The FSH Society is pleased to announce that it has received a grant of $20,000 from the FSHD Canada Foundation in support of an exciting, treatment-focused project, “Developing LNA-based therapy for facioscapulohumeral muscular dystrophy.”
“We are delighted to help Neil Camarta and his colleagues at FSHD Canada help make a huge impact on FSHD by helping to fund high-quality research, both critical and necessary—in Canada and around the globe,” said Daniel Perez, President, CEO and CSO of the FSH Society. “Projects as this one are ideal for achieving the purposes of both our organizations and helping to provide insights and hope for the constituents we serve.”
The project will be carried out by Yi-Wen Chen PhD, of Children’s National Health System Washington DC, and Toshifumi Yokota PhD, of the University of Alberta Faculty of Medicine and Dentistry. The project was reviewed favorably by the FSH Society’s scientific advisory board, and approved by Boards of the FSH Society and FSHD Canada Foundation. Its total budget of $179,104 over two years will be jointly funded by the FSH Society and FSHD Canada Foundation.
Drs. Chen and Yokota are investigating a promising method for developing a gene therapy to slow down or stop FSH muscular dystrophy (FSHD). They are studying an antisense oligonucleotide (AON) compound called LNA (locked nucleic acid) gapmer to reduce DUX4, a gene widely thought to play a key role in FSHD.
AONs are short gene-like molecules that bind to and inactivate target gene activities (in this case DUX4). LNA gapmers are designed to overcome some problems that made earlier AONs unsuitable for use as therapeutics. LNA gapmers are more stable, resistant to being degraded, more effective and can penetrate the cell membrane and get inside cells where the target DUX4 messaging molecules (mRNA) reside.
Dr. Yokota will continue to improve the anti-DUX4 LNA gapmer, testing them in FSHD cell lines, while Dr. Chen will test the safety and efficacy of the molecules in a mouse model of FSHD. If successful, this research could help advance the quest for a gene therapy that can be tried in FSHD patients.
FSHD Canada Foundation was established in 2012 with the assistance of the FSH Society to provide an avenue for Canadians who wish to support FSHD research through tax-deductible donations.
Neil Camarta, CEO, FSHD Canada Foundation, said, “since its beginning the partnership with FSH Society has helped enormously with the inception and development of the FSHD Canada Foundation and we are effectively serving the needs of Canadians with FSHD—in helping to understand FSHD and find a treatment.”