A Doctor's Plea From a Nation Asleep on Brain Disease

Newsweek

01-08-2025

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As a physician who once treated pain, I now endure the unimaginable. Amyotrophic lateral sclerosis (ALS) has left me quadriplegic, dependent on a tracheostomy to breathe and a feeding tube to eat. Diagnosed at 35, my career ended abruptly. My family's story reveals the scale of the coming storm—my father battles Alzheimer's, my uncle succumbed to Parkinson's, and my grandmother to Lewy body dementia. We are not outliers; we represent a silent epidemic. Neurodegenerative diseases are surging, yet our nation slumbers, unaware of the devastation ahead.
The public's complacency is by design, built on a statistical illusion. ALS, fatal since 1869, exemplifies this peril. With a median survival of 2-3 years, it strikes about 1 in 300 people in their lifetime, which predicts that over 1 million people now alive in the U.S. will succumb to ALS. Yet, under federal law, it is labeled "rare" because that definition is based on prevalence—a static snapshot of how many people are living with a disease at one time. Because ALS kills its victims so quickly, the number of living patients stays below the 200,000-person "rare" threshold. Its very lethality ensures it is never treated like the mainstream public health crisis it is. This paradox obscures a terrifying forecast: a projected 69 percent global increase in ALS cases by 2040.
This illusion of rarity perpetuates a deadly inaction that extends to all brain diseases. Alzheimer's already affects over 7 million Americans and is projected to strike nearly 13 million by 2050, costing our economy $384 billion in 2025—and projected to nearly $1 trillion annually by mid-century.
Exterior view of the headquarters of the U.S. Food and Drug Administration (FDA).
Exterior view of the headquarters of the U.S. Food and Drug Administration (FDA).
Getty Images
The failure to confront this crisis stems from a Tale of Two Agencies within the Food and Drug Administration (FDA). In 2017, Congress established the Oncology Center of Excellence (OCE), a dynamic hub that has revolutionized cancer treatment, accounting for 85 percent of all accelerated approvals in the last decade. This success is the result of focused will and resources; National Institutes of Health (NIH) funding for cancer has topped $7.2 billion, compared to $2.8 billion for neuroscience.
Neurology has no such center. Lacking an institutional home, it is fragmented, slow, and characterized by a risk aversion unthinkable in oncology. This disparity persists because of the tragic nature of these diseases. In the 1980s, ACT UP activists staged "die-ins" to force a reluctant government to fight AIDS. Patients with ALS, Huntington's, or Alzheimer's cannot mount a similar protest—we are physically silenced and immobilized, unable to "seize the FDA." This vulnerability places a unique moral obligation on our leaders to act proactively on behalf of the voiceless.
The new FDA leadership now arrives with bold promises of change, posing a question that haunts everyone touched by an untreatable neurological disease: Will this time be different? Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. vows to "sweep away barriers" and to "figure out new ways ... of accelerating approvals for drugs and treatments that treat rare diseases." FDA Commissioner Marty Makary has several times asked why it takes 10 years for a drug to get to market and proposes a "conditional approval" pathway based on a "plausible mechanism." Center for Biologics Evaluation and Research (CBER) Director Vinay Prasad promised to "take action at the first sign of promise for rare diseases."
For the ALS community, these words are now a crisis of credibility. In a disease that has a median survival of 2-3 years, we are asking for regulatory flexibility for a treatment that began its Phase 1 trial in 2011 and was granted FDA Fast Track designation in 2014. As a petitioner on the July 3 citizens' petition for a stem cell therapy known as NurOwn, my community has presented the FDA with a clear test. Ultimately, we are seeking accelerated approval based on new, unprecedented survival data from an expanded access program—data that exceeds the extension of survival of many approved cancer therapies. But the initial request is simpler: invite the sponsor to resubmit its application for a full review. Ours is a request for due process to give a voice to the voiceless—the lowest possible bar for the Trump administration to demonstrate its promised flexibility.
A clear, bipartisan solution has already failed once. The Neuroscience Center of Excellence Act, introduced in 2021 to replicate oncology's success for brain disease, stalled in committee. It is time for our leaders to find the political will that has been so catastrophically absent.
Congress must immediately revive and pass the Neuroscience Center of Excellence Act. The FDA, in turn, must match its leaders' promises with action by granting our petition a review. The science is poised for breakthroughs, but it is being shackled by a broken system.
For those of us on a deadline, this is not a policy debate. It is a death sentence. Awaken now, before this silent storm engulfs us all. The voiceless can't wait.
Dr. Shahriar Minokadeh, a former anesthesiologist trained at Johns Hopkins and pain management at UC San Diego, types via an eye-gaze device.
The views expressed in this article are the writer's own.