On this episode of the podcast, Terry and Bob discuss the latest medical research into ALS in conjunction with ALS Awareness Month. Dr. Sharon Hesterlee, the Chief Research Officer at the Muscular Dystrophy Association, joins the show to give the big picture on ALS research and other neuromuscular diseases.
She highlights new disease-specific, bipartisan ALS legislation. It has three main components: greater funding for pharmaceutical companies looking to offer expanded drug access, public-private partnerships to bring more people together to look for treatments, and grants for research into ALS and other neuro-degenerative research.
Dr. Hesterlee highlights the latest ALS research that targets the gene mutation that triggers the disease. She also discusses a new registry that can help track ALS symptoms and treatment data. Terry points out how iCER plans to do an ALS treatment review, which insurance companies may use to deny coverage.
Terry and Dr. Bob discuss new health legislation, the Protecting Healthcare for All Act, that would ban the QALY. This discriminatory healthcare metric hurts patients with rare or chronic diseases. Bob explains why the QALY is problematic, illustrating how it puts a price cap on treatment for an extra year of life. The QALY discriminates against the sickest and most vulnerable patients. Bob argues that it’s political theater at its worst because it deals with people’s lives.
Kate speaks with Jenny Jones, an advocate for Familial Adenomatous Polyposis (FAP), about her advocacy work for this rare genetic condition. Jenny explains her condition’s gastro-intestinal consequences and recounts her mental-health struggles as a child growing up with it. She highlights her advocacy to try to help children overcome the many challenges facing those with this condition. She concludes with a call for more colon screening.