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2004/OB/08
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OXFORD
BIOMEDICA PLC
UK SCIENTISTS
PIONEER NOVEL TREATMENT FOR MOTOR NEURON DISEASE
Oxford
BioMedica (LSE:OXB.L), the leading gene therapy company, announces
today that the Company’s scientists in collaboration
with scientists from VIB (the Flanders Interuniversity Institute
for Biotechnology) in Leuven, Belgium have published the results
of pioneering research that could lead to a treatment for
patients with Amyotrophic Lateral Sclerosis (ALS), the most
prevalent form of motor neuron disease. The results, published
in today’s Nature
magazine (Volume: 429, Issue: 6990 pp: 413-417) are based
on preclinical studies and show that using a novel gene therapy
approach, both onset and progression of disease is slowed,
and that life expectancy is extended by 30%, thereby achieving
one of the most effective therapies reported in the field
to date.
ALS causes
adult-onset, progressive motor neuron degeneration in the
brain and spinal cord, resulting in paralysis and death three
to five years after onset in most patients. There is currently
no known cure for motor neuron disease, a condition that affects
approximately 100,000 people in Europe and the US.
The research
was led by Dr Mimoun Azzouz, Director of Neurobiology at Oxford
BioMedica, in collaboration with the VIB department for Transgene
Technology and Gene Therapy in Belgium. The novel gene therapy
product – MoNuDin, delivers a vascular endothelial growth
factor (VEGF) gene, a neuroprotective factor, using the Company’s
proprietary LentiVector system. The product is injected into
muscle and mediates its therapeutic effect within the nerve
cells of the spine. It has previously been reported that reduced
levels of VEGF predispose mice and humans to ALS, but this
is the first assessment of its therapeutic potential. These
results show that a single injection of a VEGF-expressing
lentiviral vector into various muscles delayed onset and slowed
progression in an animal model of ALS. Treatment was also
found to increase the life expectancy of mice by 30 per cent.
Dr Brian
Dickie, Director of Research Development at the MND Association,
said, “These findings reflect current optimism amongst
researchers that gene therapy represents a viable strategy
for the treatment of ALS and other neurodegenerative diseases,
overcoming problems of access of drugs to the central nervous
system, which can occur with more conventional approaches
to treatment.“
Commenting
on the results, Oxford BioMedica’s Chief Executive,
Prof. Alan Kingsman said, “Although these results
published in Nature are still at a preclinical stage, the
data suggests that VEGF gene therapy could provide an effective
treatment for ALS, a debilitating disease that leads to premature
death and for which there is no current cure and current treatments
are ineffective. These results also bode well for our spinal
muscular atrophy product, which employs a similar technology.”
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| Notes
| 1. |
Oxford
BioMedica plc |
|
Oxford
BioMedica (LSE: OXB) is a biopharmaceutical
company specialising in the development
of novel gene-based therapeutics with a
focus on the areas of oncology and neurotherapy.
The Company was established in 1995 as a
spin out from Oxford University, and is
listed on the London Stock Exchange.
In
addition to its technical expertise in gene
delivery, Oxford BioMedica has in-house
clinical, regulatory and manufacturing know-how.
The development pipeline includes two novel
anti-cancer products in clinical trials;
and two neurotherapy products in advanced
preclinical development for Parkinson’s
disease and retinopathy. The Company is
underpinned by an extensive preclinical
and research portfolio and about 70 patent
families, which represents one of the broadest
patent estates in the field.
The
Company has a staff of ~65 split between
its main facilities in Oxford and its wholly
owned subsidiary, BioMedica Inc, in San
Diego, California. Oxford BioMedica has
corporate collaborations with Wyeth, Intervet,
Merck & Co, Amersham and Kiadis. |
| 2. |
MoNuDin™
and Motor Neuron Disease |
| |
MoNuDin
comprises a vascular endothelial growth
factor (VEGF) gene delivered by the Company’s
proprietary LentiVector system. The product
is designed to be injected into muscle,
where it enters motor neurons via the neuromuscular
junctions. It then travels along the nerves
to the spinal cord by a process known as
retrograde transport and mediates its therapeutic
effect within the body of the nerve cells
in the spine.
Motor
Neuron Disease (MND) is the name given to
a group of related diseases affecting the
motor neurons in the brain and spinal cord.
Motor neurons are the nerve cells along
which the brain sends instructions, in the
form of electrical impulses, to the muscles.
Degeneration of the motor neurons leads
to weakness and wasting of muscles. This
generally occurs in arms or legs initially,
some groups of muscles being affected more
than others. MND is generally a steadily
progressive disease, but the rate of progression
varies greatly from one person to another.
MND
can affect any adult at any age but most
people who have MND are over the age of
40 and the highest incidence is in the 50-70
age range. Men are affected slightly more
often than women.
The
precise figures for the incidence and prevalence
of MND are still uncertain. In the UK, three
people are diagnosed and three people die
from MND every day. The average life expectancy
of a patient is two to five years from time
of diagnosis, but half the number of people
with MND will die within 14 months of diagnosis.
The estimated number of people living with
MND in the UK is 5,000 at any one time.
There are about 100,000 patients in Europe
and the US. |
| 3. |
The
Motor Neurone Disease Association |
| |
The Motor
Neurone Disease (MND) Association is the
only national charity working on behalf
of people with MND in England, Wales and
Northern Ireland. It provides advice and
support to people affected by MND and funds
and promotes research into the disease.
The charity has its national headquarters
in Northampton and over 80 branches run
by volunteers nationwide. Further information
can be found at www.mndassociation.org.
For more information about
MND, the MND Association or to interview
people living with the disease contact Gayle
Sweet, Head of PR & Media on 07831 349382
or Richard Green, Director of Communications
on 07960 941070. |
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