Umbilical Cord Blood Transplant Study Begins for Aplastic Anemia and Hypoplastic MDS

Mathew Shanley

Gamida Cell announced today that an investigator-initiated study testing their candidate CordIn, to treat severe aplastic anemia (AA) or hypoplastic myelodysplastic syndrome (MDS) has begun, and the first patient was given the drug.

Severe AA is a condition in which the bone marrow does not produce enough blood cells for the body, and hypoplastic MDS is characterized by decreased marrow cellularity.

Cordin is a variety of stem cell transplantation that uses blood from an umbilical cord. It is a curative treatment in development for an array of rare diseases, including sickle cell disease, tahalssemia, and other bone marrow failure syndromes, such as aplastic anemia and hypoplastic MDS.

Cordin would be used in patients thought to benefit from stem cell transplantation, for whom no matching donor is available.

The study is being led by Dr Richard W Childs, Clinical Director of the National Heart, Lung, and Blood Institute’s (NDLBI) Division of Intramural Research in the National Institutes of Health (NIH) and Assistant United States Surgeon General.

"Severe aplastic anemia and myelodysplastic syndrome are life-threatening bone marrow disorders with few optimal treatment options. Many patients with these diseases fail conventional therapy. Amongst those with severe AA who respond to conventional treatment, up to 30% will suffer relapse or evolve to myelodysplastic syndrome or leukemia which is often fatal," said Dr Childs in a press release.

"Promising preclinical and clinical data have shown the efficacy of Gamida Cell's ex-vivo hematopoietic stem cell technology. Based upon exciting prior data, we are now conducting a clinical trial at the NHLBI testing whether umbilical cord blood transplantation using CordIn can be used to improve the results of conventional cord blood transplantation for patients with these life-threatening conditions who lack an available matched donor.”

This study, titled Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Imrpove Transplant Outcome, is a Phase ½ trial in which the primary endpoint is prompt and durable cord engraftment.

"The study, announced today, represents a significant milestone for Gamida Cell, as we continue our important mission of expanding access to curative transplantation in patients for which engraftment has been historically difficult," said Gamida Cell President and CEO Dr. Yael Margolin. "We highly value our collaboration with the NIH and with Dr. Childs, who is one of the world experts in the field, and we look forward to evaluating the potential of CordIn in patients with life-threatening hematologic diseases."

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