When it comes to cancer breakthroughs, there have not been many announcements as big as the one made this week by the U.S. Food and Drug Administration - the approval of the first gene therapy for cancer available in the United States.
The approved drug, made by Novartis, is called Kymriah (tisagenlecleucel) and is used to treat certain pediatric and young adult patients with a form of B-cell acute lymphoblastic leukemia (ALL). The approval is for patients up to 25 years old, with ALL that has resisted standard treatment or relapsed (roughly 15 - 20 percent of patients.) ALL is leukemia - a cancer of the bone marrow and blood. it is the most common form of cancer that affects children - with roughly 3,100 cases diagnosed each year.
This is not your ordinary breakthrough. This drug, and the technology behind it, opens doors to a world of new treatments for cancer and other serious and life-threatening diseases. It is a T-cell immunotherapy. It works by using the patients' own T cells to fight the cancer. The T cells are collected, altered to carry a chimeric antigen receptor (CAR) that allows the T cells to find and kill the leukemia cells, and placed back into the patient.
The efficacy data on Kymriah was clear - with a remission rate of 83 percent in three months of treatment in 63 patients. With data like that, FDA did not hesitate. Last month, the panel voted unanimously (10-0) to preapprove it. This week, it jumped over the final hurdle with flying colors.
The drug is not cheap - as it is tailor made for each patient - running $435,000 for the one-time treatment. But, cancer treatments are not supposed to be on a budget. One of the alternative leukemia therapies - bone marrow transplants - cost as much or more. The important thing is that they work. Additionally, Novartis has offered financial support for families of patients who lack insurance.
Kymriah also comes with life-threatening side effects, namely neurological events and a response called cytokine release syndrome (CRS) which can produce a high fever and flu-like symptoms. To help alleviate these, a second drug's approval, Actemra, which is effective at treating these side effects, was expanded. As an additional precaution, hospitals that will dispense Kymriah will be certified and specially trained in the recognition and management of these side effects.
“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, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “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.”
But, this drug is not just a boon for the B-cell ALL community, but, for the cancer field as a whole. It is the first drug of its kind of that opens up the possibility of a very effective new cancer treatment - something that does not come frequently or easily. This is nothing short of a historic moment in cancer therapies that gives long overdue hope to not only the families suffering with pediatric cancer but everyone searching for an end to this horrific disease.