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<a href="https://www.everythingals.org/">EverythingALS</a> has introduced a platform aimed at advancing <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) research using artificial intelligence (AI) and machine learning — with the ultimate goal of eradicating the disease and restoring lost motor function to those with ALS and other neurodegenerative disorders.
The collaborative and technology-enabled platform, dubbed the <a href="https://v2030.org/">Vision 2030 AI Hub</a>, will be led by Indu Navar, EverythingALS&#8217; founder and CEO, and Bill Nuti, the nonprofit’s chairman.
“We are unified in our conviction that AI is not just a technological breakthrough — it is the beacon of hope for medical research and will profoundly redefine the future of healthcare and medicine,” Navar said.
In launching Vision 2030, EverythingALS made financial gifts to the Massachusetts Institute of Technology and Massachusetts General Hospital, with the nonprofit stating in a <a href="https://www.globenewswire.com/news-release/2024/03/12/2844699/0/en/EverythingALS-Launches-the-Vision-2030-AI-Hub-Charting-the-Path-to-an-ALS-Cure-and-Advancing-Neurodegenerative-Disease-Research.html">press release</a> that these institutions are &#8220;driving innovative, ground-breaking work in this field.”
The AI hub will seek to unite investigators through a comprehensive data, technology, and industry-partner supply chain. Together, according to EverythingALS, these partners will be able to quickly translate basic science discoveries in regeneration and repair into life-changing therapies.
[recommended-reading id=85557]
<h3>Everything ALS will use AI platform to seek a cure for ALS</h3>
In addition to investigators, the platform will include biotech and pharmaceutical companies, hospital clinics, academia, government agencies, and foundations seeking an ALS cure.
The AI platform is part of the organization’s Vision 2030 initiative, which seeks to address historical impediments to treatment development, such as insufficient funding and a lack of technology infrastructure, as well as a dearth of data science expertise.
“ALS research, in both the private and public sectors, is severely impeded by slow and cumbersome funding processes, and the limited availability of grants due to ALS’s lower prevalence,” Nuti said, adding that “this scarcity of resources [forces] researchers to spend considerable time seeking financial support, often through charitable means.&#8221;
EverythingALS is looking to change that paradigm.
&#8220;This stark reality underscores a pressing need for a radical change in approach to ensure that no promising research is left on the sidelines, and we accelerate the process of developing a cure. Vision 2030 is the right operating model to address these challenges,” Nuti said.
According to Navar, the platform &#8220;will strategically harness the power of advanced technology and collective clinical know-how to foment meaningful progress.&#8221;
“Along with a solid operational plan, we have reached a pivotal moment in history, where quantum advancements in science and technology can be applied to the effort and transcend human limitations,” Navar said.
Key hub features include a unique network — an ecosystem that harnesses cutting-edge innovation in AI, generative AI, and large language models. The platform also will have a “laser focus” on research targeting repair and regeneration and will establish an extensive “data lake” of such research for global availability to scientists, the release states.
It also will feature a so-called frictionless operating framework, in that it will provide research tools and resources to eliminate the burden of grant application and funding. Further, its partners are expected to work together to ultimately streamline the therapy development process.
[quote=&#8221;center&#8221;]Along with a solid operational plan, we have reached a pivotal moment in history, where quantum advancements in science and technology can be applied to the effort and transcend human limitations.[/quote]
Ernest Fraenkel, PhD, an MIT professor of biological engineering, noted that “EverythingALS has a singular yet powerful mission — to dissect and understand the various subtypes of ALS and uncover their causes.”
The chair of the Vision 2030 AI Hub steering committee, Merit Cudkowicz, MD — also the director of the Sean M. Healey &amp; AMG Center for ALS at Mass General — says &#8220;this is the nexus where our collective strides become a giant leap for mankind.&#8221;
“Across the annals of medical research, progress often emerges in fragments, illuminating the path piece by piece,” Cudkowicz said.
“Yet, it is not until these fragments unite that we witness the dawn of a transformation. The Vision 2030 AI Hub epitomizes this momentous convergence — where the cumulative advances in ALS research and technology coalesce, heralding a new epoch of hope and discovery,” Cudkowicz said.
To encourage financial support, Vision 2030 AI Hub’s operating model will emphasize the urgency of conducting research into cures for ALS and other neurodegenerative disorders, rather than treatments that manage symptoms.
For more information on the AI platform, send an email to info@everythingALS.org.

