The Food and Drug Administration (FDA) today approved “the first gene therapy available in the United States,” a chimeric antigen receptor (CAR) T-cell therapy called Kymriah, which will be used to treat acute lymphoblastic leukemia (ALL) in some children and young adults.
“This is an exciting development for everyone in the blood cancer community,” said Jenny Ahlstrom, founder of the Myeloma Crowd, “this is proof that CAR T and T cell immunotherapy is no longer science fiction; it is moving into the clinic to benefit patients now.”
Kymriah targets the antigen CD 19, which is based on research done in the University of Pennsylvania lab of Dr. Carl June. The Myeloma Crowd Research Initiative (MCRI) is supporting two research projects that apply the same theory approved today to the treatment of myeloma patients. According to FDA Commissioner Scott Gottlieb, “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”
“I have long believed—and today’s news just increases my enthusiasm—that many of today’s myeloma patients will one day be the beneficiaries of this emerging science,” observed Jenny. “The MCRI supported projects focus on editing a patient’s own T-cells to fight their disease by focusing on the genetic targets BCMA and CS 1 and marrow infiltrating lymphocytes (MILs). “Together with other research focusing on targets like CD 19 and CD 229, which is being studied at the Huntsman Cancer Institute in Utah, the potential rewards should inspire cautious optimism for all myeloma patients.”MCRI
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about the author
Greg Brozeit has been engaged in myeloma patient advocacy since 1998. He began working with the Myeloma Crowd in 2015. Prior to that, he consulted with Dr. Bart Barlogie at the University of Arkansas after working with the International Myeloma Foundation for 15 years, where he inaugurated the public policy advocacy program, patient support group outreach and IMF Europe, organizing more than 100 physician and patient education programs. He earned his BA in political science from Loyola University in New Orleans and lives in northeast Ohio.