About Motoneurone Disease
Amyotrophic lateral sclerosis (ALS) is also known as:
- Lou Gehrig’s disease in the US
- Maladie de Charcot in France
- Motor Neuron Disease (MND) in the UK
ALS is a progressive neuromuscular degenerative disease.
The disease is characterised by the progressive degeneration of motor nerve cells in the central nervous system.
This degeneration can affect:
- Upper motor neurons in the brain
- Lower motor neurons in the spinal cord
These are very severe conditions causing a dramatic loss of quality of life and most patients with the disease die between 2-5 years after diagnosis.
Incidence Rate and Market Size
- Motorneurone Disease (ALS) is a rare disease with an incidence rate (new cases) of 2 to 2.5 in 100,000 persons in Australia with a prevalence rate of 1 to 15,000 (people living with ALS).
- The statistics in the US show a mortality rate of 1.7 per 100,000 people in 1998, with a trend showing the incidence rate rising.
- The market size is approximately $150 million US per year.
- There are no highly effective treatments for ALS.
- So far, the only approved drug is Riluzole, which prolongs life span on average by only 2-3 months.
- Riluzole generated global revenue of over $153 million US in 2006.
Science of ALS & NRPs Benefits
Causalities or strong hypotheses for the susceptibility to develop Motoneurone Disease are mainly unknown and only 2% of the patient population has inherited familial ALS with mutations/polymorphisms within their superoxide dismutase (SOD-1) gene region.
This enzyme is one of the major anti-oxidant players in the human body. The 98% patient group with unfamilial ALS have no current known genetic risk factors.
- The best validated animal model mimicking familial ALS is the G93A gain of function SOD-1 mouse mutant model.
The only FDA-approved drug Riluzole shows significant survival-promoting effects in this mouse model.
New drug approval applications are looking for combinational effects of new drugs showing synergistic effects compared to Riluzole alone.
For Motoneurone Disease:
- NRPs dosages within a range of factor 100 apart are efficacious in this model.
- NRPs dosages of ng/kg to μg/kg range are effective in the SOD-1 mouse model
- Beneficial combinational effect of NRPs in the presence of Riluzole
- NRPs are likely to be eligible for Orphan Drug Status from the FDA which would significantly shorten the clinical trial time and associated costs.
- Read more about Orphan Drug Status