EverythingALS launches AI platform to accelerate ALS research

An unprecedented $58 million donation to the <a href="https://www.als.org/">ALS Association</a> is a &#8220;beacon of hope&#8221; for the <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) community and will finance treatment centers, aid to financially strapped patients, and clinical trials of <a href="https://multiplesclerosisnewstoday.com/multiple-sclerosis-treatment/">promising treatments</a>, according to the U.S.-based nonprofit.
The donation from the estate of late philanthropist Hugh Hoffman is the single largest philanthropic gift in the history of the <a href="https://www.als.org/about-us#:~:text=Established%20in%201985%2C%20The%20ALS,fighting%20ALS%20on%20every%20front">39-year-old organization</a>, which funds global ALS research and supports patients and their families. It&#8217;s a little more than half the amount raised by the summer 2014 <a href="https://alsnewstoday.com/news/ice-bucket-challenge-dramatically-affected-als-fight/">Ice Bucket Challenge</a>, which brought in $115 million from 2.5 million donors.
&#8220;We are deeply grateful to Hugh Hoffman and his family,&#8221; Calaneet Balas, ALS Association president and CEO, said in a <a href="https://www.prnewswire.com/news-releases/hugh-hoffmans-historic-gift-to-als-association-will-elevate-fight-against-als-302059679.html">press release</a>.
The gift &#8220;will provide tangible impacts that advance our goal of making ALS livable until it is cured,&#8221; Balas said.
[recommended-reading id=20746]
<h3>Donor Hugh Hoffman wanted to provide &#8216;boost of hope&#8217; for families</h3>
Hoffman, who died last March at 91, was a Cincinnati native whose father, Herbert, died from ALS complications when Hoffman was 11. He had hoped his donation to the ALS Association would ultimately prevent other families from having to endure such a loss.
&#8220;Uncle Hugh hoped that his gift would provide an immediate boost of hope for people living with ALS and their families,&#8221; said Bert Bullock, one of Hugh Hoffman&#8217;s nephews. &#8220;This gift is a powerful testament to who he was and represents our family&#8217;s way of championing his legacy.&#8221;
Balas and her team met several times with Hoffman to help understand his desire to contribute to ALS research and to support those living with the progressive neurodegenerative disorder, the association said.
The gift will fund three programs to expand ALS research and care:
<ul>
<li>The Hoffman ALS Clinical Trial Awards program will support early-stage clinical studies to  develop experimental therapies. The association expects to fund two to three additional ALS clinical trials annually, or 20-30 new clinical investigations over 10 years.</li>
<li>The Hoffman ALS Clinic Development Awards program will help develop eight new ALS Association <a href="https://www.als.org/support/certified-centers-clinics">Certified Treatment Centers of Excellence</a> and Recognized Treatment Centers over 10 years, and bring existing clinics to Certified or Recognized Treatment Center status. These centers offer specialized care with teams typically including a neurologist, physical therapist, occupational therapist, respiratory therapist, nurse, dietitian, speech language pathologist, social worker, mental health professional and ALS Association liaison.</li>
<li>The Hoffman ALS Patient Care Expansion Awards program will increase patient access to multidisciplinary care by funding additional clinic days, expanding access to teleheath, and reducing financial barriers to care. The association expects to provide 60 grants to ALS clinics and 3,000 grants to ALS patients through the program.</li>
</ul>
Hoffman, a Yale University graduate who became a stockbroker and investment adviser, also left money to the Cincinnati Zoo &amp; Botanical Garden and the University of Cincinnati, according to the ALS association.
&#8220;Our Uncle Hugh was an incredibly generous, kind man of great integrity who wanted to give back,&#8221; said Steve Bullock, another nephew. &#8220;He lived a modest life, which enabled him to make truly transformative donations after his death. His generosity speaks volumes about the sort of person he was.&#8221;
The Ice Bucket Challenge, primarily aimed at raising ALS awareness, became a social media phenomenon when launched in the summer of 2014. The funds raised from the effort helped finance new treatments, expand care, and start research projects, the association said.
&#8220;We saw how the ALS Association was able to leverage the Ice Bucket Challenge donations and provide tangible impacts for people living with ALS,&#8221; said Steve Bullock. &#8220;We believe Hugh&#8217;s gift provides a giant step forward toward the ALS Association&#8217;s goal of making ALS livable for everyone, everywhere, until there is a cure.&#8221;

