FARA Press Release


First Human Dose Initiated of Promising New Friedreich’s Ataxia Drug

Highlights Effectiveness of New Public-Private Partnership Model

July 15, 2008 – The Friedreich’s Ataxia Research Alliance (FARA) joins its public and private partners in announcing that a phase I clinical trial of a promising new drug, designated A0001, began dosing on July 11th. This milestone achievement illustrates the power of a new model for advancing therapies, especially in rare diseases.

“FARA believes in the essential nature of public-private partnerships that involve government agencies such as the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), academic investigators, the pharmaceutical industry, and multiple non-profits like FARA and the Muscular Dystrophy Association (MDA),” said FARA President Ron Bartek. “FARA is grateful to its partners that span the spectrum from discovery through clinical development as we work together to advance promising compounds like A0001 toward the goal of approved treatments and cures for Friedreich’s Ataxia (FA) and other neurodegenerative disorders.”

A0001 is a small molecule that has the potential for treating defects in the mitochondrial respiratory chain like those believed to cause significant damage in FA and a number of other neurological and neuromuscular disorders. In April 2006, the FDA granted orphan drug designation status to A0001 for treatment of inherited mitochondrial respiratory chain diseases.

The opening of this clinical trial demonstrates the power of the public-private partnerships FARA has been fortunate to build and bring to bear on the quest for treatments and a cure for FA. FARA began its support of A0001 in 2005 with substantial research grants to and investment in Edison Pharmaceuticals. The MDA, via Seek A Miracle/MDA, joined FARA in co-funding the initial research grant to Edison.


"We all know that drug development is expensive, but this is a wonderful example of how investments can be leveraged by multiple groups to get more therapies into the clinic," says Dr. Sharon Hesterlee, MDA VP for Translational Research.

Later in 2005, Dr. Robert Wilson of the University of Pennsylvania, FARA and Edison joined forces as co-applicants to the National Institutes of Health (NIH) program called RAID (Rapid Access to Intervention Development), designed to help move drugs from the discovery bench to the clinical trial bedside. The application was successful and contract services provided by NIH RAID played an important role in completing the preclinical preparations of A0001 so the molecule could be made ready for the clinical trial.

“This is a great example of how the various stakeholders joined forces to help transform a promising laboratory discovery into a therapeutic that is now in clinical trial,” says Dr. Story Landis, NINDS Director. The NINDS manages the NIH RAID program and is also responsible for Friedreich’s Ataxia research as part of its mission to reduce the burden of neurological disorders. NINDS’ support for the A0001 pre-clinical project consisted of formulation, a genotoxicity study, a dose escalation study, synthesis of radiolabeled drug for ADME and radioautography studies, dose ranging studies in two species, a 90 day GLP toxicity study, and safety pharmacology studies.

The phase I clinical trial just initiated by Penwest Pharmaceuticals will be conducted in healthy volunteers and is designed to evaluate the safety and tolerability of A0001 at various doses, as well as to collect pharmacokinetic data. In addition, the trial is designed to determine if there is a maximum tolerated dose of the drug. This data will be used in subsequent phases of clinical trials in patients with FA and other diseases that involve mitochondrial dysfunction.

For futher information, contact FARA at                (703) 426 1576         or fara@....

About FARA
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich's ataxia. http://www.CureFA.org

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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!



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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