THE US APPROVES FIRST DRUG FOR THE TREATMENT OF CANCER

The Food and Drug Administration in the US has approved a new drug for the treatment of leukemia. It was titled “a historic action” by the agency as this is this first cell based gene therapy to be sold in the United States.

The treatment called Kymriah, is known as a “living drug” because it contains the patient’s immune cells which are used to produce a virus through genetic modification. The therapy, called chimeric antigen receptor T-cell therapy otherwise known as CAR-T allows the cell to be able to recognise, source and kill the cancerous cells.

The drug was approved, by an FDA advisory committee, for patients suffering from a form of acute lymphoblastic leukemia prominent in children and young adult, that do not respond to standard treatment after a first line of drugs do not work.

The FDA Commissioner Scott Gottlieb in a written statement said “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,”

He also added that "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses."

The Director of cancer immunotherapy at children’s hospital of Philadelphia that spearheaded the research Dr Stephan Grupp said "We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population,"

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

He also added in an FDA statement that "Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

An overreaction by the immune system of the patient known as cytokine-release syndrome, is a risk involved in taking of the treatment. As a result, the FDA is requiring strong warnings. Hence it is only going to be availale in 32 hospitals and clinics in the United States that have been specially trained to administer the treatment.

Based on available data, patients on the treatment have had an 89% chance of surviving at least six months and a 79% chance of surviving at least a year, with most being relapse-free at that point.

The company that developed the drug, Novartis, said the treatment will consist of a one tim fee of $475,000 for patients that respond. Meanwhile ptients who do not respond to the treatment after a month will not be charged. And also the company, Norvatis said it was taking additional steps to ensure that the drug was available and affordable for everyone.

"While Novartis' decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive," says David Mitchell, founder and president of Patients For Affordable Drugs.

He also added that "Let's remember, American taxpayers invested over $200 million in CAR-T's discovery."

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