My last column addressed legislation I have supported to better overall health care. This week, I want to focus on neurodegenerative diseases. The most notable of these diseases are Amyotrophic lateral sclerosis (ALS), Huntington’s, Alzheimer’s, and Parkinson’s.
On July 29, the House Energy and Commerce Committee’s Subcommittee on Health held a hearing entitled “The Path Forward: Advancing Treatments and Cures for Neurodegenerative Diseases.”
During this hearing, my colleagues and I questioned a panel of agency administrators from the National Institutes of Health (NIH) and the Food & Drug Administration (FDA). We had a second panel, which consisted of physicians and patient advocates. The two panels discussed the complexities of these diseases and the government’s role in providing resources and developing treatments.
My lines of questioning to the panels focused on ALS, also known as Lou Gehrig’s disease, and Huntington’s disease. Though these diseases are relatively rare, they affect several people I know or knew personally. Consequently, I have a special interest in furthering and perfecting research in these areas.
Neurodegenerative diseases cause nerve cells to break down over time. ALS primarily impacts physical function through deterioration of the muscles, leaving the mind intact. Huntington’s disease, on the other hand, affects physical and mental abilities by attacking the brain as well as the body. Both eventually lead to the loss of movement and speaking capacity, meaning patients require round-the-clock care.
Even after decades of investigation, no cure has been found for either ALS or Huntington’s disease, and there are very few treatment options. While unique challenges accompany the study of diseases that occur infrequently, I believe the government can do more to advance this research.
A swimming friend of mine has been battling ALS for several years. Desiring to support efforts to learn about the disease, he enrolled in an NIH study. It was a study requiring only 25 participants. And yet, recruiting a mere 25 people took nearly a year. This is unacceptable, particularly given that the average life expectancy for folks with ALS is 3-5 years. I pushed Dr. Walter Koroshetz, director at the NIH, to increase recruitment process efficiency.
Further, because medical studies rely on participants from a wide variety of backgrounds, I emphasized the need to recruit patients from all geographic areas. Patients from certain ethnic groups and those who reside in rural areas are traditionally underrepresented in research studies. But telehealth technology is allowing that to change. I was encouraged to learn NIH is beginning to use this tool.
To offer my support for expanding research for ALS, I have co-sponsored HR 3537, the Accelerating Access to Critical Therapies for ALS Act. This bill would award grants to help facilitate patients’ access to investigational medications and fund further research and development of drugs used to treat ALS.
ALS is supposed to be a rare disease, but I have known a number of people who have had this malady. Among those who lost their battle were Mike Aheron, Julie Mullins, and my friend, former Delegate Ray Robrecht. These numbers seem high based on national averages, so I wanted to know if there is any evidence of geographic outbreaks. Or if environmental exposure could be a component of diagnosis.
Dr. Jinsy Andrews, of Columbia University, responded that exposure to environmental issues might be a contributing factor, but there is not yet enough research to prove a direct correlation.
While the cause of ALS is unknown, experts have proved that Huntington’s disease is genetically transmitted.
Another friend of mine from Salem died from Huntington’s disease. I asked Kala Booth, a Huntington’s disease caregiver and patient, about the medications currently being prescribed to treat Huntington’s. She replied, “There is nothing to treat it right now, but they (current drugs) are to slow the progression of it.”
She also shared that the lack of treatment and deterioration of their brains often leads to feelings of hopelessness, putting many at risk of suicide. I commend Miss Booth’s courage and resilience and the courage of all battling this disease.
In response to her testimony, I was happy to sign on to HR 2050, the Huntington’s Disease Disability Insurance Access Act. This bill would accelerate payment of Social Security Disability Insurance (SSDI) benefits and Medicare eligibility for those with Huntington’s.
Current treatment practices do little to actually cure these diseases and provide minimal physical changes for the patients. As someone who has witnessed the devastating impacts of some of these neurodegenerative diseases, I will continue to support legislation promoting research for treatments and cures.
For questions, concerns, or comments, contact my Abingdon office at 276-525-1405, Christiansburg office at 540-381-5671, or via email at www.morgangriffith.house.gov.
