Stryka-001 treatment in the FSHD-like mouse model

Budget

• Amount awarded: US$190,000
• FSHD Canada Foundation Contribution: US$25,000
• Date awarded: June 15, 2018 (for one year)

Lay Abstract

Investigator: Dean Burkin, PhD, and Peter Jones, PhD (University of Nevada, Reno School of Medicine)

This is a project to validate the efficacy of a potential drug therapy for FSHD in a mouse model of FSHD (developed in the laboratory of Peter Jones). Data from previous use of the drug in another disease, along with laboratory studies, indicate that Stryka-001 may promote muscle regeneration and recovery after damage. Preliminary results suggest that treatments using Stryka-001 result in reduced muscle loss and increased strength recovery after DUX4 expression has caused extensive muscle damage in the Jones mouse model of FSHD. The hope is that, while other efforts are underway to develop DUX4-modifying therapeutics, in the nearer term, Stryka-001 will offer an effective treatment option to mitigate further muscle damage and improve muscle strength. Stryka-001 can also be used in combination with other treatment options to speed muscle recovery for FSHD patients. Stryka-001 is already FDA approved for another indication, and therefore these studies may quickly translate into clinical trials within a year. If successful, FDA approval of Stryka-001 for use in FSHD patients could occur within three years.