New cancer drug's 'astronomical price' only affordable for the 1%

Posted September 01, 2017

The US Food and Drug administration (FDA) has announced the approval of the first cell-based gene therapy, something that is has described as a "historic action".

"We're excited and proud to have moved this auto therapy, in collaboration with Novartis and CHOP, through all phases of development and clinical trials, established its efficacy, and now extended its reach to children across the country under this FDA approval", he added.

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. This is the first type of gene therapy to hit the USA market called the CAR-T cell treatment that was developed by Novartis Pharmaceuticals and the University of Pennsylvania.

On Wednesday, the FDA also expanded approval for another drug, tocilizumab, to treat CRS in patients 2 and older.

Novartis has suggested it will however develop a flexible pricing strategy for the therapy that will be based on the outcome of therapy, and with payment only becoming due when ALL patients have responded to Kymriah "by the end of the first month".

The one-time treatment isn't cheap: Novartis, which makes Kymriah, on Wednesday said the price would be $475,000, which is lower than many expected.

Novartis will create a registry to follow patients for 15 years after being treated to monitor their progress and any potential, future side effects. The overall remission rate within three months of treatment was 83 percent.

Dana-Farber/Boston Children's has recently participated in a similar clinical trial of vehicle T-cells - led by Silverman and pediatric oncologist Steven Margossian, MD, PhD - that evaluated the efficacy of auto T-cell therapy (short for chimeric antigen receptor T-cell therapy) in treating relapsed or treatment-resistant B-cell ALL.

Other trials with CTL019 therapy are also underway in the Abramson Cancer Center for adult ALL patients and those with CLL and non-Hodgkin lymphoma.

The treatment involves altering the patient's own immune system by removing T-cells and genetically modifying them to recognize, attack and kill cancer cells. Eventually, the therapy will be offered at 32 sites, the company said. 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. Several recent patient deaths in CAR-T clinical trials have raised serious questions over the safety of the treatment. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of vehicle T-cells causing high fever and flu-like symptoms, and for neurological events.

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

Health officials in the United States have approved a revolutionary treatment that uses genetic engineering to combat childhood leukemia. Right now, that's where most of the big successes have come from, but that could one day include solid tumors and maybe even autoimmune diseases like Type 1 diabetes, Epstein said. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years.

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. And past year, another company, Juno Therapeutics, said five people in its clinical trials had died, all from cerebral edema.