gene therapy
TRANSCRIPT
Gene Therapy for the
Treatment of Cancer
Mig’s story
Migs’ Story
2009 11 years old, boy, Filipino Painless blood in urine Cystoscopy: bleeding mass in bladder Dx: Rhabdoid tumor of the urinary bladder Tx: Partial removal of urinary bladder
Migs’ Story
Hospital in China Locoregional and systemic chemotherapy
(Gemcitabine/Cisplatin) Immunotherapy
2 mos: pelvic recurrence Cryosurgery Brachytherapy
Mig’s story
Progressive and metastatic disease
(R) flank pain Came back to Philippines Urologist: total excision of bladder and
rectum
Mig’s story
Gendicine: IT 9IA 2IV 1
+ 6 Chemo (ICE)
Outcome: No tumor seen on 2nd look surgery PET CT : no active disease
OUTLINE
WHAT IS A GENE? HOW IS GENE MEDICINE MADE HOW DOES IT WORK? HOW IS IT USED? OTHER CASES
WHAT IS A GENE ?
SEGMENT OF COLON
ORGAN SYSTEMS
ORGAN
TISSUECELLS
HUMAN BODY
THECELL
CELLNUCLEUS
CHROMOSOMES
23 pairs or 46 chromosomes in the nucleus of each cell in our body
CHROMOSOME # 3
•ONCOGENES
•TUMOR SUPPRESSOR GENES
P53chromosome 17 (17p13.1)
TUMOR SUPPRESSOR GENE
P53 tumor suppressor gene when a cell's
DNA is damaged, it acts as an "emergency brake" to stop the resulting cell division that can lead to cancer.
P53 tumor suppressor gene It also acts as
an executioner, programming damaged cells to self-destruct before their altered DNA can be replicated.
P53 tumor suppressor gene
when it mutates, p53 can lose its suppressive powers or have the devastating effect of actually promoting abnormal cell growth
p53 is the most commonly mutated gene found in human tumors
HOW IS GENE THERAPY MEDICINE MADE?
ADENOVIRUS
TWO COMPONENTS
p53 gene
Genetic Engineering Adenovirus
rAd-p53
Remove
P53 Gene
bad gene
Insert
RECOMBINANT HUMAN ADENOVIRUS p53 INJECTION MEDICINE
HOW DOES GENE THERAPY WORK?
The adenovirus containing the p53 tumor suppressor gene binds to the receptor in the cell membrane of the cancer cell
The adenovirus is then packaged in a vesicle
The vesicle proceeds to the cell nucleus
The vesicle breaks down, releasing the adenovirus near the cell nucleus
The adenovirus injects its gene, which contain the p53
The adenovirus injects its gene, which now includes the p53 tumor suppressor gene, into the cell nucleus
The cancer cell then makes p53 protein
The p53 protein causes the cancer cell to self destruct without affecting surrounding normal cells
HOW IS GENE THERAPY ADMINISTERED?
Direct injection to tumor
Injection into pleural and peritoneal cavities
Intravenous infusion
Intra arterial infusion
P53 Gene Therapy is Safe
• First used since year 2003
• Over 16,000 patients already treated
• No serious side effects
• Common side effects are self-limited fever
OTHER PATIENTS
Liver CancerMale, 60 y.o.
•治疗前 before treatment 介入 + 基因治疗后 after interventional therapy+gene therapy
Advanced Lung CancerMale, 62 y.o.
Before treatment after chemotherapy+gene therapy
Lung cancerMale, 55 y.o.
Cancer of the lower lipMale, 67 y.o..
GENE THERAPY
GENE THERAPY
