Global Down Syndrome Foundation Funding for Alzheimer’s Research Results in Milestone Clinical Trial for People with Down Syndrome

Principal Investigators Dr. Huntington Potter and Dr. Peter Pressman Receive NIH Five-Year Grant to Study Whether Leukine® is Safe & Effective in Improving Cognition in Young Adults with Down Syndrome

 

DENVER – Oct. 21, 2021 – Today, the Global Down Syndrome Foundation (GLOBAL) announced that their lead Alzheimer’s scientist and colleagues were awarded a $4.6 million, five-year grant by the National Institutes of Aging to study whether a potential Alzheimer’s disease treatment is safe and effective in improving cognition function in young adults with Down syndrome.

Huntington Potter, PhD, is a professor of neurology, Director of the University of Colorado Alzheimer’s and Cognition Center (ACC), and Director of Alzheimer’s research at the Linda Crnic Institute for Down Syndrome (Crnic Institute). AAC and Crnic Institute are GLOBAL Affiliates. Dr. Pressman is a behavioral neurologist and assistant professor of neurology.

“This is the first clinical trial in years to target cognition in people with Down syndrome,” said Dr. Potter. “We are breaking new ground in studying both Down syndrome and Alzheimer’s disease, and we hope that this therapy will greatly improve their quality of life. We are so grateful to our affiliate, the Global Down Syndrome Foundation, for making this all possible with their early investment in our clinical trial.”

In 2016, GLOBAL and the Alzheimer’s Association joined forces to fund $1 million for Dr. Potter’s Phase II clinical trial of the FDA-approved drug, Leukine®, to determine whether it is safe and can help slow or prevent the progression of Alzheimer’s in the general population. The NIH provided another $7.5 million to complete the trial work which may be complete as early as 2026.

“We are very proud of Dr. Potter, Dr. Pressman, and all our brilliant scientists at the Crnic Institute and the Alzheimer’s and Cognition Center,” said Michelle Sie Whitten, President & CEO of GLOBAL. “And we are grateful that they were able to build upon our promising results in the general population, and then be awarded this important research grant that could help our loved ones with Down syndrome.”

“People with Down syndrome have unfortunately been excluded from a number of previous clinical trials,” said Dr. Pressman. “I’m excited to be able to work alongside this underrepresented group of individuals.”

The grant provides funding to design and complete a clinical trial in adults with Down syndrome using sargramostim, which is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF). GM-CSF is used to help increase white blood cell production in some cases of medical care, including after bone marrow transplantation and after induction chemotherapy in older patients with acute myelogenous leukemia.

Previous studies by Dr. Potter and his colleagues have also found that sargramostim treatment is associated with cognitive improvements in leukemia patients. In another clinical trial, Dr. Potter and fellow researchers found that three weeks of sargramostim treatment was safe and well-tolerated in participants with mild-to-moderate Alzheimer’s disease. The treatment was associated with improvement in cognition and with reduced biomarkers of neurodegeneration.

This new study is focused on quality-of-life improvements for members of the Down syndrome community, who have cognitive challenges and are particularly vulnerable to developing Alzheimer’s disease. As they grow older, every person with Down syndrome develops the brain pathology associated with Alzheimer’s disease, often beginning during middle adulthood. It is estimated that over 50% will get the early onset of dementia associated with Alzheimer’s in their 40s and 50s. Thanks to improved medical care, people with Down syndrome are living longer. In 1983, a person with Down syndrome lived to be only 28 years old on average. Today, the average life expectancy of a person with Down syndrome is nearly 60 years and continuing to climb.

A multidisciplinary team at CU Anschutz Medical Campus will study the safety and tolerability of the treatment. “If there is any indication that it is unsafe or not tolerated, the full trial will be stopped,” said Dr. Potter. “Safety is central to the execution of this grant.”

The new trial builds on this previous work and on a laboratory study in which CU researchers, led by Md. Mahiuddin Ahmed, PhD, senior research instructor of neurology, and Dr. Potter, found that GM-CSF reverses cognitive impairment and brain pathology in a mouse model of Down syndrome.

 

About Global Down Syndrome Foundation

The Global Down Syndrome Foundation (GLOBAL) is the largest non-profit in the U.S. working to save lives and dramatically improve health outcomes for people with Down syndrome. GLOBAL has donated more than $32 million to establish the first Down syndrome research institute supporting over 400 scientists and over 2,000 patients with Down syndrome from 33 states and 10 countries. Working closely with Congress and the National Institutes of Health, GLOBAL is the lead advocacy organization in the U.S. for Down syndrome research and medical care. GLOBAL has a membership of over 150 Down syndrome organizations worldwide and is part of a network of Affiliates – the Crnic Institute for Down Syndrome, the Sie Center for Down Syndrome, and the University of Colorado Alzheimer’s and Cognition Center – all on the Anschutz Medical Campus. GLOBAL’s widely circulated medical publications include GLOBAL Medical Care Guidelines for Adults with Down Syndrome, Prenatal Testing and Information about Down Syndrome, and the award-winning Down Syndrome WorldTM magazine. GLOBAL also organizes the Be Beautiful Be Yourself Fashion Show, the largest Down syndrome fundraiser in the world. Visit globaldownsyndrome.org and follow us on social media (Facebook & Twitter: @GDSFoundation, Instagram: @globaldownsyndrome).

About the University of Colorado Alzheimer’s and Cognition Center

​The mission of the University of Colorado Alzheimer’s and Cognition Center (ACC) is to discover effective early diagnostics, preventions, treatments, and, ultimately, cures for Alzheimer’s disease and related neurodegenerative disorders. The ACC is located at the CU Anschutz Medical Campus and is part of the School of Medicine, Department of Neurology. Our center offers clinical care, clinical research, and translational research through a collaboration between the University of Colorado and the University of Colorado Health System. We emphasize both research and clinical care using a team approach, with laboratory research scientists and neurology clinicians, who are also faculty members and clinical researchers at the CU School of Medicine, all working collaboratively on the science and treatments of neurodegenerative diseases. Our center also connects with the community outside of the university with education and support programs, participating in community events, and working with community advisory boards.

About the Linda Crnic Institute for Down Syndrome

The Linda Crnic Institute for Down Syndrome is one of the only academic research centers fully devoted to improving the lives of people with Down syndrome through advanced biomedical research, spanning from basic science to translational and clinical investigations. Founded through the generous support and partnership of the Global Down Syndrome Foundation, the Anna and John J. Sie Foundation and the University of Colorado, the Crnic Institute supports a thriving research program involving over 50 research teams across four campuses on the Colorado Front Range. To learn more, visit crnicinstitute.org and follow us on social media (Facebook & Twitter: @CrnicInstitute).

About the University of Colorado Anschutz Medical Campus

The University of Colorado Anschutz Medical Campus is a world-class medical destination at the forefront of transformative science, medicine, education and patient care. The campus encompasses the University of Colorado health professional schools, more than 60 centers and institutes, and two nationally ranked independent hospitals – UCHealth University of Colorado Hospital and Children’s Hospital Colorado – that treat more than two million adult and pediatric patients each year. Innovative, interconnected and highly collaborative, the University of Colorado Anschutz Medical Campus delivers life-changing treatments, patient care and professional training and conducts world-renowned research fueled by over $650 million in research grants. For more information, visit www.cuanschutz.edu.

 

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