ALS is a horrendous disease attacking the neuromuscular system of the body, slowly and systematically rendering the person paralyzed and unable to breathe.
The journey is emotionally exhausting for family and friends as you watch your loved one wither away, becoming trapped in their bodies. As awful as this disease is and with the extraordinary medical advances giving us unprecedented views into the human body, a new drug for ALS hasn’t made it to market since 1995, leaving those with ALS and their families with little hope. ALS is classified as a rare disease with a much smaller population (20,000 US) of people affected than that of cancer (2 million US). Thus the profit potential of an ALS drug is limited and companies tend to invest where the profit potential is greatest.
Further accentuating the hopelessness is the lack of any ALS Biomarker, which is critical for drug development. There is no test for ALS. It is sadly the disease you have when you fail all the known disease tests for AIDS, MS, Parkinson’s etc. Significantly missing from our knowledge necessary to develop a cure is an understanding of the ALS disease mechanism. Knowledge of what conditions and processes trigger the start of the ALS disease and what keeps it attacking the body’s motor neurons is a critical first step to a cure.