Information revealed at the 2024 AAIC conference on the ACTC-DS-led study in DS-associated Alzheimer’s disease with Lilly’s anti-amyloid drug Kisunla.
At the 2024 Alzheimer’s Association International Conference in Philadelphia this past week, Down syndrome was on the center stage of international Alzheimer’s research. A panel chaired by Dr. Juan Fortea (Sant Pau Memory Unit, Barcelona) and Dr. Beau Ances (Washington University at St. Louis, MO) discussed the latest findings on the progression of Down syndrome-associated Alzheimer’s Disease (DS-AD). Dr. Fortea discussed fluid biomarkers of DS-AD, Dr. Patrick Lao (Columbia University Irving Medical Center, NY) discussed MRI imaging of DS-AD, Dr. Sigan Hartley (University of Wisconsin-Madison, WI) discussed cognitive changes during the progression of DS-AD, and Dr. Michael Rafii (Alzheimer’s Therapeutic Research Institute, CA) discussed clinical trials for DS-AD.
During the Q&A portion of the panel, Dr. Teresa Buracchio, Director of the Office of Neuroscience at the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration (FDA), conclusively confirmed that people with Down syndrome are included in the FDA labelling for recently approved anti-amyloid drugs Leqembi and Kisunla.
Speaking during the Q&A portion of a panel dedicated to Down syndrome and Alzheimer’s disease, Dr. Buracchio stated: “there are no contraindications to the labeling for the treatment of Alzheimer’s disease associated with Down syndrome.”
This is the first time an FDA official has publicly and clearly stated that people with Down syndrome who have Alzheimer’s disease are included in the class of patients that can be prescribed the new range of approved anti-amyloid treatments.
The FDA’s acknowledgement of people with DS-AD has been a gray area of policy since the agency approved the first anti-amyloid drug in 2021. LuMind IDSC has asked for clarification several times since then, offered guidance, and provided detailed public comments to policymakers. This week’s announcement is a big step forward in policy that will impact the lives of people with DS-AD.
The policy victory is met with an equally important acknowledgment that no one with Down syndrome has been included in any of the safety trials associated with the approved drugs. The FDA official said she recognized the difficulty doctors will have in recommending an anti-amyloid treatment to people with DS-AD without any corresponding safety data.
“There are appropriate use recommendations around safety,” Dr. Buracchio said. She suggested doctors need to use caution because no safety data currently exists for people with Down syndrome and Alzheimer’s disease.
As with any medical and therapeutic treatments, the ultimate decision to take a drug should be made by patients, their families, and their trusted medical team. LuMind IDSC does not recommend people with Down syndrome take Kisunla or Leqembi at this time, except to consider it in the case of a clinical trial. This guidance aligns with the recommendations of the LuMind IDSC /National Task Group on Dementia and Intellectual Disabilities expert panel of medical doctors and clinicians, and with recently published appropriate use criteria for this class of drugs.
In spite of the dearth of safety information to date, LuMind IDSC and other Down syndrome organizations are buoyed by the forward momentum on policy, and by the confirmation that the necessary safety data for people with DS-AD may well be on the way.
In June, an official from Eli Lilly Co (Lilly) first confirmed that the drug development company was planning to conduct a trial evaluating the safety of anti-amyloid drug Kisunla in people with Down syndrome. That message was confirmed again at this week’s AAIC conference with a scarce but important framework for the first anti-amyloid antibody drug trial to include people with Down syndrome.
During Lilly’s session at AAIC about post-FDA approval studies of donanemab, Dr. Robert Alexander, Chief Scientific Officer of the Alzheimer’s Prevention Initiative at the Banner Alzheimer’s Institute announced the first details of what will be the “Amyloid Lowering for Alzheimer’s in Down syndrome with Donanemab Investigation (ALADDIN) study in DS-AD.”
The ALADDIN study will be led by Dr. Michael Rafii of the Alzheimer’s Clinical Trials Consortium Down Syndrome (ACTC-DS), and is a randomized, double-blind, placebo-controlled, Phase 4 dose-escalation study evaluating the safety, tolerability, and efficacy of donanemab in adults with Down syndrome.
“The news that ACTC-DS are moving forward with the ALADDIN study for donanemab is hugely important for the Down syndrome community,” Hampus Hillerstrom said. He is the president & CEO of LuMind IDSC. “After many years of advocating, providing data, and breaking down policy barriers, we are finally at the threshold of seeing people with Down syndrome included in a clinical study for an anti-amyloid antibody drug. This is a watershed moment, and we are excited that researchers, clinicians, companies, the NIH and regulators are focusing increased attention to Alzheimer’s clinical trials for people with Down syndrome.”
What can I do today to influence the future of my loved one with Down syndrome?
Families will play an important role in accelerating research into DS-AD. Increased participation is critical for the next phase of Down syndrome research. Please consider:
- Signing the national petition calling for equity in access to treatment. Due to the recent efforts of our persistent and dynamic community, people with Down syndrome are beginning to be included in Alzheimer’s disease treatment trials. But full equity in research and medical care is not here yet, and we can’t stop this fight until children and adults with Down syndrome are included in research that impacts them. Everyone is invited to add their name to the community’s demand for equity for the Down syndrome community.
- Participating in research: speak to your doctor or clinic contact about upcoming clinical trials, and investigate research opportunities that are right for you and your family member with DS (LuMind IDSC opportunities).
- Getting familiar with the key policies, regulations, and barriers that prevent people with Down syndrome from being fully included in life-changing treatments. LuMind IDSC is leading multiple initiatives to break down those barriers, and your interest and support will help us see them through.
- Learning and sharing information on our web site about Down syndrome-associated Alzheimer’s disease: free, vetted resources for families available in myDSC, real-life stories, resources for medical professionals, too.
- Registering for our monthly newsletter to keep current on Down syndrome research news and the latest on clinical trials and treatment access for Alzheimer’s therapies.