Financial boost for motor neurone disease programme from USA Amyotrophic Lateral Sclerosis Association
Oxford BioMedica announced in June 2002 that its motor neurone disease product development programme, a collaboration with Nicholas Boulis of the Centre for Functional and Restorative Neurosurgery, the Cleveland Clinic Foundation in Ohio, had received a grant of $120,000 from the USA Amyotrophic Lateral Sclerosis Association, a prestigious charity dedicated to the fight against ALS or Lou Gehrig's Disease, named after the famous baseball player.
Lou Gehrig's disease is one of the most devastating human disorders affecting the function of nerves and muscles.
BioMedica is assessing a series of five genes as components of a product to address the Motor Neurone Disease (MND) market of which Lou Gehrig's disease is the most common manifestation.
The genes, which have been selected to prevent disease progression, will be delivered to motor neurones using the company's proprietary LentiVector technology.
The company has already reported that the LentiVectors are able to deliver genes to motor neurones with very high efficiency.
It is anticipated that, within a year, up to two of these genes will be selected as the component of the final product that will progress to clinical trial.
The grant from the ALS Association is to support this initial stage of the programme, and it is anticipated that, pending regulatory approvals, the phase I trials of the new product will be conducted in late stage ALS patients at the Cleveland Clinic Foundation.
In ALS motor neurones die and, as a result, the ability of the brain and the spinal cord to initiate and control muscle movement is lost.
With all voluntary muscle action affected, patients in the later stages of the disease become totally paralysed.
Early symptoms of ALS include increasing muscle weakness, especially involving the arms and legs, speech, swallowing and breathing.
When muscles no longer receive the messages from the motor neurones that they require to function, the muscles begin to waste away and limbs begin to look thinner.
Patients usually die from respiratory failure as a result of collapse of the diaphragm muscle.
Over 5,600 people in the USA are diagnosed with ALS each year (15 new case a day).
Most people who develop ALS are between the ages of 40 and 70, with an average age of 55 at the time of diagnosis.
ALS is 20% more common in men than in women.
However, with increasing age, the incidence of ALS is more equal between men and women.
Half of all people affected with ALS live less than three years after diagnosis.
Twenty percent live five years or more; only ten percent will survive more than ten years.
The incidence of ALS is approximately two per 100,000; the prevalence is seven per 100,000.
The financial cost to families of persons with ALS is exceedingly high.
It is estimated that in the advanced stages, care can cost an average of $200,000 a year.
Market estimates for a significant therapeutic product are of the order of $0.5 billion for Europe and the USA.
Commenting on the programme BioMedica's chief executive, professor Alan Kingsman said: "Over the past year we have broadened our neurobiology portfolio to include products for Parkinson's disease (ProSavin), nerve repair (Renurex) and now motor neurone disease, all based on our powerful proprietary LentiVector technology.
"The initial research costs for this new programme will be almost entirely covered by the grant.
"We are particularly pleased that the prestigious ALS Association has chosen us as the leading collaborator to bring a gene therapy approach to treating this dreadful disease, it represents a considerable endorsement of the quality of the company's LentiVector gene delivery system."
