Batting for Battens Inc.

22 Cambridge Place, Wishart, QLD, 4122
enquiries@bfbf.org.au
ABN 40 931 835 917 Endorced DGR
Donations over $2 are Tax Deductible

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.