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

From: Ronald Bartek


To: This email address is being protected from spambots. You need JavaScript enabled to view it. ; This email address is being protected from spambots. You need JavaScript enabled to view it.

Sent: Wednesday, November 05, 2008 10:51 PM

Subject: [FAPG] HDAC Inhibitors and EPO in FA



Hello All,

Wanted to provide a brief update on the two promising compounds and clinical trial plans that Linda Condon recently asked about -- HDAC inhibitors and EPO.  Wanted you to know, too, that the next issue of FARA's Advocate will be published and distributed very soon in both hard copy and electronic form.  The Advocate will contain a full update on all the drug compounds in the FA research pipeline and on the clinical trials currently under way or being planned for next year.


FARA is in steady, frequent contact with the scientists and drug companies advancing these compounds in FA.  You might recall that Dr. Joel Gottesfeld at The Scripps Research Institute is credited with discovering the potential for HDAC inhibitors being beneficial in FA.  Scripps then licensed these compounds to the Repligen Corporation near Boston.  FARA continues to fund Dr. Gottesfeld's work and has thus far provided his lab with about $1 million in support.  FARA and MDA have partnered to help support Repligen's HDAC inhibitor effort, with MDA providing about $1 million to the company and FARA providing $100,000.  As Dr. Gottesfeld and Repligen design and refine these HDAC inhibitors, they are testing them in the FA mouse models developed by Dr. Massimo Pandolfo in Belgium and Dr. Mark Pook in the United Kingdom.


FARA continues to meet with this entire team (Repligen, Drs. Gottesfeld, Pandolfo, Pook and others) regularly to stay up to date on progress being made and to see how we might all work together most effectively to accelerate it.  The next such meeting is scheduled for Saturday, November 15.  This wonderful team's plan is to work together so as to arrive at the best HDAC inhibitor it can develop and take it to the FDA as early next year as possible with a proposal to begin a clinical trial in FA.  We should be able to report to you after the November 15 meeting whether the team is on track and what the time lines might be for going to the FDA.


One way FARA has been able to help advance and accelerate this project and others beyond the direct financial support is by helping make the FA animal models more readily available.  We funded much of the early work to develop such mouse models and to distribute them to additional scientists.  More recently, thanks to generous donors in the FA community, FARA has also entered into agreement with the world's premiere animal model facility - The Jackson Laboratory in Bar Harbor, Maine (JAX).  JAX has begun importing the leading FA mouse models, improving them where possible, and breeding them so as to be able to make them available to scientists around the world conducting the tests of promising FA drug compounds. This approach has the additional advantage of freeing up the original mouse-development scientists to do their own experiments rather than spending all their time breeding and caring for the mice.


FARA's longstanding efforts to advance Erythropoietin (EPO) as a potential therapy for FA has encountered some obstacles.  You might recall that FARA supported the fine work of an Austrian team (Drs. Scheiber-Mojdehkar and Sturm) that showed significant promise of EPO in FA.  In a short, small pilot study, the Austrian team demonstrated that EPO could apparently increase frataxin protein levels in FA patients.  FARA has been working with first-rate clinicians in the Collaborative Clinical Research Network in FA and with drug companies in an effort to develop a clinical trial of EPO in FA.


This EPO effort has been impeded by recently published studies in much different conditions.  First, a study of cancer patients following chemo-therapy who were administered EPO appeared to show that those patients taking EPO did not survive at as high a rate as those not taking EPO.  Later, a study of stroke victims appeared to show serious complications in those that were administered EPO.  As a result, the FDA issued a warning letter regarding EPO.  All of this has led to a situation in which it has been more difficult to secure a donated EPO drug supply for a Clinical trial, find partners eager to help us fund such a trial and be confident that the FDA will allow us to conduct such a trial.


We continue to work constantly with FA clinicians, drug companies and funding partners toward the goal of conducting a clinical trial of EPO in FA.  We will keep you informed of progress.


Again, please look for the upcoming issue of the Advocate in which you will find an update on all FA research and all the important efforts around the country and the world to support and accelerate that research.


Warm regards to you all,



Ronald J. Bartek


Friedreich's Ataxia Research Alliance (FARA)

P. O. Box 1537

Springfield, VA 22151

Tel (703) 426-1576

FARA website: http://www.CureFA.org

Email: This email address is being protected from spambots. You need JavaScript enabled to view it.

Please register in the FARA Patient Registry at http://www.curefa.org/registry/

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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:

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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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