Following a segment on Channel 7 last Friday night on Huntington's
Disease, Prof Elliott responds to one individual's questions. We
hope this is helpful to you. If there are any other specific
questions you wish to raise, Prof Elliott would be happy to answer
these. Please feel free to send this information to people who are
seeking information.

· What did the trial involve?
This is described in the publications (see list below - can be
accessed through Pubmed on the internet - just dial in first authors
name after finding Pubmed ). We used a fairly standard rat model of
Huntington's which involves injecting quinolinic acid into the
striatum. An excitation and degeneration mimicking Huntington's in
both clinical presentation and pathology results. We injected either
rat or piglet choroid plexus cell clusters encapsulated in alginate
into the area immediately prior to inducing the lesions

· What were the results?

A remarkable prevention of the behavioural, pathological and
clinical changes - around 80% . The animals recovered extremely
well.

· When will the human trials begin and are you looking for
participants (if so, how do they get in contact with you)?

We are repeating the studies in non-human primates first, using
piglet choroid plexus - these should be completed by mid February
2005. This is a necessary step to applying for regulatory approvals
in order to begin Phase 1 human trials . How long these approvals
take is uncertain. There are strong non-scientific prejudices
against the use of animal cells, which may be difficult to overcome.
In Australia we hope to hear from the regulatory body in December,
whether or not they will be able to process an application. In the
USA the corresponding regulatory body is empowered to process such
an application.

· What will the human trials involve?

At present the trial would involve the injection of a small
volume of encapsulated pig choroid plexus cells into the brain
striatum of patients who are already experiencing severe symptoms of
the disease. A small 'burr hole' would be made in the skull and a
needle positioned by stereotactic means. An alternative might be to
inject into the ventricles of the brain - this is yet to be
determined.


· If the treatment looks promising for humans, what sort of
time-frame are we looking at?

There are two stages. We will know whether the treatment works
or not in the larger (primate) animal model, hopefully by
February. If it works, we will apply to do the human phase 1 study.
With any luck this could be underway by the middle of 2005 - but this
is in the hands of the regulators.

Our web site (lct.com.au) is up and running, but lacks the detailed
science papers as yet. These are listed below.

The web site is under construction and will be updated as the
results come in.

As you would understand, this sort of information creates a great
load of hope within the HD community – whilst we too are optimistic,
we want to be able to provide accurate information.

I appreciate that our announcement could cause some consternation. A
friend of mine died recently from the disease. A challenge that we
face is to determine how long the treatment effect will last. The
therapeutic cells are very hardy and last a long time (6 months) in
artificial conditions (cell culture). The animal experiments we did
went for 6 weeks and the restorative effect remained for that time.
We now have to determine if we can predict how long the treatment may
last.

As we are a commercial company listed on the stock exchange, we must
by law announce anything likely to influence the share price ahead of
the information becoming available to a select few.

Publications:

C V Borlongan, S J M Skinner, M Geaney, A V Vasconcellos, R B
Elliott, D F Emerich Neuroprotection of encapsulated choroids plexus
in a rodent model of Huntington's disease Neuroreport Vol 15 No 15
2004



C.V. Borlongan; S.J.M. Skinner, M. Geaney; R. Elliott; A.V.
Vasconcellos; D.F. Emerich. Neuroprotective effects of encapsulated
choroid plexus in a rodent model of Huntington's disease. 34th
Annual Meeting, Society for Neuroscience. San Diego, CA, USA Oct 23-
7, 2004



Dwaine F. Emerich; Stephen J.M. Skinner; Cesario V. Borlongan; Alfred
Vasconcellos The Choroid Plexus in the rise, fall and repair of the
brain. In Press (Nov 2004) BioEssays



Borlongan CV, Skinner SJM, Geaney M, Vasconcellos AV, Elliott RB,
Emerich DF. CNS grafts of rat choroids plexus protect against
cerebral ischemia in adult rats. Neuroreport Vol 15 No19 19 July
2004.



Borlongan CV, Skinner SJM, Geaney M, Elliott R, Vasconcellos AV and
Emerich DF. Intracerebral transplants of choroids plexus provide
structural and functional neuroprotection in a rodent model of stroke.

Eleventh Annual Conference of the American Society for
Neural Transplantation and Repair. Florida, USA May 6-9, 2004.



Borlongan CV, Skinner SJ, Geaney M, Vasconcellos AV, Elliott RB,
Emerich DF. Intracerebral Transplantation of Porcine Choroid Plexus
Provides Structural and Functional Neuroprotection in a Rodent Model
of Stroke. 2004, International Society for transplantation, Vienna,
Austria.



Emerich DF, Vasconcellos AV, Elliott RB, Skinner SJ, Borlongan CV.
The choroid plexus: function, pathology and therapeutic potential of
its transplantation. Expert Opin Biol Ther. 2004 Aug;4(8):1191-1201





Professor R B Elliott