ALS Association granted $58M in largest single donation ever

A research team at Ball State University has received a $20,000 donation from the <a href="https://www.bigtopen.com/" rel="nofollow">Todd Siebert Memorial Foundation</a> supporting work into better understanding and treating <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) caused by C9orf72 mutations.
Awarded to Philip Smaldino, PhD, a cell biology professor at the Indiana school, and his team, the gift prompted the formation of the Smaldino ALS/FTD Research Project Fund as part of the university&#8217;s foundation. A related neurodegenerative disorder, FTD or <a href="https://alsnewstoday.com/news/about-35-als-patients-show-signs-frontotemporal-dementia/"> frontotemporal dementia also can affect ALS patients</a>.
The project aims to contribute to further understand the underlying mechanisms of C9orf72-associated ALS and to find new treatments. In particular, the team will focus on developing therapeutic approaches that target DHX36, a protein whose inhibition may improve outcomes for people with this ALS subtype.
[recommended-reading id=89682]
<h3>C9orf72 mutations are the most common genetic cause of ALS</h3>
The Todd Siebert Memorial Foundation, which supports patients and research into C9orf72-linked ALS,  was established in 2021 to honor of Siebert, who died from disease complications that year.
Its support came on the heels of a campus visit last year by Tina Siebert, Todd Siebert&#8217;s widow, and Erin Eckerle, the foundation&#8217;s president.
“I was honored and inspired by the visit from Tina and Erin,” Smaldino said in a <a href="https://www.bsu.edu/news/press-center/archives/2024/02/todd-siebert-memorial-foundation-boosts-als-research-at-ball-state-university">university press release</a>. “The Siebert Foundation has been working tirelessly since 2021 to raise awareness and funds to support Indiana families affected by ALS.”
Mutations in the C9orf72 gene are the most common <a href="https://alsnewstoday.com/causes-of-als/#Genetics_of_ALS">genetic cause of ALS</a>, found in up to 50% of people with <a href="https://alsnewstoday.com/forms-of-als-familial-als/">familial ALS</a> and up to 10% of those with <a href="https://alsnewstoday.com/sporadic-als/">sporadic ALS</a>. Similar mutations also can be found in some FTD patients.
These mutations usually are characterized by the excessive repetition of six DNA building blocks, GGGGCC, in the gene’s sequence. These regions form &#8220;knot-like&#8221; structures that regulate the process of protein production in healthy cells. However, excessive or aberrant forms of these structures can help to drive disease processes.
DHX36 is an enzyme, a type of protein responsible for untying these knot-like structures. According to Smaldino, lowering its levels has shown potential in reducing the protein&#8217;s toxic forms in cell and mouse models of C9orf72-linked ALS.
The donation will support further exploration of this treatment approach, and contribute to the training of student scientists in innovative ALS research.
“I try to remind my students that what we are doing in the lab may someday help real people. The visit with Tina and Erin brought that idea to light in ways that my words could never have,” Smaldino said.

