Choosing a Therapy to Test

Our goal is to provide the best possible opportunities for our patients and their families to access experimental medications through targeted clinical trials.

Sometimes we design and organize our own clinical trials.  Other times, we participate in a trial organized by others, such as a drug company.

We have limited resources to organize and participate in clinical trials, so we try to pick the most promising therapies and studies based on a number of factors:

  • Scientific merit: 
    • Does the science behind the experimental therapy make sense?  Have rigorous experiments been done in animal models to support the proposed mechanisms of action? 
    • Did the drug have a strong benefit in the animal models?
    • Does the drug get into the brain? Has this previously been measured in humans? 
    • Is there a tool (such as a biomarker) available to make sure that the drug is having the desired biological effect in humans? 
    • Is there previous experience with the drug in humans that suggests that it might be beneficial?
  • Safety. 
    • Are there good data that suggest that testing the drug in our patients will be safe at the proposed dose? 
    • Will the potential benefits of the therapy outweigh the risks or known side effects? 
  • Participant burden.
    • Will it be easy for people to take the drug?
    • Will they be required to travel frequently to our center to receive the drug or other tests?
    • Will everyone have access to the drug at the end of the study, e.g., through an open label extension?
Evidence for Efficacy


Balancing Efficacy

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