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Trial Begins Testing Enhanced Cord Blood Stem Cells

Surgery

(Sasin Tipchai, Pixabay)

28 February 2017. A clinical trial testing transplant grafts derived from umbilical cord blood as an alternative to bone marrow stem cells for people with blood-related cancers is treating its first patient. The trial is conducted by Gamida Cell Ltd., a biotechnology enterprise in Jerusalem, Israel.

Gamida Cell develops therapies for cancer and inherited diseases derived from engineered cells, including stem cells, with sources such as umbilical cord blood. One of the company’s technologies enhances umbilical cord blood with nicotinamide, a natural compound related to vitamin B3 used in some cases as a sensitizing agent for chemo- or radiation therapy that expands blood flow to treat cancerous tumors.

The company applies nicotinamide to its NiCord product designed as an alternative to bone marrow transplants for people with blood-related cancers such as leukemia. A leading treatment for these cancers is a bone marrow transplant that delivers healthy stem and progenitor cells to the patient. But bone marrow transplants require a full genetic match that often makes it difficult to find a donor.

NiCord is derived from umbilical cord blood, which in its natural state has some stem and progenitor cells, but not enough for blood-related cancer treatments. Gamida Cell enhances umbilical cord blood with nicotinamide to expand the quantity of stem and progenitor cells, as well as improve their functionality and grafting ability to bone marrow, then added to cord blood mixed with bone marrow and lymphoid cells. NiCord treatments are produced on demand in Gamida’s manufacturing facilities then shipped frozen to the patient’s bedside, a process the company says takes 3 weeks.

The late-stage clinical trial is enrolling 120 individuals age 16 to 60 with various blood-related cancers at 9 sites in the U.S. and Europe. Participants will be randomly assigned to receive NiCord or regular umbilical cord blood treatments for comparison. The main outcome indicator of the study is time needed for treatments to graft to neutrophils, the most abundant type of white blood cell, up to 42 days following the transplants.

Gamida Cell conducted early and intermediate-stage trials of NiCord treatments, with outcomes the company hopes to confirm in the new trial. NiCord transplants are recognized by the Food and Drug Administration and European Medicines Agency as orphan treatments for rare diseases. FDA also designated NiCord transplants as a breakthrough therapy in October 2016, signifying a treatment for serious or life-threatening condition, where early clinical evidence indicates a substantial improvement over available therapies. A breakthrough designation gives the product more intensive attention from FDA and an accelerated review schedule.

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