$20K gift supports Ball State work into C9orf72-linked ALS

The <a href="https://www.als.org/">ALS Association</a> awarded $400,000 to University of Missouri-Columbia School of Medicine and its NextGen Precision Health Initiative as part of its program to encourage more <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) patients living in rural areas to take part in clinical trials.
The university&#8217;s <a href="https://www.als.org/research/funding-opportunities/trial-capacity-awards-2023">2023 Trial Capacity Award</a> was given to W. David Arnold, MD, the executive director of NextGen and a professor of physical medicine and rehabilitation at the medical school. It is expected to support efforts to break down the various barriers keeping rural Missourians from participating in ALS trials, including those of potential treatments.
Specifically, the funds will help create a new position in NextGen devoted to identifying and circumventing obstacles facing ALS patients in rural communities, like difficulties in accessing distant clinics. This person will serve as the point of contact for people interested in participating in clinical trials, in addition to boosting research collaborations.
It was <a href="https://www.als.org/blog/breaking-down-barriers-trial-capacity-awardees-work-improve-access-als-clinical-trials">one of five such awards</a>, totaling nearly $2 million, given to scientists working at institutes in Texas, Wisconsin, New York, and California, as well as in Missouri.
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<h3>Distant clinical trial sites can be a barrier for rural ALS patients</h3>
“I hope this person to be a valuable resource for ALS research at the University of Missouri and to be the champion for improving clinical research access for rural Missourians,” Arnold said in a <a href="https://medicine.missouri.edu/news/nextgen-mu-school-medicine-receive-national-als-grant">university press release</a>.
A study into <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6289079/">ALS prevalence by U.S. region for 2015</a> found the highest rate — about 5.5 for every 100,000 residents — to be in the Midwest, a region that includes Missouri.
Muscle weakness is a classic <a href="https://alsnewstoday.com/symptoms-of-als/">symptom of ALS</a>, a progressive neurodegenerative disease, and patients can find travel to distant clinical trial sites difficult at best. Rural areas generally lack the population and services needed to support large clinical centers.
The Missouri university system&#8217;s <a href="https://precisionhealth.missouri.edu/about">NextGen Precision Health Initiative</a> promotes research across its four public campuses, and has among its goals to &#8220;revolutionize health care for our citizens, [and] eliminate health care disparities,&#8221; it states on its website.
In addition to addressing the bottlenecks affecting trial participation by rural residents, the award aims to encourage greater outreach to U.S. veterans and to residents for whom English is not the primary language.
<h3>Making trials more accessible to a &#8220;wider community of people with ALS&#8217;</h3>
“ALS clinical research sites often know what they need to do to reduce or eliminate barriers to trial participation, but they don’t have the resources or the infrastructure to do it,” said Paul Larkin, director of the association&#8217;s research program.
“Through our Trial Capacity Awards, we provide the support these sites need to reduce or eliminate specific obstacles or bottlenecks, improving trial accessibility to a wider community of people living with ALS as well as increasing the efficiency and pace at which this research is conducted,” he added.
According to Arnold, the University of Missouri-Columbia is leading multiple research studies involving ALS patients. The association award will help to foster new research collaborations and connections, as well as finding ways to bring into studies more patients in outlying areas.
“We&#8217;re trying to push the envelope. A lot of patients really want to be involved in research,” Arnold said. “We want to try to make that as accessible as possible for all of our patients.”

