Akron Children’s Hospital Mito Clinic Research Update

ENERGY IN ACTION - EPISODE 090

I'm joined by Dr. Bruce Cohen, Dr. Matthew Ginsberg and Clinical Research Coordinator, Ashley Erdesky— all part of the clinical and research team at the Akron Children's Hospital Mitochondrial Center.

EPISODE HIGHLIGHTS

How does a patient discover if they qualify to participate in a clinical trial and how do they get started?

A patient can visit clinicaltrials.gov, enter their diagnosis and search for open and active trials. They can also find resources through MitoAction or United Mitochondrial Disease Foundation. More directly, a patient can visit us in Akron or visit another mitochondrial center to see what they're eligible for. Clinical trial requirements generally have a checklist of qualifications and other details and considerations. It may be difficult to know with certainty that a patient qualifies without speaking to a trial coordinator.

What is the importance of patients participating in clinical trials and why is it critical to move therapy developments forward?

It's absolutely essential. We can't know if a treatment is effective and safe without subjecting it to scientific inquiry and performing a clinical trial. Participating in a clinical trial is the greatest gift you can give to other patients who will come later. Even if a clinical trial shows that a medication is not effective, it's useful information that brings us one step closer to effective therapies. We need enough patients and enough data in trials to know whether or not a therapy is effective and every participant counts.

In a drug trial, how is it determined who will get the drug and who will get the placebo?

In most clinical trials, patients are randomized and double-blinded by computer before receiving the drug or placebo. To accurately compare patients, neither the doctor or patient will know if they're receiving the drug or placebo to ensure we get medications to market that work and don't have side effects.

What are your words of encouragement for patients that progress is being made and hope is on the horizon?

There is always a light on at our facility. Through trials, progress is being made and people are seeing the progress, so we won't stop and we'll continue to fight for our patients. This is an exciting time for mitochondrial disease. There are over 70 trials registered for mitochondrial disease and there's more reason to be helpful than ever before. Soon, we'll have treatments potentially curative for mitochondrial disease and there's so much hope.

LINKS & RESOURCES MENTIONED

North American Mitochondrial Disease Consortium Patient Registry and Biorepository

https://www.clinicaltrials.gov/ct2/show/NCT01694940

Active and Enrolling Trials at Akron Children's Hospital

https://www.clinicaltrials.gov/ct2/results?locn=Akron+Children%27s+Hospital&Search=Apply&recrs=b&recrs=a&recrs=f&recrs=d&age_v=&gndr=&type=&rslt=

Mitochondrial Care Network

https://www.mitonetwork.org/

NAMDC

https://namdc.rarediseasesnetwork.org/

United Mitochondrial Disease Foundation

https://www.umdf.org/

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