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
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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