1. Gene Therapy
for the
Treatment of Cancer

2. Mig’s story

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

8. WHAT
IS
A
GENE ?

9. ORGAN
ORGAN SYSTEMS
HUMAN BODY
SEGMENT
CELLS OF COLON
TISSUE

10. THE
CELL

11. CELL
NUCLEUS

12. CHROMOSOMES
23 pairs or 46
chromosomes in the
nucleus of each cell
in our body

13. CHROMOSOME # 3
•ONCOGENES
•TUMOR
SUPPRESSOR
GENES

15. P53 TUMOR SUPPRESSOR GENE
chromosome 17
(17p13.1)

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

19. HOW IS
GENE THERAPY MEDICINE
MADE?

20. TWO COMPONENTS
ADENOVIRUS
p53 gene

21. Genetic Engineering Adenovirus
P53 Gene
ns ert
I
bad gene Remove
rAd-p53
RECOMBINANT HUMAN ADENOVIRUS p53 INJECTION MEDICINE

22. HOW DOES
GENE THERAPY
WORK?

23.  The adenovirus
containing the
p53 tumor
suppressor gene
binds to the
receptor in the
cell membrane of
the cancer cell

24.  The
adenovirus is
then
packaged in a
vesicle
 The vesicle
proceeds to
the cell
nucleus

25.  The vesicle
breaks down,
releasing the
adenovirus
near the cell
nucleus
 The
adenovirus
injects its
gene, which
contain the
p53

26.  The
adenovirus
injects its
gene, which
now includes
the p53
tumor
suppressor
gene, into the
cell nucleus

27.  The cancer
cell then
makes p53
protein

28.  The p53
protein
causes the
cancer cell to
self destruct
without
affecting
surrounding
normal cells

29. HOW IS
GENE THERAPY
ADMINISTERED?

30. Direct injection to tumor

31. Injection into pleural and peritoneal cavities

32. Intravenous infusion

33. Intra arterial infusion

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

35. OTHER PATIENTS

36. Liver Cancer
Male, 60 y.o.
•治疗前 before treatment 介入 + 基因治疗后 after interventional therapy+gene therapy

37. Advanced Lung Cancer
Male, 62 y.o.
Before treatment after chemotherapy+gene therapy

38. Lung cancer
Male, 55 y.o.

39. Cancer of the lower lip
Male, 67 y.o..

40. GENE THERAPY

41. GENE THERAPY

42. Landline: 63-2-9618035
Mobile: +639175536300
Email: surg.onc1@tullamarine.ph