Amyotrophic Lateral Sclerosis Clinical Trials 2023

What Are Amyotrophic Lateral Sclerosis Clinical Trials?

Amyotrophic Lateral Sclerosis (ALS), or Charcot's Disease, is the most common progressive neuromuscular pathology in adults. The progressive death of motor neurons characterizes it. These neurons control walking, speaking, swallowing, and breathing, among other things. This loss of motor neurons leads to muscle atrophy and progressive paralysis in patients.

There are two types of motor neurons:

• Central motor neurons, located in a particular region of our brain, the motor cortex, transmit contraction orders to the spinal cord.
• Peripheral motor neurons, motor neurons located in the spinal cord, transmit motor information to the muscles.

The degeneration of these motor neurons during ALS leads to the loss of information transmission between the brain and the voluntary muscles, which are no longer solicited, do not contract, and atrophy.

Motor neuron damage can sometimes be associated with a loss of neurons in the brain's characteristic frontal and temporal regions, which can lead to cognitive and behavioral disorders of varying intensity. In its most severe form, this leads to frontotemporal dementia (FTD): 15% of patients with Charcot's disease also have FTD.

Dementia in FTD is not characterized, as in Alzheimer's disease, by memory impairment but by prominent behavioral changes, resulting in changes in the social behaviors and personalities of the patients.

Researchers from leading academic institutes worldwide have been studying the diseases for years to determine the origin of the disease due to hereditary genetic mutations and new therapeutic methods.

Why Is Amyotrophic Lateral Sclerosis Being Studied Through Clinical Trials?

There are different lines of research for ASL. The first axis of research is the identification of biological markers characteristic of the disease. Discovering such molecules would make it possible to treat patients more quickly and improve their quality of life.

For the moment, the mechanisms at the origin of the death of neurons and the disease initiation remain unknown. Researchers want to better understand them to open the door to developing new therapies through Amyotrophic Lateral Sclerosis Clinical Trials.

Finally, the third area of ​​research is the development of effective treatments to manage the disease. Several approaches are being studied which aim to limit neurons' degeneration or promote their regeneration.

So many avenues of research have been explored to better fight against ALS.

What Are the Types of Treatments Available for Amyotrophic Lateral Sclerosis?

To date, there is no curative treatment for Charcot's disease. However, the combination of neuroprotective treatment and multidisciplinary management, allowing the management of the different aspects of the disease, makes it possible to slow down the progression of symptoms.

What Are Some Recent Breakthrough Clinical Trials for Amyotrophic Lateral Sclerosis?

Below are some of the most notable Amyotrophic Lateral Sclerosis clinical trials:

2023: Evaluate the Effectiveness and Safety of Reldesemtiv in ASL Patients - This is an ongoing trial that aims to compare the efficacy and safety of reldesemtiv and placebo (twice daily) in alleviating the symptoms of ASL in patients aged 18 to 80. Initially, the patients will be given 300 mg of reldesemtiv, which will transition to an active drug period at the end of the 24th week. The results will determine new and improved therapeutic methods for ASL.

2021: Efficacy of AMX0035 in ASL Patients - This study evaluated the effectiveness of AMX0035 (sodium phenylbutyrate and taurursodiol) compared to placebo in terms of providing larger survival benefits to ASL patients. The results showed significant survival benefits in ASL patients given AMX0035 and found a new potential treatment that offers more time to ASL patients for whom time is invaluable.

Who Are Some of the Key Opinion Leaders & Institutes on Amyotrophic Lateral Sclerosis Clinical Trials Research?

Sabrina Paganoni, MD, PhD

Dr. Sabrina Paganoni is a physician-scientist at the Healey Center for ALS and Spaulding Rehabilitation Hospital and an Associate Professor at Harvard Medical School. Her research interests have always been in Amyotrophic Lateral Sclerosis, and she has conducted multiple Amyotrophic Lateral Sclerosis clinical trials in this regard.