• By Dana Sparks

‘A living drug’ to fight leukemia

July 13, 2017

A leukemia treatment that uses a patient's own cells to boost immunity and fight cancer has been forwarded for approval by a U.S. Food and Drug Administration (FDA) panel of experts. The unanimous recommendation for the treatment was made this week, and the FDA is expected to make its approval decision in a few months.

Dr. Yi Lin, a hematologist with the Mayo Clinic Cancer Center, says this is a groundbreaking new way of treating children and young adults with B-cell acute lymphoblastic leukemia. "Each dose is an individualized treatment made by genetically changing the patient’s own T-cells to strengthen those cells’ ability to kill leukemia cells," says Dr. Lin. "This is completely different from the other off-the-shelf drugs that are currently available to treat this disease. The response rate of this treatment seen to date is impressive, compared to other available drugs."

Dr. Lin says the treatment comes with some potentially serious side effects in the first month after infusion that are unique to immunotherapy and different from the side  effects of chemotherapy or stem cell transplant. She emphasizes that specialized expertise from a trained medical team to monitor and manage these side effects, is necessary to safely support the patients through the treatment.

The FDA requires patients who receive gene therapy be monitored for 15 years. "As this treatment begins to enter into clinical practice, it will need to start with academic centers of excellence with experience and training in these treatments," says Dr. Lin. "We will learn more about the logistics of implementing this treatment into clinical practice."


 "This is a remarkable feat in making this very complicated technology accessible to more patients. FDA approval of this first treatment of its kind will give recognition and tribute to an example of successful partnership between academia and industry to bring meaningful treatment to patients." - Dr. Yi Lin


More research is needed to understand the late effect of the treatment and to examine whether there's a role in using the treatment earlier in the disease. "We also need to improve the treatment to reduce the side-effects, but this approval is only the beginning of a new era of cellular immunotherapy in cancer," says Dr. Lin.

Researchers have worked in multiple academic centers for the past three decades to develop this genetic technology. Dr. Lin says it's good to see industry companies joining forces to expedite moving this technology into multi center clinical trial testing. She says, "This is a remarkable feat in making this very complicated technology accessible to more patients. FDA approval of this first treatment of its kind will be recognition and tribute to an example of successful partnership between academia and industry to bring meaningful treatment to patients."

The next cancer type to be reviewed by the FDA for chimeric antigen receptor T-cell (CART) therapy treatment will be non-Hodgkin lymphoma. Dr. Lin says FDA review for that research is anticipated later 2017. She also says Mayo Clinic has an active trial portfolio with trials in lymphomamyeloma, and acute lymphoblastic leukemia. Dr. Lin adds, "We are also preparing for CART treatment in clinical practice when this becomes FDA-approved."

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