Research
The Latest Research
1. Juvenile Drug Trial, USA, using CellCept:
The FDA (Federal Drug Administration) has approved the drug trial for juvenile
Batten disease. The group
that will be conducting the trial are now applying for
Institutional Review Board (IRB) approval to conduct
the trial at the
University of Rochester.
Details not yet known are;
(a)
the number of children who will be allowed to participate in the trial;
(b)
what the parameters for inclusion will be (other than the child
must be confirmed by genetic testing to have CLN3;
(c)
if open to children outside of USA and Canada.
Although the Australian Chapter has assisted with funding
to bring this trial to reality, in this early
stage it is doubtful that this
trial could be offered to Australian and New Zealand children
until the safety aspect and effectiveness of the drug is monitored.
In the meantime (whilst the USA trial is progressing),
I intend to approach a medical institution to find out the protocol of
having the drug prescribed here as I have
contacted an organisation
in Australia who manufactures the drug so I now know that it is available here.
That is one less hurdle !! We'll stay tuned on this.
2. Late Infantile Stem Cell treatments:
You would be aware of the StemCell Inc. research in Stage 2
this year (also only available in the States)
and we all will
watch closely as to its progress and effectiveness but
here is a very interesting link for
Late Infantile stem cell treatment in China
http://www.unistemcells.com/en/stemcell/20081216204518510.htm
where unlike America's (Stem Cell Inc.) Stem Cell TRIAL treatment,
anyone can participate. I believe
that the main issue is that there is no protection
for the child as to the efficacy of the treatment whilst
outside
your own country. I am in contact with a American family who is currently
in China under
going the treatment and will keep you informed of their progress.
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