The Roche Report: Notes from an Evening of Research

Much of what we do in Down syndrome cognition research necessarily focuses on the future instead of on the “now.”  The research life cycle is laborious and long, and progress is incremental.  The process can seem painfully protracted when we look at it from the outside as parents, families, and friends who are eager for breakthroughs in treatment.

That’s one reason last night’s presentation was so exciting.  More than 300 people gathered in the Chicago area for a program put together by local parents’ group UPS for DownS.  They were there to learn about the Roche study of RG1662 — early-stage clinical trials of the first potential therapy designed to improve cognition in people with Down syndrome.

The multi-site study, which is now recruiting, will test the safety and tolerability of the drug in question in a small group of subjects with DS between the ages of 18-30.  Depending on results, the next step is to test the drug’s efficacy in improving attention and memory.  Later-stage clinical trials will involve a broader group of subjects and a deep, detailed examination of how well the drug works.  Fast-forward through the process, and maybe — just maybe — the eventual outcome will be what the formal documentation dryly calls “an improvement in communication, behavior, or daily living in the subject,” and what we call greater success in school, more opportunities in the workplace, and increased independence and fulfillment for the people we love with DS.

Elizabeth Berry-Kravis, MD, PhD

The presentation, featuring investigators Dr. Cesar Ochoa-Lubinoff and Dr. Elizabeth Berry-Kravis and moderated by UPS for DownS board member Dr. Nancy Keck, was meticulously detailed.  Drs. Ochoa-Lubinoff and Berry-Kravis went into some depth to describe the isolated structures in the brain they expect BP 5186582 to target and explain the desired neurochemical effects.

Cesar Ochoa-Lubinoff, MD

DSRTF’s Margie Doyle, who also addressed the group, found an extremely interested and engaged audience, and said the excitement was palpable: “There was a great deal of buzz at the end of the program, and many people speaking about how to get involved.  It was a wonderful feeling!”

Yes, we’re still focused mainly on the future, with long-term studies still to come.  But that future feels much closer now that trials are underway. In light of this tangible evidence of progress, there was a lot of hope in the room last night.  And there’s a lot of hope here at the DSRTF, too.  Dr. Michael Harpold, our Chief Scientific Officer, says the trials represent “years of hard work and dialogue between industry executives, scientists and families coming to fruition.”  In line with our goal of identifying and supporting the most promising research in Down syndrome cognition treatment, we’re proud to be a supporter and collaborator in this effort.  We’re thrilled to be part of a process that may indeed be slow, but could bring such great rewards.

Thank you to UPS for DownS for making this event happen.  To learn more about the study or to apply as a participant, visit Roche’s Clinical Trial Protocol Registry.  We’ll keep you up to date here, too, and on

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