Saving new cancer drug given green light

Saving new cancer drug given green light

Saving new cancer drug given green light

The FDA has approved the first "living drug" that uses genetically modified immune cells to target cancer.

Known as CAR-T therapy, the FDA said it was the "first gene therapy" that has been made available in the USA and could cure patients with lethal-type blood and bone marrow cancer that has been affecting children and young adults. ACGT provided an additional $800,000 of funding in 2008.

The CAR-T cell treatment, developed by Novartis Pharmaceuticals and the University of Pennsylvania, is the first form of gene therapy to be introduced to the USA market. "We thank all those involved in this historic FDA approval, which provides renewed hope to children and young adults who have had limited treatment options thus far".

The new treatment is the first USA approval of a treatment for genetic modification, and its maker promises some unprecedented pricing options- including offering it for free if it doesn't work quickly.

In clinical trials, CAR-T therapies have shown remarkable efficacy against blood cancers. At the one year mark, most patients would be relapse-free.

Each dose of Kymriah is a customised treatment created using an individual patient's own T-cells, a type of white blood cell known as a lymphocyte.

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

The immunotherapy now can be used in children and young adults with B-cell ALL that will not respond to other therapies, the FDA announced. For those who do respond-in a trial, 52 of 63 leukemia patients went into remission within three months of treatment, though the disease often kills quickly-the cost is less than that of a bone-marrow transplant, a doctor tells the Times.

More news: About 18000 Rohingya Have Fled Myanmar Since Violence Erupted Last Week

"This transformational therapy for patients is the result of true collaboration between industry, academia, healthcare professionals, patients and caregivers", said Bruno Strigini, CEO, Novartis Oncology.

The price of the therapy is tagged at around $500,000.

"Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more".

The rights to the new cancer treatment were given to them and they would sell it under the label Kymriah for $475,000.

The U.S. Food and Drug Administration's approval came weeks earlier than scheduled. Several recent patient deaths in CAR-T clinical trials have raised serious questions over the safety of the treatment.

Kymriah is expected to be available through a network of certified treatment centers throughout the United States. The manipulated cells could cause a cytokine storm, which could present with high fever, low blood pressure, long congestion and other life-threatening issues.

"We're at the very beginning of what's going to be a big new field of medicine", David Epstein, who helped license Kymriah in from the University of Pennsylvania while at Novartis.

Other genetic therapies for cancer are also in the research pipeline.

Related news