Rare Neurology News
Amyotrophic lateral sclerosis (ALS)
6 / 100,000
Age of Onset
ALS is sometimes referred to as Lou Gehrig’s disease. Its namesake, Lou Gehrig played first base for the New York Yankees between 1923 and 1939. Pethaps the most famous person with ALS in recent times is the noted British physicist Stephen Hawking. Other famous people who reported to have ALS include Chinese leader Mao Zedong, actor Davis Niven, and football player Dwight Clark.†
5 Facts you should know
ALS is the most common type of motor neuron disease
Early symptoms include muscle twitching, cramping, stiffness, or weakness, slurred speech, and/or difficulty chewing or swallowing
The underlying neurological problems was first described by French neurologist Jean-Martin Charcot
About 10% of the people with ALS have at least one relative with the disease have the familial form of the disease
The average survival from onset to death is two to four years, though this can vary, and about 10% survive longer than 10 years
Interest Over Time
Common Signs & Symptoms
Generalized muscle weakness
(Ongoing loss of nerve cells)
(Involuntary muscle stiffness, contraction, or spasm)
Skeletal muscle atrophy
(Muscle degeneration, muscle wasting)
(Inability to move)
Fatigable weakness of respiratory muscles
(Brand name: Radicava) Manufactured by Mitsubishi Tanabe Pharma Corporation
FDA-approved indication: May 2017, edaravone (Radicava) was approved for the treatment of amyotrophic lateral sclerosis (ALS).
(Brand name: Rilutek) Manufactured by Sanofi
FDA-approved indication: December 1995, riluzole (Rilutek) was approved for the treatment of patients with amyotrophic lateral sclerosis. Riluzole extends survival and/or time to tracheostomy.
Riluzole oral suspension
(Brand name: Tiglutik) Manufactured by Italfarmaco SpA
FDA-approved indication: September 2018, riluzole oral suspension (Tiglutik) was approved for the treatment of amyotrophic lateral sclerosis (ALS).
Top Clinical Trials
|A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)|| This is a multi-center, two-part study of ION363 in up to 64 participants. Part 1 will consist of participants that will be randomized in a 2:1 ratio to receive a multi-dose regimen of ION363 or placebo for a period of 29 weeks, followed by Part 2, which will be an open-label period where all participants will receive ION363 for a period of 77 weeks.|
|Phase 3||Recruiting||Drug: ION363|Drug: Placebo||More Information|
|Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients||The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS).||Phase 3||Recruiting||Drug: Masitinib (6.0)|Drug: Riluzole|Drug: Placebo|Drug: Masitinib (4.5)||More Information|
|Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS)|| This is a Phase 3, international, multicenter, open-label, long-term extension study. The primary objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) for up to 96 weeks.||Phase 3||Recruiting||Drug: MT-1186||More Information|
|A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)|| The purpose of this study is to assess the effect of reldesemtiv versus placebo on functional outcomes in ALS.||Phase 3||Recruiting||Drug: Reldesemtiv|Drug: Placebo||More Information|
|An Efficacy and Safety Study of Ravulizumab in ALS Participants||The purpose of the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.||Phase 3||Active, not recruiting||Drug: Placebo|Biological: Ravulizumab||More Information|
|Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS||A Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase.||Phase 2|Phase 3||Recruiting||Drug: MN-166|Drug: placebo||More Information|
|HEALEY ALS Platform Trial - Master Protocol||The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.||Phase 2|Phase 3||Recruiting||Drug: Zilucoplan|Drug: Verdiperstat|Drug: CNM-Au8|Drug: Pridopidine||More Information|
|A Study of BIIB067 When Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation||The primary objective of this study is to evaluate the efficacy of BIIB067 when initiated in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability of BIIB067 and to evaluate the effect of BIIB067 on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).||Phase 3||Recruiting||Drug: BIIB067 (Tofersen)|Drug: Placebo||More Information|
|Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS||To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline up to Week 48:||Phase 3||Recruiting||Drug: MT-1186|Drug: Placebo||More Information|
|Long-Term Evaluation of BIIB067||The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), and efficacy of BIIB067 administered to participants with ALS and confirmed SOD1 mutation.||Phase 3||Active, not recruiting||Drug: BIIB067||More Information|
Top Treatments in Research
|Agent||Class/Mechanism of Action||Development Status||Company||Company Contact||Clinical Studies||More Information|
|BIIB067||BIIB067 is a second-generation antisense oligonucleotide (ASO) targeting the mRNA for superoxide dismutase 1. It mediates mRNA degradation to prevent SOD1 protein synthesis and reduce levels of SOD1 protein. BIIB067 is being developed for ALS caused by SOD1 mutations, which account for about 20 percent of all familial ALS and 2 percent of all ALS cases. Although the exact pathological mechanism remains unknown, mutant SOD1 is believed to exert a toxic action on motor neurons, and reducing its levels may be beneficial.||Pase 3||Biogen||US Biogen Clinical Trial Center 866-633-4636 email@example.com||https://clinicaltrials.gov/ct2/results?cond=ALS&term=BIIB067&cntry=&state=&city=&dist=||More Information|
|MT-1186||MT-1186 is an investigational oral formulation of edaravone being studied in patients with amyotrophic lateral sclerosis (ALS). Edaravone was discovered and developed by researchers together with Mitsubishi Tanabe Pharma Corporation (MTPC), headquartered in Osaka, Japan.||Phase 3||Mitsubishi Tanabe Pharma America, Inc. (MTPA)||MT1186@iconplc.com||https://clinicaltrials.gov/ct2/results?cond=als&term=mt-1186&cntry=&state=&city=&dist=||More Information|
|MN-166||MN-166 is a small molecule that inhibits the action of enzymes called phosphodiesterase (PDE) -4 and -10 and that of macrophage migration inhibitory factor (MIF), which all play important roles in inflammation. MN-166 also blocks the activity of cell signaling molecules that play a role in inflammation, promoting the survival, development, and function of nerve cells. Finally, it attenuates activated glial cells, which play a major role in certain neurological conditions.|
The anti-inflammatory and neuroprotective effects of MN-166 have been shown in preclinical and clinical studies.
