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A conference in July will feature &#8220;diverse perspectives,&#8221; including patient presentations and discussions featuring people living with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) along with clinical and research experts.
That&#8217;s according to the <a href="https://www.als.org/">ALS Association</a>, which said its inaugural <a href="https://www.als.org/als-nexus/registration">ALS Nexus Conference</a> will be held July 14-17 at the Gaylord Texan Resort &amp; Conference Center in Grapevine, Texas.
“ALS Nexus represents a significant step forward in our efforts to transform the experience of living with ALS,” Nicole Eck, chief strategy officer at the ALS Association, said in an <a href="https://www.prnewswire.com/news-releases/the-als-association-announces-inaugural-als-nexus-conference-302097321.html">association press release</a>. “While there have always been separate conferences focused on ALS research, advocacy, or care, this will be the first conference integrating these areas around how we specifically make ALS livable and cure it.”
Attendees will be able to connect with peers and gain insights into the needs and experiences of the ALS community, and contribute to discussions that influence research, advocacy, and patient care initiatives, the association said.
[recommended-reading id=90530]
<h3>Spotlighting people living with ALS</h3>
Sessions will spotlight novel solutions with an emphasis on the value of interdisciplinary collaboration, and will feature clinicians and researchers who specialize in ALS, as well as ALS patients and their families and friends.
<a href="https://www.als.org/als-nexus/agenda">Topics</a> will include <a href="https://alsnewstoday.com/als-treatment/">ALS treatment</a> development, care access, the changing care landscape, palliative care, insurance coverage, and clinical trial efficiency. Sessions also will cover technology and ALS, next-generation research strategies, the power of caregiving, affordable access to genetic therapies, health disparities, <a href="https://alsnewstoday.com/forms-of-als/familial-als/">familial ALS</a>, and future advancements in treatment and care.
The conference will be structured around finding new treatments and cures, optimizing current treatments and care, and preventing or delaying the <a href="https://alsnewstoday.com/als-symptoms/">manifestations of ALS</a>, the association said.
Participants are invited to attend the 10th anniversary of the <a href="https://alsnewstoday.com/news/ice-bucket-challenge-dramatically-affected-als-fight/">Ice Bucket Challenge</a> on July 16 at the nearby Paradise Springs water park. The challenge, designed to promote ALS awareness, has resulted in a marked increase in spending on research.
Patients and caregivers may attend the conference virtually or in person at no charge. The virtual option includes live-streamed and breakout sessions, as well as opportunities to connect with others. <a href="https://web.cvent.com/event/e158cb6f-97c0-4a56-b5f9-fbd4509b34a4/register">Registration</a> is open.
Registration for in-person attendance is available to scientists, healthcare professionals, advocates, patients, and others. It includes access to the July 14 welcome reception and the following day&#8217;s Heroes Award dinner, in addition to all conference sessions. Clinicians and researchers also will be welcome at networking events. Lunch will be provided daily.
Early registration, which offers lower rates, ends on May 10. Standard registration runs to July 13. On-site registration also is available, but &#8220;space is limited, and early registration encouraged,&#8221; the organization said.
For all paid registrants, there is a $250 cancellation fee, with balances refunded until May 17.
The ALS Association, the world&#8217;s largest ALS organization, funds global research collaborations, assists patients and their families, and advocates for patients.

