Riluzole, marketed as Rilutek, was the first treatment to be approved by the FDA for ALS in
1995. Rilutek is an orally swallowed pill. Its effects are modest, extending life by about
two to three months. The drug aims to address issues associated with the neurotransmitter
operates in a person's body. Destructive behavior of glutamate, called glutamate
excitotoxicity, has been linked to ALS disease progression.
Edaravone, also known as Radicava, was approved by the FDA in May 2017. Radicava is administered via intravenous infusion. The drug
aims to address oxidative stress
occurring in the cells, which is thought to
be caused by an overabundance of certain molecules of oxygen called “free radicals”. Once
infused into a person's blood stream Radicava diffuses around their body finding those oxygen molecules and helping the body to dispose of them.
In a six month Phase 3 clinical trial in Japan, Radicava was shown to slow down the loss of physical function in people with ALS
by 33% as compared to placebo. The greatest impact was seen in those who started getting infusions of the medication early on in
their disease. There are ongoing studies assessing the extent to which Radicava extends survival in people with ALS.
Today, there are dozens of clinical trials evaluating potential treatments enrolling people with ALS. For more
information on enrollment and inclusion criteria, visit our clinical trials page.
NurOwn is a proposed stem-cell
-based treatment created by
Brainstorm Cell Therapeutics (BCT). The therapy focuses on the cellular support system around a person
with ALS' motor neurons. It aims to slow disease progression by replacing the damaged system with an enhanced one.
Participants enrolled in NurOwn trials have their own stem cells harvested. These stem cells are then
programmed to secrete neurotrophic factors (NTFs), aimed at promoting growth and survival of nerve cells
when returned to the patient's spinal cord.
to read more about NurOwn clinical trial design and enrollment criteria.
Nurtec is a version of riluzole that dissolves almost instantly after being placed under the tongue (sublingual). The
originally approved formulation of riluzole, marketed as Rilutek, is administered as a pill. Though riluzole was approved
as a treatment more than 25 years ago, there are people with ALS who have not been able to access it, due to their inability
to swallow pills or their concerns about liver toxicity. Since Nurtec dissolves almost immediately when placed under the
tongue, it can be administered to people with ALS who cannot swallow pills. It is also an option for those who have been
advised against taking riluzole due to concerns about liver function, as Nurtec is approved at a smaller dose than Rilutek.
to read more about the Nurtec (BHV-0223) Expanded Access Program.
AMX0035, developed by Amylyx, is a pill that contains two small molecule compounds: sodium phenylbutyrate and tauroursodeoxycholic
acid (TUDCA). Sodium phenylbutyrate was approved by the FDA to treat urea cycle disorders. TUDCA is not approved by the FDA for
any marketing use. Amylyx is using a proprietary dose combination of these two compounds, aimed at impacting disease progression
by addressing issues with the mitochondria
and endoplasmic reticulum function of cells.
to read more about AMX0035 clinical trial design and enrollment criteria.
Reldesemtiv is a small molecule compound designed to slow the release of calcium with the intent of improving muscle function
and mobility. According to Cytokinetics, reldesemtiv is more potent than their other skeletal muscle activators, such as
tirasemtiv. This means that smaller doses of reldesemtiv can be used to result in the same, if not higher drug concentration
in a person's system. Additionally, because reldesemtiv does not cross the blood-brain barrier it should cause fewer significant
side effects than tirasemtiv.
to read more about reldesemtiv clinical trial design and enrollment criteria.
Copper ATSM (CuATSM) is a small molecule therapeutic that chaperones ions of copper into the body. The theory behind this approach
is that the body may not have sufficient levels of copper ions to support the proper folding of the SOD1 protein. Several
done on CuATSM, including work at the ALS Therapy Development Institute, Oregon State University, and the University of Melbourne. All
of these independent research efforts have shown similar results: the CuATSM treatment slowed disease in a highly effective way.
