F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000
To customize Kymriah for individual patients, white blood cells called T cells will be removed from a patient’s bloodstream at an approved medical center, frozen, shipped to Novartis in Morris Plains, N. J., for genetic engineering
and multiplying, frozen again and shipped back to the medical center to be dripped into the patient.
The therapy, marketed as Kymriah and made by Novartis, was approved for children
and young adults for an aggressive type of leukemia — B-cell acute lymphoblastic leukemia — that has resisted standard treatment or relapsed.
The approval was based largely on a trial in 63 severely ill children and young adults who had a remission rate of 83 percent within three months — a high rate, given
that relapsed or treatment-resistant disease is often quickly fatal.
Medical staff members need training to manage these reactions, and hospitals are being told
that before giving Kymriah to patients, they must be sure that they have the drug needed to treat the problems, tocilizumab, also called Actemra.
Novartis said the treatment would be available at an initial network of 20 approved medical centers
to be certified within a month, a number that would be expanded to 32 by the end of the year.
Dr. Kevin J. Curran, a pediatric oncologist at Memorial Sloan Kettering Cancer Center in Manhattan, said his
hospital was “99 percent” of the way through the certification process, and would soon be offering Kymriah.
Certification is being required because the revved-up T cells can touch off an intense reaction, sometimes called a cytokine storm,
that can cause high fever, low blood pressure, lung congestion, neurological problems and other life-threatening complications.