Stem Cell Graft Engineering to Treat Sickle Cell Disease

Award Information
Agency:
Department of Health and Human Services
Branch
n/a
Amount:
$220,500.00
Award Year:
2004
Program:
STTR
Phase:
Phase I
Contract:
1R41HL074579-01A1
Award Id:
70890
Agency Tracking Number:
HL074579
Solicitation Year:
n/a
Solicitation Topic Code:
n/a
Solicitation Number:
n/a
Small Business Information
REGENEREX, LLC, 201 E JEFFERSON ST, LOUISVILLE, KY, 40202
Hubzone Owned:
N
Minority Owned:
N
Woman Owned:
N
Duns:
n/a
Principal Investigator:
SUZANNE ILDSTAD
(502) 852-2080
SUZANNE.ILDSTAD@LOUISVILLE.EDU
Business Contact:
SUZANNE ILDSTAD
(502) 292-1740
REGENEREX1@AOL.COM
Research Institute:
UNIVERSITY OF LOUISVILLE

OFFICE OF GRANTS MANAGEMENT
KENTUCKY, KY, 40292

Nonprofit college or university
Abstract
DESCRIPTION (provided by applicant): Sickle Cell Disease (SCD) is an abnormality of red blood cells (RBC) that affects approximately 72,000 people in the United States, or approximately 1 in every 500 African-Americans. With current therapies, the mortality in the United States is 50 percent by age 40. SCD is a chronic, debilitating disease associated with frequent painful crises, acute chest syndrome, stroke, and renal failure. As a result, patients require frequent hospitalizations and experience absenteeism from work. The only cure for SCD to date is bone marrow transplantation (BMT). However, at present the morbidity and mortality associated with ablative BMT has limited the widespread application of this approach. Moreover, the requirement for a perfectly matched donor has limited BMT to only 17 percent of candidates. We have developed an approach to engineer a bone marrow graft to avoid the major complications of BMT: graft-versus-host reactivity (GVHD); graft failure; and the need for perfect matching. Our approach is superior to current therapies because it involves a proprietary process that enriches for patented facilitating cells (FC) and stem cells but removes GVHD-producing cells. In this proposal we will utilize this FCRx product, already demonstrated to be effective for treatment of leukemic patients, for treatment of patients with SCD. In phase I, we will develop a nonmyeloablative conditioning approach to establish 20 percent donor chimerism. The well defined end point will be engraftment and production of normal RBC. In the long term we will develop and market a quality controlled/quality assured FDA-approved bone marrow product (SCD FCRx) to treat individuals with SCD. Our platform technology will be provided to hospital systems worldwide as an FDA-approved bioservice. Such a treatment approach could be expanded to other blood disorders, including thalassemia. The complimentary structure and expertise of the company and the University lends itself nicely to this joint effort. The company will develop a strategy to produce and market the product while the University will conduct the clinical trial and process the marrow in its FACT-accredited cell processing facility.

* information listed above is at the time of submission.

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