Philly Hospital Critical in Groundbreaking Leukemia Treatment Approved by FDA

Philly Hospital Critical in Groundbreaking Leukemia Treatment Approved by FDA

Principal supporting data for the application came from a phase II trial of children and young adults with relapsed/refractory ALL, given a single infusion of CTL019. After the modified cells have had time to multiply, they are injected back into the patient where the receptors are free to target and destroy the cancer cells.

Many of the FDA's advisers were effusive in their praise.

A new cancer treatment endorsed Wednesday by the Federal Drug Administration's Oncologic Drugs Advisory Committee is being described by one panel member as "the most exciting thing I've seen in my lifetime". Of the 52 patients examined, 83% had complete remission, although a lot of them suffered serious side effects. Those so-called "risk mitigation" plans were one of the main concerns that the FDA, in documents released Monday, asked its advisors to consider.

Slightly fewer patients experienced neurological side effects, such as seizures and hallucinations, according to the committee's briefing document.

Novartis believes it can turn around a finished CAR-T product 22 days from receipt of leukapheresed material to final packing and shipping.

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. While patients on this engineered vehicle 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. In 2012 as a 6 year old, she was treated in a study at the Children's Hospital of Philadelphia. This would be a huge treatment option for these children and young adults with B-cell ALL.

"While CAR-T is a promising new type of immunotherapy, it is not commercially available and we have yet to complete our evaluation", said T.J. Crawford, a spokesman for Aetna Inc. "You have to be a long-term survivor to experience [long-term] toxicity", said Bruce Roth of the Washington University School of Medicine in St. Louis, MO.

The pediatric oncologists on the committee called Polanin out on her comments, saying her follow-up fears were unfounded when it comes to children.

Leukaemia patient
GETTYNew therapy 83 per cent of patients went into remission

He said in a blog: "With every paper I read, it becomes more and more clear that CAR-T is not a biotech race to be won, but rather a revolutionary therapy to be explored and applied to other cancers".

"In those settings, the downstream effect [of triggering a secondary cancer] is conceivable", said Maziarz.

Other panelists called the treatment a "major advance" and that novel therapy meets a "dire, unmet need". The drug maker cautioned that more work is needed.

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

Although the FDA is not obliged to follow the recommendations of its advisory panels, it often does so. Novartis is also vying to have CTL-019 approved to treat adults with the most common forms of non-Hodgkin lymphoma.

Juno Therapeutics abandoned its CAR-T product in March after five people died during testing. Another risk is that there are many other players that are developing treatments in the CAR-T space as well.

Such a complex system for making personalized treatments is likely to drive up their cost, and the next big hurdle (assuming an FDA approval this fall) is to win over insurers. Doctors extract a specialized type of white blood cells, known as T-cells, from the patient and re-engineer them to home in on and attack cancerous cells.

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