Association conference features living with ALS as part of program

The <a href="https://www.als.org/">ALS Association</a> is partnering with <a href="https://mytomorrows.com/">MyTomorrows</a>, a global health technology company, to make it easier for people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) to find and participate in clinical trials, and to know about &#8220;pre-approval treatment options,&#8221; the company said in a <a href="https://mytomorrows.com/blog/partnership/mytomorrows-partners-with-the-als-association-to-support-als-patients-in-accessing-clinical-trials/">press release</a>.
The collaboration will allow the nonprofit to tap into the company&#8217;s database of ongoing clinical trials — granting treating physicians, patients, and families access to information about experimental treatment options in ALS and guiding them toward relevant studies.
&#8220;We look forward to witnessing the impact of our partnership with the ALS Association, one of the most effective and impactful patient advocacy groups in the United States,&#8221; said Michel van Harten, MD, myTomorrows’ CEO, who noted the &#8220;high unmet needs&#8221; of ALS patients.
“Partnering with leading patient advocacy groups such as the ALS Association will help an even larger cohort of ALS patients and their treating physicians discover clinical trials, thereby reducing the barriers they may face in accessing their treatment options,&#8221; van Harten added.
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<h3>Collaboration to make use of myTomorrows digital platform</h3>
ALS is caused by the progressive dysfunction and death of motor neurons, the nerve cells that control voluntary movements. This results in progressive muscle weakness, with <a href="https://alsnewstoday.com/symptoms-of-als/">symptoms</a> such as trouble walking, and difficulty speaking, swallowing, and breathing.
Because ALS has such a great impact on a person&#8217;s everyday life and typically progresses at a fast rate, searching for clinical trials that patients can get involved in is a particularly difficult task, according to myTomorrows.
The company offers a digital platform that connects pharmaceutical companies and trial sites with physicians and patients — conducting extensive searches of databases worldwide to provide up-to-date information on available experimental treatments and clinical trials.
Providing patient and clinician access to such information will be helpful for the pharmaceutical companies developing novel ALS therapies, according to myTomorrows, which noted that treatment developers commonly face struggles in patient recruitment.
The longer these trials take to enroll their target number of participants, the greater the delay in treatment development and the longer it will take to bring these treatments to the market, the company noted.
&#8220;myTomorrows explores each patient’s case individually, helping ALS patients and their loved ones identify and understand all of the available clinical trial options in a well-rounded manner, guided by expert patient navigators,&#8221; the release states.
The goal, according to the company, is to help patients and their physicians to be aware of treatment options and make informed decisions on the best treatment journey.
[quote=&#8221;center&#8221;]Research participation is so impactful to finding treatments and cures as well as reducing the harms of ALS.[/quote]
The company also provides information on international expanded access programs, which allow patients who fail to qualify for clinical trials to in some cases get access to an investigational therapy.
Pam Knott, the ALS Association’s vice president of data and technology, said the nonprofit is &#8220;very excited about this new partnership.&#8221;
&#8220;Research participation is so impactful to finding treatments and cures as well as reducing the harms of ALS,&#8221; Knott said. She added that the collaboration is &#8220;an opportunity to allow more people with ALS to enroll in clinical trials faster while also reducing burden.&#8221;
The ALS Association serves more than 20,000 people living with ALS in the U.S. The nonprofit organization raises awareness of the disease, provides funding to support research and development of new treatments and clinical trials, and offers care services for both patients and caregivers.

ALS Association, myTomorrows team up to aid clinical trial access

<a href="https://coavetx.com/">Coave Therapeutics</a> has received a grant from the <a href="https://www.als.org/">ALS Association</a> to support the development of its gene therapy candidate CTx-TFEB for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The grant, made through the <a href="https://www.als.org/research/research-we-fund/our-awards-and-programs/barnett-drug-development-program">Lawrence and Isabel Barnett Drug Development Program</a>, is part of the $2.9 million the ALS Association has granted to six organizations committed to advancing novel therapies for ALS.
It will help the company conduct proof-of-concept preclinical studies of CTx-TFEB that may support moving the treatment into ALS clinical trials in the future.
“Our goal is to establish a robust preclinical proof-of-concept, laying a strong foundation for advancing to the clinic in the near future,” Lolita Petit, PhD, Coave’s chief scientific officer, said in a <a href="https://www.globenewswire.com/news-release/2024/02/29/2837821/0/en/Coave-Therapeutics-Receives-Grant-from-the-ALS-Association-to-Advance-its-CTx-TFEB-Program-as-a-Potential-Treatment-for-All-Forms-of-ALS.html">company press release</a>. “Together, we are now on the brink of a breakthrough that promises hope and progress for patients impacted by ALS.”
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<h3>Importance of moving promising treatments from laboratory to clinical testing</h3>
“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,” said Kuldip Dave, PhD, senior vice president of research at the ALS Association.
The molecular mechanisms that cause ALS aren&#8217;t fully known, but virtually all cases are marked by the accumulation of protein clumps. This is in part due to a dysregulation in autophagy — the process by which cells recycle abnormal or defective molecules, which usually happens inside specialized compartments called lysosomes.
It&#8217;s anticipated that boosting autophagy could promote the clearance of these toxic protein clumps in ALS nerve cells, thereby slowing or halting neuronal degeneration and disease progression.
The transcription factor TFEB has emerged as a key regulator of the autophagy lysosomal pathway. This protein is found at lower levels in the brains of ALS patients, and activating it is believed to be a promising approach to increase autophagy and remove the harmful protein clumps.
CTx-TFEB is a novel genetic therapy product designed to deliver the TFEB gene specifically to nerve cells in the brain and spinal cord. The gene sequence is transported inside an adeno-associated virus that does not cause disease in humans. The virus was modified with Coave’s proprietary <a href="https://coavetx.com/#meetaligater">ALIGATER platform</a> to carry certain molecules that make the vector more specific to nerve cells.
“Modulating autophagy using TFEB gene therapy is an exciting approach to promote the clearance of accumulated toxic material in affected neurons, halt motor neuron degeneration, and preserve muscular function in people with ALS,” Petit said.
The therapy is expected to activate autophagy in ALS patients regardless of their genetic subtype, potentially serving as a therapy for all <a href="https://alsnewstoday.com/forms-of-als/">types of ALS</a>.
“We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies through our Lawrence and Isabel Barnett Drug Development Program,” Dave said.

