Breakthrough gene therapy treatment for leukemia to cost N144m

Breakthrough gene therapy treatment for leukemia to cost N144m

Breakthrough gene therapy treatment for leukemia to cost N144m

"[Tisagenlecleucel] is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research, said in a statement.

The Food and Drug Administration approved a treatment that engineers a patient's own blood cells to fight cancer.

Kymriah is the first gene therapy for leukemia available in the United States.

"Before CAR-T cells, we've been able to save about 70 per cent of children with leukaemia, but those other 30 per cent of children who don't respond to chemo have died".

The therapy consists of an individualized, one-time treatment that has shown an 83% remission rate in clinical trials with patients that have not responded to standard treatments.

More than 3,000 patients aged 20 or younger are diagnosed with ALL every year, according to the National Cancer Institute.

The Food and Drug Administration (FDA) has called this a historic moment because it is considered to be the first gene therapy hitting the US markets.

The price of Kymriah - which is delivered to a patient just once - is $600,000, said Bruno Strigini, CEO of Oncology at Novartis.

Doctors remove T cells from each cancer victim and ship the material to its factory in Morris Plains, New Jersey.

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American taxpayers invested over $200 million in CAR-T's discovery.

Future uses for the drug in more cancers "could be much larger, but the offset to a larger population would be a lower price most likely", Anderson said in a note to clients.

Over the last few years, CAR-T cell research has exploded, and there are now nearly 300 clinical trials underway experimenting with the treatment.

After the FDA approval, hospitals are close to offering Kymriah to patients. It involves removing disease-fighting T cells from a patient, genetically modifying them to better recognize and attack cancer, and then replacing them, where they can circulate for years seeking out the disease.

Devoe also said that while the positive results are exciting, many questions remain about how long these novel cancer treatments - developed mainly in the past decade or so - will last. Most ALL patients respond to initial therapy, but Kymriah will be an invaluable back-up option for relapsed and refractory patients who now have poor prognosis.

CAR-T can lead to a life-threatening condition known as cytokine release syndrome, as well as severe neurological events. They'll also have to be less toxic, so more people can consider it instead of only those who have very few other options. Now Novartis has proved it is possible for one therapy to succeed, this proves others will succeed too, and this adds competition to the mix.

That first patient had been near to death, and according to BBC News has now been cancer-free for more than five years.

This side-effect can be controlled with drugs. "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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