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The Barrow Neurological Institute has received $16.7 million from the National Institutes of Health (NIH) to help coordinate a nationwide repository of clinical data of people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The Access for All in ALS (ALL ALS) Consortium will serve as a repository of biological samples and clinical data acquired across time from ALS patients and people at risk of developing the condition, as well as healthy people acting as controls. Data will be collected in in-person and remote visits, and will be made available to scientists worldwide via a centralized portal.
The hope is that the data helps scientists gain a greater understanding of ALS and develop better, more effective treatments for the progressive neurodegenerative condition.
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“Time is of the essence for patients with ALS and we are working to identify new and effective approaches to develop and test new treatments quickly,” Robert Bowser, PhD, chief scientific officer at Barrow and one of the researchers leading the consortium, said in a <a href="https://www.eurekalert.org/news-releases/1003893">press release</a>.
“It’s my hope the Access for All in ALS Consortium will lead us to potentially groundbreaking ALS discoveries and treatments that will extend the quality of life for those living with ALS,” Bowser added.
The new consortium will have 34 clinical sites across the U.S. and Puerto Rico. There will be two coordinating centers, each handling half the sites: the ALL ALS West Clinical Coordinating Center at the Barrow Neurological Institute, in Phoenix, Arizona, and the ALL ALS East Clinical Coordinating Center, located at the Massachusetts General Hospital (MGH)&#8217;s Neurological Clinical Research Institute, in Boston.
<h3>ALS research consortium personnel</h3>
Neurologist Jeremy Shefner, MD, PhD, chief medical officer of Clinical Research at Barrow is one of the lead researchers of the consortium. His team will be responsible for site monitoring activities and training of clinical outcomes for all clinical sites in the consortium.
In addition to Bowser and Shefner at Barrow, other principal investigators of the consortium include: Suma Babu, James Berry, MD, and Sabrina Paganoni, MD, PhD, from the Sean M. Healey &amp; AMG Center for ALS at MGH; Jinsy Andrews, MD, Matthew Harms, MD, Neil Shneider, MD, PhD, Hemali Phatnani, PhD, and Frank Provenzano, PhD, from Columbia University; and Brent Harris, MD, PhD, from Georgetown University.
The funding for the consortium was made possible by the Accelerating Access to Critical Therapies for ALS (ACT for ALS) Act, according to a <a href="https://www.massgeneral.org/neurology/als/news/nih-funds-all-als-consortium">press release</a> from the Healey Center.
“Barrow Neurological Institute is honored to be selected by the NIH to help coordinate this important research consortium,” Bowser said. “By studying patient-derived biosamples from diverse ALS cohorts, we hope to accelerate the development of effective new drugs to extend life for people living with ALS.”

