Welcome to our research project on developing a potential treatment for Friedreich's ataxia. Research is the only way we can achieve our mission: to see a world free of ataxia, and you can help us achieve that. Read below to discover more about this project, request a copy of a fundraising leaflet to disseminate to potential donors, or donate below to support this project. 

The project

Background

This project builds on the research team’s work on bone marrow stem cells as a potential treatment for Friedreich’s ataxia (FA). An alternative to transplanting bone marrow cells would be to use bone marrow stem cell-mobilising drugs.

These are drugs used in clinical practice that activate stem cells within the bone marrow and induce them to circulate around the body. The researchers believe that increasing the circulation of the body’s own stem cells is a promising therapeutic approach.

Aim

They have recently completed a study of a mouse model of FA in which a bone marrow stem cell-mobilising drug (granulocyte colony stimulating factor) protected mice from neurological damage. In this study they plan to: (a) Test the effect of this drug in human cells (to see if they get the same response as in mice) (b) Do a pilot trial in seven people with FA to test safety, dosage and get information for a larger trial

Impact

If this study is successful it could pave the way for a larger clinical trial testing the effect of a new treatment in Friedreich’s ataxia.

Request a leaflet

Get in touch with our research department via [email protected] to request copies of leaflets, helping you to disseminate easily to potential donors. 

We are fundraising for this project both online and offline; such funds will be added to this page as they come in. We owe particular thanks to our generous fundraiser, Richard Bradford: Richard has dedicated his own fundraising page to raise as much as possible towards this project. 

Please be aware that if we raise over the target amount for this project, we will put the extra funds towards another research project dedicated to FA.