ALS Association supports work to bring rural patients into clinical trials

The <a href="https://als.ca/">ALS Society of Canada</a> (ALS Canada) has awarded two expedited grants totaling $200,000 to international researchers seeking to advance understanding of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
Provided through the 2023 ALS Canada Acceleration Grant Program, a $100,000 grant will support Mónica Povedano, MD, at the Hospital de Bellvitge-IDIBELL in Barcelona, Spain, in conducting a population-based study of ALS in Colombia and Ethiopia.
Stefan Dukic, PhD, a postdoctoral researcher at the University Medical Centre Utrecht, in the Netherlands, will also receive $100,000 to develop a new method to subgroup people with ALS using an electroencephalogram (EEG), which measures the electrical activity in the brain.
“We are incredibly proud to be bringing forward an innovative opportunity to fund new projects at an expedited speed with the ALS Canada Acceleration Grant Program,” Tammy Moore, CEO of ALS Canada, said in a <a href="https://als.ca/blogs/als-canada-funding-new-acceleration-grant-program-2023/">press release from the non-profit</a>.
[recommended-reading id=89616]
<h3>ALS Canada seeks to fund research that will affect future discoveries</h3>
“ALS is a devastating disease; by bringing together global leaders in ALS to identify and support projects from around the world, we look to fund research that will change the outcome of an <a href="https://alsnewstoday.com/als-diagnosis/">ALS diagnosis</a> and have an impact on future ALS discoveries,” Moore said.
In ALS, the nerve cells that control voluntary movements, called motor neurons, progressively degenerate and die, preventing the brain from properly controlling muscles.
People with ALS experience <a href="https://alsnewstoday.com/symptoms-of-als/">symptoms</a> such as progressive muscle weakness, typically starting in the hands or feet, or the muscles around the throat and mouth, which leads to problems swallowing and speaking. Over time, muscle weakness can evolve into paralysis, with little to no ability to move or breathe. Eventual respiratory failure is the most common cause of death in ALS.
<h3>Program created to expedite funding process for promising research projects</h3>
The ALS Canada Acceleration Grant Program was created to expedite the funding process for promising research that relies on a unique form of global peer review. Specialists in the field take proactive measures to identify the most suitable projects to support rather than assessing applications after they are submitted. This could reduce the time to secure funding from years to a few months.
Among the selected projects, Povedano will lead a collaborative effort with colleagues in Colombia and Ethiopia to conduct an epidemiological study — the distribution of ALS in populations and the factors determining that distribution — in these countries. The project will be the first comparative study of ALS across different ethnic backgrounds while incorporating data from European individuals.
In addition, Povedano will help create the infrastructure to conduct clinical trials, which may provide access to <a href="https://alsnewstoday.com/experimental-treatments-for-als/">investigational therapies</a> in these countries. The research will also advance opportunities to discover new treatments that may help ALS patients globally if successful.
Dukic and colleagues have shown that EEG, which uses electrodes placed on the scalp, can detect abnormal patterns of brain activity related to motor and cognitive function in people with ALS. The technology may predict disease progression.
[recommended-reading id=89315]
<h3>Grant will help speed research, recruit more participants in EEG study</h3>
The grant will help speed up this work and recruit more participants. The team will also combine EEG with MRI to investigate individuals who carry mutations in the C9ORF72 gene, the most common <a href="https://alsnewstoday.com/als-and-genetics/">genetic cause of ALS</a>, and have yet to experience symptoms. This research may help the development of early or preventive treatments.
Funding for Dukic’s grant was made possible through a partnership with <a href="https://www.steviefever.com/">The Stevie Fever for ALS Foundation</a>, which contributed $100,000 to ALS Canada.
“Researchers around the globe are working tirelessly to uncover new discoveries that will advance our understanding of ALS and develop effective treatments for the disease,” said David Taylor, PhD, vice president of research and strategic partnerships at ALS Canada.
“To have an impact on people currently <a href="https://alsnewstoday.com/living-with-als-2/">living with the disease</a>, we must move faster to fund research and look at promising, innovative ideas beyond our borders to fuel discoveries that will lead to a world free of ALS,” Taylor added.

