Research into ALS with Royal Support

Spain’s former Queen Sofia (center) together with researchers and other participants in the project presentation. Right next to her is Manuel Barrós, who suffers from ALS and after whom the research project is named. He is a former head of security for the royal family. Source: Fundación Reina Sofia

Queen Sofia Foundation funds project with DZNE participation

Recently, a research project on amyotrophic lateral sclerosis (ALS) was launched in Madrid in the presence of Spain’s former Queen Sofia. Researchers from the university hospitals in Bonn and Cologne and from DZNE are playing a key role in this endeavour.

The project focuses on so-called diagnostic biomarkers. The underlying goal is early detection of ALS through blood testing. The Queen Sofia Foundation is funding this endeavor, Prof. Anja Schneider (DZNE and University Hospital Bonn) and Prof. Alfredo Ramirez (University Hospital Cologne) are involved on the German side. Their scientific partner in Spain is the CIEN Foundation (Fundación Centro de Investigación de Enfermedades Neurológicas).

A fatal disease

In ALS, neurons for movement control are gradually destroyed. This leads to progressive paralysis and usually to death within a few years. As yet, there is no cure for ALS. However, novel drugs are being developed or have recently been approved in the USA – at least for a specific subtype of ALS – that hold out the prospect of better treatment. Because early treatment generally increases the chances of success, it is important to diagnose ALS as early as possible. For this, a protein called TDP-43 may provide relevant clues, as this molecule accumulates in the brains of most ALS patients.

Tiny bubbles

It would be a significant advance, if ALS could be diagnosed by blood analysis. As TDP-43 levels in blood can reflect presence of the protein in the brain, this scenario seems realistic. The current research project, which draws on preliminary work by Prof. Schneider and Prof. Ramirez, aims to pave the way for this. Specifically, it involves detection of TDP-43 carried by extracellular vesicles: These tiny bubbles made of lipids are secreted by neurons and can ultimately reach the bloodstream. To this end, blood samples from ALS patients – including specimen from the CIEN biobank – will be analysed and the findings matched with data from other, already established biomarkers for neurodegeneration.

^{January 2024}