Successful screening of drug candidates for deadly brain diseases using worm models

  Recently, researchers at the University of Liverpool have successfully built a worm model of an incurable and fatal neurodegenerative disease, and used this worm model to screen a potential compound that can treat the disease.

  In Europe and North America, 1 in 100,000 people will suffer from neuronal ceroid lipofuscinosis (ANCL). ANCL usually develops in the 30s and causes death in the 40s. Although it has recently been identified that this disease is caused by mutations in the DNAJC5 gene, there is currently no known means to treat this disease.

  This is the first time that scientists have successfully replicated the disease in worms and successfully used a compound resveratrol to treat the disease. Since the worm's lifespan is usually only a few weeks, the symptoms of the disease will appear within a few days, which opens up the possibility of testing thousands of new compounds for the treatment of the disease in a short period of time.

  A compound resveratrol has been proven by the research team to be effective in treating diseases, and it works in an unknown way. Professor Alan Morgan, a physiologist who led the study, said: ANCL is rare and currently it is incurable. This research allows us to quickly test compounds that can be used for treatment. Among the first compounds tested using our model, one of them (resveratrol) has shown encouraging effects.

  Professor Morgan and his colleague Professor Bob Burgoyne, PhD student Sudhanva Kashyap now plan to use the worm model to test more compounds and explore how worms can be used to study a variety of neurodegenerative diseases.