Celebrating the Successes of ALS Research Today, While Working to Fund More for the Future

Research-Success-Blog-Header

UPDATE – APRIL 2024: Relyvrio was voluntarily withdrawn from the market by Amylyx following a phase 3 trial that failed to show it was effective. The ALS Association stands by its decision to push for early approval of Relyvrio given the promising phase 2 trial data and the safety of the treatment. At the time, we said that if it turns out to be ineffective, at worst, people living with ALS would have taken an ineffective therapy without risk of harm. If it was indeed effective, delaying access would have meant that people living with ALS would have lost two years of being able to take a life-extending therapy. In the interests of transparency and education, we are leaving this information up for future reference. People living with ALS need life-saving treatments and we are working as urgently as possible to advance the many more potential treatments in clinical trials.

This last year has seen incredible progress in the fight to create a world without ALS. The FDA approval of Relyvrio (AMX0035) in 2022 and Qalsody (tofersen) last month are significant steps in the effort to make ALS a livable disease. These treatments are the second and third to be approved to treat ALS in the last six years, a pace never before seen by the ALS community.

Both of these new treatments offer real-world benefits for people living with ALS. A combination of two drugs, Relyvrio has been shown in clinical trials to extend life by more than six months. Qalsody is the first gene-based therapy to be approved that targets a specific ALS gene mutation, and the underlying technology behind the drug is very promising, offering the potential for additional similar treatments in the future.

Image
Genetics-Blog-Body

But the seeds of these successes were planted years ago. The ALS Association was the first to fund research of the ALS-specific antisense (ASO) technology that led to Qalsody back in 2004. Over the last two decades, we have committed more than $1.3 million to the development of this emerging technology. And Relyvrio is the first FDA approved drug developed with funds raised from the ALS Ice Bucket Challenge.

As we celebrate the success of these long-term investments, we continue to fund the next generation of possible treatments, as well as the researchers that are searching for them.

Earlier this year we awarded nearly $800,000 to support 16 innovative research projects with the potential to significantly impact the experience of ALS by optimizing current care and treatments, finding new treatments and cures, and aiding with diagnosis and prevention. These grants—the first awards made through The ALS Association’s Seed Grant Program—provide one year of support (up to $50,000) so researchers can gather the data they need to secure follow-on funding and partnerships.

Pursuing new and innovative lines of research often becomes a ‘chicken or the egg’ scenario for investigators. They can’t secure funding without data, but it’s difficult to collect data without funding. Our Seed Grant Program helps fill that initial funding gap so researchers can produce data for early go/no-go decisions on novel and potentially high-impact research directions in ALS.”
Dr. Paul Larkin
Director of research at The ALS Association

Further down the drug development pipeline, we also awarded $1.6 million to fund four promising preclinical research projects through the Lawrence and Isabel Barnett Drug Development Program. This program provides researchers with up to $500,000 over two years to support the preclinical assessment of emerging ALS therapies in preparation for clinical testing, prioritizing projects that have a high probability of entering the clinical phase of testing within three years.

Initiatives like our Barnett Drug Development Program drive the crucial transition from academic research project to industry drug development effort. This support makes additional investments in the next stage of drug development less risky for other private and public funders, increasing our chances of finding new treatments and possibly a cure for this devastating disease.”
Dr. Paul Larkin
Director of research at The ALS Association

Expanding on our commitment to clinical trials—the most reliable and fastest way to translate promising laboratory science into new and better ways to treat and care for people living with ALS—we launched our inaugural Trial Capacity Awards.

Image
Patient-Clinic-Setting

In total, the program awarded $4.9 million to support efforts at 13 established and emerging ALS clinical trial sites to increase the number and diversity of people living with the disease who have the opportunity to participate and improve the efficiency and pace at which these studies are conducted. “Expanding clinical trial capacity will allow potential new therapies to be tested faster and will improve trial accessibility for people with ALS in currently underserved areas,” says Dr. Larkin.

Continuing the search for new and innovative ways to diagnose and treat ALS, The ALS Association and the Focused Ultrasound Foundation recently announced a new partnership to advance transformational treatments for people with ALS. Leveraging a $500,000 ALS Association Partnership Grant and $500,000 in matching funds from the foundation, this new collaboration will support research into diagnosing and treating ALS using focused ultrasound technology.

Never before in the history of this disease have we seen so many talented scientists and researchers working together, urgently looking for new treatments and cures, providing even more hope for the families we serve. And we will not stop until ALS becomes a livable disease for everyone, everywhere.

Learn more about ALS and how you can make a difference for people impacted by the disease.

To continue to follow stories about people living with ALS in the community and learn more about the disease, subscribe to receive our weekly blogs in your inbox HERE or follow us at als.org/blog.

Subscribe

If you would like to receive monthly ALS research updates, SUBSCRIBE to our newsletter, Research Matters.

Subscribe

We Want to Hear From You!
Your feedback is important to us. Please let us know what you think about our blog – take this short survey and provide your input and feedback.

Join the conversation. Please comment below.

CAPTCHA
7 + 6 =
Solve this simple math problem and enter the result. E.g. for 1+3, enter 4.
This question is for testing whether or not you are a human visitor and to prevent automated spam submissions.