It's a Breakthrough Cancer Drug-And It'll Cost You

Novartis' CAR-T gene therapy, the first approved by FDA, to be priced based on cancer patients' outcomes

Pharma giant Novartis on Wednesday won highly anticipated USA approval for the first of a new type of potent gene-modifying immunotherapy for leukemia, an exhorbitantly priced treatment that nevertheless marks the start of a potential new treatment approach for some cancers.

Today, the Food and Drug Administration approved a gene therapy known as vehicle T-cell therapy that genetically modifies a patient's own cells to help them combat pediatric acute lymphoblastic leukemia (ALL), the most common childhood cancer. This revised forecast encompasses the expected cross-industry reaction to the approval, and envisions the next stages in the development of cell therapies and the industry. Novartis has agreed a risk evaluation and mitigation strategy (REMS) with the FDA that amongst other things will see the therapy delivered only at certified treatment centres. It involves removing disease-fighting T cells from a patient, genetically modifying them to better recognize and attack cancer, and then replacing them, where they can circulate for years seeking out the disease. 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 drug Kymriah was developed by Novartis Pharmaceuticals and the University of Pennsylvania.

There are other severe side effects that can pop up as well that can require hospitalization.

Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy.

Of more than 60 ALL-suffering subjects in a clinical trial for Kymriah, 83 percent were cancer-free within three months.

Despite Kymriah's success in the clinical trial, Walid Gellad, a doctor and professor at the University of Pittsburgh, suggested to Axios that treatments with undetermined effectiveness-even if they have the potential to save lives-shouldn't cost more than proven measures such as bone marrow or kidney transplants, adding: "This is an fantastic therapy, but there has to be a limit at which point companies can no longer charge desperate patients, or taxpayers, enormous sums". Eventually, the therapy will be offered at 32 sites, the company said.

The drug, called Kymriah, is a highly personalized cancer treatment called auto T-cell therapy (CAR is short for chimeric antigen receptor). About half of the patients in a Kymriah study got cytokine-release syndrome, a response to the reprogrammed cells running loose in the body. The company did say that it's made an agreement with the USA government to pay for the drug only when pediatric or young adult patients with the cancer respond to treatment by the end of their first month.

The therapy is called Kymriah, and it is being made by Norvartis. Several recent patient deaths in CAR-T clinical trials have raised serious questions over the safety of the treatment.

"The only potentially curative option for these pediatric and young adult patients is allogeneic stem cell transplant, which costs $500,000 to $800,000 for the first year", Cooper said.

For one, the treatments will have to treat more types of cancer than the one approved on Wednesday. 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. CAR-T's side effects can be deadly. And previous year, Juno said five people in its clinical trials had died, all from cerebral edema.

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