ALS Research and Clinical Trials

The driving force behind what happens at the ALS Clinic at The Ohio State University Wexner Medical Center in Columbus, Ohio, is our unwavering dedication to finding better treatments and, eventually, a cure for amyotrophic lateral sclerosis (ALS) and other neuromuscular disorders.

That’s why we have such a strong emphasis on research and clinical trials in our clinic. Not only do many of our clinicians actively participate in research alongside their time in the clinic, but we also recognize the need of people with ALS to gain access quickly to experimental drugs for treatment. Time is of the essence, both for you and others who may be diagnosed with this disease in the future.

We offer clinical trials or recently approved therapies that other places can’t because of this focus on innovation. Our ALS experts are internationally known and regularly publish and present their findings, often setting the guidelines and standards followed at other medical centers across the country.

Active ALS clinical trials and projects

Our team is actively recruiting participants for various types of research and ALS clinical trials. A few studies are listed below, and more research is started every day.

To get the most up-to-date information, contact Cheyenne Grove at ALSResearch@osumc.edu.

Healey ALS Platform Trial

This trial accelerates the path to new ALS therapies by testing multiple treatments quickly and at once. You can enroll in regimens, which will screen different medications used for neurological disorders to see their effectiveness in treating ALS. We’re currently enrolling for several regimens. View a video about the Healy ALS Platform Trial.

Gene therapy for FUS mutations

If your ALS is caused by rare mutations in the fused in sarcoma (FUS) gene, you could be eligible for this treatment trial looking at the efficacy of a drug that could suppress the mutations in the gene.

Neuroscience Research Institute Brain Bank & Biorepository (NRI-BBB)

The goal of the NRI-BBB is to collect and store biospecimens, like blood, spinal fluid and tissue, from patients undergoing testing for clinical care.

When you see your doctor and undergo tests (such as blood and spinal tap tests) for the management of ALS, you can participate in this study by providing an extra sample of blood, spinal fluid or skin tissue to be stored for future research. These samples are essential in increasing our understanding of the disease process in ALS and other neuromuscular disorders.

National registry for neuromuscular disease data

During this natural history trial, we’ll collect clinical and health-outcome data throughout your disease. Launched by the Muscular Dystrophy Association, this information helps establish best practices, better predict progression and outcomes, and expedite research and clinical trials.

How do I enroll in an ALS clinical trial?

If you’re interested in enrolling in an ALS clinical trial, please email ALSResearch@osumc.edu. We can help you decide what trials you may be eligible for and whether joining one is right for you.

Preparing to join an ALS clinical trial

To expedite your clinical trial enrollment process, please begin assembling this necessary information:

• Medical records from your initial diagnosis
• Most recent breathing test results and other vital capacities
• List of current medications, supplements and over-the-counter drugs
• Latest neurological notes

ALS experts search for answers at Ohio State

Our research efforts in ALS involve exploring all facets of the disease, including the genetics that cause it, better and earlier diagnosis methods and possible treatments that will slow the progression of symptoms. To better understand ALS, we participate in every research phase from basic science research to preclinical testing on models to active clinical trials.

While our ultimate goal is finding a cure for this horrible disease, any discovery has the potential to positively impact those with ALS and their families — which motivates our work daily.

Defining types of research

People living with ALS can participate in the following:

• Natural history and biomarker studies – People participate in these by giving samples (blood, skin biopsies, cerebrospinal fluid) to allow us to understand how the disease progresses and to model the disease in the laboratory.
• Treatment trials – During these studies, people receive a potential therapy to see how it impacts the disease and their body. Sometimes participants are given a placebo (sugar pill, saline, other inactive medication) as a control group to test the efficacy of the treatment.

Basic science research for ALS

With basic science research, our researchers work in the laboratory to discover answers to basic questions about how ALS works. At Ohio State, you can participate in basic science research that can shed light on your own disease, as well as help others.

One way we do basic science research is through studies looking at individualized models of disease. Our researchers take your skin cells and turn them into a type of stem cell to reproduce your exact ALS condition in the laboratory. We then study the models to look for causes and develop therapies on a case-by-case basis. This research is the first step in developing personalized model systems so that in the future we can possibly discover personalized treatments for ALS.

Clinical trials at Ohio State

Ohio State initiates clinical trials, participates in trials collaboratively with other institutions (including Nationwide Children’s Hospital) and networks with other centers around the world to offer you the most effective treatment.

We are a member of the National Institutes of Health’s NeuroNEXT: Network for Excellence in Neuroscience Clinical Trials. This collaboration results in more promising treatments, providing people with ALS and their families more hope.

We also are a member of the Northeast ALS Consortium (NEALS), which provides you with access to clinical trials not available at surrounding regional medical centers. Our membership connects us to other NEALS researchers regarding disease progression and the best ways to address unusual or persistent problems.

Gene therapy for ALS at Ohio State

Our researchers have begun to focus their efforts on finding a cure for ALS in identifying genes that cause the disease and gene therapies that may be able to alter these mutations. Now that we know genetics play a larger role in ALS than originally thought, and we’ve identified various genes that are responsible for it, we believe gene therapy is the future of ALS treatments. For example, we’re one of the only places in the country administering the recently FDA-approved gene therapy Qalsody.

Many more clinical trials involving genetics and gene therapy are in the works but are not available to participants just yet.

Donating to Ohio State’s research efforts

Much of this research is made possible due to donations from people who want to see an end to this devastating disease. If you’d like, you can donate to help us discover new treatments.

Please feel free to contact Jennifer Lamb at 614-625-8931 or Jennifer.Lamb@osumc.edu if you have questions about how to make a gift or how your gift will impact our ALS research.

ALS research news

Advancements in ALS diagnosis and treatment are being made frequently at the Ohio State Wexner Medical Center. Here are some examples of the tremendous work done by our team every day:

• Ohio State researchers publish national guidelines for ALS genetic testing, counseling
• Wexner Medical Center among the first in the nation to administer new gene therapy for ALS
• New hope for neurological disorders
• Gene therapy is changing lives today; tomorrow, it could change millions
• Innovative approach centers neurological disorder care around the patient
• Pioneering gene therapy surgery changes young patient’s life, gives immeasurable hope to his parents