CHICAGO (Reuters) - A small trial conducted in China found that an experimental therapy using altered cells to recruit the body's immune system to attack cancer can induce remission in most patients with advanced multiple myeloma, a blood plasma cancer.
The study of 35 patients tested a chimeric antigen receptor T-cell (CAR-T) therapy developed by China's Nanjing Legend Biotech Co.
The drug candidate, known as LCAR-B38M, targets a protein called BCMA found on cancerous blood plasma cells - the same target being pursued by Bluebird Bio Inc and Celgene Corp with their CAR-T called bb2121.
CAR-T therapies require a complicated process of extracting immune system T cells from an individual patient, altering their DNA to sharpen their ability to spot and kill cancer cells, and infusing them back into the same patient.
The American Society of Clinical Oncology, which featured the data here at its annual meeting, said that out of 19 trial patients followed for more than four months, 14 reached complete remission. One patient had a partial response and four patients reached "very good partial remission," but the cancer did get worse in one of those patients.
Multiple myeloma "is a disease you can treat pretty well with other drugs, but this could be long-term remission," said Dr. Bruce Johnson, chief clinical research officer at Boston's Dana-Farber Cancer Institute and ASCO's incoming president.
Eighty five percent of trial patients experienced cytokine release syndrome (CRS), a potentially life-threatening inflammatory condition, but researchers said the side effect was temporary and manageable in most patients. Two people had severe CRS, but recovered after treatment with Actemra, an anti-inflammatory drug.
The study is being conducted at Second Affiliated Hospital of Xi'an Jiaotong University in Xi'an, China.
The largest investor in Nanjing Legend Biotech is Genscript Biotech, a multinational provider of contract research services to pharmaceutical companies and others, according to Dr. Frank Fan, chief scientific officer at Nanjing Legend.
He said the company plans to enroll a total of 100 patients in the Chinese trial and to start a similar trial in the United States in early 2018.
"At ASCO I will hope to find collaborators in the U.S.," Fan told Reuters. "We are open for collaboration at different levels."
So far two companies have filed for U.S. approval of CAR-T drugs targeting a different protein called CD19. Kite Pharma Inc expects the Food and Drug Administration to decide by Nov. 29 whether to approve axicabtagene ciloleucel for advanced non-Hodgkin lymphoma, a cancer that starts in white blood cells.
The FDA is also reviewing Novartis AG's tisagenlecleucel-T for pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia.
Reporting by Deena Beasley; Editing by Phil Berlowitz