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We have also defined specific intracellular mechanisms underlying these effects and shown that human stem cells can be induced to adopt a neuronal phenotype under a variety of conditions, possibly either by transdifferentiation or cell fusion. The proposed project aims to further explore this possibility by defining the precise neuroprotective role of bone marrow-derived stem cells; transplanting stem cells into an animal model of FA; and studying the process of cell fusion, which may be a novel neuroprotective mechanism. Autologous bone marrow transplantation has many advantages as a treatment, not least its safety profile, and is being trialed as a potential treatment for other neurological disorders. We believe it may be a potential therapy for FA in due course.
Freidreich’s ataxia (FA) is characterised by degeneration of a variety of cells within the body including nerve cells. Abnormalities in the balance part of the brain, called the cerebellum, give rise to the symptom of ataxia. Current evidence suggests that the genetic defect causing FA leads to reduction in amounts of energy available to nerve cells and also accumulation of toxic substances which may be poorly handled by diseased cells. Future treatments for FA will need to address these issues and provide protection for nerve cells. We are particularly interested in the potential for bone marrow-derived stem cells as treatments for a number of neurological conditions. These cells have the advantage that they may be derived from the patient’s own bone marrow, thus avoiding rejection and a large number of ethical issues concerning stem cells. Furthermore bone marrow transplants have been used for many years for the treatment of several aematological conditions and thus much safety data is available. We have carried out initial research demonstrating that in cell culture human bone marrow-derived stem cells are able to improve the survival of cells derived from the cerebellum. We have also shown that these stem cells can integrate within the nervous system following transplantation in animal models, thus establishing that bone marrow derived cells may be able to protect or even replace lost brain cells. These studies are now at a critical stage and we wish to investigate this further in an animal model of FA. Specifically, we aim to determine whether transplantation of human bone marrow-derived stem cells will protect against progression of disease in an animal model of FA. These are vital pre-requisites before commencing human trials. We hope these studies will, in time, lead to the establishment of clinical trials of stem cell therapies for FA.
For more support or information please contact: Ataxia UK, Lincoln House,
Kennington Park, 1 – 3 Brixton Road. London SW9 6DE
Helpline: 0845 644 0606 Tel: +44 (0)20 7582 1444 Fax: +44 (0)20 7582 9444
If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.
The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.
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Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:
1) Gene Therapy for Friedreich's Ataxia research project:
The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.
2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.
The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.