Coave wins grant to advance gene therapy CTx-TFEB for ALS

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 href="https://myrotx.com/">Myrobalan Therapeutics</a> will use a $400,000 grant from the <a href="https://www.als.org/">ALS Association</a> — along with $24 million in new financing — to help advance the development of its oral CSF1 receptor (CSF-1R) inhibitor program, intended to reduce neuroinflammation and promote nerve repair in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The ALS Association grant was awarded via the nonprofit&#8217;s Lawrence and Isabel Barnett Drug Development Program, which supports the preclinical evaluation of new and repurposed therapies for the neurodegenerative disorder. It follows on the heels of a <a href="https://myrotx.com/news-events/myrobalan-therapeutics-completes-series-a-financing-of-24-million-to-develop-potentially-first-in-class-cns-therapeutics-with-restorative-potential/">Series A funding by Myrobalan</a> last month that raised nearly a quarter of a billion dollars to further the development of the company&#8217;s treatment program for people with ALS.
Myrobalan&#8217;s CSF-1R inhibitor program was specifically designed for use in diseases of the central nervous system, or CNS, which comprises the brain and spinal cord. The company is advancing the clinical biomarker strategy toward the first clinical trial of its CSF-1R inhibitor, which is expected to start next year, according to a <a href="https://www.businesswire.com/news/home/20240206866405/en/Myrobalan-Therapeutics-Receives-a-Grant-from-the-ALS-Association-to-Advance-its-CSF1R-Inhibitor-Program">Myrobalan press release</a>. The new monies will keep the program on track, according to the release, which said Myrobalan was &#8220;very pleased to receive this grant from the ALS Association.&#8221;
“This recognition affirms Myrobalan’s commitment to CNS drug development and empowers us to enhance our development efforts with respect to CSF-1R and ALS,” said Jing Wang, PhD, the company&#8217;s CEO and co-founder.
&#8220;Ultimately, our goal is to make a CSF-1R inhibitor therapy accessible to all patients grappling with ALS,&#8221; Wang said.
[recommended-reading id=86121]
<h3>Myrobalan aiming for 1st clinical trial of CSF-1R inhibitor next year</h3>
ALS is characterized by the progressive death of motor neurons, the nerve cells that control muscle movement. Loss of these neurons leads to <a href="https://alsnewstoday.com/symptoms-of-als/">symptoms</a> like muscle weakness and wasting, with a progressively worsening impact on a patient&#8217;s ability to perform tasks of daily living.
Although <a href="https://alsnewstoday.com/causes-of-als/">its causes</a> are largely unknown, several bodily systems are dysregulated in ALS, contributing to the disease&#8217;s onset and progression. Known ALS-related processes include inflammation of the brain — neuroinflammation — and demyelination, or the loss of the myelin sheath that protects nerve fibers and helps them send signals efficiently.
Causing these processes are alterations in microglia, the CNS’ resident immune cells, and myelin-producing cells called oligodendrocytes.
The company is working on oral, small molecule CSF-1R inhibitors. Myrobalan&#8217;s hypothesis is that intermittent treatment with CSF-1R inhibitors depletes microglia — whose ability to adapt is then impaired, thereby reducing inflammation and allowing remyelination, or the formation of new myelin.
[quote=&#8221;center&#8221;]We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies.[/quote]
According to Myrobalan, its CSF-1R inhibitor program was specifically designed for central nervous system diseases, and has shown potency, selectivity, and favorable CNS distribution in preclinical studies.
The ALS Association award, granted to scientists worldwide, is part of the nonprofit&#8217;s philanthropic funding.
“We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies,” said Kuldip Dave, PhD, ALS Association’s senior vice president of research.
“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 said.
In addition to ALS, Myrobalan plans to develop its CSF-1R inhibitors for other neurodegenerative indications, such as <a href="https://multiplesclerosisnewstoday.com/multiple-sclerosis-overview/">multiple sclerosis</a> and <a href="https://alzheimersnewstoday.com/what-is-alzheimers-disease/">Alzheimer’s disease</a>. The company also is developing other compounds to reduce microglia-driven neuroinflammation and improve remyelination.