ALS Canada awards $200K to speed global disease research

The <a href="https://www.als.org/">ALS Association</a> has given <a href="https://www.oryzon.com/en">Oryzon Genomics</a> nearly $500,000 to advance ORY-4001, a potential treatment of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), into preclinical studies.
The $498,690 grant was provided via the association&#8217;s Lawrence and Isabel Barnett Drug Development Program. Oryzon intends for laboratory studies in mouse disease models to pave the way for ORY-4001 to be tested in clinical trials.
&#8220;We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies like ORY-4001,&#8221; Kuldip Dave, PhD, senior vice president of research at the ALS Association, said in an <a href="https://www.globenewswire.com/news-release/2023/12/04/2789939/0/en/ORYZON-Awarded-with-a-Grant-for-ORY-4001-From-the-ALS-Association-in-the-U-S.html">organization press release</a>.
&#8220;Getting promising treatments out of the laboratory and into clinical testing as quickly as possible is key to making ALS a livable disease until we can cure it,” Dave added.
[recommended-reading id=88415]
<h3>ALS Association grant supports work needed to bring ORY-4001 into a trial</h3>
ORY-4001 is a highly selective inhibitor of histone deacetylase 6 (HDAC6), part of a family of enzymes that regulate cellular activity through a chemical modification to proteins called deacetylation.
HDAC6 has been linked to cellular processes involved in neurodegenerative disease, and blocking it has emerged as a possible treatment strategy for conditions such as ALS.
Particularly, HDAC6 activity regulates alpha tubulin, a protein involved in cellular protein transport. Impaired axonal transport — the movement of proteins needed for nerve cell health and survival along a cell&#8217;s fiber-like projections (axons) — is a feature of ALS and other neurological diseases.
Preclinical studies have shown that HDAC6 inhibition <a href="https://alsnewstoday.com/news/resolving-traffic-jams-in-human-als-motor-neurons/">protects against axonal transport deficits</a> in models of ALS and may be able to slow neurodegeneration.
Oryzon has been evaluating the therapeutic potential of various HDAC6 inhibitors for neurological diseases, and earlier this year selected ORY-4001 as its clinical development candidate.
The therapy&#8217;s initial development efforts have been focused on <a href="https://charcot-marie-toothnews.com/charcot-marie-tooth-type-1-cmt1/">Charcot-Marie-Tooth disease type 1</a> (CMT1), an inherited disease affecting peripheral nerves, those that participate in communication between the brain and the rest of the body.
<h3>Therapy showed potential in model of disease also marked by muscle wasting</h3>
In a mouse model of CMT1, the molecule improved neuromuscular function, prevented axon damage, and prevented the loss of <a href="https://multiplesclerosisnewstoday.com/myelin-sheath/">myelin</a>, the substance that surrounds and protects nerve cell axons, Oryzon reported.
Both CMT1 and ALS are characterized by muscle weakness and wasting, as well as altered axonal transport. Disturbed proteostasis, the process by which proteins are regulated to maintain cellular health, also is evident in both conditions.
Given this overlap and previous preclinical studies of HDAC6 inhibitors in ALS, the scientists believe ORY-4001 may be a promising treatment candidate.
&#8220;The rationale linking ORY-4001’s mechanism of action to the disease is sound,&#8221; said Jordi Xaus, PhD, Oryzon&#8217;s chief scientific officer.
In addition to testing ORY-4001 in an ALS mouse model, the company is conducting the toxicology studies required before a therapy can be tested in humans.
&#8220;If these preclinical results are positive, we plan to extend the clinical development of ORY-4001 … to include ALS and explore its therapeutical potential for people living with this devastating disease,&#8221; Xaus said.
&#8220;It is an honor to have the support of the ALS Association,&#8221; he added.

