New candidate drug 'dials down' protein synthesis for therapy

In preclinical trials in animals, a new candidate drug called Sephin1 can markedly improve CMT and familial amyotrophic lateral sclerosis (ALS), two diseases of proteostasis (protein homeostasis, the process keeping protein production in balance in cells). Sephin1 regulates a key factor in protein synthesis, and does so by maintaining phosphorylation, or the addition of a phosphate group.
“The finding is important because proteostasis diseases are multiple and affect many people,” said Wrabetz, noting that they include neurodegenerative conditions, such as Alzheimer’s and Parkinson’s, demyelinating diseases such as multiple sclerosis and certain types of cancers and some subtypes of diabetes.
“It’s important to emphasize that further studies are necessary to confirm that the effects in these two animal models will translate to patients with CMT and familial ALS and then, that this candidate drug or similar drugs could be useful in other diseases where proteostasis is a factor,” Wrabetz explained. “Nonetheless, this study is an important first step toward developing a therapeutic strategy for these diseases with a candidate drug that could potentially be used in clinical trials.”