The FPS Public Health and the FAMHP invite you to participate in the public consultation on a clinical trial of the genetically modified medicine VTx-002 for the treatment of Amyotrophic Lateral Sclerosis (ALS). The public consultation will take place from February 9th to March 11th, 2026.
For each clinical trial application for the use of a genetically modified organism, the government organises a thirty-day public consultation, in accordance with the Royal Decree of 21 February 2005. As a citizen, you can give your opinion on this clinical trial application with a genetically modified medicine.
Amyotrophic lateral sclerosis (ALS) is a serious, progressive neurological disorder. In most cases, the disease causes the protein TDP-43, a nuclear transcriptional repressor, to become dysregulated and accumulate in nerve cells. This causes motor neurons in the brain and spinal cord to die off. This leads to loss of strength and muscle paralysis in ALS patients, making it increasingly difficult to move, swallow and breathe, among other things. There is currently a large unmet need for a therapy that can slow or halt ALS disease progression.
In this clinical trial, the study drug VTx-002 will be tested as a potential therapy for the treatment of ALS, through a single injection into the cisterna magna in adult patients.
VTx-002 is a gene therapy being developed to express a humanised anti-TDP-43 single chain variable fragment (scFv) transgene in the central nervous system. This transgene is designed to bind pathological forms of TDP-43, found in the nervous system of ALS patients. By achieving this sustained intracellular production of the anti-TDP-43 scFv, disease progression could potentially be slowed or halted by targeting the underlying pathology.
The aim of this clinical trial is to assess the safety and tolerability of two doses of VTx-002 in adult patients with ALS.
The clinical trial will take place at the University Hospital in Leuven.
How to give your opinion?
The public consultation runs from 9 February to 11 March 2026 inclusive. You can access the various data in the application file, as well as an online form to send your comments or ask your questions.
General information on GMO - public consultation.