HNF, Rarebase Project Working to Find New Therapies for CMT

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by Marisa Wexler, MS |

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The Hereditary Neuropathy Foundation, known as HNF, is joining forces with Rarebase, a biotech company, on a project that aims to identify potential treatments for multiple different types of Charcot-Marie-Tooth disease (CMT).

It is the first research initiative to tackle multiple types of CMT in one project, according to the HNF, a U.S.-based nonprofit.

“We’re thrilled to be collaborating with the HNF to help find treatments for patients with CMT,” Onno Faber, co-founder and CEO of Rarebase, said in a press release.

In the new collaboration, Rarebase will conduct a screen of thousands of compounds that are approved by the U.S. Food and Drug Administration (FDA) to treat conditions other than CMT — there currently are no FDA-approved therapies for any type of CMT.

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The goal is to identify medications that are already in use for other indications that may also hold therapeutic potential in CMT, so these treatments can be repurposed. A notable benefit of this strategy is that medications that are already in use tend to have well-established safety profiles.

“There are thousands of approved drugs, and many of them can have applications beyond their routinely prescribed indication,” Faber said. “If such treatments are out there for CMT, we will find them.”

The project specifically will look for compounds with promise in 10 subtypes of CMT. New medications that are not currently approved by the FDA for any use also will be included in the screening process.

“Our core mission is to deliver treatments to patients with CMT. We believe the Rarebase partnership will enable FDA approved drugs for clinical use for CMT,” said Allison Moore, founder and CEO of the HNF.

“If successful, HNF plans to expand the screens to other subtypes as funds become available,” Moore added.

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After promising candidate treatments are identified through the screen, they will be tested in patient-derived cellular models. Specifically, researchers will use induced pluripotent stem cell-derived neurons.

In this research model, scientists collect cells — typically skin cells — from people with CMT. Then, through a series of biochemical manipulations, the cells are “reverse engineered” into a kind of stem cell, called an induced pluripotent stem cell or iPSC.

Like other types of stem cells, iPSCs are able to grow and differentiate into other types of cells if given the right biological cues. In this case, researchers will prompt the stem cells to grow into neurons (nerve cells), the cell type that is most impacted by CMT. These patient-derived neurons will then be used to test promising therapeutic candidates.

In addition, HNF will be connecting Rarebase with its CMT researchers, clinicians, and other stakeholders around the world to potentially expand the research team.