Dr. Pastore is certainly no stranger to FARA.  FARA supported her work for several years earlier.  She is reporting in this article her conclusion that frataxin protein actually "limits" the number of iron-sulfur clusters assembled so that the lower frataxin levels in FA patients result in "more" iron-sulfur clusters being assembled.  She believes these excess clusters do not have sufficient "acceptors" so they decompose and are responsible for the damaging accumulation of excess iron in FA mitochondria.
Dr. Pastore's conclusions are contrary to those of other FA investigators.  These other investigators believe that the frataxin protein is an iron escort that provides the iron used in assembling iron-sulfur proteins.  These investigators believe that the lower frataxin levels in FA patients result in "fewer" iron-sulfur clusters being assembled - not "more."
This is a really important difference for our patients because iron-sulfur clusters are important in mitochondrial electron transport that produces cell energy and limits damage and cell death from oxidative radicals.
In fact, one key investigator who believes that the lower frataxin levels in FA patients result in "fewer" rather than "more" iron-sulfur clusters being assembled is working under a FARA grant to explore the possibility of making synthetic iron-sulfur clusters that would then be delivered to the mitochondria of FA patients so as to produce more energy and less oxidative damage.
So, FARA is discussing Dr. Pastore's recent article with a number of FA experts in an effort to clarify the differences in findings and then decide what needs to be done to figure out which are true so we can maximize the effort behind the more promising therapeutic approach.  Of course, regardless of which conclusion is proven correct, our other therapeutic approaches remain of undeminished promise and value.  Increasing frataxin protein levels, for example, remains a prime target, which is what we seek with HDAC inhibitors, EPO, TAT-frataxin and gene therapy.  Removing excess iron from FA mitochondria remains a promising approach and is what we are after with iron chelators like Deferiprone.  Improving mitochondrial function remains a valuable approach and is the reason we are pursuing Idebenone and A-0001.  And, the list goes on.
FARA will keep you posted on this issue as the experts work together to resolve it.
Warm regards and best wishes to you and your families,
(Byron - Clear, Keith - FA; Stuart - Carrier)

----- Original Message -----
From: Friedreich's Ataxia Research
To: fapg@... 
Sent: Tuesday, April 14, 2009 12:38 AM
Subject: Re: [FAPG] One troubling statement.overall, GREAT news!!


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: fara@...
Please register in the FARA Patient Registry at http://www.curefa.org/registry and for e-news at http://visitor.constantcontact.com/email.jsp?m=1101190303489

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

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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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