CAR-T Cell Approval by the FDA

A year ago we wrote extensively about the developments in CAR-T cells. We have been following this work for several years and it has finally reached a clinical level with FDA approval today of a Novartis therapeutic.

The FDA notes:

Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse. Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells. ALL is a cancer of the bone marrow and blood, in which the body makes abnormal lymphocytes. The disease progresses quickly and is the most common childhood cancer in the U.S. The National Cancer Institute estimates that approximately 3,100 patients aged 20 and younger are diagnosed with ALL each year. ALL can be of either T- or B-cell origin, with B-cell the most common. Kymriah is approved for use in pediatric and young adult patients with B-cell ALL and is intended for patients whose cancer has not responded to or has returned after initial treatment, which occurs in an estimated 15-20 percent of patients.

ALL is a deadly childhood cancer, most often, and  I recall see my first case in March 1968. Cold rainy day and a ten year old had a fever and malaise and we ran the blood work and it was a leukemia. Death was then a certainty. Now with this therapeutic survival is a viable option.

It should be interesting to see how this can be applied to other cancers. Cell markers must be available for the differing malignancies.