By using induced pluripotent stem (iPS) cells as a discovery tool, Fate Therapeutics, a La Jolla, California company, is trying to develop drugs that would activate resident stem cells in the body for specific clinical purposes. This proprietary Stem Cell Modulators (SCMs) technology is already showing promise, as the company currently conducts a Phase 1b clinical trial of FT1050, “a small molecule SCM designed to increase hematopoietic stem cell number and function in dual umbilical cord blood transplant recipients with hematologic malignancies.”
The MIT Technology Review reports:
Fate’s first clinical trial focuses on a molecule known as FT1050. The molecule appears to stimulate proliferation of hematopoietic stem cells–which give rise to blood and immune cells–and helps guide them to the bone marrow. If successful, the drug could become an invaluable companion treatment to bone-marrow transplants and cord-blood transfusions used to treat cancer and blood diseases.
In an early-stage clinical trial, Fate Therapeutics is testing FT1050 in 12 patients who’ve undergone chemotherapy for lymphoma. The patients will each receive two units of cord blood: one that’s been treated with the stem-cell-modulating drug, and another that’s been left alone. The trial is primarily a safety study, but because the two units were harvested from two different newborns, researchers can use the genetic differences to track the cells and determine if FT1050-treated stem cells can more efficiently take hold and prosper in bone marrow.
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