FDA Approves First CAR-T Cell Therapy

FDA Approves First CAR-T Cell Therapy

The country's first approved gene therapy-approved Wednesday to fight leukemia that resists standard therapies-will cost $475,000 for a one-time treatment, its manufacturer announced.

Welcoming a new era in cancer treatment, the USA health officials have officially approved a new breakthrough treatment for the patients suffering from leukemia where it genetically transforms the patient's own blood cell into an army of assassins which detects and destroys the childhood leukemia. In a press release, the FDA called the gene therapy's approval "a historic action".

This represents the first approval of this form of gene transfer therapy and as such has given rise to the International Society for Cellular Therapy (ISCT), the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell therapy sector, revising its forecast for the CAR-T and cell therapy industries. In just the last few years, progress with auto T cells and other ACT approaches has greatly accelerated, with researchers developing a better understanding of how these therapies work in patients and translating that knowledge into improvements in how they are developed and tested. Once the cells are modified, they are infused back into the patient to kill the cancer cells. The FDA approval of this therapy is based on the results of the Phase II ELIANA trial, which was sponsored by Novartis and included 25 centres in the US, EU, Canada, Australia and Japan. The REMS program specifies that patients be informed of the signs and symptoms of CRS and neurological toxicities following infusion - and of the importance of promptly returning to the treatment site if they develop fever or other adverse reactions after receiving treatment with Kymriah.

Kymriah has a $475,000 price tag; however, patients who do not respond within a month of treatment will not be charged, according to Novartis.

However, this type of therapy carries risk of severe side effects. Despite the potential of major side effects, clinical trials of Kymriah have displayed overall remission rates of up to 83 percent. The organisation forecasts that considerable investment will be made in the cell therapy field, particularly as there are other companies in the process of submitting applications to regulatory bodies for similar therapies.

Tisagenlecleucel (Kymriah), Novartis' newly approved vehicle T-cell therapy, will be put on the market with a price of $475,000 for a single infusion, an amount that is within the range anticipated by oncologists and that Novartis characterized as well below a price level that could be justified on cost.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells.

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

Epstein said he envisioned cell therapies having much shorter life cycles than traditional drugs.

For one, the treatments would have to treat more types of cancer than the one Kymriah was approved for on Wednesday.

It's not hyperbolic to say that the development of CAR-T immunotherapy has been a revolution in cancer research over the past few years.

Kymriah is the first CAR-T cell therapy to get approved, and there are several more in the works.

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.