Experimental Treatments for ALS

AMX0035

AMX0035 is a combination of two compounds — sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA) — that work together to minimize cellular mechanisms linked to cell death in ALS. While TUDCA improves mitochondria energy production, PB helps proteins acquire their normal shape, preventing the formation of protein clumps that cause nerve cell death. It is planned to move into Phase 3 testing.

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Arimoclomol

Arimoclomol is a potential therapy that triggers an increase in the production of heat shock proteins, or HSPs, which are involved in the body’s response to stress. In ALS, one of these HSPs can bind to faulty SOD1 protein and remove it. In this way, arimoclomol has the potential to reduce the rate of motor neuron death and slow disease progression. However, it recently failed a Phase 3 trial in ALS, and its future development for the disease is unknown. 

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AstroRX

AstroRx is a stem cell therapy comprised of fully matured human astrocytes derived from human embryonic stem cells, given by intrathecal (into the fluid surrounding the spinal cord) injection to support damaged motor neurons. Treatment is aimed at compensating for the malfunctioning astrocytes and help to prevent the loss of motor neurons, slowing ALS progression. The potential therapy has completed a Phase 1/2 trial.

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AT-1501

AT-1501 is an antibody against a protein called CD40 ligand (CD40L), which plays a role in regulating the immune response and that can trigger inflammation in the spinal cord. This pathway is overactive in ALS patients. Researchers think that by inhibiting CD40L, the potential therapy could block or delay the activation of the damaging inflammatory immune response in ALS patients. A Phase 2 trial is currently enrolling ALS patients. 

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Cannabis

Cannabis-derived products are being evaluated for their potential in treating ALS in various clinical trials. The active ingredients in cannabis — tetrahydrocannabinol (THC) and cannabidiol (CBD) — are called cannabinoids. They are believed to work as antioxidants and as anti-inflammatory and neuroprotective agents and thus might be able to slow or prevent further damage to nerve cells in ALS.

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CNM-Au8

CNM-Au8 is an experimental therapy being developed to treat patients with ALS, Parkinson’s disease, and multiple sclerosis. It contains a suspension of nanocrystalline gold that acts to support biological reactions within cells that generate energy, as well as help remove the destructive byproducts of cellular metabolism. Preclinical studies have demonstrated that CNM-Au8 is able to protect motor neurons from severe damage and death. It is being studied in Phase 2 trials.

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CNS10-NPC-GDNF

CNS10-NPC-GDNF is an experimental stem cell-based therapy. It is a type of stem cell called a human neural progenitor cell that has been genetically engineered to produce GDNF. It is hoped that when transplanted into the spinal cord of the patients, the cells will grow into astrocytes and provide GDNF to motor neurons. This approach may increase the survival of motor neurons, improving motor function and delaying disease progression. It has completed a Phase 1 trial in ALS patients.

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Engensis

Engensis (VM202) is a DNA-based gene therapy carrying the instructions to produce more of a protein called hepatocyte growth factor (HGF), which aids in the formation of new blood vessels, prevents muscle atrophy (shrinkage), and participates in the growth and survival of nerve cells. By boosting HGF production, Engensis may help lessen the progressive loss of motor control that characterizes ALS. A Phase 2a trial is currently recruiting ALS patients.

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GM6

GM6 is an artificially created peptide being developed for the treatment of ALS and other neurodegenerative diseases. In contrast to other therapies, GM6 does not have a distinct target, but rather affects multiple pathways to treat ALS and promote motor neuron survival by slowing degeneration through an unknown mechanism. It has completed Phase 2 trials in ALS patients. 

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IPL344

IPL344 is an experimental treatment being developed to slow ALS progression. IPL344 is a molecule that researchers designed to activate the Akt signaling pathway, which plays a role in the survival of cells. Studies have shown that the activity of the Akt pathway is reduced in patients with ALS. By activating the pathway, researchers think IPL344 may help protect nerve cells and slow the progression of the disease. A Phase 1 trial is currently recruiting ALS patients.

