The Walk on Project (WOP) nonprofit foundation, in collaboration with BBK Fundazioa, will fund with 98,000 euros a research project led by Dr. Aurora Pujol, ICREA research professor in Bellvitge Institute for Biomedical Research (IDIBELL), who is an expert in Human Genetics and Neurodegenerative Diseases. The project, entitled "Pharmacological strategies for the regeneration of myelin and axonal protection in X-linked Adrenoleukodystrophy", will be carried out in close collaboration with Dr. Robin Franklin, a Neurobiologist specialized in Stem Cell and Regenerative Therapies for Myelin Diseases at the Cambridge Stem Cell Institute.
Dr. Pujol, with a long experience in the study of the Leukodystrophies, in particular, the X-linked Adrenoleukodystrophy (X-ALD) and Dr. Franklin, with an extensive knowledge in demyelination and remyelination processes, have decided to face a new challenge: the research of effective treatments for patients with demyelinating diseases such as cerebral X-ALD.
The X-ALD is an inherited neurodegenerative disease with minimum incidence of 1 in 17,000 males. One of the most common phenotypes in this disease is the cerebral X-ALD, characterized by demyelination and cerebral inflammation, which has a lethal outcome approximately two years after the diagnosis.
"In order to find effective treatments for this disease is essential to generate an animal model, nonexistent today, which mimics the molecular mechanisms and the symptomatology of patients with the demyelinating variant. Thus, we will be able to perform preclinical trials in mice before starting clinical trials in humans. Moreover, the study of the molecular changes underlying demyelination and neurodegeneration in the animal model will pave the way to more effective therapeutic approaches", said Dr. Pujol.
The molecular mechanisms underlying remyelination deficiency in Leukodystrophies are still unknown, but some studies, mainly from Dr. Franklin’s laboratory, suggest that the main problem resides in the inability of stem cells to differenciate in oligodendrocytes; the only cell type in the central nervous system able to produce myelin. Therefore, the project will be focused on therapeutic approaches able to induce oligodendrocytes differentiation and myelin regeneration that can be applied in patients in medium-term time in order to cure or mitigate the devastating effects of demyelination.
The results of the study may also be applied in other neurodegenerative diseases related to demyelination such as Multiple Sclerosis, Parkinson's Disease, Alzheimer's Disease or Leukodystrophies.
Jone Lopez Erauskin, predoctoral researcher at IDIBELL, attended the presentation of the aid, in representation of Dr. Pujol’s group.
In addition to this ambitious project, WOP has also funded another project led by Professor Salvador Martinez from Neurosciences Institute of Alicante, with 58,500 euros. Dr. Martinez research is focused in cell therapies to regenerate myelin. Diseases associated with myelin damage have no cure yet. The aim of the project is to block demyelination and try to regenerate damaged brain areas using cell therapy in order to recover neuron’s functional capacity.
Walk on Project was founded in 2010 by Mikel Renteria and Mentxu Mendieta, Jontxu’s parents, a child affected by cerebral X-ALD. During the last two years, WOP has collected almost 160.000 euros for research in treatments against Leukodystrophies by performing concerts together with public and private donations. After a scientific evaluation by experts, WOP has funded the two previously mentioned projects.