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US Approves First Gene Therapy for Young Leukemia Patients

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Swiss drugmaker Novartis AG is seen at its headquarters in Basel

USA regulators approved a revolutionary new treatment from Novartis that modifies the body's own immune-system cells and reinjects them to treat, and in many cases cure, patients with deadly blood cancers who have run out of other options.

"We are very encouraged by today's news that vehicle T-cell therapy will now be available for pediatric and young adult patients with relapsed acute lymphoblastic leukemia (ALL.) This treatment provides renewed optimism for some of the most challenging cases we face in pediatric ALL", says Lewis Silverman, MD, clinical director of the pediatric Hematologic Malignancy Center, in a statement issued by Dana-Farber/Boston Children's. In 2012, Penn and Novartis entered into a global collaboration to further research, develop and commercialize Kymriah and other CAR-T cell therapies for the treatment of cancers. Beyond the cost of the procedure, patients would need to pay for traditional chemotherapy, which is given before auto T-cell therapy to improve its odds of success.

The United States Food and Drug Administration (FDA) on Wednesday announced that it has approved a cell-based gene therapy in the United States. Meanwhile, this week Roche secured FDA approval to use its arthritis drug Actemra (tocilizumab) as a treatment for CAR-T-induced CRS.

This breakthrough allows scientists to manipulate a patient's blood cells, turning them into an army that can seek and kill cancer cells.

"There has been an urgent need for novel treatment options that improve outcomes for patients with relapsed or refractory B-cell precursor ALL", Novartis, the drug company that makes Kymriah, said in a statement.

"Of course, we have to talk about pricing from a national standpoint", he said.

The FDA has recognized these dangers in approving the Kymriah treatment, noting that the approval is only directed at patients that have not responded to other, more common, initial treatments.

Of more than 60 ALL-suffering subjects in a clinical trial for Kymriah, 83 percent were cancer-free within three months. Novartis, the company behind Kymriah, claims it takes around 22 days to produce an individual treatment for a patient. The hope is that one day doctors will be able to prescribe a cell therapy and use it that same day instead of waiting weeks to get it back. "It will be several years before cell therapies are widely accessible for patients in more indications beyond leukaemia and lymphoma", said Dr Miguel Forte, PhD, chief commercialization officer, ISCT and chief medical officer, Bone Therapeutics. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years.

Those updates could potentially expand the number of patients cell therapies are able to treat. They'll also have to be less toxic, so more people can consider it instead of only those who have very few other options. CAR-T's side effects can be deadly. In May, Kite disclosed that one person had died in a clinical trial for its late-stage CAR-T therapy from cerebral edema, a condition in which excessive fluid causes the brain to swell.

With the treatments available now, fewer than 10 percent of patients with the relapsed, hard-to-treat leukemia are alive five years after diagnosis.

And then, to impact more people, the cell therapies will need to go beyond the blood cancers.

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