Research at Sutter Health Brings New Hope to People With ALS
SAN FRANCISCO, Calif. — Stephen Hawking—one of the world’s most accomplished physicists— lived most of his life with amyotrophic lateral sclerosis (ALS). His lifetime of accomplishments and worldwide renown cast an international spotlight on the debilitating disease.
ALS, commonly called Lou Gehrig’s disease, is a progressive neuromuscular disease that affects nerve cells in the brain and spinal cord, causing progressive paralysis. The disease usually strikes people between the ages of 40 and 70. Approximately 16,000 Americans have the disease at any given time.
80% cases of ALS are called “sporadic,” meaning the cause(s) of the disease are unknown, and 10-20% of cases are due to genetic mutations. If two or more family members in a given family have ALS, the disease is considered “familial”.
We interviewed Liberty Jenkins, MD, a neuromuscular and general neurologist at the Forbes Norris ALS/MDA Research and Treatment Center at California Pacific Medical Center. She shared her perspectives on the latest ALS research and clinical trials throughout Sutter Health—and how they may lead others to pursue their goals, their dreams, their life’s work despite receiving a devastating diagnosis.
What are the most pressing challenges in treating ALS today, despite advances in research and new treatment approaches?
The most pressing challenge clinicians and patient advocates are grappling with is finding a treatment that is effective in slowing the progression of disease. Despite decades of scientific research, there are still only two FDA-approved medications to treat ALS. Both approved treatments have limited effectiveness in halting the persistent degeneration of motor nerves in the brain and spinal cord associated with ALS.
To make real progress in the treatment of ALS, I think researchers need to focus on clinical trials of novel treatments that involve the simultaneous evaluation of multiple agents. These trials are sometimes called platform trials and have been done successfully in testing treatments for cancer. Combining platform trials with the identification of robust biomarkers (a measurable indicator of the severity or presence of disease) for early diagnosis and disease progression will allow researchers and clinicians to more effectively and rapidly evaluate potential new therapies for the treatment of ALS.
you most excited about when we look to new advances in treatment on the horizon, or
advances in our understanding of ALS?
At the moment, the most exciting advances in ALS treatment are in the treatment of hereditary or genetic ALS. Newer treatments called “anti-sense oligonucleotides” (ASO, some that are only in the early stages of development) are designed to block the effects of genetic mutations linked to the illness. Unfortunately though, ASOs only benefit a minority of people with ALS because only 10-20% of cases are due to genetic mutations.
But for those individuals with a genetic form of ALS, ASO therapies may well prove to be transformational. The same mutation blocking technique underlies two recently FDA-approved therapies for neuromuscular diseases that were previously fatal in children and young adults. At the Forbes Norris ALS/MDA Research and Treatment Center at California Pacific Medical Center, we are excited to be actively recruiting for a Phase 1 trial that will test the safety of a treatment that blocks a genetic mutation associated with the disease.
How are neurology researchers throughout Sutter working to develop new therapies?
We are participating in the NurOwn Brainstorm stem cell clinical trial, a trial evaluating a treatment for patients who have sporadic ALS. This is a Phase III, randomized, double-blind, placebo-controlled study of stem cells harvested from the patient’s own bone marrow that have been engineered to secrete special growth factors.
The trial has been received with great excitement in the ALS community because of promising early-phase results. The Forbes Norris ALS Clinic at California Pacific Medical Center is one of only six sites nationwide that was selected to participate in the trial. To date, we have enrolled approximately 75% of study participants nationwide.
In addition to the Phase 1 trial of ASO therapy for an inherited/familial form of ALS mentioned above, we also have several other active ALS studies at the Forbes Norris ALS Clinic at California Pacific Medical Center. Collectively, these trials and new approaches to studying diverse neuromuscular disease represent Sutter’s commitment to participating in cutting edge clinical research and trials.
What sparked your latest “ah ha” moment in your work/ALS research?
It’s been illuminating to collaborate with colleagues aligned with the general, growing understanding that ALS is not just one illness but instead develops because of multiple abnormal pathways. We are slowly chipping away at the complexities that underlie ALS, and this gives me renewed hope for the patients we care for.