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
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
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
This is a world wide study being run in Europe, Australia and Canada.
Criteria seems to be "7 to 35 years of age, has a confirmed genetic
diagnosis of FRDA with number of GAA repeats greater or equal to 400 on
both copies of DNA, has a score between 20 and 85 on a neurological exam
known as Friedreich's Ataxia Rating Scale (FARS), has a negative urine
pregnancy test (if applicable) before the research study starts and is
currently not participating in another research study. "
further information, on
Name: Ms Geneieve Tai
Role: Research Coordinator
Telephone: (03) 8341 6374
cheers all
--
Laurel Hosking
Help FA in SA
mob: 04204 98963
ph: 08 85221167
fax: 08 85221313
laurel@linvid.com
Next month I will be attending the Grants round meeting of FARA(A) in
Melbourne. I recently had a wonderful luncheon meeting with the
Executive Director Varlli Beetham. She had come across to SA to speak
to Trinity College students, who have embraced FARA as their yearly
fundraiser. During this Assembly, the students presented Ms Beetham
with an initial chq for almost $400.... not bad for a couple of gold
coins thrown in a classroom jar.
As a part of this presentation, Billys slide show on FA was shown to the
entire school, and now the major fundraiser of Tea towels is about to
kick off.
As part of our meeting, we discussed the importance to get neurologists
on side here in SA. This is vital for the establishment of any drug
trials here in SA, plus the long term goal of an SA clinic.
As you can guess, I " dobbed in" billys neurologist, who has accepted an
invitation to attend the grants meeting and meet with FARA and be
involved first hand.
As you can see, things are still ticking along.
cheers everyone
Laurel
