The ALS Association, ALS Finding A Cure, and MDA Co-Fund Clinical Trial Grant to Study Restoring Immune System Function to Treat ALS

The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The principal investigator is Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of the Stanley H. Appel Department of Neurology and the Peggy and Gary Edwards Distinguished Chair in ALS at Houston Methodist Hospital, and professor of Neurology at Weill Cornell Medical College. Dr. Appel is an internationally renowned researcher and neurologist and is one of the country’s foremost experts on amyotrophic lateral sclerosis (ALS), a progressive neuromuscular disease that destroys muscle-controlling nerve cells called motor neurons, eventually causing muscles to become nonfunctional.

Dr. Stanley H. Appel

The funding award will allow Dr. Appel to expand the first-in-human study that demonstrated promising results leveraging patients’ own immune cells to treat ALS. The clinical trial grant is a collaborative study between the ALS team at Houston Methodist Hospital led by Dr. Appel and the ALS team at Mass General Hospital led by Merit Cudkowicz, MD, chief of Neurology at Mass General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School. Dr. Cudkowicz is the director of the Sean M. Healey & AMG Center for ALS (Healey Center) and the Neurological Clinical Research Institute (NCRI) at Mass General Hospital. She is the co-founder and former co-chair of the Northeast ALS Consortium (NEALS) and serves as the chief medical officer of ALSFAC. Drs. Sabrina Paganoni and James Berry are co-investigators on the team from the Healey Center.

“We are proud to support a dedicated community of scientists and clinicians who are refining promising research approaches in the exciting new field of neuro-immunology to further define the causes of ALS and develop strategies to treat or block the disease process,” says Peter Foss, president of ALSFAC.

“Dr. Appel’s work gives us great hope,” says Neil Thakur, executive vice president of mission strategy at The ALS Association. “We are grateful to Dr. Appel and Dr. Jason Thonhoff [at Houston Methodist] as well as all the scientists in the lab, and especially grateful to the volunteers in the studies. These volunteers are putting themselves on the front line of science to fight ALS.”

Regulatory T-lymphocytes (Tregs) are a type of T-lymphocyte (a white blood cell that plays a central role in the immune response) that reduces immune system activation. Tregs are decreased in both numbers and function in the blood of people with ALS. Because Tregs are reduced in people with ALS, harmful immune processes can occur. In fact, people with more rapid ALS progression have even lower Treg function.

Building on positive preclinical results showing that it was possible to restore Treg function outside the body, the first clinical trial in humans demonstrated that Tregs could be extracted from people living with ALS, expanded in vitro, and then safely infused back into the same patients. In the three people who received this treatment with “expanded Tregs” in the first trial, a significant improvement in their Treg suppressive function was observed, as well as an apparent slowing of disease progression.

“Now is a time of unprecedented promise for the field of ALS research and drug development,” Dr. Cudkowicz says. “This collaboration among the three foundations has made possible the support needed to conduct a trial of this nature. It is only with this support that we will be able to continue to explore the possibility of harnessing the immune system to slow disease progression in ALS — possibly bringing us closer to a breakthrough that may one day lead to a cure.”

About the clinical trial

Drs. Appel and Thonhoff will expand on the promising results of the first-in-human trial by collaborating with Drs. Cudkowicz, Berry, and Paganoni on a randomized, placebo-controlled phase 2a clinical trial of infusions of expanded regulatory (EPAR) T-cells followed by an ascending-dose, open-label extension trial. The Tregs will be expanded with the expert assistance of Dr. Fabio Triolo and his team in the GMP facility at the UT Health Science Center at Houston. The primary goal of the phase 2a trial is to enhance Treg suppressive function and thereby slow disease progression in patients with ALS. Enrollment will begin in the next month.

About ALS

ALS is a progressive neuromuscular disease that destroys muscle-controlling nerve cells called motor neurons. As these cells deteriorate and are eventually lost, the muscles they control become weak and then nonfunctional. Each person’s disease course is unique, but eventually most patients will not be able to stand or walk, get in or out of bed on their own, use their hands and arms, or breathe without assistance. Currently, there is no cure for ALS and approved therapies only moderately slow the progression of the disease.

About ALS Finding a Cure

ALS Finding a Cure is dedicated to being a game changer in discovering a cure for this fatal disease. Founded in 2014 by noted philanthropist and Conair Founder and Chairman, Leandro P. Rizzuto, as a tribute to Christie Rizzuto, his daughter-in-law who was diagnosed with ALS in 2009 at the age of 41, and under the leadership of Denis Rizzuto, Peter N. Foss, and a team of renowned ALS researchers, ALS Finding a Cure is focused on identifying the gaps in the scientific understanding of ALS that are preventing the development of a cure. The organization, a program of The Leandro P. Rizzuto Foundation, collaborates with a wide range of companies, ALS organizations, other disease research organizations, and ALS patients and families to ensure research efforts are non-duplicative, synergistic, and focused on the ultimate goal: finding a cure.

