New treatment for deadly blood cancers expected to be approved soon – STLtoday.com

Cancer doctors in St. Louis are ready to use a new therapy using a patients own blood to fight their disease.

The therapy, called CAR-T, for chimeric antigen receptor T-cell, involves removing immune cells from the blood, reprogramming them genetically to find and destroy cancer cells and then returning the immune cells to the patient. So far, the therapy has been tested on patients with hard-to-treat advanced blood cancers such as leukemia, lymphoma and myeloma that kill more than 58,000 Americans a year.

In one small study sponsored by Novartis Pharmaceuticals, 52 of 63 pediatric and young adult patients with relapsed acute lymphoblastic leukemia went into remission after undergoing CAR-T therapy. The 11 other patients died, seven from the cancer and four from side effects of the treatment.

Acute lymphoblastic leukemia, the most common childhood cancer, can be effectively treated with chemotherapy, but survival rates drop below 30 percent if the patient relapses. Candidates for CAR-T therapy include an estimated 600 children each year who relapse or do not respond to traditional chemotherapy.

At least 16 of the 20 people who have received CAR-T therapy for leukemia or lymphoma through clinical trials at Washington Universitys Siteman Cancer Center have seen their cancers disappear after treatment.

Ive never seen anything in cancer history with that kind of response, said Dr. Armin Ghobadi, an assistant professor in oncology at Washington University. These are the basically bad, incurable, deadly, unstoppable cancers and patients usually die quickly when we dont give them this treatment.

If approved as expected by the Food and Drug Administration, CAR-T therapy could be available locally within a year. Currently no patients at St. Louis Childrens Hospital qualify for the therapy, but patients are expected to come from neighboring states, said Dr. Robert Hayashi, director of hematology/oncology at the hospital.

This advancement is significant and has already demonstrated that it can be an effective form of therapy, Hayashi said. The ability of being able to show a clear success opens the door in terms of what other cancers can benefit from this exact same strategy.

So far the therapy has shown the most effectiveness in cancers of the blood. Another small trial in China involved 33 out of 35 patients experiencing remission from relapsing multiple myeloma, a plasma cancer, after receiving CAR-T therapy.

For decades, scientists have tried to corral the bodys immune system to fight cancer the way it attacks harmful bacteria or viruses. The immune system has a harder time recognizing cancer cells, allowing them to grow. Re-engineering immune cells to fight cancer cells is like turning on the cars headlights at night, Ghobadi said.

A main challenge with CAR-T therapy is the length of time it can take to reprogram the patients blood cells up to three weeks. Researchers are studying ways to reduce the time frame, including engineering universal CAR-T cells derived from donor blood or umbilical cord blood.

CAR-T therapy is expected to cost up to $500,000 for a one-time treatment. Scientists at Washington University are working to engineer the cells in-house, which could lower the price.

The side effects of the treatment can be severe as the immune system is amplified to fight cancer. A complication called cytokine release syndrome can cause life-threatening reactions including brain swelling. In early studies, one-third to one-half of patients treated with CAR-T therapy developed the syndrome. Because patients will need to be closely monitored, drug companies will limit the treatments availability to a few dozen cancer centers nationwide, including Siteman.

Marie Miceli, 64, was one of the first to be treated with CAR-T cell therapy in a trial at Siteman after several rounds of chemotherapy and a stem cell replacement failed to knock out non-Hodgkin lymphoma.

A year later, Miceli is in remission and just celebrated the birth of her fourth grandchild. Miceli, a real estate agent and branch manager at Berkshire Hathaway in St. Louis, said she was blessed to receive the experimental treatment.

You have to trust those doctors and have faith, she said.

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New treatment for deadly blood cancers expected to be approved soon - STLtoday.com