$400K ALS Association grant will advance CSF-1R inhibitor program

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

To reflect a new era of commitment and determination in the fight against <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), The ALS Association Greater New York Chapter has become <a href="https://als-ny.org/">ALS United Greater New York</a>.
The rebranding of the New York City-based nonprofit represents a break from the <a href="https://www.als.org/">ALS Association</a>, to which the former chapter group belonged <a href="https://als-ny.org/who-we-are/what-we-do/">for 28 years</a>. ALS United Greater New York joins with 14 other former association affiliates to form ALS United.
The new, independent New York organization called the change a &#8220;transformation&#8221; and a bold step forward in its ongoing mission to find a cure for the progressive neurodegenerative disorder and support patients, families, and caregivers. The move also reflects advances in ALS care and research and will enable broader advocacy of policy changes of importance to the ALS community, the organization said.
ALS United Greater New York will continue to provide local support to ALS patients and help to drive innovation and accelerate research.
&#8220;This is more than a name change; it signifies an evolution for the organization. It represents an exciting future, building upon our decades-long legacy as a trusted resource and re-emerging as one of the largest, local ALS patient service organizations in the nation,&#8221; Kristen Cocoman, president and CEO of ALS United Greater New York, said in a <a href="https://www.prnewswire.com/news-releases/transforming-lives-als-association-greater-new-york-unveils-striking-evolution-as-als-united-greater-new-york-302033112.html">press release</a> from the organization. 
&#8220;As science advances so has our approach to care services, including the launch of more support groups, educational programs, financial support grants, and equipment and communication resources for those living with ALS and their families. Our partnership with 10 regional ALS clinics and centers allows us to take part in providing state-of-the-art care and clinical management services to families living with ALS.&#8221;
[recommended-reading id=88468]
<h3 class="p1"> Other founding ALS United members</h3>
The launch follows the organization&#8217;s 2023 legal separation from the ALS Association, an act joined by 14 other association chapters across the U.S., to form ALS United, a membership organization for patients and their families. A website for ALS United is being developed, ALS United Greater New York told ALS News Today.
In addition to ALS United Greater New York, the founding members of ALS United include ALS Arizona, ALS United Central &amp; Southern Ohio, ALS Golden West, ALS of Nevada, ALS New Mexico, ALS United North Carolina, and ALS United Orange County. Others are ALS United Rhode Island, ALS United Rocky Mountain, ALS United Greater Chicago, ALS United Connecticut, ALS United Georgia, ALS United Mid-Atlantic, and ALS Northwest.
ALS United Greater New York, which collaborates with fellow chapters, services Greater New York-area patients and families, including those in New York City, Long Island, the Hudson Valley, Westchester County, and northern and central New Jersey.
Formerly known as ALS Greater New York City, the organization joined the ALS Association in 1995 and changed its name to the ALS Association Greater New York Chapter.