About The ALS Association

The ALS Association is the only national nonprofit organization fighting ALS on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit the website at www.alsa.org.

39 thoughts on “The ALS Association, ALS Finding A Cure, and MDA Co-Fund Clinical Trial Grant to Study Restoring Immune System Function to Treat ALS”

  1. Please can you contact myself re the trial , I have been looking at your trial on twitter and I have been talking to Ammar Al chalabi from kings college London . He told me to contact you to see if my father would be eligible for the disease trial .
    My contact number is 07399174950

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  2. This is William D Hatch. I live in Denver Colorado area. I would like to be considered for inclusion in one of your clinical trials in 2020. My address is:
    7944 S. Depew St., Unit D, Littleton, Colorado, 80128 and my phone number is: 925-337-0853

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  3. I HAVE ALS and would like to be part of this trial. My name is Susan Grucci and I am a patient of Dr. Katherine Nicholson at Mass. General Hospital. I can’t speak but you can text me at 860-810-9084. You can also contact my son Kyle Grucci at 413-244-6014. I look forward to hearing from you.
    Thank you!

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    1. My Mother died of ALS and my sister now cannot move neck down. She is 69 years old. She has good swallowing, speech and respiratory. My mother lost swallow, speech and breathing but could always walk until she died at 66 years old. Please keep me informed if it works, I would happily help my sister with stem cells if that is found to work.

      Mary

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  4. Diagnosed September 2018 when I had pain in right hand. 67 years old still walking and good use of my left arm and can make a fist with my right hand. A biker 🚴‍♀️ for many years. Would love to take part in this trial.

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  5. Thank you for your important work. My brother in law has ALS. He’s 56, a Veteran of the US Navy and fighting this disease like hero. If any possible consideration for participation in this study, please contact me.

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  6. I am 44 and was diagnosed on March 21 2019. I started having twitching in my shoulders Dec 2017 that has progressed to drop foot, and weakness in my hands. I would like to participate in your trial also.

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  7. Hello I’m Maria, I was diagnosed with als in May 2019 it’s been a year already and my insurance Medicaid Superior-will not cover my treatment for Radicava IV fusion . My doctor and insurance case manager are trying to get me approved for this treatment but they keep being turned away. Please I beg that you choose me for your studies I would benefit a lot I’m still mobile and I wish to preserve some of my mobility as much as I can . I’m 55years old and I would like for you to consider me for your testing. I don’t understand how people can come out with medication for illness and then restrict the only people who can benefit from it or make it impossible for someone like me to receive this treatment. Please help me in Jesus Christ name I pray Amen

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  8. I am 76 years old. I felt something was wrong in March of 2018 – I had
    a lot of saliva and lisped a little. They thought I had a mild stroke – if it were only so. I was diagnosed with ALS in August of that year.

    At this point – I have more saliva, and my words are getting very slurred. My right hand, and arm, are weak, My left arm is starting to feel a little weak.

    I live in Tucson, AZ – there are no test trials going on here – I would really like to participate in one.

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  9. My mother passed away on June 25.2019 and had bulbar palsy . one of my older sister s has als..its all the same family and I’ve never heard of two family members having it. Im learning as much as I can and hope I our nieces don’t get it. My prayers are with you all. We need a cure!

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  10. I just want to say thank you to Dr. Appel and ALS team for giving hope for future candidates that will be diagnosed with ALS. My youngest sister was diagnosed five years ago and lost her speech, walking and all other motor skills. It’s too late for my sister but she’s fighting hard everyday to stay positive with this horrific disease. God bless you.

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  11. I am a 73 year-Old retired Lutheran pastor, who was diagnosed with ALS in 2018. Though in a motorized wheelchair I otherwise remain reasonably healthy. I make myself available in whatever way to fight this awful disease. I’m working as an advocate, but I’m open to participating in clinical trials for which I might qualify.

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  12. My husband was diagnosed with Als in may of 2017, he’s been getting Radicava since may of 2018, still walking around the house, but outside with a walker and some times wheelchair, limited use of his arms, No problem with speech Or Respiratory! But I see he is deteriorating little by little, he is 70, he is a veteran, we are trying to fight this monster call ALS, if you can help us! Thank you!

    Like

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