South Florida Bone Marrow Stem Cell Transplant Institute (SFBMSCTI), has received IND (Investigational New Drug) approval from the FDA and WIRB (Western Institutional Review Board) approval to conduct a Phase I/II clinical trial using granulocytes for treatment of solid tumors in cancer patients. The clinical trial designation is 08001-BMSCTI.
I. BACKGROUND:In its simplest terms the protocol involves recruiting donors with a matching ABO/Rh (blood type) to the cancer patient and a significant HLA (tissue typing) mismatch, screening them on the basis of a predetermined set of study criteria, collecting their granulocytes from their peripheral blood, and transfusion of the fresh product to the matching cancer patient. On average, it requires 4 to 6 qualified donors to yield sufficient numbers of granulocytes to treat one cancer patient. Again on average, it takes 25 willing donors to yield those 4 to 6 qualified donors.
II. NEED FOR FUNDINGThe FDA and WIRB approvals to conduct this clinical trial are based on a sponsor providing the funding for this treatment. The total budget is currently estimated to be $10 million dollars to complete the study.
SFBMSCTI has the experience, expertise, ability and treatment setting to effectively undertake such a clinical trial. However SFBMSCTI does not have the financial resources to do so.
This proposal examines the budgetary costs involved to undertake and complete this study in 36 months. The mechanism for funding of this study is based on Stem Cell Cancer and Regenerative Medicine Research (SCCRMR), a 501(C) (3), a non -for profit foundation, raising funds in sufficient amounts to fully sponsor the study at SFBMSCTI.
III. DONOR RECRUITMENT COSTS:
To reach the yield of matching donors for 29 patients, it is projected that approximately 450 willing donors have to be screened. To attract a pool of 450 donors, a deliberate advertising, canvassing and screening effort has to be conducted in the local community throughout the 36-month study period. Once a donor is matched to a patient who is ready for treatment, the donors return for Visits 2, 3, and 4.
Five to six donors (on average) are required per patient. The donors go through a complete history and physical examination, infectious disease testing as well as routine blood tests to confirm their current health status and eligibility.
The granulocyte product costs involves the physical examination of the donor and dosing with G-CSF and oral dexamethasone. This is done on the day prior to collection (Day -1)
IV. GRANULOCYTE PRODUCT COSTS
On the day of collection (Day 0) another nursing and physician assessment is made prior to the apheresis of the granulocytes from the donor’s peripheral blood. The apheresis procedure is a short run lasting about 3 to 4 hours. In addition to the kits and materials needed, medications for cell separation and to prevent clotting (hetastarch and tricitrate) are used. Nursing coverage is continuous throughout the procedure. A technician remains in the room the entire time. An RN checks vitals and connections every 30 minutes. The physician checks in at least once an hour. This ensures the donor does not have any complications and that the product collected meets the highest standards.
At the end of the apheresis procedure a sample from the collection bag is taken for the lab for a CBC. In accordance with AABB standards, a repeat infectious disease panel is also taken from the donor.
V. PATIENT TREATMENT
The patient will be seen at the institute over a period of up to 14 days prior to the first infusion and up to 14 days after their last transfusion. These pre-treatment and post-treatment phases are based on the Institute’s protocol in dealing with cancer patients with high acuity levels as anticipated with the patients selected for the study. Because of the acuity of the cancer patients, pre-treatment visits every other day from Day –14 to Day 0 may be planned for patient evaluations and disease evaluation including blood tests and CT/MRI scans as well as biopsies in some cases.
Treatment from Day 1 up to Day 14 will be the granulocyte transfusions. It is planned that the transfusions will take place over a 2- week period, without any being on successive days. The timing will depend on the patient’s tolerance of the granulocyte product and the ability to schedule the corresponding donor on a successive basis.
Post treatment visits for patient evaluations are planned for every day during the treatment period, and will consist of full nursing and/or physician assessments for patient.
Follow-up visits will then occur on Days 30, 60, and 90 days after the patient’s final infusion.
VI. NEED FOR PUBLIC FUNDING
SFBMSCTI is conducting the clinical trials. Funding for the protocol is being collected through a not-for-profit foundation, the Stem Cell Cancer Regenerative Medicine Research (SCCRMR) Foundation. SCCRMR depends on charitable donations to continue funding the study. At this time, the Foundation does not have sufficient funds to continue treating patients.
For additional information please contact:
South Florida Bone Marrow/Stem Cell Transplant Institute
10301 Hagen Ranch Road; Suite 600
Boynton Beach, FL 33437
Ph: (561) 752-5522
Fax: (561) 752-5446