ALS Association helping to move ORY-4001 into animal studies

One important habit I enjoy doing every day is keeping up with the latest <a href="https://alsnewstoday.com/category/news/">ALS-related news</a> and research. But every once in a while, I come across an article that has me muttering, “Was this research even needed?” And more often than not, I answer my own question with, “Well, no.” The researchers could’ve just asked me or any other <a href="https://alsnewstoday.com/what-is-als/">ALS</a> patient for our opinion.
What pushed my buttons was <a href="https://alsnewstoday.com/news/anxiety-als-patients-family-soon-after-diagnosis/">reading about a recent two-year study</a> that found that many ALS patients and their relatives experience anxiety and depression after a <a href="https://alsnewstoday.com/als-diagnosis/">diagnosis</a>. Really?
Plus, I knew this study was just one more in a long line of variations on the same theme, published over and over. Several years ago, I <a href="https://alsnewstoday.com/columns/als-depression-link-anxiety-prognosis/">wrote</a> about this same topic. In it, I referenced several studies dating back to 2005, all looking at the possible association between anxiety-depressive disorders and ALS. Really?
Why are medical experts still trying to figure out if ALS causes patients to feel negative emotions, especially when concise and useful <a href="https://alsnewstoday.com/als-mental-health/">resources on the subject</a> are already available?
[recommended-reading id=86545]
<h3>What we want</h3>
What do ALS patients want? My advice: Don’t guess; ask us and we’ll gladly tell you.
An excellent example of asking is the recent survey that <a href="https://alsnewstoday.com/">ALS News Today</a> reported on in &#8220;<a href="https://alsnewstoday.com/news/survey-offers-look-life-treatment-priorities-those-living-als/">Survey offers look at life, treatment priorities of those living with ALS</a>.&#8221;
Even though the survey represented ALS patients from nine European countries, I believe similar results would be found in the U.S.
Among the many insights shared in the survey, this statement stood out to me: “When asked about what they valued more about current and new <a href="https://alsnewstoday.com/approved-treatments-for-als/">treatments</a>, most patients wanted therapies that could stop disease progression.”
In other words, the next best thing to a cure.
<h3>Looking forward</h3>
While this past year brought us improved ALS treatments, support services, and increased focus on mental health, let’s continue to keep our focus on the goal: a cure for ALS.
I believe it&#8217;s possible. I look forward to the day when I read the grand announcement that there will be a world without this disease.
While we wait for that day, let’s help each other learn to live well while we&#8217;re <a href="https://alsnewstoday.com/living-with-als-2/">living with ALS</a>.
<hr />
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.

Just ask and ALS patients will tell you what we want: a cure

A neuroscientist with a specialty in <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) is joining the faculty at Vanderbilt University Medical Center (VUMC) and will establish an ALS research center at the Nashville school.
Véronique Belzil, PhD, whose work is focused on developing tailored treatments for people with ALS, also will serve as director of the first basic science lab in Tennessee exclusively dedicated to the progressive neurological disease.
She comes to Vanderbilt from the <a href="https://www.mayo.edu/research/faculty/belzil-veronique-ph-d-m-s/bio-20480537">Mayo Clinic in Jacksonville, Florida</a>, where she led work into characterizing the mechanisms that damage and kill motor neurons — the specialized nerve cells that control muscle movement — to <a href="https://alsnewstoday.com/causes-of-als/">cause ALS</a> and the progressive muscle weakness that is a hallmark <a href="https://alsnewstoday.com/symptoms-of-als/">disease symptom</a>.
“The Vanderbilt ALS Research Center will become a natural extension of my own laboratory’s mission: develop patient-centered approaches to quickly bring findings from bench to bedside so ALS becomes curable,” Belzil said.
[recommended-reading id=87929]
<h3>Planned ALS research center sets as its mission finding a disease cure</h3>
The lab will be one of the few U.S. centers focused on translational ALS research, according to Dane Chetkovich, MD, PhD, a professor and chair of the neurology department at VUMC, which Belzil is joining on Jan. 8. Translational research refers to basic science specifically aimed at moving discoveries &#8220;from bench to bedside&#8221; as treatments or the like.
&#8220;Recruiting Dr. Belzil is an important step forward for Tennessee, Vanderbilt, and most importantly the patients and families who need us to do everything we possibly can to find a cure for ALS,” Chetkovich said in a <a href="https://news.vumc.org/2023/11/20/belzil-to-establish-vumc-als-research-center/">university press release</a>.
Belzil has been an assistant professor of neurology at the Mayo Clinic since 2014, where she leads a lab working in ALS,  frontotemporal dementia, a related disease, and other neurodegenerative conditions.
Belzil and her team&#8217;s current research uses human tissue and integrative approaches to identify disease-specific genetic variants and uncover their mechanisms of action in a search for disease biomarkers and potential therapeutic targets. The scientists also use patient-derived cells in work toward gene therapies for ALS and its related disorders.
“Dr. Belzil is a recognized leader in ALS research whose state-of-the-art experimental approaches are well suited to synergize with VUMC’s strengths in genomics and bioinformatics and our goal of making health care personal,” Chetkovich said.
Belzil’s was recruited through a partnership between VUMC with the <a href="https://www.livelikelou.org/">Live Like Lou Foundation</a> — named for Lou Gehrig, the baseball player who died of ALS — that works to support families affected by the disease and research into potential <a href="https://alsnewstoday.com/approved-treatments-for-als/">ALS treatments</a>.
“I am looking forward to establishing a world-renowned collaborative and translational ALS research center by prioritizing open science, teamwork, interdisciplinary expertise, and national and international collaborations,” Belzil said.

