Medicine

FDA Votes In Favor Of Gene Therapy To Treat Childhood Leukemia

FDA Votes In Favor Of Gene Therapy To Treat Childhood Leukemia

If the FDA agrees with the panel's recommendation and moves to approve the treatment for commercial use, it would be the first such gene-altering treatment to make it to market. Others are expected: Researchers and drug companies have been engaged in intense competition for decades to reach this milestone.

Novartis expects to get a decision from the FDA by October.

Approval of tisagenlecleucel would have significant implications not only for Novartis but for companies developing similar treatments, including Kite Pharma Inc, Juno Therapeutics Inc and bluebird bio Inc. After the cells are removed by medical personnel, they are frozen, shipped to Novartis (the therapy's maker), processed, refrozen, and shipped back to the medical center.

Despite unknowns surrounding the therapy, "it is hard to argue with the unprecedented clinical success we have seen in this population of patients who do not have other viable treatment options", added Grzegorz S. Nowakowski, MD, of the Mayo Clinic in Rochester, Minn.

This therapy is a one-time treatment for children and young adults affected by the most common form of childhood cancer, acute lymphoblastic leukemia or ALL. Since that time she has been cancer free.

Novartis conducted a study of 63 patients and treated them between April 2015 and August 2016. Of the 52 patients whose results were analyzed, 83 percent had complete remission, meaning their cancer vanished.

However, Connor underwent the new treatment at Duke University and has since recovered so well that he is back to playing hockey, his father said.

Indians will be able to access the treatment at USA centres, which will also accepting worldwide patients.

But 15% of the cases of LAL are resistant to treatment. For one thing, the manufacturing process is extremely intensive since each individual batch of the drug needs to be tailored to each individual patient.

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.

Another parent at the meeting, Don McMahon, described his son Connor's grueling 12 years with severe and relapsing leukemia, which started when he was 3. Those prior treatments involved multiple spinal taps and painful bone marrow testing, all of which was tough to tolerate and left his son infertile, McMahon explained.

The drug enables patients' own immune cells to recognize and kill the source of the cancer: a different immune cell gone awry. He has since returned to playing hockey.

The FDA is not obligated to follow the recommendations of its advisers, but it often does.

The Food and Drug Administration panel is holding a hearing Wednesday to discuss the treatment developed by the University of Pennsylvania and Novartis Corp. Use will not be widespread at first, because the disease is not common.

Patients who fail to respond to standard treatments have a low chance of survival - usually 10% to 20%. Although Novartis did not provide a price tag, analysts told the Times that individualized therapies could cost more than $300,000. "Today is about advancing an exciting technology that has the potential to transform cancer treatment".

In addition to understanding that process, the staffers wrote, Novartis will need to understand critical product quality attributes unique to autologous auto T-cell products-as well as to understand and address sources of variability seen in the products.


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