This
article has been drawn from the Eldorado
(publication of April 2006).
FARA (Friedreich’s Ataxia Research Alliance). His president
Ron Bartek, makes very promising researches coming from scientific
projects being actually directed including those that will make
the object of clinical trials in the next year.
-
Idebenone: The trial in phase 2 at the (National Health Institute)
goes off smoothly with the 48 participants and some more in
case they are needed. Idebenone will enter the phase 2 and
3 of the trial in Europe in few months. This trial will be
conducted by the Laboratory Santhera who provides Idebenone
used at the NIH. We have often qualified Idebenone as being
an antioxidant, "a free radicals extractor" which
without it will damage the cells. Even if it is an antioxidant
in a certain way, it seems to be more evident that it can
also be something else. It seems to be probable that Idebenone
can also help so well electrons along the mitochondria inner
membrane that more energy and less free radicals are produced.
-
Mitoquinone: Clinical Trials in the phase 11 are planned for
the first semester in the next year in Australia (Martin Delatiky
at the Mordoch Institute) and in California (Sue Perlman UCLA
Los Angeles). Mitoq is Coenzyme Q 10 more a little additive
that is the same to Mitochondria. Mitoq's specialists are
hoping that will have the effect of a very efficient antioxidant
because it concentrates at more elevated doses a way more
than in the mitochondria to block the effects of free radicals
where they are produced and before they cause any damages.
-
AOOO1 (Edison Pharmaceutical Laboratory). An attempt is made
in the elaboration of a clinical trial in the phase 2 on this
molecule in the half-next year. FARA has granted 3 million
dollars for the development of this point. At the head of
this particular effort of pharmaceutical development, are
the Dr. Guy Miller d'Edison, Dr. Rob Wilson of Pennsylvania's
University and Dr. Sid Hetch of Virginia's University. These
scientists claim that AOOO1 is introduced inside the mitochondria,
which facilitate the transport of electrons with more fluidity
along the mitochondria membrane in a way to produce more energy
and less free radicals. Such flux of electrons is normally
accompanied with frataxin protein. Because the electrons along
the mitochondria membrane have the tendency to accumulate
and to escape prematurely free radicals are produced and the
process of energy production does not arrive in time. In fact,
these scientists believe that there are big chances for the
molecules as AOOO1 can compensate the deficit of frataxine
and produce a significant rise of energy and an important
reduction of oxidative stress.
-
Substances that are able to increase fratixin level. FARA
closely follows and works head onto 2 promising projects of
the same type. One is leaded by DR. Joel Gottesfeld at Scripps
Research Institute of Jolia, California with the help of several
bourses from FARA. His project is promising because the compounds
he is using are seems to increase the frataxin level in cell
cultures in patient blood at equal levels or even higher,
that the ones of their brother and sisters, healthy bearers.
These compounds act upon the mitochondria but inside the cell
nucleus on the chromosome that contains the gene of AF.
Dr.
Barbara Shelber conducts the other project in Austria with the
help of many grants from FARA and "Seek a Miracle".
In this project, a substance, called EPO, is utilized on blood
cells, cardiac, nervous, with apparently resulting with the
elevation of 2 to 5 times the level of frataxin. The doctor
is still not sure how is acting this substance that allow reaching
promising results. She works hard to put in place a clinical
study with AF patients. She just wrote FARA asking them to send
her scientific articles.
Those
two projects aiming to raise up the frataxin level use compound
already employed in other diseases or are being the object of
other clinical trials for other diseases, thus if they continue
to show such promising results for AF, scientists could utilize
it more efficiently in specific trials. Already, innocuity test
have been perpetrate on animals and have been administrated
to humans. Clearly this approach of frataxin increment could
progress rapidly.
The
potential benefit for frataxin increment in these two projects
is enormous. As we know carriers have a level of frataxin inferior
to non-carriers but they are healthy. Since then our patients
could enormously benefit from a possible increase of their frataxin
level reaching of even exceed the one of their siblings, healthy
carriers.
Not
realized yet but we are on the right trail!
Although we shall keep in mind that culture cells are not people.
There is a lot to do yet, so that culture can be reproduced
on patients without danger, these projects are extremely promising
and are going fast.
Since
then, we can imagine to what extent it will be exalting to see
the development of a "therapy cocktail" involving
anti-oxidants "cleaning up" into free radicals even
in the case of unhabitually low case of frataxin and its compounds
increasing the level of frataxin. These scientists begin to
think that such cocktail could stop the progression of the disease.
Ron
Bartek - FARA 11.05
(American Association for Research on Friedreich’s Ataxia)
fararesearch@yahoo.com