When you have a disease like ALS, you learn how slowly medical research moves, and how often it fails the people it is supposed to save. You also learn how precious time is.

For decades, the dominant pathway for developing new drugs has relied on animal testing. Most of us grew up believing this was unavoidable: that laboratories full of caged animals were simply the price of medical progress. But experts have known for a long time that data tell a very different story.

The Los Angeles Times reported in 2017: “Roughly 90% of drugs that succeed in animal tests ultimately fail in people, after hundreds of millions of dollars have already been spent.”

The Times editorial board summed it up in 2018: “Animal experiments are expensive, slow and frequently misleading — a major reason why so many drugs that appear promising in animals fail in human trials.”

Then there’s the ethical cost — confining, sickening and killing millions of animals each year for a system that fails 9 times out of 10. As Jane Goodall put it, “We have the choice to use alternatives to animal testing that are not cruel, not unethical, and often more effective.”

Despite overwhelming evidence and well-reasoned arguments against animal-based pipelines, they remain central to U.S. medical research. Funding agencies, academic medical centers, government labs, pharmaceutical companies and even professional societies have been painfully slow to move toward human- and technology-based approaches.

Yet medical journals are filled with successes involving organoids (mini-organs grown in a lab), induced pluripotent stem cells, organ-on-a-chip systems (tiny devices with human cells inside), AI-driven modeling and 3D-bioprinted human tissues. These tools are already transforming how we understand disease.

In ALS research, induced pluripotent stem cells have allowed scientists to grow motor neurons in a dish, using cells derived from actual patients. Researchers have learned how ALS-linked mutations damage those neurons, identified drug candidates that never appeared in animal models and even created personalized “test beds” for individual patients’ cells.

Human-centric pipelines can be dramatically faster. Some are reported to be up to 10 times quicker than animal-based approaches. AI-driven human biology simulations and digital “twins” can test thousands of drug candidates in silico, with a simulation. Some models achieve results hundreds, even thousands, of times faster than conventional animal testing.

For the 30 million Americans living with chronic or fatal diseases, these advances are tantalizing glimpses of a future in which we might not have to suffer and die while waiting for systems that don’t work.

So why aren’t these tools delivering drugs and therapies at scale right now?

The answer is institutional resistance, a force so powerful it can feel almost god-like. As Pulitzer Prize–winning columnist Kathleen Parker wrote in 2021, drug companies and the scientific community “likely will fight … just as they have in past years, if only because they don’t want to change how they do business.”

She reminds us that we’ve seen this before. During the AIDS crisis, activists pushed regulators to move promising drugs rapidly into human testing. Those efforts helped transform AIDS from a death sentence into a chronic condition. We also saw human-centered pipelines deliver COVID vaccines in a matter of months.

Which brings me, surprisingly, to Robert F. Kennedy Jr. In December, Kennedy told Fox News that leaders across the Department of Health and Human Services are “deeply committed to ending animal experimentation.” A department spokesperson later confirmed to CBS News that the agency is “prioritizing human-based research.”

Kennedy is right.

His directive to wind down animal testing is not anti-science. It is pro-patient, pro-ethics and pro-progress. For people like me, living on borrowed time, it is not just good policy, it is hope — and a potential lifeline.

The pressure to end animal testing and let humans step up isn’t new. But it’s getting new traction. The actor Eric Dane, profiled about his personal fight with ALS, speaks for many of us when he expresses his wish to contribute as a test subject: “Not to be overly morbid, but you know, if I’m going out, I’m gonna go out helping somebody.”

If I’m going out, I’d like to go out helping somebody, too.

The post Animal testing slows medical progress. It wastes money. It’s wrong appeared first on The Hemet & San Jacinto Chronicle.

WASHINGTON (AP) — For two years, Becky Mourey pushed the Food and Drug Administration to approve an experimental drug for her Lou Gehrig’s disease.

She went to members of Congress and health regulators to make the case for Relyvrio, until patient-advocates finally prevailed.

In September, Relyvrio became only the third drug approved in the U.S. for ALS, or amyotrophic lateral sclerosis, an incurable neurodegenerative disease that is usually fatal within five years. But patients and physicians who celebrated Relyvrio’s approval several months ago are now contending with the obstacles posed by the U.S. health care system.

Their odyssey is an object lesson in the soaring cost of specialty drugs and the byzantine systems that insurers have created to try and control them.

Patients with insurance coverage say the $158,000 per year price tag set by drugmaker Amylyx Pharmaceutical is fueling insurance delays or denials, and sometimes exorbitant out-of-pocket expenses.

Mourey’s insurer initially refused to cover Relyvrio, but reversed itself seven weeks later after online pressure— including social media posts by Mourey and other ALS advocates.

“Amylyx priced it way too high,” said Mourey, 58, a former music teacher, who now communicates using a vision-controlled speech computer. “They could cut the price in half and still make a hefty profit.”

Even when Relyvrio is covered, some patients see themselves shouldering co-payments between $1,000 and $4,000 per month.

Unlike those patients, the Moureys have been exempt from copays due to a combination of company-managed Medicare and Medicaid coverage.

Some ALS patients are covered by private plans, while others receive government coverage through programs like Medicare.

In both cases, patients and their doctors can face onerous requirements to document their condition before insurers will pay for an expensive new treatment. Additionally, many insurers require patients to pay a percentage of their pharmacy costs. With many drugmakers now charging upwards of $100,000 for drugs approved for hard-to-treat illnesses, even a 5% copay can become unaffordable. The trend has increasingly made the drug industry deeply unpopular in the U.S.

Drugmakers say their prices reflect the years of costly clinical trials needed to win FDA approval.