NIH grant will help launch the ALL ALS research consortium

<a href="https://alsnewstoday.com/what-is-als/">Amyotrophic lateral sclerosis</a> (ALS) patients not eligible to participate in clinical trials of <a href="https://alsnewstoday.com/pridopidine-experimental-treatment-als/">pridopidine</a> — a small molecule developed by <a href="https://www.prilenia.com/">Prilenia Therapeutics</a> to slow disease progression in ALS — will soon be able to receive the experimental therapy via an expanded access program (EAP).
EAPs, also known as compassionate-use programs, are aimed at people with serious or life-threatening conditions that have few or no adequate treatments. Such programs allow patients to access investigational therapies available outside of a clinical trial. They typically run in parallel with late-stage clinical trials and provide additional clinical data that may support an approval of the given treatment by regulatory agencies.
Led by researchers at the Sean M. Healey &amp; AMG Center for ALS at Massachusetts General Hospital in collaboration with Prilenia, this EAP will enroll about 200 patients at as many as 45 centers across the U.S. Participants will receive the medication for up to two years.
The program is being launched thanks to a grant from the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), and is supported by the Accelerating Access to Critical Therapies (ACT) for ALS act.
“Prilenia is proud to work together with The Sean M. Healey &amp; AMG Center for ALS at Massachusetts General Hospital to make this important EAP possible for the many people living with more advanced stages of ALS,” Michael R. Hayden, Prilenia’s founder and CEO, said in a <a href="https://www.massgeneral.org/neurology/als/news/healeyamg-awarded-ninds-grant-for-eap-pridopidine">press release</a>, adding, &#8220;We share the great appreciation for the NIH and NINDS for supporting this work through the Act for ALS.&#8221;
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<h3>EAP for pridopidine expected to provide real world data</h3>
In the release, the Healey &amp; AMG Center staff said the expanded access program &#8220;will provide real world safety, biomarker, and clinical data in two hundred (200) ALS individuals treated with pridopidine for up to two (2) years, thus contributing to our understanding of the effects of pridopidine in a broad population.&#8221;
The EAP&#8217;s lead investigators are Suma Babu, an assistant professor of neurology at Harvard Medical School, James Berry, MD, director of Massachusetts General Hospital&#8217;s Neurological Clinical Research Institute, and Sabrina Paganoni, MD, PhD, a physician-scientist at the Healey Center and co-director of the Neurological Clinical Research Institute.
“We are grateful to the NIH for this award, and the opportunity to launch the pridopidine EAP,&#8221; Babu, Berry, and Paganoni said, adding, &#8220;Programs like this help advance research and innovation in ALS.”
Pridopidine is an orally available small molecule designed to bind and activate the sigma-1 receptor (S1R), a protein that&#8217;s broadly produced in the brain and spinal cord and <a href="https://www.prilenia.com/about-pridopidine/">regulates a number of processes</a> needed for nerve cell function and survival.
By activating S1R, the therapy boosts the clearance of toxic molecules, increases energy production, and limits inflammation and cellular stress — all known hallmarks of ALS. In doing so, the therapy is expected to ease certain disease mechanisms, thereby slowing disease progression.
[quote=&#8221;center&#8221;]We are grateful to the NIH for this award, and the opportunity to launch the pridopidine EAP. &#8230; Programs like this help advance research and innovation in ALS.[/quote]
The candidate therapy was the focus of one arm of the <a href="https://clinicaltrials.gov/ct2/show/NCT04297683">HEALEY ALS Platform trial (NCT04297683)</a> — a study testing multiple potential <a href="https://alsnewstoday.com/als-treatment/">ALS therapies</a> at the same time against a shared placebo group. The study was led by the Healey Center.
Pridopidine&#8217;s <a href="https://clinicaltrials.gov/ct2/show/NCT04615923">Phase 2/3 trial arm (NCT04615923)</a> enrolled 163 adults with ALS, whose symptoms had started less than three years before screening. Each was randomly assigned to receive either oral pridopidine, at a dose of 45 mg, or a placebo, twice daily for 24 weeks, or about six months.
The trial ultimately <a href="https://alsnewstoday.com/news/pridopidine-shows-some-potential-in-healey-trial-but-misses-main-goal/">failed to meet its primary goal</a> of showing that pridopidine could significantly slow disease progression, as measured by changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), a standardized measure that assesses a patient&#8217;s ability to perform daily activities.
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<h3>Pridopidine fails primary trial goal, but shows some benefits</h3>
There were significant benefits seen in certain speech measures in participants given pridopidine, however, as well as positive trends in respiratory function for these patients.
The medication also showed <a href="https://alsnewstoday.com/news/pridopidine-shows-some-potential-in-healey-trial-but-misses-main-goal/">promising trends</a> in a subgroup of patients with rapidly progressing disease, all of whom were enrolled less than 18 months after symptom onset.
In this group, pridopidine-treated patients had a significantly smaller decline in ALSFRS-R scores than those given the placebo (7.5 vs. 12.7). These patients also had an average 40% reduction in neurofilament light chain (NfL) levels, a marker or nerve damage, compared with patients given the placebo.
Based on these findings, Prilenia has decided to <a href="https://alsnewstoday.com/aan-2023/aan-2023-als-treatments-idd-healey-trial-move-toward-phase-3-tests/">move pridopidine into</a> Phase 3 clinical trials.
Participants also are being followed in the trial&#8217;s open-label extension, in which all are receiving the experimental therapy. Data from this open-label part and from the upcoming EAP may help to better understand the effects of pridopidine in ALS patients.
“We &#8230; want to express our gratitude to the participants and their families as well as the site healthcare providers and staff who will make this EAP possible, further advancing our understanding of pridopidine as a potential therapy for ALS,” Hayden said.
Pridopidine has been granted orphan drug designation for ALS in both the U.S. and Europe. This designation aims to encourage therapies for rare and serious diseases, through benefits such as market exclusivity and exemption from certain fees.

Tag: NIH grant

NIH grant will help launch the ALL ALS research consortium

New EAP will allow pridopidine use for ALS patients not in trial