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
is less than $5,000 short of its $500,000 goal to support its two research projects. Donate Now
about the author
Greg Brozeit has been with the HealthTree Foundation since 2015 when he began volunteering for the Myeloma Crowd. Prior to that he worked with Dr. Bart Barlogie and the International Myeloma Foundation, inaugurating many myeloma patient advocacy and education programs.