FDA approval for Gene Therapy in the horizon

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FDA Panel unanimously recommends Novartis CAR-T cell therapy in a big win for gene therapy.

Overview of a general CAR T-Cell Therapy . Image courtesy: Novartis Pharmaceuticals Corporation -http://bit.ly/1QVlQ02

On Wednesday as most of us went about our lives, Emily Whitehead, 12 and her family sought to move one step closer to history, a second time. After all, she was one of the first children to be treated with CAR-T cells. 

Emily is one of the first children to be treated with genetically modified immune cells which target cancer cells. She has been in remission from acute lymphoblastic leukemia for five years.

On Wednesday, the Whitehead family appeared before a panel of FDA advisors to advocate for this approach to combat cancer. First developed by Carl June at the University of Pennsylvania and licensed to Novartis, CAR-T cells offer hope to many patients for whom conventional therapy has been ineffective.

The latest approval recommendation comes from an FDA advisory panel for Tisagenlecleucel from Novartis. Under this process, cells extracted from the patient’s blood will be genetically engineered to attack cancer cells which give rise to lymphoma and leukemia.

Cancer cells typically overexpress a protein called CD19 on their surfaces which act as an effective bait for CAR-T cells to target and eliminate. The genetically modified cells are infused and multiply within the patient and set out to hunt. The entire process, termed as “Vein to vein” takes a mere 22 days. However, questions about the costs and potential insurance coverage still remain unanswered. Experts predict that the personalized treatment could cost about $300,000.

For many cancer patients, conventional therapy is a prolonged financial and emotional toll that often leads to very poor results. In contrast, CAR-T cell therapy offers a chance of complete remission and has less toxic side effects compared to conventional chemotherapy which comprises of numerous diagnostic test, spinal taps, bone marrow tests and drugs with high toxicity and side effects including infertility.

The FDA panel was convinced about the efficacy about the effectiveness of the therapy despite concerns regarding the “cytokine release fever” that patients experience upon infusion of CAR-T cells. In particular, an impactful Novartis trial in which 83 percent of patients went into remission has gone a long way in expediting the approval process, which sources predict will be complete by September. Despite concerns regarding the short term toxicities including neurological symptoms seen in some patients, the panel unanimously recommended an approval giving patients all over the world a fighting chance at remission.

The panel recommended approving CAR-T cell therapy for patients with B-cell acute lymphoblastic leukemia aged 3 to 25 years of age.

Despite all the excitement that this potential approval holds, the FDA panel expressed concerns regarding quality and long term effects of the treatment as Novartis scales up the operation. To ensure high quality, Novartis will limit the therapy to 35 medical centers employing personnel with extensive training. In addition, Novartis employees will track patients up to 15 years.

“We believe that when this treatment is approved it will save thousands of children’s lives around the world,” Emily’s father, Tom Whitehead, told the panel.

“I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.”