I wanted to give you some general feedback into what was discussed at
the FARA Review Meeting last Thursday.
The Researchers and Scientists covet their studies very closely, so
anything I say here will be of a general nature out of respect for their
work.
Stem cells were the major theme for this years meeting. With many
interesting developments, including research into avoiding rejection of
stem cells by using one owns stem cells,and removing the affected FA
part.
Other Stem Cell work uses Virus to help with the reproduction of cells.
Apparently the virus stops dead after initiating cell growth.
EpiGenetics is fascinating. Instead of using regular gene therapy, it
uses an inhibiting agent to attach to the gene and turn the gene off. A
compound to turn the FA gene off, was found in UCLA last year... so that
is very exciting.
lab work is continuing on the Frataxin protein with promising findings
on Frataxin levels in FA patients, Carriers and non carriers.
Trials abound. Word on Idebenone ( for those not already using it) is
that it should be available to patients by the end of 2009.
Deferiprone trial is about to start in australia. Unfortuately there
are only 2 definate participants with 2 more maybes. they had hoped for
10, but will add this data to the worlds. If you know anyone between
7-17 with FA they may be interested in participating.
Other trials are happening around the world with some held up in
Ethics...
Antioxidants... some research is suggesting that antioxidants are not
the panacea we had hoped, however, idebenone DOES target the heart in a
positive way, and help some neurological aspects so there is a
suggestion there are factors at work other than straight antioxidants.
FA testing models were discussed with the conclusion that "the wheel" is
still not perfect and still upto fine tuning.
Audiology. WOW:- there was a presentation on this front which was
eyeopening. It has been found ( for the most part) that FA sufferers
have normal hearing on general sound testing... even cochlear testing
brings normal results.
However, when the time delay of hearing was tested, it was found that FA
sufferers often had problems with the speed they " hear" the sounds...
this then leads to the speed of processing the sound, and it was found
in general, that FA sufferers depending on their hearing speed had
problems with processing what they hear.... for eg, the more background
noise there is, the harder it is to distinguish certain aspects of
speech.. What is not being tested generally is this hearing speed/
processing speed..... So if you attend the clinic, it may be worth
asking Louise ahead of time if you can get this tested.
On the clinic, they have about 120 attending the clinic, with most from
Vic, NSW and QLD, and 4 from SA ( its a start)... none from WA as yet,
but distance is a considerably factor. On that front... you do all
realise that you can apply for distance rebate on your travelling and
accommodation to attend the clinic. Just ask Louise Corben about it.
Unfortunately, as the day was running a bit overtime, I had to leave to
catch a plane, and missed most of Louise Kenetic presentation, but as
you can see above there is HOPE, and they ARE getting closer.... the
fact that Idebenone is so close to being available and that we now have
a trial happening in Australia is a huge step.
Hope this has provided you with something interesting... if you have
further questions I will try and shed some light on them... still
processing it all myself.
Merry Christmas to you all, and hope and happiness for 2009
--
Laurel Hosking
Help FA in SA
mob: 04204 98963
ph: 08 85221167
fax: 08 85221313
laurel@linvid.com
