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The first chimeric antigen receptor (CAR) T-cell therapy was approved just a year ago, changing the face of treatment for certain types of leukemias and lymphomas but carrying with it the downsides of toxicity and cost. A year later, scientists from a major cancer center said that they’ve made headway to discovering more about the T-cell signaling patterns and that understanding more about the biological pathways could help design the next generation of CAR-T treatments.

Next week, a CMS committee will hold a day-long meeting to discuss a national coverage determination (NCD) for chimeric antigen receptor (CAR) T-cell immunotherapies, and in Thursday’s New England Journal of Medicine, Peter B. Bach, MD, MAPP, reviewed several strategies open to CMS as it continues to try to determine how to pay for CAR T.

For insights on this issue, Evidence-Based Oncology™ spoke with Lynda Kwon Beaupin, MD, a pediatric hematologist-oncologist who recently became the director of CanSurvive, the pediatric cancer survivorship program at Johns Hopkins All Children’s Hospital in St. Petersburg, Florida. Beaupin and her colleagues in the Consortium of Adolescent and Young Adult Cancer Centers, which includes oncologists from major cancer centers including Johns Hopkins, are working to address the nuances that come with treating adolescent and young adult cancer patients and looking for ways to increase their quality of life.

Thea Danze lights up the room wherever she goes; she loves space and music and fire trucks. Since 2012, she has been the namesake and spokesperson for a foundation created to raise funds and awareness about pediatric brain tumors. Called Thea’s Star of Hope, the foundation supports development of therapies that will treat brain tumors without the toxic adverse effects that Thea has endured.

In July 2018, the FDA approved iobenguane I 131 (Azedra), the first treatment for patients with unresectable pheochromocytoma or paraganglioma, which are rare tumors of the adrenal gland, that require systemic anticancer therapy. These tumors, which typically appear at an early age and are associated with premature death, can increase the production of epinephrines and norepinephrines, leading to a host of symptoms, including hypertension, vomiting, weakness, and chest pain.

Celyad, a biopharmaceutical company that focuses on the development of chimeric antigen receptor (CAR) T-cell therapies, recently announced that the FDA has accepted its Investigational New Drug (IND) application for CYAD-101, the first non–gene-edited allogeneic clinical program.

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