Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects motor neurons, or nerve cells that control muscle movement, causing their death.
Rilutek (riluzole), Tiglutik (riluzole oral suspension), and Radicava (edaravone) are the only medications currently approved to treat ALS, but they have limited efficacy. Thus, many experimental compounds are being investigated as potential treatments. These include BHV-0223, H.P. Acthar Gel, MN-166, GM604, AMX0035, Gilenya (fingolimod), IONIS-SOD1Rx (BIIB067), Arimoclomol, AT-1501, NP001, VM202, Reldesemtiv, Masitinib, NurOwn, NSI-566, CNS10-NPC-GDNF, and Mexiletine.
BHV-0223 is a new sublingual (placed under the tongue) formulation of the glutamate-modulating agent riluzole — the first medication approved by the U.S. Food and Drug Administration (FDA) to treat ALS. The orally dissolving medicine is being developed by Biohaven Pharmaceuticals and is designed to address the shortcomings associated with the solid oral dosage form, such as difficulty in swallowing.
H.P. Acthar Gel is an injectable, investigational treatment for ALS, being developed by Mallinckrodt Pharmaceuticals. It contains a highly purified preparation of adrenocorticotropic hormone (ACTH). Preclinical studies in mouse models suggest that H.P. Acthar Gel could act by affecting the immune system and preventing neuroinflammation, a hallmark of ALS.
MN-166, also known as ibudilast, is an investigational small molecule oral medicine being developed by MediciNova for the treatment of ALS and other neurological conditions. The anti-inflammatory and neuroprotective effects of MN-166 have been shown in preclinical and clinical studies.
GM604, a potential treatment for ALS being developed by Genervon, is designed based on a naturally occurring master regulatory peptide involved in the nervous system. It is thought that GM604 may prolong motor neuron survival in ALS patients.
AMX0035, from Amylyx, is a combination of two compounds, sodium phenylbutyrate and tauroursodeoxycholic acid. It is designed to reduce nerve cell death and inflammation by blocking key cellular pathways.
Gilenya (fingolimod) is a therapy developed by the ALS Therapy Development Institute (ALS TDI). It is already approved for the treatment of multiple sclerosis (MS) and is currently being investigated for the treatment of ALS. It works by inhibiting signaling from a receptor called sphingosine 1-phosphate (S1P) receptor, thereby reducing inflammation.
IONIS-SOD1Rx (BIIB067) is an investigational antisense therapy, being developed in a collaboration between Ionis Pharmaceuticals and Biogen, to slow the progression of familial ALS caused by mutations in the superoxide dismutase (SOD1) gene. The therapy acts by reducing the levels of toxic SOD1 protein.
Arimoclomol (Orph-001) is an investigational therapy being developed by Orphazyme and the University of Miami for the treatment of ALS. It acts by increasing the production of heat-shock proteins that bind and remove the toxic SOD1 protein.
AT-1501 is an investigational drug being developed by Anelixis Therapeutics to treat ALS. It is an antibody that binds and blocks a protein called CD40 ligand (CD40L). Thus, AT-1501 works by damping the CD40L pathway that has been shown to be overactive in ALS patients.
NP001 is an investigational, injectable therapy being developed by Neuraltus to slow or stop the progression of ALS. It contains chlorite, which can reduce neuroinflammation by changing the pro-inflammatory immune macrophages, a type of immune cell, back to their inactive normal state.
VM202 is an investigational DNA-based therapy being developed by VM Biopharma for the treatment of ALS. VM202 is a plasmid — a piece of circular DNA that contains the genetic information necessary to produce a protein called hepatocyte growth factor (HGF), which improves the survival and growth of nerve cells.
Reldesemtiv is an experimental therapy being developed by Cytokinetics to increase muscle strength in patients with ALS. It is a fast skeletal muscle troponin activator (FSTA) designed to slow the release of calcium from the regulatory troponin complex — a protein essential for the contraction of fast skeletal muscle fibers – increasing the ability of muscles to contract.
Masitinib is an oral therapy being developed by AB Science to treat symptoms of ALS. It targets immune cells and works by blocking the signaling proteins called tyrosine kinases, which play a role in inflammation.
NurOwn is an investigational mesenchymal stem cell (MSC)-based therapy, being developed by BrainStorm Cell Therapeutics, for the treatment of ALS. It uses MSCs taken from the patient that are then modified to develop into cells that promote nerve cell growth and survival.
NSI-566 is an experimental stem cell-based therapy being developed by Neuralstem for ALS. It consists of human spinal cord-derived neural stem cells (HSSC) designed to nurture, protect, or repair the patients’ remaining motor neurons.
CNS10-NPC-GDNF (human neural progenitor cells secreting glial cell line-derived neurotrophic factor) is an investigational stem cell-based therapy being developed by Cedars-Sinai for the treatment of ALS. It consists of neural progenitor cells that have been genetically engineered to produce a growth factor called glial cell line-derived neurotrophic factor, or GDNF.
Mexiletine, approved by the FDA to treat ventricular arrhythmias (irregular heartbeat), is being investigated to treat muscle cramps in ALS patients. It works by inhibiting the flow of charged sodium ions in the nerves, slowing the conduction of a nerve impulse.
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