Enzyme blocker may reverse nerve damage
http://www.abc.net.au/science/articles/2009/10/27/2724996.htm?
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here is the latest FARA ( US) treatment pipeline... you will see there
is alot happening, and the funding is spread all over the world.....
let me stress, this is what FARA (USA) is funding... not what FARA (A)
is funding here in Australia...... however it gives you a world idea of
what is going on, and you will notice that the team from the Murdoch
research centre is included.
cheers everyone
Laurel Hosking
Help FA in SA
mob: 04204 98963
ph: 08 72212045
fax: 08 85221313
laurel@linvid.com
laurel
http://www.scripps.edu/newsandviews/e_20090928/gottesfeld.html
well we have booked and locked in the 2010 Charity Dinner.
I am please to announce that the Lakes Resort Hotel has welcomed us
back. ( good thing too, as they are a hard act to follow... believe me
I spent most of August looking around).
I am also happy to announce that the Flaming Sambucas have agreed to
return too. ( I DID look at other bands, but at the end of the day,
they TOO are a hard act to follow:->)
So the date for next year is.... drum roll please
August 14th 2010
put it in your diaries and spread the word.
cheers
Laurel
Laurel
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DANBURY, Conn., July 16, 2009 (GLOBE NEWSWIRE) -- Penwest
Pharmaceuticals Co. (Nasdaq:PPCO) today announced the results of its
Phase Ib clinical trial of A0001, a compound the Company is developing
for the treatment of mitochondrial diseases. Drs. Amale Hawi, Senior
Vice President, Pharmaceutical Development, and Thomas Sciascia, Senior
Vice President & Chief Medical Officer, will be presenting these results
at the Friedreich's Ataxia Therapeutics Meeting on Friday, July 17,
2009, in Philadelphia, PA.
In the Phase Ib trial, the drug was well tolerated by subjects, and no
serious adverse events were reported. There was a dose-dependent
increase in exposure approaching steady state within 2-4 days following
repeat-dosing, and a maximum tolerated dose was established.
Based on these results, Penwest plans to advance A0001 into Phase IIa
studies in patients with mitochondrial diseases. The Company intends to
commence two Phase IIa trials - one focused on patients with
Friedreich's Ataxia and the second focused on patients with the A3243G
mitochondrial DNA point mutation associated with the "MELAS" syndrome -
in the fourth quarter of 2009. The Phase IIa trials will be conducted
with twice daily oral dosing. The Company expects data from both of
these trials in the first half of 2010.
Jennifer L. Good, Penwest's President and CEO, said, "We are very
pleased to have completed the Phase I safety work on A0001 so that we
can advance this compound into patients. We believe A0001 is promising
for patients who suffer from mitochondrial diseases and currently have
very limited treatment options, and we are eager to begin to evaluate
the clinical benefit in patients."
Source/Read more:
cheers
laurel
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By Guy D'Anjou, M.D. FRCPC
Neurologist, Sainte-Justine Hospital
Friedreich's ataxia (FA) is one of the most frequent neurodegenerative
diseases. Its clinical manifestations appear at a critical stage in the
development of an individual and affect his autonomy and quality of
life.
A number of years ago it was discovered that FA is among others caused
by a deficit of frataxin in the cells of the heart and of the nervous
system.
Until very recently there was no medication available to improve the
condition of FA patients. Idebenone as well as Catena, which is its new
high dose formulation, seem to cause an improvement at the neurological
and cardiac levels. Two ongoing studies have yet to prove their
effectiveness for all patients. Hopefully other drugs will appear on the
market and contribute to ease the clinical symptoms of a maximal number
of patients.
Multiple ongoing studies on inhibitors of the histone deacetylase enzyme
(HDACIs) are trying to demonstrate their effectiveness in several types
of cancer or neurodegenerative diseases. It seems that only a specific
group of HDACIs is able to significantly increase the level of frataxin
in human body cells, particularly in immature white blood cells as well
as in organs singled out by Friedreich's ataxia.
At this moment one of these molecules was chosen to be a clinical
candidate and should reach phase 1 before the end of the year in Europe
(study of the drug: absorption, elimination, etc.) If everything goes
well, phase 2 (clinical study of patients) should start somewhere in
2010.
A meeting in Europe is scheduled in July to sum up the situation on the
development of this molecule. Dr Massimo Pandolfo, a world expert in
Friedreich's ataxia, tells us that the principal researchers will start
planning clinical trials during that meeting.
Obviously FA patients in Quebec could eventually take part in such a
clinical study because the disease is so much more prevalent in Quebec
and because of the experience we have acquired during these years
studying neurodegenerative diseases. In this regard Dr Pandolfo has
already mentioned that he will propose our clinical environment as a
possible North American partner. An exciting story worth to keep an eye
on!
Source: L'Eldorado, June 2009, p.11
Translated by Sofia Arenzon
Canadian Association for Familial Ataxias – Claude St-Jean Foundation
http://www.lacaf.org/index.php?option=com_content&task=view&id=76&Itemid=138&lang=english
this came across my desk this morning...
cheers
Laurel
------------------
Idebenone Well Tolerated in Patients With Friedreich's Ataxia: Presented
at ENS
By Judith Moser, MD
MILAN, Italy -- June 25, 2009 -- In patients with the rare autosomal
recessive disorder Friedreich's ataxia (FRDA), idebenone is generally
well tolerated, according researchers here at the 19th Meeting of the
European Neurological Society (ENS).
Principal investigator Jörg B. Schulz, MD, Department of Neurology,
University Medical Center Aachen, Aachen, Germany, presented the results
of a blinded interim analysis of the first phase 3 trial conducted in
Europe to evaluate a potential treatment for this disease on June 23.
As Dr. Schulz explained, the randomised, placebo-controlled,
double-blind, 12-month, multicentre phase 3 Mitochondrial Protection
With Idebenone in Cardiological or Neurological Outcome Study (MICONOS)
is being conducted to further evaluate the efficacy and safety of
idebenone in children and adults with FRDA based on phase 2 data.
A total of 232 patients aged >=8 years at baseline with a diagnosis of
FRDA and confirmed FRDA mutations are participating.
The patients were randomised to idebenone 180 or 360 mg/day (depending
on body weight), 450 or 900 mg/day, or 1,350 or 2,250 mg/day or placebo.
The threshold regarding body weight was 45 kg.
"It has been shown before that even higher doses are safe in the
treatment of FRDA," Dr. Schulz stressed.
Source/read more:
http://www.docguide.com/news/content.nsf/news/852571020057CCF6852575E100649CE8
Article kindly supplied by Mari Luz González Casas
