FDA advisers say yes to experimental personalized cancer therapy

FDA advisers say yes to experimental personalized cancer therapyā€¯

There are still concerns about side effects, but the FDA is expected to follow the panel's advice and approve the "living drug" therapy for people ages 3 to 25 whose bodies have resisted other treatments like chemo or who've relapsed after receiving chemo for the most common U.S. childhood cancer, B-cell acute lymphoblastic leukemia, reports NPR.

The committee voted unanimously (10-0) in favor of the therapy, which the FDA will certainly weigh in when making the final decision on approval in October.

A breakthrough cancer therapy that can wipe out leukemia in some deathly ill children and young adults comes with an expensive, long-lasting side effect.

On CTL019's safety, surprisingly the bulk of the discussion centered on two legacy gene therapy issues that have not been seen in CAR-T: insertional mutagenesis and replication-competent retrovirus.

The gene therapy, also known as CTL019, uses a new technology called chimeric antigen receptor T-cell therapy, or CAR-T for short.

The FDA is not required to follow the panel's recommendation but often does.

Yesterday, the FDA's Oncologic Drugs Advisory Committee (ODAC) held a hearing to evaluate Novartis CTL019.

Samit Hirawat, Novartis' head of oncology global development, explained the process by which the treatment is created with patients' own cells that are transferred from the patient, moved to a dedicated manufacturing facility in Morris Plains, New Jersey where the cells undergo enrichment and are then cryopreserved and returned to the clinical site where patients are infused. The immune cells from the patient's blood are being reprogrammed to focus on cancer cells and completely destroy them without doing any damage to the healthy cells that are around it.

In a unanimous vote on 12 July, the panel determined that the benefits of the treatment, called CAR-T therapy, outweigh its risks.

Observers crowded into the FDA's presentation room this morning, rubbing shoulders with CAR-T celebs like Penn's Carl June with many, many more looking on online.

Brody said personalized immunotherapy treatments like this one require that patients use their own immune cells because they would "almost never (find) a match" in an off-the-shelf product.

"When other organizations, including the (National Institutes of Health), considered gene therapy too risky, ACGT believed in the science and funded us when no one else would".

Alan Rein from the Center for Cancer Research cited the drug's "remarkable clinical successes", though he noted some lingering concerns with the long-term risks involved.

While many patients can be cured with existing treatments, around 15%-20% see their disease return, and most of these relapsed patients do not survive.

The treatment is also complex and can lead to serious complications, so Novartis plans to initially limit the use of CTL019 to just 30 or 35 approved centers in the United States. They then multiply and gradually replace the cell not treated. A total of 82.5% of the patients went into remission, which is a high rate for the disease.

Novartis officials acknowledged the adverse events associated with auto T-cell therapy in general, including the potential for serious adverse events. It's risky and can come with severe side effects.

Immunotherapy is a growing field in cancer treatment and medications that alter the body's immune system to fight cancer are already on the market.

Novartis disbanded its cell and gene therapy group past year but vowed to press on with CTL-019.

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