April 23, 2018

FDA Approves First "Living Drug" To Treat Childhood Leukemia

31 August 2017, 01:45 | Franklin Nunez

First gene therapy coming to US after FDA approval

Novartis' CAR-T gene therapy, the first approved by FDA, to be priced based on cancer patients' outcomes

Some 83 percent of those given the therapy were in remission within three months of treatment, the agency said.

The therapy was evaluated in clinical testing involving 63 children and young adults with relapsed or refractory B-cell ALL.

Kymriah is a type of personalized immunotherapy known as CAR-T, or chimeric antigen receptor T-cell therapy. "Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials".

In response to the possibility of CRS development, the FDA has also expanded the approval of tocilizumab (Actemra; Genentech) to treat CAR-T cell-induced severe or life-threatening CRS in patients 2 years of age and older.

"We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population", Dr Stephan Grupp of Children's Hospital of Philadelphia said in a statement. Kymriah is intended for people with B-cell ALL whose cancer hasn't responded to or has returned after treatment, the FDA said.

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It's too soon to tell how widely applicable CAR-T will be - it's especially suited to leukemia since infusing patients with T-cells is relatively simple. However, there were some severe side effects observed in study.

"Through our collaboration with Novartis, we are creating the next wave of immunocellular cancer treatments, and are eager to progress CAR-T therapy in a host of hematologic and other cancer types", said Carl June, MD, Professor of Immunotherapy, Director of the Center for Cellular Immunotherapies in Penn's Perelman School of Medicine.

Kymriah treatment also has the possibility of serious side effects, including cytokine release syndrome (CRS), which consists of high fever and flu-like symptoms and can be life-threatening, and neurological events, which are also life-threatening.

A spokeswoman for Switzerland-based Novartis declined to say how much the drug will cost, though analysts have estimated a price of $500,000 or more. Research is therefore focusing for now on refining the therapy for patients of this disease. It is the first gene therapy of its kind to hit the USA market. The agency said that 69% of patients developing the condition during vehicle T-cell trials "had complete resolution of CRS within 2 weeks following one or two doses" of tocilizumab, which inhibits interleukin-6. More than half were children and teens.

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