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FDA panel endorses potential 1st United States gene therapy

13 Juillet 2017

Others are expected: Researchers and drug companies have been engaged in intense competition for decades to reach this milestone. That could create problems if demand surges because of Novartis's cancer treatment. After one year 79 percent of patients were still alive.

"The panel's unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need", Novartis Oncology CEO Bruno Strigini said in a news release.

Twelve-year-old Emily Whitehead is the success story.

Dr. Esquilin thinks it'll be a long time before this kind of treatment becomes a mainstream option. Her father said his family was more afraid of the side effects of the full-body radiation that's used ahead of a transplant than of the experimental immunotherapy. But she emerged cancer-free, and has remained so.

The FDA is expected to make a decision on approving Novartis' treatment within the next few months. The dramatic effect of the treatment, known for years as CTL-019, was never questioned at the meeting. She had relapsed twice with acute lymphoblastic leukemia. And he added that chemotherapy had left his son infertile.

Notably for CAR-T, Novartis saw no cases of cerebral edema in its clinical program for tisagenlecleucel. Connor underwent the cell treatment at Duke University, and he has since returned to playing hockey.

Even so, several of the committee members were unusually enthusiastic in explaining their 10-0 vote recommending approval.

Cripe was a member of the FDA advisory panel that voted Wednesday to support the drug's approval.

Use will not be widespread at first, because the disease is not common.

Almost 5,000 people were diagnosed with acute lymphoblastic leukemia in 2014, the most recent year on record, according to the US Centers for Disease Control and Prevention. ALL is the most common childhood cancer in the US. Although analysts predict that the cost of these unique treatments may exceed $300,000, a Novartis spokesman declined to specify a price when NYT inquired. Kite Pharma also has a CAR-T therapy in FDA review and Juno Therapeutics and others are in late stages of testing. "We are working on bringing CTL019 to other countries around the world".

Norvartis plans to seek approvals in the European Union in 2018. Novartis' therapy is one of two cutting-edge treatments for blood cancers are poised to get approved by the end of the year. But Novartis says trial clinicians were able to manage the reaction successfully in all cases.

Image of a CAR-T cell (reddish) attacking a leukemia cell (green).

CTL019 is a lentivirally transduced CAR-T B cell therapy in which a patients B cells are isolated by cryopreserved leukapheresis, and genetically engineered to express a chimeric T cell receptor containing the CD19 B cell antigen linked to a co-stimulatory domain including the CD3zeta and 4-1BB proteins. First, a process specific to each patient is conducted where millions of immune system T-cells are removed and frozen at an approved medical center.

"I'm happy most of all for the patients who will benefit from this therapy", said Dr. Carl June of the University of Pennsylvania Perelman School of Medicine, who developed the therapy. Once infused back into the patient, the cells reproduce at a fierce pace to attack the cancer. To generate a batch of tisagenlecleucel, white blood cells are purified from a sample of a patient's blood and shipped to a central processing centre. It can be deadly, with spiking fevers and other symptoms, if medical staff aren't properly trained for it. Novartis said it would train staffers at 30 to 35 medical centers and take other measures, as well. But the approach also is being tested for a range of diseases from non-Hodgkin lymphoma and multiple myeloma to solid tumors.

FDA panel endorses potential 1st United States gene therapy