First Patient Treated in Clinical Trial for Hematopoietic Stem Cells

Fate Therapuetics, located outside of San Diego in La Jolla, California, announced today that the first patient has been treated in a clinical trial for hematopoietic stem cell support. The Phase 1b study is testing FT1050, a proprietary small molecule stem cell modulator (SCM) which is being administered in a dual cord blood transplant for hematologic malignancy. Among other properties, FT1050 has been designed to activate specific pathways that determine the "fate" (the direction of differentiation and ultimate cell type, i.e.) of a stem cell, the purpose of which in this particular context is to increase hematopoietic stem cell number and function in the treatment of hematologic malignancies such as leukemia and lymphoma in adult patients who have undergone nonmyeloablative conditioning. The study is being conducted at the Dana-Farber Cancer Institute in Boston, where FT1050 is being administered during the standard course of dual umbilical cord blood transplants.

As Dr. Paul Grayson, president and CEO of Fate Therapeutics, explains, "The mission of Fate Therapeutics is to develop small molecules and biologics that modulate adult stem cells within the body for regenerative medicine. As our first SCM clinical candidate, FT1050 represents the initial step in our approach – using a small molecule to treat cells ex vivo but creating an in vivo regenerative effect. With FT1050, we are trying to affect stem cell biology in the body, improving the reconstitution of a patient’s blood and immune system."

According to Dr. Corey Cutler, assistant professor of medicine at Harvard Medical School and the director of the clinical study at Dana-Farber, "For patients who need hematopoietic stem cell support, time is of the essence. However, many patients do not have a suitably matched donor, either from a sibling or from an unrelated volunteer in the worldwide registries. Because umbilical cord blood units are readily available from cord blood banks, and the matching criteria for cord blood are less stringent than with adult donors, the ability to increase cord blood use by enhancing its efficiency has the potential to help thousands of patients waiting for a match." Twelve patients are expected to be enrolled in the trial for FT1050, which has been demonstrated to mediate not only stem cell differentiation pathways but also the ability of stem cells to "home in" on, and target, the bone marrow, thereby repopulating the patient’s own blood and immune system.

Patients with hematologic malignancies such as leukeia and lymphoma who do not respond to conventional therapies are often treated with intensive radiation, chemotherapy and additional forms of high-dose immunotherapy which often destroy the patient’s healthy blood and immunological systems in addition to the cancer cells. Bone marrow restoration through hematopoietic reconstitution is therefore necessary for the survival of such patients. In this as well as other types of clinical settings, adult stem cells obtained from umbilical cord blood have a number of therapeutic and logistical advantages over bone marrow or peripheral blood transplants, including but not limited to ease of matching HLA (human leukocyte antigen) markers as well as "faster availability, lower incidence and severity of acute graft-versus-host disease, lower risk of transmitting infections by latent viruses, lack of donor attrition and no risk to the donor", among other advantages, as described in the official press release of Fate Therapeutics. Additionally, as described on the company’s website, when compared to bone marrow and peripheral blood transplants, "cord blood is less immunogenic, which means it is less likely to be rejected by the patient’s immune system", and "cord blood is more readily available, which reduces medical costs and procedures necessary with obtaining stem cells from bone marrow or a patient’s or donor’s blood." Additionally, "cord blood has been used in hematopoietic stem cell support since 1988", and its safety and efficacy are well documented in the medical literature.

As also stated in the press release, "FT1050 was discovered by Leonard Zon, M.D., director of the Stem Cell Program at Children’s Hospital Boston and a scientific founder of Fate Therapeutics and is part of an exclusive license granted to Fate by Children’s Hospital Boston and Massachusetts General Hospital."

(Please see the related news article on this website, entitled, "Children’s Hospital of Boston and Fate Therapeutics Sign IP Deal", dated May 20, 2009).

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