Cancer study sees 100% response from gene therapy

CHICAGO— Doctors are reporting unprecedented success from a new cell and gene therapy for multiple myeloma, a blood cancer that’s on the rise. Although it’s early and the study is small — 35 people — every patient responded and all but two were in some level of remission within two months.

In a second study of nearly two dozen patients, everyone above a certain dose responded.

Experts at an American Society of Clinical Oncology conference in Chicago, where the results were announced Monday, say it’s a first for multiple myeloma and rare for any cancer treatment to have such success. Chemotherapy helps 10 to 30 per cent of patients; immune system drugs, 35 to 40 per cent at best, and some gene-targeting drugs, 70 to 80 per cent, “but you don’t get to 100,” said Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society.

Multiple myeloma affects plasma cells, which make antibodies to fight infection. More than 30,000 cases occur each year in the United States, and more than 115,000 worldwide. It’s the second-fastest-growing cancer for men and the third-fastest for women, rising 2 to 3 per cent per year, according to the National Cancer Institute. Nine new drugs have been approved for it since 2000, but they’re not cures; only about half of U.S. patients live five years after diagnosis.

The treatment, called CAR-T therapy, involves filtering a patient’s blood to remove immune system soldiers called T cells. These are altered in a lab to contain a gene that targets cancer and then given back to the patient intravenously.

Doctors call it a “living drug”— a onetime treatment to permanently alter cells that multiply in the body into an army to fight cancer. It’s shown promise against some leukemias and lymphomas, but this is a new type being tried for multiple myeloma.