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Gene therapy - a Powerpoint Presentation
1. Gene Therapy
for the
Treatment of Cancer
DAVID Y. DY, MD, MS, MBE, FPCS, FACS
Surgical Oncologist
AMY G. DY, MD, FPPS, FPSMO, FPSPO
Pediatric Oncologist
3. Migsâ Story
2009
ď§ 11 years old, boy, Filipino
ď§ Painless blood in urine
ď§ Cystoscopy: bleeding mass in bladder
ď§ Dx: Rhabdoid tumor of the urinary bladder
ď§ Tx: Partial removal of urinary bladder
4. Migsâ Story
ď§ Hospital in China
ďş Locoregional and systemic chemotherapy
(Gemcitabine/Cisplatin)
ďş Immunotherapy
ďş 2 mos: pelvic recurrence
ď Cryosurgery
ď Brachytherapy
5. Migâs story
ď§ Progressive and metastatic
disease
ď§ (R) flank pain
ď§ Came back to Philippines
ď§ Urologist: total excision of bladder and
rectum
6. Migâs story
Gendicine: IT 9
IA 2
IV 1
+ 6 Chemo (ICE)
Outcome: No tumor seen on 2nd look surgery
PET CT : no active disease
7. OUTLINE
ď§ WHAT IS A GENE?
ď§ HOW IS GENE MEDICINE MADE
ď§ HOW DOES IT WORK?
ď§ HOW IS IT USED?
ď§ OTHER CASES
16. P53 tumor suppressor gene
ď§ when a cell's
DNA is
damaged, it
acts as an
"emergency
brake" to stop
the resulting
cell division
that can lead
to cancer.
17. P53 tumor suppressor gene
ď§ It also acts as
an
executioner,
programming
damaged cells
to self-destruct
before their
altered DNA
can be
replicated.
18. P53 tumor suppressor gene
ď§ when it mutates, p53 can lose its
suppressive powers or have the
devastating effect of actually
promoting abnormal cell growth
ď§ p53 is the most commonly mutated
gene found in human tumors
34. 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