New Gene Therapy For Childhood Leukemia Gets Approval

Novartis has developed the ground-breaking leukaemia therapy

Novartis has developed the ground-breaking leukaemia therapy

The FDA approved the first-ever gene therapy in the U.S. on Wednesday.

The complexity of the treatment, involving the harvesting the patient cells and the subsequent genetic editing, is undoubtedly time-consuming and labor-intensive, so it remains to be seen how widely this kind of customized gene therapy could be rolled out. 5 years ago, for the first time, he used the CAR-T cell therapy to treat a girl child who was close to death but now she is completely ok.

"We're at the very beginning of what's going to be a big new field of medicine", said David Epstein, who helped license Kymriah from the University of Pennsylvania while at Novartis. They call the one-time infusion CAR-T cells and they will not charge the patient if he/she does not show signs of improvement within a month.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The company is carefully training hospitals and staff to provide the treatment, which can cause a life-threatening immune reaction, as well as long-term complications. Nevertheless, researchers caution that, in many respects, it's still early days for auto T cells and other forms of ACT, including questions about whether they will ever be effective against solid tumors like breast and colorectal cancer. A multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL found that the overall remission rate within 3 months of treatment was 83%.

Despite Kymriah's success in the clinical trial, Walid Gellad, a doctor and professor at the University of Pittsburgh, suggested to Axios that treatments with undetermined effectiveness-even if they have the potential to save lives-shouldn't cost more than proven measures such as bone marrow or kidney transplants, adding: "This is an unbelievable therapy, but there has to be a limit at which point companies can no longer charge desperate patients, or taxpayers, enormous sums".

Kymriah belongs to a new class of treatments called CAR-T therapies.

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Dana-Farber/Boston Children's has recently participated in a similar clinical trial of vehicle T-cells - led by Silverman and pediatric oncologist Steven Margossian, MD, PhD - that evaluated the efficacy of auto T-cell therapy (short for chimeric antigen receptor T-cell therapy) in treating relapsed or treatment-resistant B-cell ALL. "The established path to commercialization taken by Novartis will provide considerable motivation for cell therapy scientists and development companies as well as investors looking to benefit from the ongoing success in the industry".

The therapy is called Kymriah, and it is being made by Norvartis. The treatment carries a boxed warning for cytokine release syndrome (CRS) and for neurological events. Kite, which was recently acquired by Gilead Sciences, is expected to hear back from the FDA about its treatment for aggressive B-cell non-Hodgkin lymphoma by November.

"The only potentially curative option for these pediatric and young adult patients is allogeneic stem cell transplant, which costs $500,000 to $800,000 for the first year", Cooper said. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years.

The U.S health officials have approved a treatment which genetically turns the patient's blood cells into assassins seeking to destroy leukemia.

There could be side effects. The patient was a little girl nearly dying and is now five years cancer free.

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