Ataxia UK and FARA collaborate for new study into a potential treatment for Friedreich’s ataxia
A new research project on Friedreich’s ataxia (FA) is starting at the University of Bristol, supported by ATaxia UK, FARA and the University of Bristol. This project builds on Dr Alastair Wilkins’ research team’s work on bone marrow stem cells as a treatment for Friedreich’s ataxia (FA). They have recently completed a major study of a mouse model of FA in which the bone marrow stem cell-mobilising drug, GCSF, protected mice from neurological damage. These types of drugs have potential in neurodegenerative conditions and represent a novel therapeutic possibility for FA. Read more here.
New partnership between Ataxia UK and the Spanish organisations R+SCAs and ACAH
Ataxia UK is working for the first time with the Spanish ataxia patient group R+SCAs (PLATAFORMA DE APOYO A LA INVESTIGACIÓN DE LAS ATAXIAS ESPINOCEREBELOSAS) in funding a research project on a range of ataxias. An existing partner ACAH (L’Associació Catalana d’Atàxies Hereditàries) is also co-funding this project.
The project is led by a team at University College London (UCL) Institute of Neurology and it is a genetic study looking at the DNA repair mechanism in the modulation of age onset in a range of ataxias.
Advances in Huntington’s disease research could be of relevance to ataxias
A landmark trial for Huntington’s disease has announced positive results, suggesting that an experimental drug could become the first to slow the progression of the condition. This was a phase I trial using an antisense drug which was found to be safe and lowered the levels of the toxic disease-causing protein. The trial was too small, and not long enough, to show whether patients’ clinical symptoms improved, but a larger trial is planned to test this. You can read more here.
A similar antisense approach is relevant to some inherited ataxias, thus the advances in Huntington’s research are exiting for ataxia research too. In April this year we reported on positive findings for SCA2, using this same antisense approach in a mouse model.