BREAKING: Potential Therapeutic Approach to Treatment for Pelizaeus-Merzbacher Disease Announced

In breaking news regarding the treatment of Pelizaeus-Merzbacher disease (PMD), Case Western Reserve University and Ionis Pharmaceuticals, Inc. have signed an exclusive license agreement to advance and commercialize a therapeutic approach discovered by university medical researchers. 

Antisense treatments are a therapeutic approach discovered by a team of medical researchers, led by principal investigator and PMD Scientific Advisory Board member, Dr Paul Tesar, DPHIL. Dr. Tesar is also the Dr. Donald and Ruth Weber Goodman Professor of Innovative Therapeutics in the Department of Genetics and Genome Sciences at the Case Western Reserve School of Medicine.

Originally reported in the journal Nature in 2020, Tesar and his research team targeted a toxic protein resulting from the genetic mutation. Working with Ionis Pharmaceuticals, the research team successfully used a family of drugs known as antisense oligonucleotides to target the RNA strands that created the abnormal protein to stop its production in brain cells. As reported in Newswise, “this treatment with Ionis-developed antisense oligonucleotides reduced PMD’s hallmark symptoms and extended lifespan in mouse models of PMD, establishing the clinical potential of this approach.”

Dave Manley, PMD Foundation Board Chair, is ecstatic at the news. 

“On behalf of the entire Board of Directors for the PMD Foundation, Inc., we are excited to share this news with our PMD families and PMD community worldwide.

We would also like to congratulate Dr. Paul Tesar and his lab for the work that they have done developing this potential therapeutic treatment. Dr. Tesar was the Recipient of our $100,000 research grant in 2012 which played a key part in this research.  

Friends and families of those with PMD contributed to raising those funds to help make this news possible. Your continued generosity over the years is bearing fruit! We thank you! 

The continued support of the PMD community will be needed to assist with advancing this research. The PMD Foundation is committed to doing its part to help move this research forward. We are very thankful for our relationship with Dr. Paul Tesar and his lab, Case Western University, and Ionis Pharmaceuticals. 

We look forward to continuing to work together to develop this potential treatment and change the course of Pelizaeus-Merzbacher disease forever.”

Read the full story on Newswise.com: CWRU grants exclusive license to Ionis Pharmaceuticals to advance antisense therapy for Pelizaeus-Merzbacher disease