Research

I know that this is the part of the website that offers all of us hope. So I will do my best to keep this up to date. In the meantime, please also check the FARA Australia and FARA USA research pages.

FARA USA have published 6 short utube editions that details current research in USA, Europe and Australasia.(There is still 1 more to come) Here are the links, watch with some excitement that we have some hope that something will be available to us FAers soon.

Part 1 of 7:

Part 2 of 7:

Part 3 of 7:

Part 4 of 7:

Part 5 of 7:

Part 6 of 7:

Update Re: Drug Trials: There is an update on HDAC / EPO / Varenicline and Sulforaphane. Notes on this were available at our recent AGM. I will post these as soon as I receive an electonic version of the information. (03Dec2010)

Idebenone

•  Phase III of the Idebenone trial in the U.S. shocked many when the trial was deemed unsuccessful. However, this does not mean Idebenone isn't effective for FA'ers.

All participants in the trial showed improvement, including those on the placebo. However, the overall improvement of those taking Idebenone was not statistically significant enough, compared to those on the placebo, to validate the benefit of Idebenone.

•  The results from a longer-duration trial of Idebenone in Europe, plus the data from a U.S. extension study, may be more conclusive.

Idebenone supplies can be ordered from the US via this website: www.smartpowders.com or for larger orders (10 bottles or more) by emailing Mike from Smart Powders at this address: My65Cuda@aol.com A discounted price can be negotiated for larger orders.

Deferiprone

•  Australia was included in this multi-centre global trial, under the supervision of Assoc. Prof. Martin Delatycki in Melbourne (Monash/MCRI). The other sites were France, Belgium, Italy, Spain and Canada.

•  Eight participants enrolled, all under the age of 18.

•  Two young patients became very ill early in the trial, and the trial was immediately paused, and ultimately the Australian leg of the trial was aborted. It continues in other locations using lower dosages.

•  Results of the trial are expected in late 2009.

Varenicline (brand name in Australia is Champix/in the U.S. it's Chamtix)

•  Refer to FARA newsletter (Page 6) for more details, or visit www.CureFA.org/registry and select “Clinical Trials”.

EPO (Epoetin Beta)

•  A full-scale double-blind trial was planned for EPO, but the funding application (for US$3 million) to MDA was rejected after two different pieces of research suggested there is an elevated risk of specific cancers reoccurring in patients who have previously had those types of cancer.

•  FARA USA continues to seek funding for an EPO trial. Our view is that the above research is largely irrelevant to FA sufferers, and that EPO has significant potential as a treatment for FA.

Update re New Research

Stem Cells

•  Stem cell research has moved beyond the highly-controversial debate of a few years ago re the use of “embryonic” stem cells. The latest techniques grow stem cells from skin scrapings.

•  Stem cell research is consistently coming in ahead of schedule, under budget and exceeding expectations! (The opposite experience we've had with conventional research).

•  However, clinics around the world offering treatments for conditions such as FA should be regarded with a great deal of scepticism.

•  A team in Melbourne, led by Mirella Dottori and Alice Pebay, is being co-funded by FARA Aust. & FARA USA, to develop stem cell lines from FA patients, and is making fantastic progress. These stem cell lines will be made available to researchers around the world and will be an invaluable tool to ‘fast-track' research.

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