Unanimous Advice To FDA: Approve Landmark CAR-T Cancer Therapy

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While this gene therapy treatment for leukemia, known as CTL019, will be the first to reach the market, there are more on the way.

If CTL019 is approved, the Swiss pharmaceutical giant plans to dole it out from about 30 preapproved sites, each trained in the multi-step process of harvesting cells, handling the product, and treating patients for the feverish and often life-threatening immune response that usually accompanies CAR-T therapy. CTL019 demonstrated consistent safety across three separate clinical trials involving children and young adults with relapsed/refractory B-cell ALL, the officials added.

Novartis has lagged some other big drugmakers in the first wave of immunotherapy drugs that are revolutionizing cancer treatment. CTL019 is an investigational chimeric antigen receptor T cell therapy that represents a new therapeutic approach to fill the lack of treatment options for patients with this disease, according to a press release.

All Novartis requires, in order to start selling the treatment, is the stamp of approval from the FDA, which it is most likely to get.

The treatment was developed by researchers at the University of Pennsylvania and licensed to Novartis.

The panel claimed the treatment would prove to be useful for children and young adults, between ages of 3 and 25 years, suffering from B-cell acute lymphoblastic leukemia.

When a patient is treated under the Novartis process, T cells are extracted from a patient's blood, frozen and sent to the company's plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.

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The FDA is also reviewing a CAR-T from Kite Pharma for the treatment of adults with advanced and aggressive lymphoma. The committee's unanimous positive vote will go a long way towards that approval.

The Emily Whitehead Foundation, which raises funds for immunotherapy research, tweeted pictures on Wednesday of Emily before and after her treatment, along with a quote from her father's comments to the panel, saying the treatment killed Emily's cancer in "just 23 days".

The agency is expected to decide by November 29 whether to approve Kite's CAR-T, axi-cel, for treatment of adults with advanced and aggressive lymphoma.

Tisagenlecleucel's benefit for ALL patients who have relapsed from other treatments was clear before the meeting. The treatment beat back her daughter's cancer and brought back "the sparkle" in her eyes.

First developed by the University of Pennsylvania, CTL019 uses the 4-1BB co-stimulatory domain in its auto to enhance cellular responses as well as persistence of CTL019 after it is infused into the patient, which may be associated with long-lasting remissions in patients.

"It is not uncommon for the companies who make these products to manage supply closely", said Elizabeth Kalina, a spokeswoman for Shire, which is building a new manufacturing facility that's slated to open in Georgia in 2018. Most patients suffered serious side effects.

Spokeswoman for Novartis, Julie Masow, has said the company will file for approval of the treatment in the European Union later this year. 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.

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