Cerebral palsy is the most common movement disorder in the United States, affecting 10,000 babies born every year. Andrea Domenighetti, a research scientist at Shirley Ryan AbilityLab, has discovered that an FDA-approved drug used to treat blood cancers shows promise for treating muscle contractures in cerebral palsy, and in April published his findings in American Journal of Physiology-Cell Physiology.
Now at a crossroads, Dr. Domenighetti has laid out a detailed plan to shepherd his research into clinical trials (summary below), and is looking for partners to make it a reality.
- Are you an industry partner that can provide translational research expertise or other in-kind resources?
- Are you an impact investor who wants to help children with cerebral palsy?
- Are you a patient or patient advocacy group who can help spread awareness?
Provide your information below to connect with Dr. Domenighetti.
Abridged Research Proposal
Milestone 1:
- Goal: Evaluate effects of drug on contractured muscle tissue from children with cerebral palsy
- Timeline: 2 Years
- Funding Needed $400,000
Milestone 2:
- Goal: Determine in vivo effect of drug on muscle satellite cells (MuSCs)— a population of resident stem cells indispensable for postnatal muscle development and repair
- Timeline: 1 Year
- Funding Needed: $200,000
Milestone 3:
- Goal: Design a multimodal rehabilitation strategy consisting of instrumented measurement of neuromuscular function in contractures subjected to the drug
- Timeline: 1 Year
- Funding Needed: $200,000