Research into treatment for SPG11 started with support from Life4HSP and Euro-HSP.

Starting in March 2025, Euro-HSP, together with Life4HSP, launched support for a new research project for SPG11. This project focuses on finding a treatment for SPG11, a rare and hereditary form of HSP (Hereditary Spastic Paraplegia). Related forms such as SPG15 and SPG48 are also included in this research. With this, we are expanding our efforts beyond the ongoing euroSPG4 project.

The medicine Venglustat may be a hopeful treatment

The research is taking place at the Paris Brain Institute in France, under the leadership of Dr. Frédéric Darios. The researchers are testing an existing medicine, Venglustat, which may help slow the progression of the disease in SPG11. The initial laboratory results are promising. They are now investigating whether the medicine is safe and effective in the long term. This entire process will take two years.

It has now been discovered that in SPG11, SPG15, and SPG48, a problem occurs in the nerve cells: they are unable to properly break down certain substances – gangliosides. These substances accumulate in the cells and cause damage to the nervous system. Venglustat appears to help by reducing the production of these harmful substances.

Thanks to donors, the project was able to get started.

To carry out this research, nearly €200,000 is needed over two years. Thanks to our donors, and with a major contribution from Life4HSP, Euro-HSP was able to launch the project with an initial payment of €60,000. We are incredibly grateful for that.

In addition, funding is also needed for other important research: the development of a biomarker. This is a kind of ‘measurement tool’ that allows doctors to see whether a treatment is having an effect. Without such a biomarker, it is difficult to determine whether a medicine truly works. Life4HSP and our partner organization stopp-HSP from Austria are jointly supporting biomarker research at the renowned Children’s Hospital at Harvard (USA). That project is receiving a contribution of €10,000.

In addition to these new activities, the euroSPG4 project continues to develop as well. On behalf of Life4HSP, we would like to thank all our donors.

Would you like to contribute to more research?

Then visit our donation platform: https://life4hsp.community-fundraising.com/donate?locale=en