Last week, I attended the Alzheimer's Association International Conference (AAIC) along with the science team. Here are my top five takeaways from the conference:
Encouraging Results in an Amyloid Drug Trial
The buildup of beta-amyloid proteins is a hallmark of Alzheimer's disease. Despite several recent clinical trial failures with amyloid drugs, Japanese drug company Eisai and its US partner Biogen presented encouraging results for an early trial of the experimental drug BAN2401. The drug cleared amyloid in the brains of study participants at all doses tested and demonstrated a statistically significant slowing of clinical decline at the highest dose. While the results are promising, several questions still need to be answered and larger trials are needed to reproduce these findings. Nevertheless, these results highlight the emerging role of modern clinical trial design, where novel tools like the amyloid PET scan Amyvid™ (developed in part by ADDF) can greatly improve the overall quality of trials. By using Amyvid™ to measure brain amyloid, the BAN2401 study ensured the right patients were enrolled and provided convincing evidence that the drug was efficacious.
Progress in Developing Affordable and Accessible Biomarkers
Last month, the ADDF announced a partnership with Bill Gates and other investors to create the Diagnostics Accelerator, an initiative that advances the development of novel biomarkers from blood and other peripheral fluids and tissues for Alzheimer's disease and related dementias. We are entering a new age of biomarker development and are closer than ever before to bringing affordable and accessible tests to the clinic. This was evident by the large focus on biomarker research at this year's AAIC meeting. One of the most interesting developments comes from Dr. Elisabeth Thijssen of the VU University Medical Center in the Netherlands. Dr. Thijssen is developing a highly sensitive blood test for beta-amyloid that can help to differentiate Alzheimer's disease from healthy subjects.
Reducing Agitation in Patients with Alzheimer's Disease
Approximately 50% of patients with Alzheimer's disease will experience agitation. This serious complication is particularly burdensome for caregivers and is one of the leading causes for placing patients into long-term care facilities. There are no approved drugs for agitation in Alzheimer's disease patients. Although certain antipsychotic drugs are currently prescribed off-label for agitation, they have significant side effects.
But there is encouraging news. Krista Lanctôt, PhD, of Sunnybrook Health Sciences Center in Toronto, Canada, reported that the synthetic cannabinoid nabilone significantly reduced agitation in patients with moderate to severe Alzheimer's disease. Nabilone is FDA-approved for the management of nausea and vomiting associated with cancer chemotherapy in patients who have not responded to conventional treatments. Dr. Lanctôt's study was funded in part by the ADDF.
Further development is still needed to determine the positive and negative effects of nabilone for agitation in patients with Alzheimer's disease. But the results of this research are encouraging, especially given the numerous failures in the field to date.
Intensive Blood Pressure Control May Reduce the Risk of Dementia and Mild Cognitive Impairment (MCI)
Preliminary results of the SPRINT MIND trial provided additional evidence that an intensive course of blood pressure treatment may reduce risk of MCI and dementia. Individuals receiving intensive hypertension management (targeting systolic blood pressure to 120mmHg or lower) had less risk of developing MCI than patients with standard hypertension treatment (targeting systolic blood pressure of 140mmHg or lower). These results echoed an observation study published earlier this year showing that individuals at age 50 with blood pressure over 130mmHg had an increased risk of dementia later in life.
Some researchers have expressed concern that aggressively lowering blood pressure could reduce blood flow to the brain and harm patients' mental abilities. And other trials have not shown the same benefit of aggressively lowering blood pressure. Researchers agree that further studies are needed to confirm these results.
New Treatment Fast-Tracked for Progressive Supranuclear Palsy (PSP)
Swiss-based company Asceneuron SA, presented results from a Phase 1 clinical trial for their investigational compound ASN120290, which reduces toxic levels of tau protein. The abnormal buildup of tau is one of the major culprits behind Alzheimer's disease and other related disorders, like PSP. PSP is a rapidly progressing rare neurodegenerative disease that affects movement, speech, vision, mood and behavior, and thinking. The FDA recently granted Orphan Drug Designation to ASN120290 for the treatment of PSP, which has the potential to become a first in class treatment for PSP and other tau-related dementias. The drug was shown to be safe in healthy subjects and plans are underway for the Phase 2 trial in patients. ADDF invested in the preclinical development of the drug.
The AAIC meeting highlighted significant progress towards the diagnosis and development of treatments for Alzheimer's disease. The ADDF is proud to support programs in pursuit of finding a cure.
Howard Fillit, MD is the Founding Executive Director and Chief Science Officer at the ADDF.