Expanded Access Opportunities

Pridopidine is a highly selective Sigma-1 receptor (S1R) agonist developed by Prilenia for the treatment of neurodegenerative and neurodevelopmental disorders. S1R regulates key cellular pathways, commonly impaired in neurodegeneration. Of particular interest is its role in the pathogenesis of ALS which is supported by human genetic and postmortem studies as well as by preclinical models. Pridopidine demonstrates robust neuroprotective effects in numerous preclinical models of neurodegenerative diseases including models of ALS. Compelling preclinical data supports the therapeutic potential of pridopidine in ALS. In ALS SOD1^G93A motor neurons (MNs), pridopidine exerts neuroprotective effects via activation of the S1R. Specifically, pridopidine increases MN survival, improves BDNF and GDNF axonal transport, and restores the neuro-muscular junction (NMJ) synaptic activity. In vivo, pridopidine treatment of SOD1^G93A mice reduces toxic protein aggregates and ameliorates muscle fiber wasting.

View the Pridopidine EAP on ClinicalTrials.gov

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In people with ALS, the body’s immune system becomes imbalanced and appears to hasten the loss of motor neurons in the brain and spinal cord. Regulatory T-cells help reduce inflammation and could lead to a more balanced immune system in people with ALS. The goal of this study is to reduce neuroinflammation, potentially slowing ALS progression, using specially prepared regulatory T-cells, called RAPA-501 cells. RAPA-501 cells are created through a series of steps by first taking the participant’s own blood though a specialized IV (apheresis), then isolating regulatory T-cells from the blood. Next, these regulatory T-cells are grown under special conditions in a petri dish, becoming RAPA-501 cells. The RAPA-501 cells are then returned to the participant through an intravenous infusion. Prior to the IV infusion of RAPA-501 cells, a low dose of chemotherapy is given to reduce the body’s immune response and potentially heighten the effects of the RAPA-501 cells.

View the RAPA-501 EAP on ClinicalTrials.gov

Download the RAPA-501 EAP Brochure

Motor neurons consume significant energy in order to function normally. In ALS, corrupted energy metabolism together with increased cellular stress lead to motor neuron degeneration. CNM-Au8 is a new class of medicine that provides an energetic assist to impaired motor neurons, helping them improve their ability to function more normally. CNM-Au8 acts catalytically to both support bioenergetic reactions inside cells and eliminate the harmful waste byproducts of cellular metabolism. Oral delivery of CNM-Au8 resulted in both neuroprotection and remyelination in multiple animal studies. Each 2 oz dose of CNM-Au8 is a concentrated, liquid suspension of pure gold nanocrystals that study participants drink every morning. These extremely small nanocrystals travel through the body and enter the brain and motor neuron cells where they enhance the ability of these cells to survive and communicate by supporting cellular metabolism. CNM-Au8 was demonstrated to be safe and well-tolerated by healthy volunteers in a Phase 1 study.

View the CNM-Au8 EAP on ClinicalTrials.gov

Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood-brain barrier, stabilizes proteins and, importantly, activates autophagy, which is the process that clears pathologic material from cells. In animal models of ALS, treatment with trehalose delayed onset of disease, prolonged survival and preserved motor function and motor neurons in the spinal cord. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a myriad of diseases with pathologic accumulation of storage material. The drug is manufactured by Seelos Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases.

View the Trehalose EAP on ClinicalTrials.gov

Watch a webinar about the science behind Trehalose