Ground Breaking Discovery into causes of Motor Neuron Disease, an Opportunity for the Development of new Therapeutic Strategies

Amyotrophic Lateral Sclerosis (ALS) is the most common neurodegenerative disorder of young and middle aged adults, is incurable and invariably fatal. The only disease-modifying therapy currently available is Riluzole®, which only extends survival by 3-6 months and has recently come off patent. This treatment is expected to reach sales off about US$129 million this year. Novel disease-specific mutations in a particular gene have recently been identified in ALS patients. This gene has not previously been associated with ALS thereby opening up a whole new avenue of possible treatments to slow down or even reverse the process of cell degeneration in this condition. As there are similar pathways in all neurodegenerative diseases, disease-modifying drugs in ALS may have therapeutic effects in other more common neurodegenerative disease such as Parkinson's disease (PD) and the Dementias. The total dollar value for drugs to treat PD alone in the seven major markets predicted to exceed $3 billion in 2012.


ALS is a disease with an incidence of approximately two per 10,000. The gene studied (Angiogenin) is a hypoxia-inducible gene. Recent animal evidence indicates that the hypoxia-inducible factor vascular endothelial growth factor (VEGF) may be implicated in the pathogenesis of ALS. These findings have led to a series of planned clinical trials of VEGF in patients with ALS.

Technology Description

  • Dr Orla Hardiman of the Royal College of Surgeons in Ireland (RCSI) and her collaborators have studied ALS-specific mutations in angiogenin, soon to be published in Nature Genetics (Advanced On-line Publishing, 26 February 2006 doi:10.1038/ng1742).
  • Prof Jochen Prehn also of the RCSI and his collaborators have carried out key studies building on these findings showing that angiogenin is expressed preferentially in the nervous system and is neuroprotective to motorneurons. Ongoing studies are being carried out to test whether treatment with angiogenin can rescue injured neurons.

    Competitive Advantage and IP Status

  • This opportunity points to the development of new therapeutic products for ALS, an incurable and invariably fatal disease where only one disease-modifying therapy currently exists. Given that ALS is a relatively rare disease there is the potential that new agents will be given 'orphan drug' status
  • The new approach is based on breakthrough work on a gene not previously associated with ALS
  • There is the potential that disease-modifying drugs in ALS may have therapeutic effects in other more common neurodegenerative diseases such as Parkinson's disease

    A patent application has been filed to protect the use of angiogenin for the treatment or prevention of neuro-degenerative diseases.

    Type of Business Sought

    This opportunity is available for discussion with potential commercial partners and/or interested investors.

    Principal Investigators:

    Prof. Jochen Prehn & Dr. Orla Hardiman,
    Royal College of Surgeons, 123 St Stephen's Green, Dublin 2, Ireland

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