen for their social, psychological and physical well-being. Paediatric patients may be referred to other relevant specialists, if necessary, Prof Tan said.

All young patients at KKH have consistent follow-up checks and are transferred to an adult hospital if they need treatment then. Late effects can go undetected initially or it takes time for endocrine defi ciencies to manifest, said Dr Cindy Ho, associate consultant at the division of paediatric endocrinology at NUH.

For example, radiation damage to the pituitary gland - also known as the “master gland” as the hormones it produces control many different processes in the body, including growth and maturation — is indirect and can be very gradual. It may take up to 20 years after radiation treatment for the full spectrum of hormonal defi ciencies to evolve, said Dr Ho.

Studies have shown that some cancer survivors have a slightly higher risk of secondary cancer, due to certain chemotherapy drugs and receiving radiation at a young age, said Dr Tan. If secondary leukaemia occurs, it is usually within the fi rst 10 years following treatment of the original cancer. Secondary solid tumours more commonly happen 10 or more years after treatment. Less than 10 per cent of patients have secondary cancer due to gene mutations.

NUH is currently collecting data on all the different types of childhood cancer, their treatment and the types of late effects caused. It has found the top fi ve late effects in children who had received a bone marrow transplant and had survived one to fi ve years post transplant.

The results, presented in 2010 at the 15th Congress of Asia-Pacifi c Blood and Marrow Transplantation meeting in Thailand, showed that six in 10 survivors had iron overload,


which has ill effects on the heart and growth. Four in 10 suffered from poor bone health. Two in 10 had high cholesterol and lipids. Between one and two in 10 were low in thyroid hormone. And six in 100 had cataracts.

WATCHING AND WAITING

Ally crossed the fi ve-year mark after her second cord blood transplant three years ago, said her mother, Mrs Lourdes Samson. The family was originally from the Philippines and are now permanent residents here.

At that point, doctors started doing comprehensive tests, including tests to examine her eyesight, teeth, bones, lungs and heart. She also had a bone mineral density test to fi nd out how strong her bones were, said Mrs Samson, 42, a housewife.

Before Ally had a transplant, she underwent radiotherapy to kill the cancer cells.

“She had a very small amount of it, but we don’t know what the late effects would be,” said Mrs Samson. “It’s all about her development now and she has had a check-up every year since. So far, everything is good, but we have to keep watch.”

The main concern is that the radiation may have affected her physical development. If Ally, an only child, does not grow normally, doctors may give her growth hormones to help boost her growth. They will also watch for signs of early or delayed puberty.

NUH’s Dr Ho said before the use of growth hormones is considered, the cancer should be in remission.

Most studies have shown no safety issues with long-term use of growth hormones, but there is still limited data and it demands continued surveillance, she added.

“We are hoping for the best for Ally, and if possible, no growth hormones,” said Mrs Samson.

Growing up in cancer’s shadow (cont’d)


Media : The Straits Times, LIFE!

Date : 24 November 2012

Child has benefi ted from cord bloodBy Mrs L. Conn

I read with interest and sadness the report Public Or Private Bank? (SundayLife!, Nov 4) and a subsequent letter to Mailbag (Limitations Of Cord Blood Banking, Life!, Nov 10).

Conservative comments do great damage to the researchers and progressive medical teams around the world using stem cells successfully for all sorts of conditions.

As a mother of 5½ year-old girl who suffers from cerebral palsy, I can say that my family has benefi ted greatly from our decision to store and use our child’s umbilical cord blood here.

We cannot “cure” her cerebral palsy but we have searched the globe for therapies and treatments that may help her gain control over her movements and give her better quality of life.

The treatment using her own stem cells was safe and effective, and has made an enormous difference to our daughter’s health and future, and we would do it again a thousand times over.

Having easy and direct access to her own cells was the key to the success in our case.

Doctors need to be very careful with very generalised comments about blood banking.

Parents have the right to do their own research and protect their child as they see fi t, especially if that child has an illness or injury that regular, conservative medicine offers little hope for.


Media : Mind Your Body, The Straits Times

Date : 02 December 2010

The stem-cell hopeBy Lee Hui Chieh

Stem cells, which have the potential to develop into some or all of the specialised cells in tissues and organs provide hope that they can replace damaged cells. The race is on to fi nd new ways to harness them to treat various diseases.

