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SPEAKER 1: There were previous trials with gene therapy, also evaluating the intraparenchymal administration. In some of these trials they had great hope, but unfortunately some of the results weren't so good. Do you have an explanation for what reasons it led to failure, and what we can do better in the future?

SPEAKER 2: I think we've learned the hard way, really, because one of the first clinical trials that we initiated was in patients with Parkinson's disease, with advanced stage of the disease. And we've delivered a [INAUDIBLE]. And we just did not infuse enough vector. We did not get the correct coverage. And so instead of getting-- in those that [INAUDIBLE] infusion, 50% or so of the coverage, we only got probably less than 10%.

And that was with the volumes which exceeded dramatically what other trials had been using at the time. So one very significant lesson that we've learned over the years, really, is that the delivery of [INAUDIBLE] using intraparenchymal route is directly related to the volume of infusion. And that translates into coverage of the targeted structure.

So unfortunate, the earlier trials have failed to realize and recognize that the effect and the success of gene therapy is really dependent on the gene delivery strategy. So that really took a long time for us to realize that, establish specific technology to allow us now to be able to use it in the patients which are undergoing the clinical trials.

Video

Lessons from Previous Experiences

Aristide Merola, MD, PhD, and Krystof Bankiewicz, MD, PhD, explain what physicians learned from previous gene therapy trials in order to improve the treatment.

Related Presenters

Aristide Merola, MD, PhD.

Aristide Merola, MD, PhD

Neurology
Associate Professor of Neurology

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Krystof Bankiewicz, MD, PhD.

Krystof Bankiewicz, MD, PhD

Neurosurgery
Professor of Neurological Surgery

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