It is important to note that this therapeutic is delivered in a highly specific method. Simply swallowing raw copper will not
produce the same biological effect and could be extremely harmful.
to read more about CuATSM clinical trial design and enrollment criteria.
Biogen is developing specific antisense oligonucleotides (ASOs) to target SOD1-mediated ALS. Mutations in the SOD1 gene
result in misfolded SOD1 proteins, which in turn hinders the body's ability to rid itself of harmful, charged oxygen
molecules and causes significant cell stress. The ASO treatment targets RNA transcription, an important process that
occurs before a piece of the genetic code is translated to form a protein. In theory, ASOs are able to enter a cell
and break up the RNA before the harmful, misfolded proteins can be formed. Tofersen is a particular ASO treatment
that aims to help with the SOD1 subset of ALS by targeting the destruction of RNA produced by the SOD1 gene and
preventing the creation of misfolded SOD1 proteins.
to read more about Tofersen clinical trial design and enrollment criteria.
Some forms of ALS have been associated with inherited genetic mutations, including mutations in the C9orf72 gene. This
atypical type of genetic mutation —known as a repeat expansion—is thought to be the most common inherited gene mutation
causing ALS. BIIB078 targets and degrades specific messenger RNA from the C9orf72 gene. It then removes the RNA to prevent
further production of the toxic repeat expansion protein that the gene encodes. Importantly, BIIB078 largely preserves the
normal (not expanded) C9orf72 protein coming from the gene.
to read more about BIIB078 clinical trial design and enrollment criteria.
AT-1501 was discovered and developed at the ALS Therapy Development Institute.
began a Phase 1 clinical trial investigating
the safety of AT-1501 in 2018. AT-1501 (antiCD40L) is an antibody therapeutic with comprehensive and promising preclinical data. It acts
in a highly targeted, disease specific way to tamp down the immune system and aims to protect nerves against the progression of ALS
and Alzheimer's disease.
to read more about AT-1501 clinical trial design and enrollment criteria.
MediciNova's ibudilast is a potential small molecule therapy. It aims to help motor neuron survival by suppressing cytokines – amino
acids which cause motor neurons and supporting cells to become inflamed. Ibudilast is approved in Japan for the treatment of Asthma
and cerebrovascular disorders.
to read more about ibudilast clinical trial design and enrollment criteria.
Recent data suggest that the SOD1 mutation reduces the availability of a variety of molecular chaperones. Arimoclomol is a small molecule
which aims to amplify the production of these molecular chaperones, which are critical in the cellular response to stress and protein
misfolding. Arimoclomol has long been studied as a potential treatment for ALS, among other disorders. However, it has not yet been
approved as a treatment by either the European Medicines Agency (EMA) or the FDA.
to read more about arimoclomol clinical trial design and enrollment criteria.
Levosimendan aims to help muscles contract more easily by sensitizing the skeletal muscle to calcium signaling, making them contract
more easily. Levosimendan, marketed by Orion, was approved as a treatment for congestive heart failure in 2000 by the EMA. To date,
the use of levosimendan has not been approved by the FDA for the treatment of any disorder.
to read more about levosimendan clinical trial design and enrollment criteria.
H.P. Acthar Gel is FDA-approved for use in several diseases, such as multiple sclerosis, rheumatoid arthritis, sarcoidosis, and many
others. The potential treatment is delivered subcutaneously via an injection into the lower layers of skin. Research suggests that a
person with ALS' immune system may be causing significant damage to their own nervous system. H.P. Acthar Gel contains the adrenocorticotropic
hormone (ACTH), which stimulates the production of naturally produced steroid hormones, and may modify immune cell activity and reduce neuroinflammation.
to read more about H.P. Acthar Gel clinical trial design and enrollment criteria.
There are also a growing number of medicines that might help alleviate key symptoms of the disease. Baclofen may reduce
muscle spasms. Nuedexta
might help with
might reduce painful muscle cramps. A number of medicines including Robinul, Elavil, and Botox may help reduce salivation.