Life expectancy for someone with ALS ranges from two to six years. A small percentage may live longer but typical symptoms are debilitating over that span and most with ALS die from respiratory failure.
Recent news of a drug test:
The drug is not a cure, but it may help slow the inexorable disability caused by ALS, which rapidly destroys the nerve cells that control the muscles that allow us to move, speak, eat and even breathe.
Researchers are working to slow down the disease progression in the hopes of finding a cure.
Hope can be fleeting. Imagine having a disease with a prognosis of living for six more months, then to read about a potential cure in the last trial stage; a medicine likely to be released in six months.
Timing is everything.
The multicenter, randomized, double-blind study is the second step — a phase 2 trial — in a three-step process required by the Food and Drug Administration for drug approval. In a double-blind study, neither the patients nor the researchers know who is receiving the drug. If a phase 2 study generates positive results, the FDA typically requires a larger and longer phase 3 trial.
See the problem?
Hope is on the horizon. Rather, hope is just over the horizon for those who have less than a horizon to live.
Neurologists use an assessment scale to determine how ALS has affected someone and a score determines approximately how much longer they will live. The scale starts at 48 points. Those in the study had reached 36 points so debilitation had already begun.
Patients taking the medication lost an average of 2.32 points less than those receiving placebos, a 25 percent better functional outcome.
My progression score already is in the 20s and moving quickly so I look at such drug test results with a mixed plate of emotions.
I’m thankful progress is being made, pray that some will benefit, yet I understand the nature of such testing procedures. They take time. Time is exactly what those with ALS do not have. I’m saddened to realize that many ALS patients do not have enough time left to live to benefit from a medicine that may not get to market until long after they have died.