The initial focus will be on Alzheimer’s disease in adults with Down syndrome.
Marlborough, MA – December 28, 2017 – LuMind Research Down Syndrome Foundation announced today that it has funded a grant to support the planning effort required to establish a Down Syndrome Clinical Trial Consortium, focused initially on advancing Alzheimer’s therapies for individuals with Down syndrome.
The goal of this consortium is to enable research sponsors, both pharmaceutical companies and academic, to conduct clinical trials with promising therapeutic molecules targeting Alzheimer disease in people with Down syndrome. Such targets include amyloid beta plaques and tau tangles as well as the processes that lead to their formation. These targets are characteristic of Alzheimer’s disease in the general population as well as in people with Down syndrome.
The consortium planning effort will define the oversight, infrastructure and resources required to carry out Phase 2 and Phase 3 trials. In so doing, it hopes to lay the foundation for a consortium that serves to enhance the design and management of clinical trials to combat Alzheimer’s disease in the Down syndrome population. This important initiative will include experts from institutions across the United States and in London, UK.
Initially these experts will be Dr. William Mobley (University of California San Diego), Dr. Wayne Silverman (Kennedy Krieger Institute), Dr. Ira Lott (University of California Irvine), Drs. Diana Rosas, Florence Lei, and Brian Skotko (Massachusetts General Hospital) and Dr. Andre Strydom (King’s College London/Horizon 21 Consortium). They will collaborate, and work with other colleagues, to set the structure and build the groundwork for a larger international consortium while coordinating closely with a similar endeavor called Horizon 21 in Europe. Each of the experts recruited to the planning effort are from clinical research centers that have focused on advancing research and care initiatives for adults with Down syndrome. As part of this initiative, during the first year of this grant, the consortium will establish and harmonize internationally a set of criteria of clinician-generated information that will be catalogued in a common database to support the future rapid enrollment of clinical trials.
The decision to fund this grant was an outgrowth of a June 2017 Translational Key Opinion Leader (KOL) meeting initiated by LuMind RDS Foundation and supported by the Trisomy 21 Society on the topic of Alzheimer’s Disease in Down syndrome and that included 20 Down syndrome and Alzheimer’s experts, and pharma representatives.
“Establishing this consortium is a first important step to the ambitious goal to stimulate industry and academic sponsors to launch in the next years clinical trials in Alzheimer’s disease in the adult Down syndrome population with promising drug candidates, including some currently already in late stage trials in the general Alzheimer’s population. As many people with Down syndrome are getting older and nearly all have the pathology of Alzheimer’s already at age 40, there is a highly pressing need for Lumind RDS and other organizations to do all we can to prepare the Down syndrome population for such trials while providing important resources – such as this consortium – that sponsors need to effectively conduct trials in this large and important group of people affected by Alzheimer’s”, said Hillerstrom.
The lead principal investigator Dr. William Mobley will coordinate the consortium which will start in January 2018. “We are excited that this effort to plan the consortium has come together in only a few months since the meeting in June. It will fill a major need by expediting the creation of a network of sites with expertise in clinical trials and participant enrollment in the adult Down syndrome population. As large trials are likely to be complex, international in nature, and costly to conduct, the consortium planning group looks forward to working closely with key stakeholders and to a robust dialogue with pharma companies and academic sponsors in the US and internationally to enable and enhance multi-site, multi-country trials.” wrote Dr. Mobley.
For more information about the LuMind RDS’s research grants and the work we do to prevent Alzheimer’s onset in Down syndrome, visit www.lumindrds.org or contact us at research@lumindrds.org. To help LuMind RDS fund these research projects, visit events.lumindrds.org/donate.
Founded in 2004, the sole focus of LuMind Research Down Syndrome Foundation (LuMind RDS) has been the discovery, development, and translation of groundbreaking Down syndrome research. We have defined a clear path to drugs and interventions. We’re focused on exciting research that targets the root cause. Our objective is to improve health and independence of those with Down syndrome throughout their lifetime. In order to best accelerate the translation of research breakthroughs in Down syndrome, we are constantly exploring ways to collaborate with industry, clinics, researchers, and foundations across the globe.
Based on recent scientific findings, LuMind RDS has established four priorities for allocating funding during the next decade of research. These four priorities are: preventing Alzheimer’s onset, improving cognition, developing gene therapies, and advancing understanding. Current research studies — funded either in whole or part by LuMind RDS — target each of our four strategic scientific priorities.
Media contact: Marly Chevrette > mchevrette@lumindrds.org | 603-828-8857