ALS research center planned to open at Vanderbilt University

Continuing its annual support, <a href="https://www.goblusky.com">BluSky Restoration Contractors</a> has raised $140,000 to advance research at the <a href="https://millerlab.wustl.edu/">Miller Laboratory</a> at Washington University School of Medicine in St. Louis, Missouri, for new and better <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) treatments.
The funds were raised through the sixth annual <a href="https://app.eventcaddy.com/events/6th-annual-rakers-classic">Rakers Classic</a> charity golf tournament, the signature fundraiser of the <a href="https://www.lylerakers.com/">Lyle Rakers Charitable Foundation</a>. Since 2018, when the foundation was established, the tournament has generated $638,000 for the St. Louis, Missouri research lab. The goal this year was $125,000.
Jim Rable, senior vice president of corporate development at Denver, Colorado-based BluSky, helped to create the foundation in honor of Lyle Rakers, his longtime friend and fellow BluSky employee.
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Rakers learned in 2017 he had brachial amyotrophic diplegia, a rare form of ALS also commonly known as fail arm ALS, or man-in-the-barrel syndrome, due to its progressive upper limb weakness and atrophy. The diagnosis rocked the company.
&#8220;We at BluSky support each other in the good times, and the not so good times,&#8221; said Rable in a <a href="https://www.prweb.com/releases/blusky-philanthropic-event-raises-140-000-for-als-research-in-st-louis-301953467.html">BluSky press release</a>. &#8220;We are truly grateful for the overwhelming amount of generosity and kindness from all [who] have participated, sponsored, donated, and volunteered at our events. Our goal is to grow our foundation to really raise significant funds to make a bigger impact in the world of ALS research.&#8221;
Research at the Miller Lab focuses on understanding the mechanisms underlying neurodegeneration in ALS, dementia, and related disorders, and to develop novel therapies. The lab employs RNA-targeted therapeutic strategies, primarily using antisense oligonucleotides, which are pieces of RNA designed to silence specific genes and prevent protein production.
&#8220;The generous support from the Lyle Rakers Foundation allows us to push research on the cutting edge and bring new RNA-targeted therapies to clinical trial,&#8221; said Timothy Miller, MD, PhD, the principal investigator at the Miller Lab. &#8220;We are so honored to be associated with the Lyle Rakers Foundation and the terrific group of people that have put together another very successful golf tournament.&#8221;
<h3>Working with the Washington University School of Medicine ALS Center</h3>
The Miller Lab functions in conjunction with the <a href="https://alscenter.wustl.edu/">Washington University School of Medicine ALS Center</a>, comprised of physicians, clinicians, scientists, and other experts. The center treats ALS patients and offers access to clinical trials focused on personalized medicine to enhance the understanding, treatment, and prevention of ALS.

Tag: ALS Research