Hope for blood cancer patients

Research into stems cells is yielding results that could change the treatment of certain cancers

By ADAM GONN
Gamida Cell studying 521 (photo credit: Courtesy Gamida Cell)
Gamida Cell studying 521
(photo credit: Courtesy Gamida Cell)
Close to half the patients suffering from illnesses such as leukemia and lymphoma are treated by undergoing bone marrow transplantations, in which damaged or dead marrow is replaced with bone marrow from a healthy individual, usually a family member.
Leukemia and other blood diseases are also often treated with chemotherapy, which destroys the cancer cells in the bone marrow.
However, the bone marrow is also needed for the production of both new red and white blood cells. But, in more than 50 percent of all cases, a fully matched donor isn’t found, and the patient’s survival chances drop drastically.
However, there is now hope that a new transplant method being developed by Israel-based bio-medical firm Gamida Cell (www.gamida-cell.com) could help those patients for whom donors can’t be found.
A Phase II/III study of the company’s StemEx product showed that the mortality rate for the test group that used StemEx was 15.8 percent and 24.5 percent in the control group. The study was conducted at 25 bone marrow transplant centers worldwide.
StemEx uses stem/progenitor cells that are extracted and expanded from a portion of a single unit of umbilical cord blood and transplanted in combination with non-expanded cells from the same unit. The advantage of using cord blood is that it has less matching requirements than bone marrow or peripheral blood transplants, providing the potential to increase the number of suitable transplant matches and to shorten the time it can take to find a match, according to Gamida Cell.
While stem cells are useful due to their ability to morph into other types of cells, there is only a relatively small quantity of them in the cord blood, usually only enough for treating small children. By using the technology behind StemEx, the amount of stem cells can be increased so there are enough stem/ progenitor cells to treat adolescents and adults as well. More specifically, the technology stimulates the production of a high count of CD34+ cells, which have been seen to reduce the risk of transplant-related mortality and increase the production of new blood cells.
Speaking to The Jerusalem Report, Dr. Yael Margolin, president and CEO of Gamida Cell says she had become involved with the company “to save lives.” A veteran of the pharmaceutical industry, having worked for pharmaceutical giant TEVA Pharmaceutical Industries, Margolin is also experienced in the world of venture capital, with several years at the Denali Fund under her belt.
Assuming all goes well with the regulatory process in the United States and Europe, she says the company hopes to have the product on the market in two years. Gamida Cell is also looking to market StemEx in India and China.
The product’s market potential is estimated at more than $1 billion.