Treating cerebral palsy with a repurposed cancer drug

Treating cerebral palsy with a repurposed cancer drug


Andrea Domenighetti at the Shirley Ryan AbilityLab discovered an FDA-approved cancer drug can slow muscle contractures in cerebral palsy. Now he’s at a crossroads.

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.

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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

Written by
Kevin Leland
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