hope abounds

this is very very exciting.

laurel
http://www.scripps.edu/newsandviews/e_20090928/gottesfeld.html

2010 event

Hi everyone,

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

and this too

Sorry, this one as well, looks really great too.

Laurel

-------------

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:

http://www.globenewswire.com/newsroom/news.html?d=169097

Clinical study on HDACIs…

HI, this came across my desk and looks exciting and worth keeping an eye
on.
5 more sleeps till the Charity Dinner..... gosh this has come fast.
Fingers crossed for a great result...

cheers

laurel
-------------

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

idebenone update

Hi everyone,

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

Donation news

Hi everyone

just a couple of announcments. The following have come on board for
this years event.

AMP Foundation ( pledged 50c for every $1 raised)
Charlesworth Nuts ( festival of flavours basket)
Dowdall Family ( doll house again )

Thanks to financial planner Brett Davy who managed to arrange the AMP
Foundation application for us.

What a great start to this years event

Laurel

Sydney Stem Cell Breakthrough

Hi, this came across my desk today... very very exciting for all of us,
and an example of how fund raising directly helps research

Stem cell breakthrough by Sydney scientists

Louise Hall
March 5, 2009

SCIENTISTS in Sydney have become the first in the world to use adult
stem cells to regrow damaged muscle tissue, offering hope to sufferers
of incurable diseases such as muscular dystrophy.

The breakthrough procedure has been proven to regenerate muscle in a
mouse engineered to have an injured skeletal muscle, but the concept
could also be applied to human diseases such as lung disorders, chronic
liver disease, and types I and II diabetes.

The team of gene therapy, cancer and muscle disease experts solved one
of the biggest hurdles involving stem cell therapy in solid organs -
getting the donor cells to survive for more than an hour after they are
inserted into the damaged host tissue.

The lead author, Peter Gunning, the head of the Oncology Research Unit
at the University of NSW, said until now, the new healthy cells had no
survival advantage over the dominant existing damaged tissue.

Furthermore, injected donor cells were almost immediately wiped out by
the immune system.

"In muscle, most stem cells die in the first hour or are present in such
low numbers that they are not much help," Professor Gunning said.

The most well-established form of cell replacement therapy, bone marrow
transplants, have been performed successfully for 40 years. However
solid tissue such as muscle is much more complicated and previous trials
have yet to work successfully.

Scientists from the Children's Hospital at Westmead and Sydney and NSW
universities tried to enhance the stem cells' survival chances by
inserting an artificial, harmless virus - called a vector - into the
cells, making them resistant to chemotherapy.

The diseased tissue is then killed off by chemotherapy, leaving room for
the healthy cells to engraft and propagate.

"It's the first strategy that gives the good guys the edge in the battle
to cure sick tissues," Professor Gunning said.

The experimental technique, funded by the Oncology Children's Foundation
and published in the journal Stem Cells, is still at the pre-clinical
stage but Professor Gunning said human clinical trials could start
within three to five years.

This story was found at:
http://www.smh.com.au/articles/2009/03/04/1235842487806.html