3. 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
4. Migs’ Story
Hospital in China
Locoregional and systemic chemotherapy
(Gemcitabine/Cisplatin)
Immunotherapy
2 mos: pelvic recurrence
Cryosurgery
Brachytherapy
5. Mig’s story
Progressive and metastatic
disease
(R) flank pain
Came back to Philippines
Urologist: total excision of bladder and
rectum
6. Mig’s 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
WHAT IS A GENE?
HOW IS GENE MEDICINE MADE
HOW DOES IT WORK?
HOW IS IT USED?
OTHER CASES
16. 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.
17. P53 tumor suppressor gene
It also acts as
an
executioner,
programming
damaged cells
to self-destruct
before their
altered DNA
can be
replicated.
18. 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
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