FDA panel endorses potential 1st United States gene therapy

Eye of Science  SPL	The benefits of CAR-T therapies make them attractive options for certain cancer patients

Eye of Science SPL The benefits of CAR-T therapies make them attractive options for certain cancer patients

If the F.D.A. accepts the recommendation, which is likely, the treatment will be the first gene therapy ever to reach the market. Ten committee members voted in favor, and one left early without voting.

Each patient would have a copy of the drug suiting their body, their cells used in the manufacturing of their own drug. Diefenbach, who was not involved in researching the drug and has no ties to its manufacturer, Novartis, described its results as "astounding". ALL accounts for a quarter of all cancers in children under age 15.

[T] he annual cost of managing the disease is projected to rise to $5.1 billion by 2025, up from $740,000 million in 2011, based on current pricing. But if the cancer comes back, the prognosis can be dire. Of the 52 patients whose results were analyzed, 83 percent had complete remission, meaning their cancer vanished.

CAR-T therapies also present a gnarly regulatory challenge for the FDA: how do you assure the potency and purity of a complex, living drug that must be made fresh for each patient?

Slightly fewer patients experienced neurological side effects, such as seizures and hallucinations, according to the committee's briefing document. These patients had mostly failed to respond to standard treatments or had relapsed. The drug maker cautioned that more work is needed. Novartis said Wednesday that overall survival in its pivotal trial was 89% at six months and 79% at 12 months. The majority were also relapse-free in the same time frame. While patients on this engineered auto cell therapy have been repeatedly hit by potentially lethal cases of cytokine release syndrome and neurological toxicity with the threat of new malignancies, advisers were clearly willing to accept the risk in order to achieve the potential gains.

The CART-T therapy developed by Novartis involves extracting special immune system cells from the patient, then reprogramming them.

A single one can destroy up to 100,000 cancer cells, the New York Times reported.

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Several committee members expressed concern about the uncertainties that still swirl around the therapy.

At the meeting, the panel of experts did not question the lifesaving potential of the treatment in hopeless cases. Brody said it could take decades to conclusively say this does not happen.

"This is probably the most exciting thing I have seen in my lifetime", said Tim Cripe, chief of hematology and oncology at Ohio State University's Nationwide Children's Hospital. "It's not an opinion". Four patients treated Juno Therapeutics' CAR-T cell treatment JCAR015, died in July 2016 by the condition in which excessive fluid causes the brain to swell.

"You can put someone else's red blood cells into you", he said. Baldrick's Pediatric Cancer Dream Team.

Loncar said approval of tisagenlecleucel would represent an inflection point for investors.

Licensing a product without significant data is an unnecessary risk, Polanin said, adding that new treatments shouldn't be approved on the hope that they will be shown to be effective. They are also tackling a far more hard challenge - using the therapy for solid tumors in the lungs or brain, for example. There, staff use a virus to insert genes into the T cells that encode a cellular receptor that will recognize leukaemia cells. She was the first child to receive the treatment. Gene therapy may soon be authorized by the Agency to the u.s. food and drug administration (FDA).

Although the FDA is not obliged to follow the recommendations of its advisory panels, it often does so.

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