To most people, a bone marrow transplant is as familiar as any Japanese, Korean or Taiwanese tearjerker, which often features a protagonist who is dying of leukaemia and needs such an operation.

Stem-cell therapy, on the other hand, sounds experimental, almost the stuff of science fi ction.

Yet, the bone marrow transplant, which has been in the medical lexicon for decades, is really a form of stem-cell therapy. It is a transplant of haemopoietic stem cells, or immature blood cells, found in the bone marrow.

Bone marrow transplants have been used here since 1983 to treat blood diseases such as leukaemia.

These and other stem-cell treatments here have been done mostly with adult stem cells.

These are immature cells found in the body’s tissues or organs that can eventually develop into some or all of the specialised types of cells found in those tissues and organs.

The appropriate stem cells are injected into the body to replace diseased or damaged cells.

The race is on to fi nd new and more effective ways of harnessing stem cells for the treatment of various diseases.

Miss Cho, 56, a procurement offi cer, still played at club level after leaving the national team in 1989.

In the last few years before she called it quits, her knees became swollen and painful after each game.

In 2003, the pain worsened and her knees hurt when she climbed the stairs. One day in 2005, she woke up to fi nd them locked.

She sought help from Associate Professor James Hui, National University Hospital’s (NUH) senior consultant

orthopaedic surgeon, who sliced her left kneecap and implanted cartilage grown from stem cells taken from her bone marrow into the joint. The cartilage cells grew to fi ll the spaces between the bones, he said.

Miss Cho had the procedure done on her right knee two years later.

After a year of physiotherapy, she is now playing badminton weekly. She said: ‘Now I can play normally but I don’t go all out like before.’

She is among more than 200 patients who have paid $4,000 to $8,000 to undergo this therapy, on trial since 2003. Prof Hui hopes to recruit 300 patients aged below 65.

NUH is also testing a second method using stem cells injected directly into the knee.

Patients fi rst undergo an existing treatment where microfractures, holes made with a tiny pick, are made through keyhole surgery in the bone of the worn-out joint. This stimulates the bone marrow into releasing a type of stem cell that later grows into cartilage.

Stem cells are also retrieved from the hip, grown and injected into the knee with hyaluronic acid, which protects the cells.

NUH hopes to enrol 100 patients in this trial, which began last June. All of them will get microfractures and the acid, but only some will be given the additional stem cells for comparison purposes. The 42 patients treated so far paid between $4,000 and $7,000.

Former national netball player Sally Cho (above) was forced to give up the sport 10 years ago when the pain in her knees became unbearable.


Cerebral palsy baby helped by cord bloodBy Judith Tan



Infusion a fi rst in S’pore; clinical trial planned for next year

AN ACCIDENT at birth 2½ years ago deprived Georgia Conn of oxygen to her brain, resulting in cerebral palsy.

The damage to the toddler’s brain caused her to move involuntarily, suffer from muscle spasms and have up to 50 seizures a day. But in September, after Georgia was infused with her own cord blood in Singapore, her condition improved.

Her Australian parents, who have lived here for fi ve years, noticed changes in her temperament and concentration. She also regained some muscle strength. Said her 35-year-old mother, Mrs Louise Conn: “These changes happened over the past 2½ months, so we are intrigued as to what might happen over the next year or so.”

The procedure marked the fi rst time that stem cells from a baby’s own cord blood have been used to treat cerebral palsy here.

The 15-minute infusion was carried out by neurosurgeon Keith Goh at the Conns’ home after getting the green light from the Ministry of Health (MOH).

“There were no ethical issues as the cord blood was Georgia’s own. The worst that could happen was nothing. At least her body would not reject her own cells,” Dr Goh said.

Mrs Conn said: “It took us more than 18 months and an enormous amount of research, mainly on the Internet, to fi nd information about the potential for using stem cells.”

Mrs Louise Conn, with husband Michael and daughters Georgia (left) and six-month-old Sybilla.

ST PHOTO: JOYCE FANG

At the time, the Conns thought their best option was at Duke University in North Carolina in the United States. There, a professor of paediatrics and pathology was conducting a study in which 97 children with cerebral palsy were injected with their own cord blood cells.

Mrs Conn and her husband Michael, 37, got in touch with CordLife, a private cord blood bank at which Georgia’s had been banked at birth. It was after Cordlife put them in touch with Dr Goh that they realised the infusion could be carried out here.

Eighty per cent of Georgia’s banked cord blood was used for the $10,000 infusion – a payment that the Conns felt was “worth its weight in gold”.