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Masitinib

Masitinib is an experimental oral tyrosine kinase inhibitor that targets cells of the immune system called the mast cells and macrophages. It works by blocking the activation of proteins called tyrosine kinases, which play a role in inflammation and chronic inflammatory states. A Phase 3 trial testing masitinib as an add-on therapy is currently recruiting ALS patients.

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MN-166

MN-166 is a small molecule that inhibits the action of enzymes called phosphodiesterase -4 and -10 and that of macrophage migration inhibitory factor, 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. A Phase 2/3 trial is currently recruiting ALS patients. 

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NSI-566

NSI-566 is an experimental stem cell-based therapy aimed at decreasing motor neuron loss and improving motor function in ALS patients. It consists of human spinal cord-derived neural stem cells, when injected into the spinal cord, differentiate into mature neurons. They surround and support the impaired motor neurons in ALS by integrating into the neural network and forming connections (synapses) with the patient’s own neurons. A Phase 3 trial is being planned.

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NPT520-34

NPT520-34 is an investigational oral small molecule that can penetrate the blood-brain barrier. It is able to reduce the production of proteins associated with astrocytes and microglia. In this way, the potential therapy may slow down ALS disease progression by reducing neuroinflammation. The potential therapy has completed a Phase 1 trial in healthy volunteers with positive results.

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NT0502

NT0502 is an experimental oral treatment being developed to reduce excessive salivation — drooling — a common problem in patients with ALS. The small molecule contained in NT0502 is an inhibitor that binds to the receptor to which acetylcholine would normally bind. By blocking this receptor (called a muscarinic receptor), NT0502 reduces the amount of saliva that is produced and secreted by the salivary glands. The potential therapy has completed a Phase 1 trial.

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NurOwn

NurOwn is an investigational therapy that uses a mesenchymal stem cell (MSC) platform to treat ALS. Using MSCs harvested from the patients themselves, it is aimed at helping nerve cells survive by fighting the neurodegeneration process. NurOwn’s technology encourages MSCs to develop into cells that secrete neurotrophic factors, which promote the growth of nervous tissue and also can help nerve cells survive through their own neuroprotective function.

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Pridopidine

Pridopidine is an investigational sigma-1 receptor agonist for ALS treatment. It is a small molecule that binds to the sigma-1 receptor on nerve and glial cells, which are the cells that support nerve cells. Scientists think that the sigma-1 receptor may play a role in protecting the nervous system against neurodegenerative diseases, including ALS. A Phase 2/3 trial is currently enrolling by invitation.

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Reldesemtiv

Reldesemtiv is an experimental therapy intended to improve muscle function and physical performance in people with diseases that cause muscle weakness and/or muscle fatigue, such as ALS. It is a type of fast skeletal muscle troponin activator (FSTA) aimed at slowing the decline of muscle function in people with ALS. A Phase 3 is being planned in ALS patients. 

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Tofersen

Tofersen is an investigational therapy aimed at slowing the progression of familial ALS. It is a type of antisense therapy in which an artificially created piece of DNA is designed to specifically bind to SOD1 mRNA, stopping the production of the misfolded SOD1 protein. In this way, tofersen may be able to slow the progression of this form of ALS. It is currently being studied in Phase 3 trials. 

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Tregs

Cell therapy using regulatory T-cells (Tregs) has shown promise in suppressing inflammation and slowing the progression of ALS. Scientists think there is an inverse link between levels of Tregs and the rate of ALS progression. This suggests that enhancing the Tregs level in people with ALS could delay the disease’s progression. The potential therapy is being investigated in a number of trials.

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TUDCA

TUDCA (tauroursodeoxycholic acid) is a small molecule that may be able to decrease nerve cell death, a hallmark of ALS, through its ability to act as an antioxidant that prevents toxic reactive oxygen species from accumulating inside cells. The molecule also has the ability to control immune function. It is currently being studied as an add-on treatment in Phase 2 and 3 trials.

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Ultomiris

Ultomiris contains an antibody that is injected into the bloodstream and that inhibits a protein called C5, a member of the complement system, which is thought to be implicated in neurodegeneration in ALS. By inhibiting C5, Ultomiris interrupts the overreaction of the complement system, potentially slowing ALS progression. It is currently being tested in Phase 3 trials in ALS patients.

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