F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000<br />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<br />and multiplying, frozen again and shipped back to the medical center to be dripped into the patient.<br />The therapy, marketed as Kymriah and made by Novartis, was approved for children<br />and young adults for an aggressive type of leukemia — B-cell acute lymphoblastic leukemia — that has resisted standard treatment or relapsed.<br />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<br />that relapsed or treatment-resistant disease is often quickly fatal.<br />Medical staff members need training to manage these reactions, and hospitals are being told<br />that before giving Kymriah to patients, they must be sure that they have the drug needed to treat the problems, tocilizumab, also called Actemra.<br />Novartis said the treatment would be available at an initial network of 20 approved medical centers<br />to be certified within a month, a number that would be expanded to 32 by the end of the year.<br />Dr. Kevin J. Curran, a pediatric oncologist at Memorial Sloan Kettering Cancer Center in Manhattan, said his<br />hospital was “99 percent” of the way through the certification process, and would soon be offering Kymriah.<br />Certification is being required because the revved-up T cells can touch off an intense reaction, sometimes called a cytokine storm,<br />that can cause high fever, low blood pressure, lung congestion, neurological problems and other life-threatening complications.