Rich stem-cell source

DON’T EXPECT MIRACLES

As a parent, you secretly hope that this will be a magical cure for your daughter and she will

get up and say, ‘Wow, that was a wild ride’. Of course, this was not ever going to be the case and we went into the procedure with our

eyes wide open.

MRS LOUISE CONN

went into the procedure with our eyes wide open.

As a parent, you secretly hope that this will be a magical cure

Dr Goh has since applied to MOH to conduct clinical trials on using cord blood stem cells in the treatment of cerebral palsy here. “The type of trial I am looking at involves about 10 cases. I hope to start the trial early next year. Three children with cerebral palsy have come forward to be treated,” he said.

Dr William Hwang, medical director of Singapore’s only public cord blood bank, said a current Duke University study found that the infusion of such cord blood cells shortly after birth will facilitate cellular repair of perinatal brain injury, reducing the incidence and severity of cerebral palsy.

“Our neonatologists at public hospitals are seeking grant funding and obtaining approval from the Institutional Review Board to conduct an extension of Duke trial,” he said.

BLOOD from the umbilical cord is collected and stored in cryogenic tanks soon after a baby is born. It is a rich source of stem cells – immature cells that can develop into a wide range of blood cells. They can be used to replace those ravaged by diseases like cancer and anaemia.

More than 80 diseases can be treated with cord blood and the number is growing rapidly. They include sickle cell anaemia, thalassaemia, leukaemia, metabolic storage disorders which cause the brain and nerves to deteriorate, and genetic disorders that hamper the ability to fi ght infection.

New areas where research into cord blood stem cells is currently being done include brain injury, juvenile diabetes, amyotrophic lateral sclerosis or Lou Gehrig’s disease, cerebral palsy, congenital heart defects, hearing loss, liver disease and spinal cord injury.

Cerebral palsy baby helped by cord blood (cont’d)




Ms Kymn Yee shows the device she wears for 20 hours a day. It administers a chelating agent via a needle to deal with excess iron in her system due to frequent blood transfusions.

ST PHOTO: DESMOND LIM

She has lived a life of countless hospital visits and needles inserted in her body.

When she was six months old, Ms Kym Yee was diagnosed with thalassaemia major, an inherited form of anaemia.

Now the 30-year-old schoolteacher still goes for monthly blood transfusions at the National University Hospital (NUH).

She also undergoes chelation therapy to deal with the excess iron in her system from the transfusions. This involves wearing a small device strapped around her body that administers the chelating agent via a needle.

She usually wears the device for eight-hour stretches, but has recently extended the time to over 20 hours due to the high iron levels detected in her blood.

“I started putting in the needle myself when I was 21. My mum taught me how to do it,” she said. “I always fi nd it traumatising.”

On the frequent transfusions, she said they usually take about 12 hours to complete, during which she reads, catches up on work or naps.

Even after all these years of enduring the treatment process, she still fi nds it “quite painful” at times, not to mention inconvenient. “It has been a great interruption to my life as I always have to make time for transfusions,” she lamented.

It is costly too. Ms Yee said she spends about $800 a month on her treatments.

Things took a turn for the worse in April 2005 when she tried a new form of oral medication for her chelation therapy and developed a severe allergic reaction to it.

Blood disease not a dead endSchoolteacher with thalassaemia tells DHANY OSMAN how she is determined not to let the monthly blood transfusions she needs get her down.By Dhany Osman


What she initially thought was just fever turned into seizures. This led to her shuttling to and from the Intensive Care Unit (ICU) over a period of two months. “The two months seemed to last forever. I’d get better, then get sick again. It was like a roller-coaster ride,” she recalled.

However, once her immune system picked up, she recovered quickly and was back at work after the June school holidays that same year. She said: “People say that I’m strong to have been through all that, but it’s not like I had a choice.”

Around the same time, she joined the Thalassaemia Society (Singapore), or TSS, and attended her fi rst international conference held in Dubai in late 2005.

Meeting fellow thalassaemia patients from around the world was an eye-opening experience. It reinforced her determination not to let the disease be a “dead end” in her life.

As the vice-president of TSS, she helped organise the International Thalassaemia Conference held here last month.

She inherited the condition from her parents, who are both carriers of the thalassaemia gene. “Those days, I don’t think there was any test for the condition,” she said.

