Multiple ongoing studies on inhibitors of the histone deacetylase enzyme (HDACIs) are trying to demonstrate their effectiveness in several types of cancer or neurodegenerative diseases. It seems that only a specific group of HDACIs is able to significantly increase the level of frataxin in human body cells, particularly in immature white blood cells as well as in organs singled out by Friedreich’s ataxia.

At this moment one of these molecules was chosen to be a clinical candidate and should reach phase 1 before the end of the year in Europe (study of the drug: absorption, elimination, etc.) If everything goes well, phase 2 (clinical study of patients) should start somewhere in 2010.

A meeting in Europe is scheduled in July to sum up the situation on the development of this molecule. Dr Massimo Pandolfo, a world expert in Friedreich’s ataxia, tells us that the principal researchers will start planning clinical trials during that meeting.

Obviously FA patients in Quebec could eventually take part in such a clinical study because the disease is so much more prevalent in Quebec and because of the experience we have acquired during these years studying neurodegenerative diseases. In this regard Dr Pandolfo has already mentioned that he will propose our clinical environment as a possible North American partner. An exciting story worth to keep an eye on!

Source: L’Eldorado, June 2009, p.11  - Translated by Sofia Arenzon
 
Canadian Association for Familial Ataxias – Claude St-Jean Foundation

http://www.lacaf.org/index.php?option=com_content&task=view&id=76&Itemid=138?=english

By Guy D’Anjou, M.D. FRCPC
Neurologist, Sainte-Justine Hospital 

The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

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.

Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!

https://www.amazon.com/Legacy-Marie-Schlau-collective-Friedreichs-ebook/dp/B01N28AFWZ

 

Research projects currently being financed by BabelFAmily

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:

https://www.irbbarcelona.org/en/news/international-patient-advocates-partner-to-fund-spanish-gene-therapy-project-to-treat

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:

https://www.irbbarcelona.org/en/news/new-research-front-to-tackle-friedreichs-ataxia
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.

 

 

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