Guest Voice: ALS advocacy matters now more than ever

The United States Congress recently passed legislation to support five more years of ALS research and expand access to promising experimental therapies. We cannot afford to waste a moment.

On Sept. 28, Congress passed the Accelerating Access to Critical Therapies for ALS (ACT for ALS) Reauthorization Act, which would extend key research, infrastructure, and expanded-access programs through 2031. The bill now awaits the president’s signature.

For families who confront the devastating realities of this disease each day, reauthorization cannot be the end of the story. If the act is signed into law, the U.S. Food and Drug Administration (FDA) must match Congress’ urgency by using the tools it already has to advance safe and promising therapies for patients.

Recommended Reading

June 23, 2023 Columns by James Clingman

Let’s step up our advocacy efforts for ALS research

Time is critical

An ALS diagnosis can upend an entire family overnight, thrusting loved ones into intense caregiving roles with little warning and even less support. On average, people with ALS survive two to five years after symptom onset, forcing families into an around-the-clock caregiving reality almost immediately. As patients lose the ability to walk, speak, eat, or breathe independently, family members can become the ones coordinating care, managing medical equipment, administering treatments, and making difficult decisions.

ALS also sends households into financial spirals. Families shoulder an average of $122,000 per year in late-stage ALS care costs, largely because health insurance rarely covers the round-the-clock support that ALS requires.

But the cost cannot be measured in dollars alone. Caregivers sacrifice sleep, work, relationships, and their own health as they watch someone they love lose function. And, unlike many other serious conditions, ALS can progress so quickly that families are constantly rushing to adapt to new levels of need.

For caregivers, any advancement in treatment represents the possibility of more time. More time before a loved one loses a critical function, more time for families to spend together, and possibly more time for the caregiver to continue working, parenting, and maintaining their own health and responsibilities.

Many caregivers report emotional strain, physical exhaustion, financial pressure, social isolation, and reduced time for their own self-care. The result is a disease that places enormous pressure not only on patients but also on the families and communities supporting them.

That’s why the ACT for ALS Reauthorization Act matters to caregivers as much as it matters to researchers and clinicians. The bill recognizes a fundamental reality of ALS: Patients can run out of time while science and drug development continue to advance. It supports ALS research and infrastructure and provides a pathway for certain patients who cannot participate in clinical trials to access investigational therapies. Reauthorizing these programs would give researchers, clinicians, and patients another five years to turn promising science into meaningful treatment options.

Making room for innovation

But congressional action is only part of the equation. We also need a drug-development and regulatory system capable of recognizing and responding to innovation. The FDA has tools, such as the Accelerated Approval Program, that allow earlier sign-off for drugs that treat serious conditions and address unmet medical needs, based on evidence that it’s reasonably likely to demonstrate real benefit. This pathway has already helped patients with other rare and devastating diseases access treatment years sooner, offering a proven model the agency could apply more consistently to ALS.

With ALS, every month of delay can bring more physical decline for patients and more strain for caregivers and families who are already operating at capacity. Expanding treatment access doesn’t just unlock possibilities for patients; it can ease the load on caregivers and the systems supporting them, from hospital staff to home health aides to family members filling in the gaps.

Congress has now reaffirmed the urgency of the fight against ALS. If the president signs the bill into law, the next step will be to make those five years count: by continuing to invest in innovative research, strengthening the path from discovery to clinical trials, and implementing regulatory tools to evaluate promising therapies so that patients can access innovative treatments when appropriate.

Families facing ALS do not need promises of “someday.” They need a system committed to finding new answers while there is still time to benefit from them.

Hopefully, ACT for ALS will give the ALS community another five years to make progress. For people living with ALS and the caregivers who stand beside them, the focus must be to use those years to turn innovation into hope, and, ultimately, more time.

Government regulators and agencies should use every available tool to advance safe, promising therapies. Congress’ approval should also inspire the FDA to consider the needs of caregivers and the 35,000 Americans living with ALS by fast-tracking promising treatments for neurodegenerative diseases. Patients and caregivers can’t wait.

To submit your own Guest Voice for publication on ALS News Today, please email your idea to our community editorial manager at [email protected] with the following included in the subject line: “Guest Voice: ALS News Today.”

Note: ALS News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of ALS News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to ALS.