She maintains an active lifestyle now and goes scuba-diving and exercises in the gym.

She has learnt to accept her condition but it is still not easy. “There are moments when I get depressed and ask why I have to go through all this,” she said.

Ms Yee, who is currently single said that she has also started to reconsider the prospect of having her own child as she fears passing on the disease.

Even with non-thalassaemic partner, her child will carry the trait as a thalassaemia minor. Should her partner have thalassaemia minor, there is a 50-per-cent chance of their offspring having thalassaemia major.

“I don’t think it’s easy for any parent to have to see their child go through repeated hospital visits,” she said.

On her hopes for the future for local thalassaemia patients, she said: “I’d like to see more inter-patient interaction. We need a stronger support network to share with one another what we go through.”

What is thalassaemia?

Thalassaemia is an inherited blood disorder and, depending on its severity, patients can have symptoms of anaemia. It is particularly prevalent along the Mediterranean coast, hence the Greek origin of its name, “thalassa” for sea and “haema” for blood.

Thalassaemics can be divided into two groups – those with thalassaemia major and those with thalassaemia minor. The latter carry the thalassaemia gene, or trait, but do not necessarily exhibit any symptoms.

Those in the more serious category may start exhibiting signs of anaemia at around six months of age, appearing pale and having diffi culty putting on weight.

Dr Angeline Lai, head of the genetics service department at KK Women’s and Children’s Hospital (KKH), said the only known cures for thalassaemia major patients are either a bone marrow or a cord blood transplant.

Given the risks in such procedures and the diffi culty in fi nding a matching donor, most patients opt for a treatment process that involves regular blood transfusions and iron

Blood disease not a dead end (cont’d)


If a couple both have thalassaemia minor, there is a 25% chance of getting thalassaemia major.major.

If a couple both have

To fi nd out more about the Thalassaemia Society ( Singapore), visit www.thalsociety.org

chelation therapy in which medications help manage the excess iron in the blood from repeated transfusions. Desferrloxa mine, the chelating agent, is delivered over several hours by a device with a syringe attached to a pump that is attached to the body. Wearable versions of this device are available but expensive, said Dr Lai, adding that an average unit costs about $2,000.

In the last few years, new oral medications have become available here but at signifi cantly higher cost than the desferrioxamine treatment.

In all treatment cases, the amount of blood and medication required will vary with the patient’s weight. Dr Lai estimated that a 40kg patient will need to spend between $700 and $2,400 a month, depending on the type of chelation therapy pursued and the subsidies available.

She said that because the disease is genetic, if a couple both have thalassaemia minor, their child has a 25 per cent chance of getting thalassaemia major and 50 per cent chance of having thalassaemia minor.

A recent study conducted by the Ministry of Health found that about 4.5 per cent of Singapore’s population, or roughly 189,000 people, carry thalassaemia genes. Set up in 1992, the National Thalassaemia Registry has been screening family members of thalassaemia patients. It has thus far screened 19,000 people.

Social support network for patients

Founded in 2003 with just 10 members, the Thalassaemia Society (Singapore) is a small charity group that provides a social support network for thalassaemia major patients and their families.

Its president, Mr Derrick Lim, 31, said the society also helps to raise public awareness of the condition, disseminates the latest medical information to members and provide fi nancial aid to those who cannot afford treatment.

Medical bills for thalassaemia patients, he noted, can range between $600 and $4,000 a month.

Mr Lim said that currently, the society has about 50 members.

While the number of new thalassaemia patients has been kept low thus far – thanks to prevention programmes such as screening of patients’ family members for the gene by the National Thalassaemia Registry – there are still those in need of support, he emphasised.

Given the small size of the charity, raising enough funds has been a constant challenge.

Early last month, the society organised an International Thalassaemia Conference, the fi rst of its kind in Singapore. The event brought together 1,200 medical professionals, patients and family members from all over the world.


Media : TODAY

Date : 28 August 2007

A 19-year-old girl has successfully battled leukaemia following a stem cell transplant using umbilical cord blood from two unrelated individuals. Polytechnic student Candy Yeow underwent a novel procedure known as double cord blood transplant after there was no match for a bone marrow transplant (see other story).

Singapore General Hospital’s consultant haematologist and Deputy Director for the Centre for Transfusion Medicine at Health Sciences Authority Dr Mickey Koh said double cord blood transplant is an option for Singaporean adult patients suffering from leukaemia. This is because due to small family size, many may have only one sibling or none at all. Therefore a patient may not have a suitable match.

