2. Our goal for 2011 is to fund the following
3 research projects with Memorial-Sloan
Kettering Cancer Research Center
3. New Approaches to childhood leukemia
Childhood leukemia (acute lymphoblastic leukemia, ALL) is the most common cancer
affecting children.
Currently, very few options exist for children with relapsed acute lymphoblastic leukemia
Strategy : Genetically modify the human immune system to fight leukemia.
T cells (human immune cells) can be re-directed in the lab to target leukemia cancer cells
Cells can be made from patient’s own blood OR from a stem cell donor
Cells are infused into the patient and eradicate the leukemia
Project 1: Children with leukemia who have failed chemotherapy
Project 2: Children with leukemia who have failed a bone marrow transplant
4. Leukemia Project 1
Project Name:
A phase 1 trial of pediatric B cell ALL treated with autologous T cells genetically
targeted to the B cell specific antigen CD 19
Objective:
1. Evaluate the ability genetically modified T cells to eradicate leukemia in children
2. Define the overall survival in children with leukemia treat with genetically modified T cells
Significance:
Chemotherapy has toxic side effects for children and is ineffective in curing all children with leukemia.
These re-directed T cells will provide more options for children with leukemia with side effects.
Timeline:
Currently, at Memorial Sloan-Kettering Cancer Center there are 2 trials with adult leukemia
patients using this therapy. The technology has been established and extension into
children will require 6 more months of pre-clinical research and 6 months to obtain the
necessary Institutional and governmental approval.
Costs:
Preclinical testing will cost $150,000 to complete. To complete a clinical trial with 12
children will require $480,000 at a cost of $40,000/child.
5. Leukemia Project 2
Project Name:
A phase 1 trial of pediatric B cell ALL patients who have relapsed after bone marrow transplant treated
with donor derived T cells genetically targeted to the B cell specific antigen CD 19
Objective:
1. Evaluate the ability genetically modified T cells to eradicate leukemia in children
2. Define the overall survival in children with leukemia treat with genetically modified T cells
Significance:
Relapse leukemia following a bone marrow transplant is very difficult to treat and side effects
from traditional chemotherapy are too toxic. These children require novel approaches, such as
this trial provides, to cure their leukemia.
Timeline:
We have already obtained initial institutional approval and are awaiting governmental approval.
The trial should be opened for accrual in the next 6 months.
Costs:
Preclinical testing is already underway. To complete a clinical trial with 12 children
will require $480,000 at a cost of $40,000/child
6. Leukemia Project 3
A phase I study of convection-enhanced delivery of 124-I-8H9 for patients with non-progressive diffuse
pontine gliomas previously treated with external beam radiation therapy Ira Dunkel & Mark Souweidane,
MSKCC pediatrics & neurosurgery
Goal and objectives:
Diffuse pontine tumors are invariably fatal malignant pediatric brain tumors. The only known effective
therapy is radiation therapy, and while that often helps temporarily, these tumors recur about 6 months
later and the children die shortly afterwards.
How will this help?:
We hope & believe (based on animal experiments) that directly infusing this radioactive antibody into the
tumors will prove to be a safe & promising new treatment.
Time line:
Already approved by the MSKCC departments of pediatrics & neurosurgery and the Research Council, the
protocol is about to be reviewed by the MSKCC IRB and will then need to be reviewed by the FDA. Once
open, the trial will take about 2 years to complete.
Additional funding required:
$486,000. We have already obtained commitments for about $200,000.
