Our team tested over 75,000 different compounds and identified a number that could eliminate stress granules. This is especially exciting news for patients because it has the potential to give them something close to their old lives back, nothing that any of the sufferers of ALS or Alzheimer’s currently dare to hope.
While there is agreement among the scientific community that stress granule proteins are the cause of ALS in more than 90% of patients and an estimated 50% of Alzheimer’s patients, we are the only company doing drug discovery and development to eliminate persistent stress granules. This breakthrough could turn around years of disappointment in ALS and Alzheimer’s research.
Before the discovery of TDP-43 stress granules, researchers spent 20 years studying SOD1, the first gene discovered that caused ALS. The problem was that SOD1 only occurs in 2% of patients with ALS. Despite many attempts, none of the trials targeting SOD1 was successful in finding an effective therapy for ALS.
For Alzheimer’s disease, the field has been focused on trying to break up the plaques. Until now, scientists have only known that the plaques and tangles accumulate, they haven’t known how or why, or why they are bad. For the first time, the stress granule provides a user’s manual for understanding the biology of how tangles form.