Dr Koh explained, “Bone marrow transplants work better if the donor is a sibling. There’s a 25 per cent chance that their cells make a perfect match with the patient’s and this minimises

Leukaemia survivor Candy Yeow remained optimistic even while she was undergoing double cord blood transplant.

side effects. If the sibling is unsuitable, we can search bone marrow registries worldwide for unrelated donors. But the Asian registry is under-represented and fi nding a match is relatively more diffi cult. Moreover, this process may also take considerable time.

Novel double cord blood transfusion

The alternative to a bone marrow transplant is cord blood transplant. Pioneered in France in 1998, the procedure was then confi ned to a single cord blood unit and initially used on children age 12 and below. Cord blood is collected at childbirth and donated to public cord banks or stored by parents with private cord blood companies. Donated cord blood can be obtained from an international network of healthcare facilities including the Singapore Cord Blood Bank at KK Women’s and Children’s Hospital.

As a single unit of cord blood may not contain suffi cient stem cells needed for an adult, a novel procedure would be to combine two cord blood units to increase the stem cell dose. However, this initially raised the issue on whether the two separate units would react with one another or against the patient.

Dr Koh said, “A double cord blood unit transplant is an innovative concept which enables us to use cord blood in adults. The best thing about cord blood transplants is that differences in blood group between recipient and donors do not matter as much. The two units of cord blood can also be of different blood groups.

Recipient rid of diseased cells

Before a cord blood transplant is carried out, a recipient undergoes chemotherapy and radiotherapy to clear his bone marrow of leukaemic cells. The donated two units of cord blood are then transfused into the recipient.

Cord blood gives hope to adultsTwo unrelated cord blood units can be used to treat adults affl icted with leukaemia.Stories by Hazel Yong and Karen Teng [email protected]


“The stem cells from the donors’ blood interestingly then migrates to the recipient’s ‘empty’ bone marrow. In time, the cord blood units would ‘engraft’ or grow in the recipient’s bone marrow and produce new platelets, white and red blood cells. It is very much like waiting for a seed to germinate.”

“Biologically speaking, there were three ‘people’ fl owing in one body after the transfusion – Candy’s cells and the two different cord blood units. But invariably, we always see one of the two cord units taking over, sometimes as soon as six weeks so that eventually, one cord predominates while the other fades away. Candy’s blood type has also changed from her original to that of the new cord unit. Research is still undergoing to understand more of this process and to predict which of the two units would eventually engraft”.

Possible new treatment option

SGH’s department of haematology runs the largest adult stem cell programme in Singapore. Since last year, doctors have performed three cases of double cord transplants. Candy Yeow is the longest survivor with the best results as she is in remission and does not require any medication.

While double cord blood transplant is still a relatively new procedure, Dr Koh and the rest of the transplant team are also embarking on a parent-child bone marrow transplant programme in a bid to increase treatment options. “This does present with additional new problems and risks as the match in such instances is usually only about 50%. The risks are therefore much higher. The parent-child bone marrow transplant is a possibility as the genetic match may reach 50 per cent.”

Her sweet victory over leukaemia

A year after undergoing double cord blood transplant, a young leukaemia suvivor is back in school and off all medication.

All eyes were on Candy Yeow when the 19-year-old arrived at a conference room packed with journalists gathered to hear how she battled leukaemia successfully with a double cord blood transplant.

Casually dressed in a grey top and black pants with chunky black earrings, she fl ashed an abashed smile before girlishly covering her face with her hands, overwhelmed by the media attention.

Her year-long struggle with Philadelphia positive acute lymphoblastic leukaemia, a rare and aggressive form of the cancer began early last year. “I don’t fall sick easily. So I knew something was not right when I felt faint, wheezed while going up short fl ights of stairs and red dots appeared on my body.”

A month later, Candy went for a check and was found to have an increased heartbeat of 120 beats per minute. That, together with her symptoms, warranted an immediate referral to a hospital. Test results of her white blood cells showed 238,000 per microlitre of blood. The normal results are between 4,000 and 10,000.

“I was admitted to SGH even though it was near midnight. I didn’t realise its signifi cance then.”

Candy Yeow and her parents (second and fourth from left) celebrated the fi rst anniversary of her successful double cord blood transplant with Dr Mickey Koh and the SGH doctors and nurses who looked after her.

