Ricolinostat (ACY-1215) is an experimental therapy being developed by Regenacy Pharmaceuticals to treat diabetic and chemotherapy-induced peripheral neuropathy.

The company also entered a partnership with the Charcot-Marie-Tooth Association (CMTA) in 2018 to advance the development of ricolinostat to possibly treat Charcot-Marie-Tooth (CMT) disease.

How does ricolinostat work?

CMT is characterized by peripheral neuropathy, or damage to the nerves that carry messages between the brain and spinal cord and the rest of the body. This damage leads to weakness, numbness, and pain in the hands, feet, arms, and legs.

Nerve cells in the peripheral nervous system often have axons, or extensions, that cover long distances. Most cellular components are manufactured close to the body of the cell, near the nucleus. Proteins, as they are made, need to be transported from their manufacturing center near the nucleus to a cell’s far reaches. Because the axons of peripheral nerves are so long, this transport is especially challenging. Vesicles (a type of small “shipping” compartment) and proteins travel along a cellular “railway” made up of tubulin proteins woven together like fibers to make a thick, more stable transit path.

In at least some cases of CMT, the disease may be caused by the destabilization of this tubulin supply network. A particular chemical modification called acetylation changes the way tubulin folds, so that the fibers stick to each other instead of existing as individual subunits; the loss of the chemical modification makes the tubulin proteins unstable, and the network falls apart. Without the supply network, vesicles cannot get to the far reaches of a cell’s axon, and nerve cells cannot work properly.

Ricolinostat is a small molecule that inhibits an enzyme called histone deacetylase 6 (HDAC6), which removes acetyl groups from proteins. By blocking this enzyme, the acetylation on tubulin remains and stabilizes the supply network in the nerve. If the supply network is stable, the neuropathy caused by CMT may ease or end altogether.

Ricolinostat in clinical trials

Ricolinostat has been shown to be safe and well-tolerated in Phase 1 and Phase 2 clinical trials involving healthy volunteers.

Regenacy has announced that it is planning to begin Phase 2 clinical trials of ricolinostat in treating peripheral neuropathy caused by CMT. The company, however, has not published updated data on this potential therapy since 2018.

 

Last updated: Feb. 26, 2020

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Charcot-Marie-Tooth News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Emily holds a Ph.D. in Biochemistry from the University of Iowa and is currently a postdoctoral scholar at the University of Wisconsin-Madison. She graduated with a Masters in Chemistry from the Georgia Institute of Technology and holds a Bachelors in Biology and Chemistry from the University of Central Arkansas. Emily is passionate about science communication, and, in her free time, writes and illustrates children’s stories.
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Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.
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Emily holds a Ph.D. in Biochemistry from the University of Iowa and is currently a postdoctoral scholar at the University of Wisconsin-Madison. She graduated with a Masters in Chemistry from the Georgia Institute of Technology and holds a Bachelors in Biology and Chemistry from the University of Central Arkansas. Emily is passionate about science communication, and, in her free time, writes and illustrates children’s stories.
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