Original Message -----

From: Mdown4@...

To: fapg@...

Sent: Tuesday, July 01, 2008 10:23 PM

Subject: [FAPG] Clinical update questions

 

 

Two questions were asked as a result of the recent email I sent. The questions asked and the responses from Jen Farmer (FARA) are below.  I hope this helps.

 

1. The first question was about an update on the HDAC inhibitors.

 

The entire HDAC team (Repligen team, Joel Gottesfeld, Massimo Pandolfo, Mark Pook, David Lynch, FARA reps and GoFAR reps) met May 31st in Boston. In addition to a briefing, the group outlined next steps and goals for the upcoming six months. As you know both the Repligen and Gottesfeld teams have been advancing the compound identified by the Gottesfeld lab through the drug development process and doing all of the necessary testing in cellular and animal models. In addition they have also been using different approaches to identify additional targets and to select a lead candidate to take forward in an IND to the FDA. They have identified additional targets and are in the process of doing the necessary experiments to be able to select a lead candidate. Repligen feels that they are on target to submit an IND in the late fourth quarter of 2008. The team is now beginning to outline clinical and biomarker studies with Dave Lynch to begin laying the necessary ground work for a clinical trial. The next team meeting is planned for October 2008.

 

Below is taken from Repligen press release June 12th 2008

 

“HDAC Inhibitors for Friedreich's Ataxia”

We are currently developing compounds which may have utility in treating Friedreich's ataxia. Over the past year, we made significant progress in identifying advanced leads through multiple rounds of novel compound library sythesis and screening for potency, specificity, metabolism and pharmacology. Over the next year these advanced leads will be further characterized in animal models for their pharmacologic, toxicologic and pharmacodynamic profiles to identify an appropriate candidate for the clinic. We have also received a grant from Go FAR to develop a biomarker tool which may be useful in monitoring the biochemical activity and guiding the dosing frequency of a clinical candidate in patients. Go FAR (Friedreich's Ataxia Research) is a fundraising organization headquartered in Turin, Italy formed by the non-profit group RUDI Onlus Committee, dedicated to raising donations to fund research and development of treatments for Friedreich's ataxia.”

 

Of note, FARA proved funding to both Repligen and Dr. Gottesfeld in late 2007 to support the work described in the press release above – “identifying advanced leads through multiple rounds of novel compound library synthesis and screening for potency, specificity, metabolism and pharmacology”

 

2nd question:   Are parents allowed to participate in the Phase I studies or does it have to be someone without a defective gene.  If parents are allowed, is there a registry we can sign up for? (Penwest A0001 study)

 

We have limited knowledge on the specifics of the study.

I do know that Penwest has arranged for the study to be completed by a third party – basically a commercial clinical research company that basically does these type of Phase I safety studies for a living. I don’t know the actual location or logistics of the study. I did get confirmation that it is anticipated to start “on-time” July 10th and that Penwest and FARA will be issuing press releases and announcements to the public and patient community very close to this date.

 

I don’t think that FA carriers would be eligible.

 

(response from Jen Farmer)

 

 


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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:

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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