Gene modifying therapy gets U.S. approval

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

First gene therapy coming to US after FDA approval

"This is the first-ever true gene therapy treatment made available to the United States population and will help accelerate the speed at which we will see even more gene-based therapies come to fruition".

"This decision marks the first commercialization in the world of a gene therapy....based on a new approach a fully custom one that uses their own immune cells of the sick", has meanwhile congratulated the CEO of Novartis, Joseph Jimenez, during a press conference call.

But medical experts have described the price as unreasonable and say questions remain about the long-term effectiveness of gene therapy type treatments. The patient was a little girl nearly dying and is now five years cancer free. In a key clinical trial of 63 people, 83 per cent of participants were cancer-free after three months.

The drug, called Kymriah, is a highly personalized cancer treatment called vehicle T-cell therapy (CAR is short for chimeric antigen receptor).

The Food and Drug Administration called the approval historic, this being the first gene therapy on the American market, ABC News reports.

"It is critical to have a realistic outlook and to have a measured response to the Novartis CAR-T approval".

The price per person is likely to be much higher when accounting for additional costs, such as drugs to prepare the patient for the treatment and medicines to control potential side-effects. 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.

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The FDA said hospitals and clinics must become certified to distribute the treatment, meaning they are prepared to recognize and treat CRS and other potentially fatal neurological events. The FDA on Wednesday expanded the approval of Actemra, a rheumatoid arthritis drug that can also treat cytokine release syndrome.

Ghobadi says this treatment genetically engineers a patient's own blood cells, turning them into assassins to seek and destroy childhood leukemia. The treatment is for those who have not responded to initial treatment, which occurs in an about 15-20 percent of patients, or those who have relapsed.

The breakthrough has been welcomed by Australian medical practitioners and researchers as a promising sign not only for leukaemia, but for the future treatment of many other forms of cancer.

The approval of tisagenlecleucel was based on phase II results from the single-arm, worldwide ELIANA trial of 63 patients who received a single dose of tisagenlecleucel.

His current research is focused on similar reprogramming of cells but for the detection of lung cancer cells.

There are other severe side effects that can pop up as well that can require hospitalization. "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".

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