Former ALS Association affiliate is now ALS United Greater New York

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

Some $2 million in new grants from two U.S. nonprofits are seeking to help scientists globally to find ways to slash the time it takes for an <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) diagnosis.
It&#8217;s now estimated to take up to 16 months from symptom onset before most individuals receive a <a href="https://alsnewstoday.com/als-diagnosis/">diagnosis of ALS</a> — too long, according to the <a href="https://www.als.org/">ALS Association</a> and <a href="https://www.alsfindingacure.org/">ALS Finding a Cure</a>, also known as ALSFAC.
To shorten that timeframe, the two organizations have collaborated on seven new grants, awarded to researchers worldwide following the ALSFAC&#8217;s Early ALS Diagnostics Initiative.
That initiative initially was launched by ALSFAC in the U.S. to find promising digital, molecular, imaging, and electrophysiological diagnostic approaches in ALS. A subsequent <a href="https://www.als.org/research/research-we-fund/our-awards-and-programs/partnership-grants">partnership grant</a> from the ALS Association earlier this year has now enabled an expansion of the number of projects to be funded and extended the initiative&#8217;s reach internationally.
&#8220;The longer it takes for someone to be diagnosed, the longer they are waiting to access the multidisciplinary care, treatments and trials that may help them live longer or with a better quality of life,&#8221; Kuldip Dave, PhD, the ALS Association&#8217;s senior vice president of research, said in a <a href="https://www.prnewswire.com/news-releases/als-finding-a-cure-the-als-association-partner-to-reduce-time-to-initial-als-diagnosis-301903955.html">press release</a>.
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<h3>Goal is to shorten time to diagnosis, now at 10-16 months</h3>
More than 150 years after ALS was first defined, there remains no definitive, universally accepted process for diagnosing the neurodegenerative disorder. This is largely because ALS shares common signs and symptoms with other conditions.
A diagnosis confirmation instead is largely dependent on an observation period to track symptom progression, combined with a series of clinical tests. As such, a diagnosis often can take more than a year. Indeed, it is estimated that patients generally wait about <a href="https://www.sciencedirect.com/science/article/pii/S0022510X20303919">10-16 months</a> from their first symptoms to receive an ALS diagnosis.
&#8220;That&#8217;s why this partnership with ALS Finding a Cure to support the development of new diagnostic approaches is so important,&#8221; Dave said, adding, &#8220;By combining our dollars, we can have a larger impact — funding more projects from researchers all around the world.&#8221;
While on their diagnostic journey, individuals ultimately determined to have ALS often receive unnecessary and expensive surgical procedures and medications. Patients also can become ineligible for clinical trials, which usually enroll people in the first years after symptom onset.
Diagnostic delays also can affect family planning and prevent early administration of <a href="https://alsnewstoday.com/als-treatment/">ALS treatments</a> that can help with disease management.
[quote=&#8221;center&#8221;]Now is a time of unprecedented promise for the field of ALS research and drug development. &#8230; This collaboration between foundations has made possible the support needed to shorten the time to diagnosis.[/quote] 
From developing new diagnostic blood tests to identifying possible biomarkers, the selected grant recipients are working on ways to quicken the time to ALS diagnosis, according to the two nonprofits.
The awardees hail from the U.S. and Europe, and come from academia, healthcare, and industry:
<ul>
<li>Erez Eitan, PhD, the co-founder and chief scientific officer at NeuroDex, with offices in the U.S. and Israel, is working to develop a blood test that measures the activity of genes controlled by the protein TDP-43. This protein forms abnormal clumps in the brain and spinal cord in about 97% of all ALS patients. As such, measuring the genes it controls could potentially be used to diagnose ALS early in the disease.</li>
<li>Mark Garret, MD, a neurologist at Massachusetts General Hospital and Harvard Medical School, aims to develop a decentralized and open innovation platform that will enable remote assessments of limb activity, speech, cognition, and behavior. Such assessments would be done in individuals at higher risk of developing ALS.</li>
<li>Roisin McMackin, PhD, a neuroscientist at Trinity College, in Dublin, Ireland, is seeking to find nerve function markers that help predict ALS in people carrying C9orf72 mutations — the most common genetic cause of ALS. These markers would be used in patients who have such mutations but no ALS symptoms and may help predict disease development.</li>
<li>Patrick Oeckl, PhD, from the Ulm University Hospital, in Germany, aims to study spinal fluid samples from asymptomatic individuals with ALS-linked genetic mutations. The goal of Oeckl&#8217;s research is to identify small protein fragments associated with early-stage ALS that could be used as the basis for a new diagnostic test.</li>
<li>Ghazaleh Sadri-Vakili, PhD, a professor of neurology at Massachusetts General Hospital and Harvard Medical School, is hoping to identify specific gene fusions that can be measured in the blood and spinal fluid and may serve as biomarkers of early ALS. The project specifically targets so-called breakpoint gene fusions.</li>
<li>Ian Thrippleton, PhD, chief technical officer at NemDx, in Massachusetts, aims to develop a comprehensive blood test that will assess TDP-43 activity, in addition to several other biomarkers associated with familial and sporadic ALS. His aim is to determine whether a combination of these biomarkers can help divide ALS patients into patient subgroups.</li>
<li>Philip C. Wong, PhD, a professor of pathology and neuroscience at Johns Hopkins University, in Maryland, will use the funding to develop a test that measures an abnormal protein that is produced when TDP-43 is not working normally.</li>
</ul>
&#8220;Now is a time of unprecedented promise for the field of ALS research and drug development,&#8221; said Merit Cudkowicz, MD, chief medical officer of ALS Finding a Cure.
&#8220;This collaboration between foundations has made possible the support needed to shorten the time to diagnosis. This has been a long-time interest for ALSFAC, and we look forward to advancing a solution towards this key need for the field,&#8221; added Cudkowicz, who also is chief of neurology at Mass General Hospital and the Julieanne Dorn professor of neurology at Harvard Medical School.
The ALS Association funds ALS research globally and supports patients and their families through advocacy and its nationwide clinical care network. ALS Finding a Cure, founded in 2014, seeks to identify scientific gaps in ALS understanding that impede cure discovery.

Tag: ALS Association