ApoE4 Alzheimer’s Alliance launched as US advocacy group
Organization aims to garner support for patients with ApoE4 gene variant
A new patient advocacy organization, the ApoE4 Alzheimer’s Alliance, has launched in the U.S., with a goal of helping advance both supportive policies and the development of treatments for people with Alzheimer’s disease associated with the ApoE4 gene variant.
According to the National Institutes of Health, this variant of the Apoe gene is one of the strongest genetic risk factors for Alzheimer’s among individuals of European descent.
As one of its first actions, the new alliance sent a letter to the commissioner of the U.S. Food and Drug Administration (FDA), asking the agency to take specific actions related to potential risks among this patient population associated with available Alzheimer’s therapies.
“The ApoE4 community faces unique challenges that demand immediate attention,” Jamie Tyrone, a board member of ApoE4 Alzheimer’s Alliance, said in an organization press release. “Our goal is to drive meaningful change by advocating for research, access to care, and policies that address the specific needs of ApoE4 patients and their families.”
Alliance to advocate for specific funding into ApoE4-related research
Alzheimer’s disease is the most common cause of dementia, a group of cognitive symptoms that include memory loss, confusion, and impaired reasoning skills — ones essential for daily life. The disease’s symptoms are believed to be tied to the accumulation of clumps of beta-amyloid and tau proteins inside nerve cells in the brain, leading to their death.
Although the cause of Alzheimer’s remains unknown, genetic factors — including the presence of the ApoE4 gene variant — increase a person’s risk of developing the disease. People with this gene variant often experience an earlier disease onset and rapid progression, and have limited therapeutic options.
The alliance plans to advocate for increased federal funding for specific research into ApoE4-related Alzheimer’s. The group also will exhort U.S. policymakers and the FDA to prioritize targeted therapies and promote equal access to diagnostic testing and care.
Additionally, the ApoE4 Alzheimer’s Alliance will provide resources and support to patients and their families, and engage with healthcare professionals to improve care for these patients.
The organization also intends to collaborate with academic institutes and pharmaceutical companies for ApoE4-targeted research and the advancement of new therapies for patients. To aid these efforts, the group will share real-life experiences of people and families affected by the condition.
The current one-size-fits-all approach to Alzheimer’s treatment does not account for the substantial genetic heterogeneity of the disease. … Implementing these [suggested] recommendations will help ensure that ApoE4 carriers receive safe, effective, and personalized treatment strategies that reflect their unique medical needs.
In the letter to the FDA, the alliance calls for a regulatory mandate for genetic testing for ApoE4 among all patients eligible for anti-amyloid antibody treatments. These include Leqembi (lecanemab) and Kisunla (donanemab). The organization says such testing is needed to assess a patient’s risk profile.
These treatments work by binding to beta-amyloid, preventing its accumulation in the brain and slowing patients’ cognitive decline. However, such therapies may be associated with amyloid-related imaging abnormalities, or ARIA, a side effect marked by bleeding and/or swelling in the brain. The risk of ARIA is higher for people who carry the ApoE4 variant.
Additionally, the letter highlights the need to ensure all participants in clinical trials are aware of their ApoE4 status, and seeks that labeling and prescribing guidelines specifically refer to the elevated risk for ARIA in ApoE4 carriers. The organization also urged the FDA to facilitate clinical trials addressing the safety and efficacy of anti-amyloid therapies in people with ApoE4-related Alzheimer’s disease.
“The current one-size-fits-all approach to Alzheimer’s treatment does not account for the substantial genetic heterogeneity [variability] of the disease,” the letter states. “Implementing these recommendations will help ensure that ApoE4 carriers receive safe, effective, and personalized treatment strategies that reflect their unique medical needs.”
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