Ancora Funding Vanderbilt Research Into Potential CMT Treatments

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by Diana Campelo Delgado |

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LRSAM1 gene and CMT2P

Ancora Innovation is funding a research project at Vanderbilt University aiming to realize potential new treatments for Charcot-Marie-Tooth disease (CMT), according to a university press release.

The research will be led by Charles Sanders, PhD, a biochemistry professor and associate dean for research at Vanderbilt. Sanders and his team are focused on understanding how defects in membrane proteins contribute to disease.

In CMT, the team has helped to explain how duplications in the PMP22 gene, the genetic cause of CMT type 1A, ultimately lead to damage to the myelin sheath — the protective layer that coats nerve cells and helps them efficiently transmit nerve signals — and cause disease symptoms.

The PMP22 gene codes for a protein of the same name that plays a role in the maintenance and development of myelin. This protein is normally found at the cell surface, but a duplication of its gene significantly increases protein levels inside cells, which overwhelm the cells’ protein folding machinery. Ultimately, not enough PMP22 is folded properly and transported to its proper place, affecting myelin integrity.

“Reviewing the work of Professor Sanders’ lab was a strong reminder of the caliber and translational quality of research that comes from our colleagues in Basic Sciences,” said Peter Donofrio, chief of the neuromuscular division at Vanderbilt University Medical Center. “It was clear that the lab benchwork deserved to progress to clinical study, so I am very pleased to see this partnership come to life.” 

Sanders will be joined by Bruce Carter, PhD, a biochemistry professor and associate director of the Vanderbilt Brain InstituteCarter’s lab investigates the role of apoptosis — which refers to “programmed” cell death, as opposed to death caused by injury — in neural development.

Ancora Innovation was founded through the partnership of Vanderbilt University, a private research university in Nashville, and Deerfield Management, a healthcare firm advancing care through investment and philanthropy. The amount and length of funding given this project, the third at Vanderbilt supported by Ancora, was not released.

It connects Vanderbilt’s research portfolio with Deerfield’s drug development and commercialization expertise to provide support for high-risk, early stage drug discovery research that might not be possible otherwise.

“Our collaboration leverages the academic innovative life science research and Deerfield’s expertise in accelerating state-of-the-art drug development,” said William Slattery, a Deerfield partner. “We are thrilled to work with Professors Sanders and Carter and to support the translation of promising early discoveries into a potential treatment.”