Blood disease specialists like Dr Koh are currently doing research into the post-transplantation process.


Intensive chemotherapy

The next day, a bone marrow test was done and the results showed that Candy had leukaemia. Following that she underwent her fi rst course of chemotherapy through a catheter inserted into a vein in her chest. To destroy as many leukaemia cells as possible, the fi rst phase of treatment was intensive and stretched over four days, leaving her faint and nauseous.

Candy shared, “On the second day, while I was in the toilet, I couldn’t see or hear anything. I pulled my pants up and yelled. When I came to, I was on the bed with nurses around me.

Support from family and friends

The effects of treatment affected Candy physically and emotionally. She had to be shaved bald to prevent her hair from falling out in clumps as a result of chemotherapy. Her mother Jane shuttled between hospital and home to watch over her daughter. “Candy had beautiful hair fl owing to her chest. As her mother, I felt the pain too.”

At that time, Candy’s father Eric was working in Australia. The oil fi eld engineer returned to Singapore every two months but remained on tenterhooks when he was away. “My heart jumped each time the phone rang. I couldn’t concentrate at work and felt like a zombie. But I was comforted that the SGH staff took good care of my daughter. Some even came back into her ward late at night to keep her company.”

Candy’s friends made her cards fi lled with messages of encouragement and hope which decorated the walls of her hospital room. But when her friends came, it was the patient who ended up consoling the visitors. “I believe I will recover faster if I remain cheerful,” she declared proudly.

Double cord blood transplant

Candy needed a bone marrow transplant but her younger brother and seven-year-old cousin were not suitable matches. A search with bone marrow registries here and overseas also failed to fi nd a suitable donor. During her fi ve-month wait for a bone marrow transplant, Candy underwent fi ve courses of chemotherapy and took 50 tablets daily.

As her condition was deteriorating and there was no suitable bone marrow donor, Dr Mickey Koh decided Candy’s only chance was in a double cord blood transplant (see cover story). In July last year, he found two units of cord blood from a cord blood bank in the US. Only one unit matched Candy’s blood type of ‘A’ while the other was of blood group ‘O’.

Double cord blood transplant is not commonly practiced but for Candy, the novel procedure paid off. “The risks of contracting infections are high during the engraftment period but I thought if I can pass this ‘fi nal exam’, I can get on with life.”

The two units of cord blood were transplanted into her body through an intravenous drip. Candy remained in hospital over the next few weeks as her immune system was then very low and susceptible to infection. After about 3 to 4 weeks, the cord blood started to grow and engraft in her and her blood tests results improved. She lost 7 kg during this procedure.

From blood type ‘A’ to ‘O

But the suffering was not in vain as her body has fully accepted the ‘new’ blood. Candy now has type ‘O’ blood coursing through her veins, courtesy of one of the cord blood donors. Tests results showed all leukaemia cells have been eradicated. In April this year, she resumed her second-year Business Management studies at Nanyang Polytechnic. Eleven months after the transplant, Candy no longer needs to be on medication.

A year after her life-saving procedure, Candy has held a party to thank the medical and nursing staff of SGH Ward 72 where she spent two months battling leukaemia. Her experience is meticulously recorded in an online diary which can be accessed by the public. “I am proud to win the battle and want people to know that contracting leukaemia is not the end of the world.”

Besides blogging, she’s catching up on her studies and planning a long-awaited trip to Perth to visit her father. “I’ll be a very busy girl.”

And healthy, too.

Cord blood gives hope to adults (cont’d)


CANCER OF THE WHITE BLOOD CELLS Acute leukaemia is a type of blood cancer which results in the overproduction of abnormal white blood cells in the body. This suppresses the production of normal white and red blood cells and platelets. This results in anaemia or low red blood cells, increases one’s risks of infection and blood clotting problems.

Chances of recovery are now possible with intensive chemotherapy or transplantation.

Leukaemia can be chronic and acute, depending on its rate of progress and abnormal cell maturity. Specifi c causes are unknown for most of the time and it is not hereditary.

Treatment Options

Chemotherapy Intravenous or oral methods to kill leukaemia cells.

Bone marrow transplantChemotherapy and radiation therapy to destroy diseased cells before infusing healthy new ones from a suitable donor.

Peripheral blood stem cell transplant

Stem cells obtained from the patient’s circulating blood are returned to the bone marrow after intensive chemotherapy and radiotherapy.

