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The FDA’s decision to delay its highly anticipated review of AMX0035, a new drug for treating amyotrophic lateral sclerosis, until September highlights an ongoing rift between the needs of people directly affected by ALS and those responsible for bringing forth treatments and cures.

ALS is a devastating neurodegenerative disease that typically kills within three years of diagnosis. People living with it do not have time to waste. Many of them also have strong opinions and keen insights into treating the disease. They and their family members must be engaged as partners — not just as patients or “subjects” — before decisions are made about treatment options, clinical trial design, research, health care policy, and anything else that affects their lives.

My father, Jacob Gamer, was diagnosed with ALS in October 2020, and died of it a year later. Not long after his diagnosis, I joined a team of people living with ALS and their caregivers led by trailblazing advocate Sandy Morris. Our mission was to address barriers faced by people living with ALS to meaningful participation in forums that could affect their treatment, care, and support.

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Members of the team played leadership roles in important achievements, such as the recent passage of the Accelerated Access to Critical Therapies for ALS Act. Many of them, however, have also experienced instances of exclusion or tokenistic engagement by health care providers, the policy community, researchers, and nonprofits. The status quo approach has failed to produce treatments and cures since ALS was first identified more than 150 years ago.

Decision-making processes that lack inclusivity and transparency impede progress. History has shown that when people take charge of their own disease, the road to treatments and cures is faster and more equitable. HIV/AIDS is a good example of this. Meaningful collaboration with people affected by it was essential to developing more effective systems of care, while shaping research and drug approval processes that paved the way for groundbreaking discoveries. That movement was driven in part by the Denver Principles, a simple but powerful statement of rights and recommendations for people living with AIDS written in 1983 that opens like this:

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We condemn attempts to label us as “victims,” a term which implies defeat, and we are only occasionally “patients,” a term which implies passivity, helplessness, and dependence upon the care of others. We are “People With AIDS.”

The systemic challenges bogging down ALS must be addressed from a fresh, more inclusive perspective.

Inspired by the Denver Principles, our team met weekly over the course of several months to map out our own social contract with professionals and organizations focused on ALS. We named this contract The Morris ALS Principles in honor of Sandy Morris, whose tenacity, wisdom, and courage are an inspiration to continue fighting until treatments or cures are found.

The Morris ALS Principles set out five key expectations for all stakeholders that will improve partnerships with the ALS community by centering and amplifying the needs of people with the disease. They include:

  • Protect our intellectual, physical, and financial dignity.
  • Be global stewards of our disease and respected partners in the science of treatments and cures.
  • Act as trusted peers with clinicians, researchers, and policy-makers.
  • Fight for equity in decision-making.
  • Lead to end ALS and other motor neuron diseases.

The principles call for health care providers to share timely, comprehensive information about approved and investigational therapies, genetic testing, and counseling to maximize opportunities for admission into clinical trials. The scientific and research communities are expected to foster participation by people living with ALS in conferences, meetings, clinical trial protocol teams, and other advisory bodies. The policy community, including regulatory bodies, is expected to exercise appropriate urgency and flexibility to approve new treatments while executing pathways to access experimental therapies. Nonprofit organizations should be guided by people living with ALS in all of their efforts, and involve them in every facet of operations.

Community empowerment is critical to shaping a timely government response and, more broadly, a health care system that adequately addresses the needs of people living with ALS.

The Morris ALS Principles team aims to enforce these boundaries and expectations by measuring the degree to which the community feels empowered and involved in their own decisions. We meet weekly to issue accolades for actions demonstrating adherence to the principles and discuss areas where citations for non-compliance are warranted. Institutions at the cutting edge of ALS care enthusiastically embrace the principles because they recognize that empowering people living with ALS is directly correlated to their own reputations, influence, and success.

The process of creating, writing, and implementing a guide such as the Morris ALS Principles could be worth emulating by other organizations that have yet to effectively advocate for their priorities. Lived experience must be afforded appropriate weight in a range of health care decisions. Without clearly defining boundaries, it will be hard to expect regulators, clinicians, researchers, nonprofits, or other influential stakeholders to change their approaches.

Irene Shapiro is an attorney and a volunteer advocate for I Am ALS’s clinical trials and legislative affairs teams.

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