Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that progressively destroys the neurons responsible for movement. As it advances, patients gradually lose their ability to move, speak, eat and even breathe.

There’s currently no cure and the available treatments only manage to slow the progression of the disease slightly. Developing new therapies is therefore one of the major challenges facing biomedical research today.

With this aim in mind, the team led by Ana Martínez and Carmen Gil at CSIC’s Margarita Salas Biological Research Centre has spent years studying TDP-43, a protein that’s altered in over 97% of ALS patients and is closely linked to the disease’s progression.

heir strategy has been to develop AP-2, a molecule designed to restore the normal function of this protein. And the results obtained so far in cell and animal models have been promising: following the administration of AP-2, TDP-43 returns to its normal behaviour, which could help to slow down the progression of ALS. 

Ana Martínez and Carmen Gil tell us all about the latest advances in this research in this News from The “Lab”.

[Vídeo News From the Lab]

A milestone in this research has been following news: Spain’s Agency for Medicines and Health Products has authorised the start of a Phase I clinical trial for AP-2. This trial, which began in April, involves 70 healthy volunteers and aims to assess the treatment’s safety.

Furthermore, the European Medicines Agency has granted AP-2 orphan drug status, a recognition that promotes the development of new therapies for rare diseases.

If the results are positive, 2027 will see the start of a new phase of the trial involving ALS patients. 

Although there’s still some way to go, these advances represent an important step forward in the development of treatments capable of changing the future of this disease.