Novartis Is First To Finish Line With CAR-T Cancer Drug Nod

The U.S. Food and Drug Administration (FDA) made history this morning when they approved the first-ever gene therapy, Kymriah (tisagenlecleucel), developed by Novartis.

FDA called the approval historic. In clinical trials in patients treated with vehicle T cells, 69% of patients had complete resolution of CRS within two weeks following one or two doses of Actemra.

The decision clears the Novartis drug, called Kymriah, for treatment with children and young adults whose illness doesn't respond to traditional approaches. But, the company said there would be no charge if the patient didn't show a response within a month.

The treatment does carry risks, however, including a risky overreaction by the immune system known as cytokine-release syndrome. We now have proof that it is possible to eradicate cancer by harnessing the power of a patient's own immune system.

"The approval of auto T-cell therapy for pediatric leukemia marks an important shift in the blood cancer treatment paradigm".

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA Commissioner Scott Gottlieb, MD.

In one multicenter clinical trial of 63 pediatric and young adult patients with relaped or refractor C-cell precursor ALL, the safety and efficacy of Kymriah were confirmed as the overall remission rate was 83% within 3 months of treatment.

While most survive, about 15% relapse despite the current best treatments, and their prognosis is bleak. The REMS program specifies that patients be informed of the signs and symptoms of CRS and neurological toxicities following infusion-and of the importance of promptly returning to the treatment site if they develop fever or other adverse reactions after receiving treatment with Kymriah.

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We recently asked you to support our journalism. "I wouldn't say we know for sure how many will be cured yet by this therapy".

Most patients suffered side effects that can be grueling, even life-threatening.

"This is remarkable technology", said Dr. Mikkael Sekeres of the American Society of Hematology.

In this type of therapy, patients' own immune cells are modified ex vivo to include a gene for the auto protein, which directs them to attack leukemia cells carrying the CD19 antigen.

Unlike traditional gene therapy, the new treatment doesn't replace disease-causing genes with healthy ones.

"I don't want to be an apologist for high drug prices in the U.S.", Grupp stressed. In addition, clinicians involved in the prescribing, dispensing, and administering of the therapy will need training to recognize these side effects. Patients' collected immune cells will be frozen and shipped to a Novartis factory in New Jersey that creates each dose, a process the company says should take about three weeks.

Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse. But the customized treatment is also being tested for a range of diseases from non-Hodgkin lymphoma and multiple myeloma to solid tumors, and its approval signals a new chapter in treating cancer by mobilizing the body's own immune system.