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From: TSS ()
Subject: MAJOR ACADEMIC/INDUSTRY COLLABORATION TO FIND A DRUG TO TREAT CJD
Date: April 13, 2005 at 6:33 am PST

-------- Original Message --------
Subject: MAJOR ACADEMIC/INDUSTRY COLLABORATION TO FIND A DRUG TO TREAT CJD
Date: Wed, 13 Apr 2005 08:09:05 -0500
From: "Terry S. Singeltary Sr."
Reply-To: Bovine Spongiform Encephalopathy
To: BSE-L@KALIV.UNI-KARLSRUHE.DE


##################### Bovine Spongiform Encephalopathy #####################

Reference: MRC/24/05

For immediate release on 12 April 2005

MAJOR ACADEMIC/INDUSTRY COLLABORATION TO FIND A DRUG TO TREAT CJD

Today, an unprecedented collaboration between the UK's leading academic
research group working on CJD - the Medical Research Council's Prion Unit
based at the Institute of Neurology, University College London - and the
UK's largest pharmaceutical company - GlaxoSmithKline (GSK) - is announced.

The occurrence of variant Creutzfeldt-Jakob disease (vCJD) and the
recognition that this is caused by exposure to the infectious agent or prion
responsible for bovine spongiform encephalopathy (BSE) has led to fears of
an epidemic of human infection with BSE prions - as many in the UK
population will have been exposed to BSE prions prior to the introduction of
rigorous controls to limit dietary exposure in 1996. Although the number of
patients developing vCJD so far is thankfully relatively small (around 150),
incubation periods for prion infections are known to span decades in humans
and the number who may be silently infected and eventually develop disease
is unknown, as are the numbers of secondary infections that may occur via
contaminated blood products or surgical instruments. Because of these
uncertainties, the development of an effective treatment to eradicate prion
infection during the window of opportunity provided by the prolonged latent
period of prion infection is considered a strategic priority. However, the
rarity of CJD makes it an "orphan" disease which means normal commercial
drug development is unrealistic.

A major effort has been underway for some years at the MRC Prion Unit to
understand the fundamental and unique processes involved in the propagation
of prions - the remarkable infectious agents that cause CJD in humans and
BSE and scrapie in animals. Much of this laborious and long term work has
been to identify and validate the best target against which to develop a
drug to block prions replicating. Prions are misfolded or rogue forms of one
of the body's own protein molecules - the prion protein - and the Unit has
shown in prion-infected laboratory animals that the disease process in the
brain can be halted by targeting this protein, without apparent deleterious
effects. The aim is now to develop a drug that is capable of readily
entering the brain and binding to the normal prion protein blocking the
change in shape involved in it turning into the rogue prion.

To do this requires screening literally hundreds of thousands of potential
drug-like molecules from large compound collections or "libraries". One of
the largest and best characterised such libraries in existence is that of
GSK - which contains over a million such compounds. Such libraries form one
of the most important commercial assets of any pharmaceutical company and
making such a library available to an academic group in this way is
unprecedented. Peter Machin, Senior Vice President Chemistry & Screening
Sciences at GSK said:

"We are delighted to provide our compound collection of high quality,
drug-like molecules to accelerate the search for compounds that may prove
useful in the treatment of CJD"

The programme of work towards development of a candidate drug for potential
trial in humans is estimated to take at least a further six years and the
first phase of this work, which has followed a large scale initial pilot
study will start soon. The UK Department of Health has agreed to fund the
work through a grant award to the Institute of Neurology,UCL. The
partnership now forged between GSK, the MRC Prion Unit,and UCL, brings
together for the first time all the relevant expertise and resources to make
such a development feasible and may act as a stimulus to such
academic-industy collaborations in other areas of medicine.

Professor John Collinge, Director of the MRC Prion Unit and Head of the
Dept.of Neurodegenerative Disease at the Institute of Neurology,UCL, said:

"Many person years of very difficult work at the MRC have been invested to
get to the stage where development of a drug to completely block prions
appears realistic. My colleagues and I, at the Unit, are absolutely
delighted at the opportunity now to translate these advances in laboratory
research into real benefit for patients affected by these dreadful diseases.

"There is only so far we can take things on our own as academic scientists
and, while there is no guarantee of success, now having the enormous
resource and expertise of GSK with us is extremely exciting".

ENDS

For further information contact the MRC Press Office on 020 7637 6011

The Medical Research Council (MRC) is a national organisation funded by the
UK tax-payer. Its business is medical research aimed at improving human
health; everyone stands to benefit from the outputs. The research it
supports and the scientists it trains meet the needs of the health services,
the pharmaceutical and other health-related industries and the academic
world. MRC has funded work which has led to some of the most significant
discoveries and achievements in medicine in the UK. About half of the MRC's
expenditure of £450 million is invested in its 40 Institutes, Units and
Centres. The remaining half goes in the form of grant support and training
awards to individuals and teams in universities and medical schools. Web
site at: http://www.mrc.ac.uk

The Institute of Neurology is a specialist postgraduate Institute of
University College London. The Institute is closely associated in its work
with the National Hospital for Neurology & Neurosurgery, and in combination
they form a national and international centre at Queen Square for teaching,
training and research in neurology and allied clinical and basic sciences.
Website at: http://www.ion.ucl.ac.uk

TSS

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