|Phase 2/3||MediciNova, Inc||Kazuko Matsuda, MD, PhD, MPH 858-373-1500 firstname.lastname@example.org||https://clinicaltrials.gov/ct2/results?cond=ALS&term=mn-166&cntry=&state=&city=&dist=||More Information|
|Ravulizumab||Ravulizumab contains an antibody that is injected into the bloodstream and that inhibits a protein called C5, a member of the complement system. The complement system is part of the immune system and is thought to be implicated in neurodegeneration in ALS.|
Under normal conditions, the complement system acts as part of the innate immune system to enhance the function of antibodies and the immune cells that recognize threats like infections. The complement system is activated at a low level at all times. Under some circumstances, however, an ALS-triggering event, for example, such as increased inflammation around nerve cells in the brain, the complement system overreacts.
By inhibiting C5, Ultomiris interrupts the overreaction of the complement system. In that way, the therapy may be able to slow disease progression in ALS.
|Phase 3||Alexion Pharmaceuticals||n/a||https://clinicaltrials.gov/ct2/results?cond=&term=Ravulizumab&cntry=&state=&city=&dist=&Search=Search&flds=abcfy||More Information|
|Reldesemtiv||Reldesemtiv is a type of fast skeletal muscle troponin activator (FSTA) that aims to slow the decline of muscle function in patients with ALS. Muscles are complex structures made up of multiple muscle fibers. These fibers contain sarcomeres, the basic mechanical unit that contracts to make the muscles work, and is activated by calcium. The release of calcium is triggered by signals from the nervous system — signals that are reduced in conditions such as ALS.|
Reldesemtiv is designed to bind to and slow the rate of calcium release from the troponin complex, a regulatory protein that plays an essential role in the contraction of fast skeletal muscle fibers. By doing this, the treatment boosts the sensitivity of the sarcomere to calcium, which should increase the ability of the muscles to contract despite reduced nerve signaling due to ALS.
|Phase 3||Cytokinetics, Inc.||Cytokinetics, MD 650-624-2929 email@example.com ||https://clinicaltrials.gov/ct2/results?cond=&term=Reldesemtiv&cntry=&state=&city=&dist=||More Information|
|ION363||ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the FUS gene3. Mutant FUS causes motor neuron degeneration through a toxic gain of function mechanism2. In patients, mutant FUS protein aggregates in motor neurons4. Antisense-mediated reduction of mutant FUS protein in a FUS-ALS mouse model prevents motor neuron loss. It is hypothesized that reduction of FUS protein will reverse or prevent disease progression in FUS-ALS patients.||Phase 3||Ionis Pharmaceuticals, Inc.|| Ionis Pharmaceuticals 1-800-679-4747 firstname.lastname@example.org ||https://clinicaltrials.gov/ct2/results?cond=&term=ION363&cntry=&state=&city=&dist=||More Information|
|Masitinib||Because of its potent and selective activity against CSF1R, masitinib is able to inhibit the CSF1/CSF1R signaling pathway thereby regulating CSF1R-dependent cells such as microglia. By merit of its activity against c-Kit, LYN and FYN, masitinib is also able to inhibit the function of mast cells. The development of masitinib in Amyotrophic Lateral Sclerosis is therefore based on the pharmacological action of masitinib in microglia cells and mast cells, thereby slowing microglial-related disease progression, reducing neuro-inflammation, and modulating the neuronal microenvironment in both central and peripheral nervous systems.||Phase 3||AB Science||Clinical Study Coordinator +33(0)147200014 email@example.com ||https://clinicaltrials.gov/ct2/results?cond=ALS&term=Masitinib&cntry=&state=&city=&dist=&Search=Search&flds=abcfy||More Information|