Wednesday, March 7, 2018

New Cell Therapy Treatment for Leukemia Receives FDA Approval


Dr. Mark Fesen serves as a medical oncologist at Central Care Cancer Center in Great Bend and Wichita, Kansas. Experienced in the treatment of all cancers, including leukemia, Dr. Mark Fesen has participated in a number of phase 2 clinical trials.

In February of 2018, the New England Journal of Medicine published the results of a global multi-center study that resulted in the first FDA approval of chimeric antigen receptor T (CAR-T) cell therapy. This gene therapy approach uses white blood cells from the patient's body that have undergone genetic reengineering, so that they specifically target cancer cells. The therapy received approval from the US Food and Drug Administration as a treatment for pediatric acute lymphoblastic leukemia (ALL) in August of 2017.

The recent phase 2 study took place at 25 clinical sites across 11 countries and featured the participation of 75 eligible patients between the ages of three and 21. All participants had relapsed or treatment-resistant B-cell ALL, and 61 percent had relapsed following the transplantation of donor hematopoietic stem cells. Although failure of this treatment has in the past signaled an overall failure of treatment and a prescription of hospice care, the new CAR-T treatment led to a remission rate of 81 percent over a period of three months.

The treatment resulted in a survival rate of 90 percent after six months and 76 percent after 12 months, while the relapse-free survival rate stood at 80 percent six months following treatment and 59 percent after a full year. 

Side effects included cytokine release syndrome (CRS), a condition that involves severe flu-like symptoms and often requires a stay in intensive care. This and other adverse effects did prove treatable and reversible, although the team is planning clinical trials in an effort to reduce the incidence of serious treatment-related symptoms.