Pioglitazone has been selected because this PPARgamma ligand is already distributed on the market as an anti-diabetic reagent, considerably reducing the delay to trial the drug in Friedreich ataxia. Noticeably, the drug has been provided to a number of diabetic individuals and has proved to have limited side effects, although not null. These side effects imply that any attempt to use the drug should be done with extreme caution under the supervision of a well-informed MD. 

At variance with Idebenone, which we proposed exactly 10 years ago as a first mean to counteract Friedreich ataxia4, Pioglitazone should counteract the disease both by reinforcing the natural cell antioxidant defenses and favouring the synthesis of depleted mitochondrial proteins, including frataxin.

I ended this short information letter by wishing you a “Bonne Année” 2009 and thanking again AFAF (Association Française contre l’Ataxie de Friedreich), REVAmoto (association française Rechercher Espérer et Vaincre l’Ataxie) ACHAF (Association Suisse contre l’Ataxie de Friedreich) AFM (Association Française contre les Myopathies) and FARA (Friedreich's Ataxia Research Alliance; USA) for their constant support in our fight against Friedreich ataxia,

Sincerely yours,       

                                             Pierre Rustin

1EuroAtaxia, AFAF and FARA meetings; London September 2005, Nouans-le-Fuzelier Avril 2005 and Bethesda November 2006 respectively; Eldorado  April 2006; Parcours May 2006;  – Journals of the Canadian and the Swiss association of FRDA patients, respectively.

2 Paupe V, Dassa EP,Goncalves S, Auchère F, Lonn M, Holmgren, Rustin P. 2008. Impaired nuclear Nrf2 translocation undermines the oxidative stress response in Friedreich ataxia. PLOS One (in press)

3See Gene Expression Omnibus Dataset (GEO profiles, National Center of Biotechnology Information, National Health Institute,USA).

4Rustin, P., von Kleist-Retzow, J.-C., Chantrel-Groussard, K., Sidi, D., Munnich, A., Rötig, A. (1998) Effect of idebenone in Friedreich's ataxia: a preliminary study. The Lancet 354: 477-479

----- Original Message -----

From: Gian Piero Sommaruga (casa)

To: Prof. Pierre Rustin

Sent: Tuesday, December 30, 2008 7:38 PM

Subject: Fw: [FAPG] ENG: Pubmed abstract - PPAR-gamma Agonist Azelaoyl PAF Increases Frataxin Protein and mRNA Expression. New Implications for the Friedreich's Ataxia Therapy.

Dear Prof. Rustin,

would you please be so kind to give an answer to the following questions to be posted through the ataxia related mailing-lists?

I'd really appreciate your help.

My warmest regards,

Gian Piero Sommaruga

----- Original Message -----

From: Bart Rupel

To: Gian Piero Sommaruga (casa) ; FARA AUSTRALASIA ; FAPG ; Internaf ; FA_babelFAmily

Sent: Tuesday, December 30, 2008 6:13 PM

Subject: Re: [FAPG] ENG: Pubmed abstract - PPAR-gamma Agonist Azelaoyl PAF Increases Frataxin Protein and mRNA Expression. New Implications for the Friedreich's Ataxia Therapy.

OK, this is very interesting, rosiglitazone, is from the thiazolidinedione class of drugs. There is another thiazolidinedione drug on the market today, pioglitazone.

Pioglitazone is about to start a phase I study in FA. It is being studied in FA because of its reported affects on ataxia symptoms in other ataxias.

Would rosiglitazone be an even better choice for FA than pioglitazone because of the frataxin level increases?

Or does pioglitazone also increase frataxin levels?

Bart Rupel

Matt 18, FA

Katie, 15 carrier


For  the Pubmed abstract see:  http://health.groups.yahoo.com/group/FA_babelFAmily/message/2481 (ENG)
Dear Gian Piero,
Please find the letter which I am sending today to several concerned persons informing them about the Pioglitazone trial which by the way seems to answer the question you asked me to deal with. The different PPARg ligands predictably share similar properties in term of protein expression regulation, various glitazone derivatives having various side effects with Pioglitazone having proved to have less, explaining our choice.
Bonne Annee,
Pierre Rustin

Paris, December 31, 2008



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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:


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.



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