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About Motoneurone Disease

Amyotrophic lateral sclerosis (ALS) is also known as:active healthy neurone

  1. Lou Gehrig’s disease in the US
  2. Maladie de Charcot in France
  3. 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:

  1. Upper motor neurons in the brain
  2. 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.
    CNS

Treatments Available

  • 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.

Animal Models

  • 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:Microscope

  • 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

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