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SPECIFIC DEPLETION OF STEM CELLS FACILITATES ENGRAFTMENT

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R43CA096457-01
Agency Tracking Number: CA096457
Amount: $100,000.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: N/A
Solicitation Number: N/A
Timeline
Solicitation Year: N/A
Award Year: 2002
Award Start Date (Proposal Award Date): N/A
Award End Date (Contract End Date): N/A
Small Business Information
BIOTRANSPLANT, INC. BLDG 75, 3RD AVE, NAVY YARD
BOSTON, MA 02129
United States
DUNS: N/A
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 JULIAN DOWN
 (617) 241-5200
 JULIAN.DOWN@BIOTRANSPLANT.COM
Business Contact
 MARY SCHARF-WHITE
Phone: (617) 241-5200
Email: LISA.LINEHAN@BIOTRANSPLANT.COM
Research Institution
N/A
Abstract

DESCRIPTION (provided by applicant): Allogeneic stem cell transplantation has
broad implications as a therapeutic strategy for treating diseases such as
cancer, autoimmune disease, and genetic disorders as well as for induction of
specific immune tolerance for transplanted solid organs. A number of previous
experimental studies have shown that depletion of primitive stem cells is often
required in the host before long-term engraftment (chimerism) from donor stem
cells is achieved. In myeloablative protocols, this is accomplished with high
doses of irradiation or pharmacological agents such as busulfan. Many
non-myeloablative protocols currently being evaluated depend, at least in part,
on mature T cells present in the donor graft to provide this function.
Engraftment achieved in this manner, however, is often associated with
graft-versus-host disease (GVHD). Our goal in this Phase I proposal is to
demonstrate proof of concept that a monoclonal antibody specifically directed
against hematopoietic stem cells of the recipient can be substituted for toxic
agents such as busulfan, thus eliminating the requirement for donor T cells in
non-myeloablative transplant protocols and reducing the likelihood of GVHD.
Phase II will be proof of concept in a relevant primate model of allogeneic
stem cell transplantation.

* Information listed above is at the time of submission. *

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