Title: Advocacy Groups Push for Access to Alzheimer’s Treatments for People with Down Syndrome
Subtitle: Efforts to Include Down Syndrome Individuals in Clinical Trials and Access to Promising Drugs Gain Momentum
CHICAGO, Oct 10 (Reuters) – A growing movement spearheaded by advocacy groups is calling for equal treatment for individuals with Down syndrome, to provide them with access to potentially life-saving drugs in the fight against Alzheimer’s disease. The push comes as pharmaceutical companies race to address the unique needs of this population.
Hampus Hillerstrom, former biotech executive and Chief Executive of the Down syndrome research organization LuMind IDSC, is leading the effort to gain parity for his 9-year-old son Oskar and others with Down syndrome. Hillerstrom is advocating for the inclusion of people with Down syndrome in clinical trials of Alzheimer’s treatments, as well as access to recently approved drugs like Eisai and Biogen’s Leqembi and Eli Lilly’s experimental donanemab.
Atypical Alzheimer’s Treatment Experts: Advocacy Groups at Odds with Prominent Neurologists
The movement, however, finds itself at odds with prominent neurologists who warn against immediate use of these drugs in the Down syndrome population. Five top neurologists interviewed by Reuters emphasize that these drugs have not been tested in this group and may pose additional safety risks, given their unique predisposition to Alzheimer’s. The neurologists express caution, stressing the need for thorough safety trials before treating people with Down syndrome.
Advocacy Groups Seek Medicare Policy Changes
Advocacy groups have taken their message directly to the U.S. government’s Medicare health program, urging changes to written policies that they believe may disqualify people with Down syndrome from reimbursement for the treatments. By including validated cognitive assessment scales in the Medicare coverage policy, advocates hope to ensure that memory changes in individuals with Down syndrome are detected. Currently, Medicare does not prohibit access to Leqembi, but it remains unclear whether the agency is considering changes to the Alzheimer’s registry requirements.
High-Risk Factors for Individuals with Down Syndrome
In Down syndrome, individuals inherit a third copy of chromosome 21, resulting in overproduction of beta amyloid, which leads to the accumulation of amyloid plaques in the brain. By age 30, most people with Down syndrome exhibit these abnormal clumps of amyloid. Furthermore, this population has higher-than-average rates of cerebral amyloid angiopathy (CAA), a condition that weakens artery walls and increases the risk of brain bleeding. Concerns have been raised that treatments like Leqembi may further compromise artery walls, exacerbating this risk in people with Down syndrome.
Longer Lifespan and Rising Alzheimer’s Risk
Advances in care have significantly improved the lives of people with Down syndrome, leading to a more than doubling of life expectancy, from an average age of 25 in 1983 to 60 today. This longer lifespan, however, has resulted in an increased likelihood of developing Alzheimer’s disease. Currently, almost all individuals with Down syndrome who live beyond age 60 are afflicted with Alzheimer’s.
Lobbying for Safety Trials and Inclusion in Future Drug Designs
Hillerstrom and fellow advocates are strongly urging pharmaceutical companies Eisai and Lilly to conduct safety trials specifically designed for the Down syndrome population. While these trials are in the planning stages, it may take three to four years before conclusive data is obtained. Advocates argue that individuals with Down syndrome should not have to wait years for FDA-approved drugs and that barriers to access should be removed.
Bioethicist and health policy expert Emily Largent highlights the importance of allowing individuals with Down syndrome to make informed decisions about the risks and benefits of treatment. In a bid to gather data and involve people in clinical trials, the National Institute on Aging is conducting screening trials that measure changes in memory, language, and attention specifically in people with Down syndrome.
Overall, the movement seeks to ensure that individuals with Down syndrome are given equal opportunities to receive potentially life-saving Alzheimer’s treatments, while also considering their unique risks and needs.
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