May Cure Champion

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On Saturday, May 7 at the 40th Annual JDRF Greater New England Gala–One Night, JDRF will be honoring Doug Melton Ph.D. and Family, as well as Vertex Pharmaceuticals, for the incredible research and development work both are doing in type 1 diabetes (T1D) research, specifically in cell therapies.

For over 30 years, T1D has been a family affair for Doug Melton, Ph.D., his wife Gail O’Keefe, daughter Emma, and son Sam. In 1991, Sam was unexpectedly diagnosed with T1D at the age of 6-months. Sam’s diagnosis, while initially terrifying for Gail and Doug, ultimately set the course for the family. Doug’s work in the lab changed and he put his entire focus on finding a cure for diabetes. He looked to embryonic stem cells with the ultimate goal of turning them into islet cells to treat patients. Gail became the expert of day-to-day diabetes management, and her focus went to caring for her infant son. Emma, four years older than her brother, would herself be diagnosed with T1D ten years later.

With help from a JDRF grant in 2000, Dr. Melton found success in 2014, creating an insulin-producing beta cell from stem cells. In 2015, Semma Therapeutics (named after Sam and Emma) was founded to develop transformative therapies for insulin-dependent patients, with a focus on generating large quantities of these beta cells, that others could repeat. In 2017, the JDRF T1D Fund made a catalytic investment in Semma, and by 2019, Vertex Pharmaceuticals acquired Semma.

Douglas Melton, Ph.D., Gail O’Keefe, and Family
It’s wonderful to be recognized by the organization that shares my own research mission, namely to find a way to relieve the burden of glucose monitoring and insulin injections by providing type 1 diabetics with their own insulin factories: an islet cell transplant.  – Douglas Melton, Ph.D.

Thank you to JDRF for honoring my husband Doug Melton, and to Doug for his tireless work to end this disease. We also want to express our gratitude for the supportive community that JDRF provides, which was so important to us when type 1 diabetes turned our lives upside-down.  – Gail O’Keefe

Vertex is focused on discovery, developing, and commercializing innovative medicines so people with serious diseases can lead better lives. They are investigating and advancing cell therapies aimed at treating an underlying cause of T1D – the absence of insulin producing cells. In February 2021, they announced a clinical trial for the stem cell-derived therapy for use in people with T1D. In June, the first patient got an infusion of insulin producing cells, derived from stem cells, just like the cells in his pancreas that he had lacked for many years. By October, it was announced that after 90 days, he now needed 91% less insulin than before, and this was with half the target dose. While this was an early phase safety trials, the results are more than encouraging and next phase trials are in motion.

Vertex Pharmaceuticals
Vertex is honored to be recognized by JDRF at the upcoming 
One Night Gala. We are working tirelessly to bring potentially transformative cell therapies to people with type 1 diabetes. Our work would not be possible without the patients who participate in clinical studies, the health care professionals who conduct those studies, and JDRF, whose long-time leadership and commitment to advancing research and advocating for people with type 1 diabetes and their families is changing lives.  – Reshma Kewalramani, M.D., CEO, Vertex Pharmaceuticals

It’s a busy and exciting time for the development of these therapies – and JDRF continues to support the field and a variety of tactics to get these cells safely and effectively into people with T1D. For the laser-focused efforts of Dr Melton, the support of the Melton family, and the resources put into this effort by Vertex, we are excited to honor you on May 7.

Be the Momentum. Be the Progress. Be the Cure.

We hope you will join us on Saturday, May 7, 2022 as we return to the ballroom and raise critical funds for T1D research. The evening will feature exciting silent and live auctions, cocktails and dining, live entertainment, and inspiring messages from members of our T1D community. Unable to attend in person? Register to become a Virtual Participant by visiting Bid on the incredible silent auction packages and stream the Gala program live!

JDRF is committed to event safety and is following all CDC, local, and organizational guidelines. For guests joining us in the ballroom, we will continue to communicate any changes to our COVID policy.