Clinical trials are essential tools for advancing research and development of more effective treatments and diagnostic methods.
BLOG | The importance of clinical trials in Alzheimer's disease
Alzheimer's is a complex disease that affects millions of people worldwide and whose prevalence continues to increase with the ageing of the population. In this context, clinical trials are essential tools for advancing research and development of more effective treatments and diagnostic methods for this disease and other dementias. At Ace, applied research, a line of clinical trial work focused on solving practical problems or specific needs, is one of our pillars for developing new treatments and improving care.
What is a clinical trial?
Clinical trials are research studies that evaluate the safety, efficacy and possible side effects of new treatments, drugs, devices or prevention strategies.
Before reaching the human clinical phases, a laboratory investigating a new formulation must conduct pre-clinical studies in animal models to determine its safety and effectiveness. Only after this stage, where the maximum tolerated dose is determined, is it allowed to move on to human clinical trials, which are divided into four main phases:
- Phase I: focuses on assessing the safety of the treatment in a small group of volunteers (20-80 participants) to determine dosage, side effects and other relevant issues such as interaction with food, etc.
- Phase II: aims to quantify the impact of the drug on the desired therapeutic target (e.g. reducing glycosylated haemoglobin in antidiabetics or removing amyloid plaque in disease-modifying treatments for Alzheimer's disease), analyse the efficacy of the treatment in a larger group (100-300 participants) and further evaluate its safety.
- Phase III: involves a larger number of participants to confirm efficacy, monitor possible side effects and compare with existing treatments.
- Phase IV: this is conducted after approval of the treatment, assessing its effectiveness in the real world and detecting possible long-term adverse effects.
In the case of Alzheimer's disease, these trials are not only focused on finding treatments that can slow or halt the progression of the disease, but also on improving methods of early diagnosis, a key tool to act before brain damage becomes irreversible. Plasma-based biomarkers for Alzheimer's disease are a clear example, as they have been used in clinical trials for years to enrich the trial population and thus reduce the overall number of patients screened during the initial screening.
Advantages of participating in clinical trials
Participating in a clinical trial offers multiple benefits for the patient:
- Access to innovative treatments: participants can access therapies not yet available on the market, which could lead to an improvement in their quality of life.
- Specialised medical follow-up: to ensure their well-being throughout the treatment, patients receive personalised and comprehensive care throughout the study process.
- Contribution to science: participating in a clinical trial is a way to contribute to the advancement of medicine and help future generations.
A prime example of the importance of clinical trials is the recent success story of Lecanemab, a treatment approved by the European Medicines Agency (EMA) that has been shown to slow the progression of early-stage Alzheimer's disease.
In addition, the results of the Phase 3 APOLLOE4 clinical trial will soon be presented at the AD/PD™ 2025 International Conference on Alzheimer's and Parkinson's Diseases on 1-5 April in Vienna. This clinical trial has assessed the safety and efficacy of Valiltramiprosate, a molecule that could become the first disease-modifying treatment administered orally to homozygous ApoE4/4 patients (people with two copies of a genetic variant associated with an increased risk of developing Alzheimer's disease).
Challenges in applied research
Despite their remarkable progress, clinical trials face significant challenges. One of these, in the case of Alzheimer's, is ensuring the representativeness of participants. According to a study by our researchers at Ace, women are under-represented in some studies, despite the fact that they have a higher prevalence of the disease. This is partly due to selection criteria that may be indirectly biased, such as educational level or medical history.
In addition, applied research has other challenges, such as the difficulty in recruiting participants, especially in pathologies such as Alzheimer's, the high costs and limited funding, the heterogeneity in the progression of neurodegenerative diseases, which makes it difficult to assess the efficacy of treatments, and the need to ensure adherence to the protocol by participants.
Ace and its commitment to clinical research
At Ace, we have 30 years of experience in applied research, which has contributed significantly to the understanding and treatment of Alzheimer's disease and has earned us international prestige and recognition from the scientific community. These studies have allowed us to validate plasma biomarkers and advance in the development of therapies targeting neuroinflammation.
In addition, since our foundation, we have participated in 173 clinical trials, where we have ensured an efficient patient flow and personalised care through cognitive assessments in each of them.
Our multidisciplinary team is composed of 21 members, including experts in neurology, neuropsychology and biomarkers, among others, allowing us to approach studies from a comprehensive and advanced perspective.
Our aim is not only to develop more effective treatments, but also to raise public awareness of the importance of research and encourage participation in clinical trials as a way to move towards a future where Alzheimer's is history.
XAVIER MORATÓ ARUS
Deputy Director of Clinical Trials at Ace Alzheimer Center Barcelona
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