Second Congressional Briefing Held on Sickle Cell Disease
November 19, 2019
On November 19th, 2019, the second congressional briefing on sickle cell disease (SCD) was held in Washington, DC. The American Society of Gene and Cell Therapy (ASGCT) co-hosted with Congress members Barbara Lee (D – CA), Michael Burgess (R – TX), and Danny Davis (D – IL). Briefing updates included ways Congress can support SCD, progress with gene therapy, and a patient’s view about the topic. Providers, Advocates, Patients, and Lawmakers participated in the briefing.
The briefing began with lawmakers being asked to co-sponsor a bipartisan resolution that calls for new sickle cell trait research. Featured in the resolution are the need for more research, better data collection, access to screening, better public education, and counseling for sickle cell trait. At present, only Ms. Lee, Mr. Davis, and Mr. Burgess sponsor this motion.
Dr. Matthew Porteus then gave an overview of SCD and Gene Therapy, a new approach to treating the disease. Dr. Porteus is an Associate Professor at Stanford School of Medicine and is partially funded by the National Institutes of Health (NIH) to conduct research on these topics. He also is an active participant on a scientific committee in the Cure Sickle Cell Initiative, a program of the NIH.
Next, Jenelle Stephenson shared a patient view of gene therapy. She lives with sickle cell disease and recently participated in a gene therapy clinical trial. Ms. Stephenson, who was featured on the CBS TV show 60 Minutes, discussed the issues she faced before going through gene therapy. She also shared how much her life has changed two years after and the resolution of many of her symptoms.
Mr. Burgess and Mr. Davis then shared how and why they are involved with SCD.
Finally, Remy Brim, Ph.D. and Vice President of BGR Group, shared how lawmakers can support SCD by:
- Supporting NIH funding and initiatives.
- Helping set up a strong public health system to support issues like newborn screening.
- Joining the motion discussed.
- Supporting the CDC Sickle Cell Data Collection Program.
- Urging for payment models that can be used now so the system is prepared when new treatments are ready.
The first briefing on SCD was hosted on June 18, 2019. To read more about both briefings, visit the American Society of Gene & Cell Therapy (ASGCT).