This month represented an important step forward in the treatment of Alzheimer’s disease with Eli Lilly announcing the successful results of their phase III clinical trials for their monocolonal antibody (mAB) donanemab. In addition to decreasing the overall amyloid burden, patients on the drug showed slowed disease progression and sustained performance on standardized dementia screenings.
This trial and others have given credence to the amyloid hypothesis of Alzheimer’s disease which posits that the accumulation of beta-amyloid protein in the brain is a central contributor to the disease. This hypothesis arises from the identification of genetic variants in genes associated with beta-amyloid production that greatly predispose individuals to Alzheimer’s disease. Additionally, it has been observed that individuals with Down’s Syndrome (who have an additional copy of the APP gene on chromosome 21) are at a dramatically higher risk for developing Alzheimer’s disease, often with an earlier onset than the general population.
Unfortunately, despite the high incidence of Alzheimer’s disease among persons with Down’s Syndrome, this population has historically been excluded from clinical trials due to complexities with data analysis and interpretation, among other reasons. The impact of these exclusions is exacerbated by CMS rulings that only those patients participating in clinical trials will be able to get the cost of their prescriptions covered by Medicare. Michelle Sie Whitten makes a compelling case in her recent article for #stat news that this must change. https://www.statnews.com/2023/05/05/alzheimers-research-down-syndrome-monoclonal-antibodies/
This story highlights the urgent need to develop novel approaches for clinical trial management that allows greater inclusivity and enables everyone to have more equitable access to healthcare.
At Velsera, we’ve been on the front lines of efforts to make sure that individuals with Down Syndrome can take part in the crucial research needed to better understand the genetics underlying their differential clinical profiles. Since 2020, we have partnered with the NIH on the INCLUDE (INvestigation of Co-occurring conditions across the Lifespan to Understand Down syndromE) Project. The goal of this project is to create a world-class resource to share data that enables novel investigations into all Down Syndrome comorbidities across their lifespan and empowers clinicians, researchers, and policymakers to elicit change all the way from the clinic to society at large.