Cell and Gene Therapy Shows Early Promise Against Alzheimer’s, But Challenges Remain ###
Promising Therapies:
Lomecel-B:
Developed by Longeveron, this therapy uses allogeneic medicinal signaling cells to reduce inflammation and promote regeneration in the brain. It has shown positive results in Phase IIa trials and received FDA designation.
Gene Therapy:
Researchers have used a modified virus to deliver a gene that boosts levels of NF-alpha-1, protecting neurons and improving cognitive functions in mice with Alzheimer's-like symptoms.
Challenges:
High Failure Rates:
Alzheimer's clinical trials have historically high failure rates, ranging from 98% to 99.6%, making it challenging to advance new treatments.
Scalability:
Moving from small-scale trials to larger patient cohorts in Phase III trials is a significant challenge due to stringent inclusion criteria and high screening failure rates.
Safety Concerns:
Active immune therapies, while promising, come with potential safety risks, such as adverse reactions and the presence of amyloid in blood vessels.
Emerging Approaches:
Active Immune Therapies:
Companies like AC Immune and Vaxxinity are developing therapies that stimulate the immune system to generate antibodies against amyloid beta or tau proteins, which are associated with Alzheimer's.
Combination Therapies:
Researchers are exploring the efficacy of combining multiple treatments, such as monoclonal antibodies, tau protein targeting, and neuroprotective agents, to manage Alzheimer's disease effectively.
Current Landscape:
Approved Treatments:
Current treatments for Alzheimer's, such as Biogen’s Leqembi, have significant shortcomings, including adverse events like amyloid-related imaging abnormalities and infusion reactions.
Ongoing Research:
There are 141 drugs being tested in clinical trials for Alzheimer's, with 78% aimed at slowing disease progression. Promising drugs include lecanemab, donanemab, and remternetug.
Future Directions:
Early Diagnosis:
Early diagnosis using blood-based biomarkers and other diagnostic tools is crucial for the success of active immune therapies and other treatments.
Precision Medicine:
Tailored treatments based on a person’s genetic and molecular profile are emerging as a personalized approach to Alzheimer’s treatment.