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While Longer-Term Results Await, T-Cell Cancer Therapy Holds Promise

While Longer-Term Results Await, T-Cell Cancer Therapy Holds Promise
Doctors have raised hopes of saving the lives of more children with the most common type of childhood cancer after a novel cell treatment that saved the life of 9-year-old Austin Schuetz was given the green light by U.S. regulatory advisers.
Approval of Novartis AG's tisagenlecleucel for treating B-cell acute lymphoblastic leukemia (ALL) in children and young adults who relapsed or failed chemotherapy was unanimously voted for approval by an advisory committee to the U.S. Food and Drug Administration. While the recommendations of its advisers are not binding on the FDA, it typically does so.
After leukemia cells were detected in his brain just two months after a bone marrow transplant from an anonymous donor, Austin Schuetz, who lives in Wisconsin, was treated in 2013 with the Novartis therapy as part of a clinical trial.
"We knew it was risky ... but it was the only option for us," said Kim Schuetz, Austin's mother. Austin today shows no signs of cancer, even though Austin, then age 6, suffered fever and severe headaches after the engineered T-cells were administered.
The cell DNA is altered to sharpen their ability to spot and kill cancer cells, and infusing them back into the patient by a complicated process that extracts immune system T-cells from an individual patient and this is the process of functioning of the new drug class, known as chimeric antigen receptor T-cell or CAR-T.
"The majority of calls we are getting are from patients looking for immunotherapies - particularly CAR-T therapies," said Dr. Gwen Nichols, chief medical officer at the Leukemia and Lymphoma Society.
In the first wave of immunotherapy drugs that are revolutionizing cancer treatment, Novartis has lagged some other big drugmakers. Through the sale of drugs known as "checkpoint inhibitors" that fight cancer by removing a brake on the body's immune system, companies including Merck & Co Inc and Bristol-Myers Squibb Co are logging billions of dollars in annually.
"The word is out there that this is different from traditional chemotherapy," Dr. Nichols said.
While intense chemotherapy or bone marrow transplant are able to cure between 80 percent and 90 percent of children with ALL, there are very few options for the rest of the patients. "If your child is in that 10 to 20 percent, you are desperate," Dr. Nichols said, while cautioning that it is too early to tell whether CAR-Ts offer lasting remissions for patients.
A year after 79 percent of patients given the Novartis therapy, were alive during a clinical trial. There is only a 16 percent to 30 percent chance of survival for patients with ALL who fail chemotherapy typically.
Risks including a potentially life-threatening inflammatory condition are posed by CAR-Ts.
Biweekly infusions of immunoglobulin to replace B-cells wiped out by the engineered cancer-killing T-cells are required by Austin Schuetz, one of a handful of CAR-T patients to achieve multi-year remission of his cancer. His body would not be able to fight off common infections without B-cells.
Costing up to $500,000 and generating billions of dollars for their developers would be possible if CAR-T therapies is approved by the FDA.
Health insurers have placed tighter restrictions on patient access as the cost of cancer drugs increases.
"While CAR-T is a promising new type of immunotherapy, it is not commercially available and we have yet to complete our evaluation," T.J. Crawford, a spokesman for health insurer Aetna Inc said in a statement.
According to the American Cancer Society, every year about 6,000 people are diagnosed with ALL in the United States and about 60 percent are children.

Christopher J. Mitchell

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