Historic FDA Advisory Committee
On Wednesday, March 30, an FDA Advisory Committee voted 6 to 4 that the current data on Amylyx Pharmaceuticals’ investigational treatment AMX0035 does not support it as an effective treatment for ALS. While we are disappointed, we have not lost hope.
This is not the final chapter of this story. It is important to remember the Advisory Committee’s vote is nonbinding. The FDA can exercise regulatory flexibility as provided by the FDA Drug Guidance issued in 2019, by its stated determination date, which is June 29, 2022. Due to the close vote, there is room to change minds. ALS organizations have committed to join together to help counter arguments raised by the Advisory Committee. We are not giving up hope!
We are grateful to the 27 people with ALS, ALS clinicians and other advocates, including our CEO, Andrea Pauls Backman, who participated in the open public hearing. Showing 35 Faces of ALS, one by one, to the committee, she stated, “There is an urgent need for access to safe and effective therapies and regulatory flexibility for unmet medical needs in ALS. We have no time to waste.” You can read her full comments here. The FDA received nearly 600 written comments, including one from the Foundation.
A Phase 3 study for AMX0035 is underway, which Senda Ajroud-Driss, MD is conducting at the Les Turner ALS Center at Northwestern Medicine. We are grateful for those people living with ALS who are participating in this study, the results of which will help inform the efficacy of this compound.
We’re proud to have worked closely with our fellow ALS organizations: MDA, I AM ALS, and the ALS Association. We will continue to work together for the benefit of the ALS community.
We need more options to treat ALS. We have no time to waste.
Research News from the Les Turner ALS Center at Northwestern Medicine
The 40+ research scientists at the Les Turner ALS Center at Northwestern Medicine investigate a wide range of biologic mechanisms in ALS basic research. We are pleased to announce the recent publication of new research from three of these extraordinary researchers: Robert G.Kalb, MD, Joan and Paul Rubschlager Professor of Neurology; Hande Ozdinler, PhD, Associate Professor of Neurology; and Evangelos Kiskinis, PhD, Assistant Professor of Neurology and Neuroscience.
Read the full article at our blog. We are proud of all the novel research coming from the Les Turner ALS Center at Northwestern Medicine, all thanks to our loyal donors.
Robert G. Kalb, MD, et al., Neurons undergo pathogenic metabolic reprogramming in models of familial ALS, Molecular Metabolism.
Hande Ozdinler, PhD, et al., Mitochondrial dysregulation occurs early in ALS motor cortex with TDP-43 pathology and suggests maintaining NAD+ balance as a therapeutic strategy, Nature Scientific Reports.
Evangelos Kiskinis, PhD, et al., Homozygous might be hemizygous: CRISPR/Cas9 editing in IPSCs results in detrimental on-target defects that escape standard quality controls, Stem Cell Reports.
April ALS Learning Series: Veterans Helping Veterans with ALS: Department of Veterans Affairs Benefits and Advocacy
Mark your calendars for April 28 from 12pm – 1pm CT for the next webinar in our ALS Learning Series, “Veterans Helping Veterans with ALS: Department of Veterans Affairs Benefits & Advocacy.” If you are a veteran living with ALS and need help navigating the Veterans Administration system, this webinar is for you!
The ALS Learning Series webinars are produced as a free service by the Les Turner ALS Foundation.
Presenters Robert Statam, Sr., National Service Office, Paralyzed Veterans of America, and Winston Woodard III, Senior Benefits Advocate, Paralyzed Veterans of America, are experienced in VA benefits and legally represent veterans, dependents, and survivors.
Presenter Shelly Hoover is an author, veteran, gratitude guru, lover of life, and an ALS warrior and a member of the I AM ALS Veterans Team.
This webinar will cover VA benefits such as disability and caregiver compensation; grants for home and vehicle modifications; medications and supplies; and home health care and equipment.