Treating cerebral palsy with a repurposed cancer drug

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.

Support Cerebral Palsy Research
Learn more about Dr. Domenighetti’s breakthrough and how you can help bring it to patients

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

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