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Think back to the summer of 2014. Social media feeds were filled with videos of people getting drenched with ice-cold water to raise money and awareness for ALS, the neurodegenerative disease that affects about 20,000 people in the U.S. each year. It was an ingenious campaign, providing an opportunity for laughter and bonding, all while raising funds at a scale for ALS that had not been seen before.
The Ice Bucket Challenge ultimately raised more than $115 million for the ALS Association. Those funds have since been invested in more than 130 research projects around the world. This money has been critical in developing treatments for ALS and improving the quality of care for people who live with the disease.
ALS is a devastatingly cruel disease. It gradually robs people of their ability to walk, talk, and eventually breathe. Most people who are diagnosed only survive for two to five years. While the disease was first identified in 1869, the medical and scientific fields still lack critical knowledge about its causes and origins in most people. There is no cure and currently available medications for ALS have limited effectiveness.
In late September 2022, Amylyx Pharmaceuticals' AMX0035 became only the third drug to win approval by the U.S. Food & Drug Administration as a treatment for ALS and philanthropy played a major role. Justin Klee and Joshua Cohen, co-founders and CEOs of Amylyx, credit multiple philanthropic partners for support at critical junctures in their journey to bring a new ALS drug to the market.
The organization Target ALS invited Klee and Cohen to its annual conference, which helped them make connections that would help fund their biotechnology startup. Amylyx collaborated with Project ALS to conduct pre-clinical studies of AMX0035 at the organization's Therapeutics Core based at Columbia University. A $3 million grant from the ALS-ACT Program, funded by The ALS Association and ALS Finding a Cure, helped the company start its Phase 2 clinical trial. And in turn, this clinical trial, called the CENTAUR trial, was conducted within the Healey & AMG Center Platform Trial for ALS - an innovative clinical research platform made possible through a transformative philanthropic gift to Massachusetts General Hospital by Sean M. Healey and the AMG Group.
Amylyx -- which will sell its new drug under the name Relyvrio -- is only the third drug approved in the 150-plus year history of ALS. But it exemplifies the outsize impact that philanthropy can have for an uncommon disease with critical unmet medical need. It's clear there's still much more to do - the three drugs now available are important tools in the ALS therapeutic toolbox but are not yet the transformative treatments needed by people living with ALS.
But philanthropy can serve as a critical lever to chart new paths forward. How? The Milken Institute Center for Strategic Philanthropy conducted a landscape analysis to understand the ALS research landscape and uncover where additional philanthropic focus and investment are needed. In conversations with more than 40 experts from around the world, review of the scientific literature, and examination of public and private funding patterns and trends, there are six key opportunities.
- Support basic research and discovery science. To diagnose, treat, cure and eventually prevent ALS, scientists and doctors must have a basic understanding of the disease across the full diversity of people living with ALS. Robust, sustained commitment to basic and discovery-based ALS research will support the entire continuum of ALS research and care that relies on this knowledge.
- Improve the understanding of ALS epidemiology and etiology across the full diversity of people living with ALS. Experts across the ALS field emphasized the need for a more thorough accounting of the prevalence and incidence of ALS and a clearer understanding of the disease's causes and course in people living with ALS from diverse backgrounds. Funding for research of the epidemiology and study of non-inherited risk factors for ALS has been chronically underfunded, and more support is critically needed here.
- Expand and improve the effectiveness of the therapeutics toolbox. Public and private funders are increasingly focusing their efforts on developing biomarkers and therapeutics for people living with ALS, and this area of research has a great deal of momentum behind it. Experts emphasized three primary needs here: more rigorous validation of biomarkers, more rational therapeutic development against mechanistic targets that have solid evidence of their involvement in ALS pathology, and an increase in the number of clinical trials that use innovative design and improve accessibility.
- Facilitate expanded access to clinical research initiatives and high-quality care. Multiple experts emphasized the need to increase equitable access to clinical research opportunities and quality ALS medical solutions within and outside of the U.S. People living with ALS experience barriers accessing timely diagnosis, clinical trials and other research studies, multidisciplinary care, and assistive technologies to improve quality of life. In addition to posing essential ethical considerations, these disparities hinder scientific progress because they limit a full understanding of ALS and the development of treatment options that work for everyone. Philanthropy is a critical resource that can sustain a commitment to increase access and inclusion to research and care.
- Build consensus and cohesion within the ALS research ecosystem. Our analysis revealed a near-unanimous desire for more cooperation, cohesion, and consensus within the ALS research system. Philanthropy is uniquely suited to help build and sustain collaborative research efforts, integrate new voices and perspectives into the field, and facilitate partnerships built with transparency of funding and scientific priorities at the forefront.
- Invest in scientific talent from diverse and multidisciplinary backgrounds. The challenges presented by ALS demand new perspectives, multifaceted expertise, and an "all hands-on deck" approach. Philanthropy can catalyze investment in the people who drive innovation and generate the knowledge that can lead to medical breakthroughs.
Relyvrio might only be the third drug approved for the treatment of ALS, but its path of development and approval is a testament to the power of philanthropy to make measurable progress in a rare disease field. Supporting new ideas and fresh perspectives, building critical research infrastructure to accelerate progress, and spurring cohesion and community-building by bringing people together are just some of the ways in which biomedical philanthropy can be transformational.
Candid.org published a version of this article on Oct. 27, 2022.