Cord blood transplantUmbilical cord blood rich in stem cells are infused into the patient. Matching need not be as close as for the bone marrow or peripheral blood transplants. Less risk of contracting graft-versus-host disease.


The Cord Blood ControversyBy Jeannette Moninger from Parents Magazine

7 Smart Questions to Ask a Cord-Blood BankObviously, price and a compelling website aren’t the only factors to consider when choosing a bank. Be sure to do your homework before signing up.

1. Is the facility accredited by the American Association of Blood Banks? This means its labs, banks, and procedures meet set standards.

2. How long has the company been in business? At least fi ve years is best.

3. How many units does it store? Look for a bank that has thousands.

4. How many successful transplants have been performed with its stored samples? Having at least one demonstrates that its process works, but two or three is better.

5. Is the blood stored as a single unit or in several samples? Freezing in portions is preferred so the blood can be tested for potential transplant use without thawing – and wasting – the entire sample.

6. Is the company private or publicly held? If it’s a publicly traded company, you’ll have the advantage of reviewing its fi nancials. Many banks are part of larger biotech companies.

7. Is the storage fee fi xed or subject to increases? You want to know the total price tag up front.

Media : Parents Magazine

Date : April 2006


Couple conceives baby, so her umbilical cord blood can be used to treat dying son. Eighteen months later, he is well.

To give their gravely ill son a chance at life, a couple here conceived a second child to save him.

Little Rachel Fu’s* umbilical cord blood proved a lifeline to her brother, three-year-old Ray Fu*, who received an infusion of her stem cells after other treatment efforts had failed.

That was 18 months ago.

Now fi ve, Ray Fu* has started kindergarten and his leukaemia is in remission, said the biotech company CyGenics yesterday, which operates Cordlife, a private cord-blood bank here that processed and stored the blood.

A Cordlife spokesman said they announced the transplant only now, because they wanted to be sure the cancer was in remission.

Late in 2001, doctors had given Ray Fu* only a 10-per-cent chance of recovery, unless, he had a bone-marrow transplant. Neither of his parents were a match, and as an only child, Ray Fu* had a one in 20,000 chance of fi nding a non-related donor.

An appeal through The Straits Times in Dec 2001 saw hundreds of donors come forward, said Mr Fu*, 35, but none was suitable.

When the Fus* heard about cord blood had been used to treat various blood-related ailments, they made up their minds to have a second child to help Ray Fu*.

Cord blood is a rich source of what doctors call haematopoietic stem cells, “blank” immature cells that can become all sorts of blood cells: red, white or platelets.

These can then replace diseased cells in patients who suffer from leukaemia or severe anaemia, for example.

“We were elated when Rachel’s* blood was shown to be a 100-per-cent match for Ray Fu*,” said Dr Tan Ah Moy, head and senior consultant for haematology and oncology services


Date : 1 July 2004

at the KK Women’s and Children’s Hospital, where transplant was performed.

The perfect match meant the transplant would have a higher chance of success.

She said: “The healthy cells from his sister re-populated in his bone marrow and revived his immune system, depleted by the chemotherapy and radiotherapy used to treat the leukaemia.”

The fi rst successful cord blood stem-cell transplant was done in France in 1988, to help a boy with serious form of anaemia.

Here, the public Singapore Cord Blood Bank, where babies’ umbilical cord blood will be processed, frozen and stored, aims to set up its facilities and start freeze-storing cord blood by the last quarter of this year. It plans to build a registry of at least 10,000 samples, to help about 100 patients a year suffering from blood disorders.

Currently, over 2,500 people have stored umbilical cord blood in two private banks here, in case they or family members have need of it in the future. Only a handful has needed to use it so far for blood-related illnesses.

Donors can be charged several thousand dollars for extraction, tests and storage.

As for Ray Fu*, who has just started kindergarten, he is doing well now, said Mr Fu*, an engineer. His mother, Mrs Fu*, 34 is a part-time accounting and clerical worker.

Although the disease cost him his sight in one eye, and he has trouble concentrating, it is nothing compared to the dark days when his life hung in the balance, Mr Fu* said.

“W e’re still living day by day, and there’s no guarantee he won’t have a relapse. But our main aim is to make sure Ray Fu’s* happy, and nowadays we can more or less enjoy normal family life.”

Leukaemia boy saved – by baby sister

*Names have been changed to protect the privacy of the individuals involved.



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