Amylyx said the delays in coverage for Relyvrio are normal because many insurers are still writing their policies for the drug. The company also highlighted its multiple programs to help patients with costs, including providing the drug for free to the uninsured and covering copays for those who qualify for assistance.

The issues surrounding Relyvrio’s price highlight industry practices that have long been blamed for driving up U.S. health costs: drugmakers can patent new combinations of old ingredients and then charge whatever price the market will bear.

Insurers have responded with extra review hurdles. Those delays weigh heavily on patients with ALS which is measured in the monthly loss of functions like walking, speaking and swallowing.

“It’s just stressful and aggravating to get these multiple denial letters,” said Jim Mourey, who cares for his wife full time at their home in Hopkinton, Massachusetts.

Relyvrio is a powder-based formulation that combines two older ingredients: a prescription drug for liver disorders and a supplement used in traditional Chinese medicine that’s available on Amazon.com for about $1 per day’s supply.

Insurers typically won’t pay for ALS patients to receive the prescription ingredient, because it’s not approved for their disease. But some patients have gotten their plans to cover it or have found lower-price versions — for less than $600 a month— at specialty pharmacies. That’s led some patients to skip the new drug entirely by obtaining its two ingredients separately.

Analysts expect Relyvrio to generate $450 million in annual sales for Amylyx by 2024. It’s the company’s first product.

One group that analyzes drug pricing said a fair price for Relyvrio would be between $9,100 and $30,700 per year. The drug showed some effectiveness in slowing the progression of ALS, based on patient questionnaires. Amylyx is conducting a larger study to answer lingering questions about its effectiveness, including whether it extends survival.

Layne Oliff, 63, hasn’t yet decided whether to seek coverage from his Medicare plan. Under the federal program, he would likely owe $7,400 in copays for Relyvrio before reaching his out-of-pocket maximum. That’s on top of monthly administration fees of over $600.

But if he pays cash, Oliff says he can buy the prescription ingredient in the drug for $550 per month, or $6,600 per year, from a specialty pharmacy. He can get the supplement online for about $30 a month.

“You’re talking about a drug that’s not a cure, though it has helped slow the progression” said Oliff, who trained as a pharmacist. “But I don’t want to leave my wife someday in a financial hole; I’m not going to spend all my money on this drug.”

Amylyx has set up a program to cover the copay costs for patients with private insurance. But that financial assistance cannot be used for federal plans like Medicare and the Veterans Administration, which cover many ALS patients. After initially restricting coverage, the VA recently broadened its eligibility.

Other insurers are initially rejecting coverage or requiring multiple rounds of documentation from doctors.

“When someone’s lifespan is measured in months, making people go through these multiple rounds of review is just cruel,” said Neil Thakur of the ALS Association. The group invested in Amylyx’s early research and stands to recoup that investment.

Dr. Terry Heiman-Patterson, a neurologist at Temple University, says it’s taken about four to six hours of phone calls with insurers to get each new patient on Relyvrio.

Heiman-Patterson warns her patients to expect weeks of back-and-forth before getting the drug.

“This is the process we’re going to have to go through and I don’t know what your copay is going to be in the end,’” she says.

Some patients who are already taking a do-it-yourself approach to the treatment see little reason to change.

Sarah Nauser, a former police officer, says her insurance has covered the prescription ingredient in Relyvrio — sodium phenylbutyrate — for a $10 copay since 2020.

“After seeing all the difficulties that people are having, I’m not going to mess with what’s working,” said Nauser, 34, who credits the combination with slowing her disease and helping her to maintain her ability to walk, talk and feed herself.

The co-founders of Cambridge, Massachusetts.-based Amylyx theorize that the two chemicals in their drug work together to shield cells from premature death. But there’s little agreement among scientists about how the drug works, or even which ingredient is responsible for its benefit.

A small European study of the supplement contained in Relyvrio showed a larger effect on slowing ALS than Amylyx’s study. And researchers there are conducting a larger, 300-patient study of the ingredient.

“That study could be hugely important in telling us whether this one ingredient–which is very inexpensive– is actually effective,” said Diana Zuckerman of the non-profit National Center for Health Research, which analyzes medical research.

___

Follow Matthew Perrone on Twitter: @AP_FDAwriter

Find your latest news here at the Hemet & San Jacinto Chronicle

The post ALS patients contend with $158K price tag on new drug appeared first on The Hemet & San Jacinto Chronicle.

The alliance aims to improve care for Veterans diagnosed with amyotrophic lateral sclerosis — and increase awareness of resources and treatment options available.

ALS is a progressive neurodegenerative disease affecting nerve cells in the brain and spinal cord. While there are no known cures, treatment options can reduce damage caused by the disease, relieve symptoms and improve one’s quality of life. Research shows those who served in the military are more likely to develop ALS than those who have not.

“ALS Association’s continuing education resources give VA health care providers and patients access to educational opportunities,” said VA National Center for Healthcare Advancement and Partnerships Nurse Executive Tracy L. Weistreich, Ph.D. “By promoting local participation in nationwide chapters, we ensure Veterans have greater access to ALS programs and services at VA and in their communities.”

Veterans can also enroll in the National ALS Registry which helps health experts enhance the standards of ALS care and seek a cure.

VA has 11 Treatment Centers of Excellence that exceed nationally accepted standards of care. To be certified, clinics must meet high level of treatment standards, participate in ALS-related research and successfully complete a comprehensive review.

Learn about VA Healthcare Advancement and Partnerships. For caregiving tips for Veterans diagnosed with ALS, visit the VA Caregiver Support Program.

Find your latest news here at the Hemet & San Jacinto Chronicle

The post VA improves quality of care for Veterans diagnosed with ALS appeared first on The Hemet & San Jacinto Chronicle.