The drug aims to restore the function of the protein that causes motor neuron death, and the new molecule could slow the progression of amyotrophic lateral sclerosis

Consalud.es

This coming April, the Community of Madrid is launching a groundbreaking clinical trial at the Institute of Health Research at La Princesa Public Hospital, paving the way for the development of new treatments for Amyotrophic Lateral Sclerosis (ALS). The research is scheduled to continue in January next year, with a second phase involving patients affected by the disease.

Specifically, the Clinical Trials Unit at the Madrid centre will conduct this study using the drug AP-2, a potential treatment for ALS. This will mark the first administration in humans (First-in-Human) and has recently been authorised by the Spanish Agency for Medicines and Health Products (AEMPS), following the successful completion of the preclinical phase at the Spanish National Research Council (CSIC).

In this regard, 72 healthy volunteers are expected to take part; they will be given the drug so that its safety and pharmacokinetics—that is, how it is absorbed, distributed, metabolised and eliminated from the body—can be assessed.

The Clinical Trials Unit at the Madrid centre will conduct this study using the drug AP-2, a potential treatment for ALS

AP-2 aims to restore the function of TDP-43, a protein that is pathologically altered in ALS patients, leading to the death of motor neurons (nerve cells responsible for transmitting signals from the brain or spinal cord to the muscles) and, consequently, the progression of the disease. To date, it has succeeded in reversing the TDP-43 abnormality, thereby restoring its natural balance, in both cellular models and transgenic animals.

In the same vein, this type of trial is based on the gradual administration of the drug at different dose levels. Each dose escalation is carried out only after safety has been confirmed in the previous patient cohort, thereby ensuring the safety of participants and enabling the necessary safety data to be established for future evaluation in patients with ALS.

The launch of this trial reinforces the Madrid public health service’s commitment to biomedical innovation, research excellence and the development of advanced therapies

The conduct of this study sets a benchmark in the transition from basic research to clinical application, consolidating its capacity to lead complex analyses in early-stage trials. Beyond safety assessment, it will enable the determination of appropriate doses and paves the way for analysing, in later phases, whether the molecule can help slow the progression of the disease, which would represent a significant step forward in the search for solutions to this condition.

The launch of this trial reinforces the commitment of Madrid’s public health service to biomedical innovation, research excellence and the development of advanced therapies, placing its public hospitals, such as La Princesa, at the forefront of clinical research in Spain, and particularly in the field of First-in-Human trials.