Drug successfully slows progression of Charcot disease in clinical trials in Kyoto

Presentation of the results of the experiments at Kyoto University on June 12.

A Japanese team announced Wednesday that a drug treating leukemia succeeded in slowing the progression of Charcot’s disease in at least 13 of 26 patients who participated in the clinical trial.

This result was obtained during the second phase of testing of the product called “Bosutinib”, carried out among others by scientists from the iPS Cell Research and Application Center at Kyoto University.

Charcot disease, or amyotrophic lateral sclerosis (ALS) is a currently incurable pathology which causes progressive paralysis of all members of the body up to the respiratory muscles. Around 10,000 people suffer from it in Japan.

Bosutinib had been identified as a drug useful for the study of iPS (induced pluripotent stem cells). In 2017, the team discovered its effectiveness in stopping nerve cell death during research using iPS nerve cells created from cells from ALS patients.

iPS cell technology, developed by Professor Yamanaka Shinya in 2006 (and awarded the Nobel Prize in Medicine six years later), involves taking cells from an adult from a tiny sample of skin or blood to reprogram and cultivate them to create an unlimited supply of cells capable of differentiating and generating cells specific to tissues such as those of muscles, bones, heart, liver, blood vessels and nerves . It is one of the major hopes for treating diseases that are still incurable to date.

(Extract from an article from Kyodo News, published in French by Nippon.com)

See the article in Japanese on the Kyodo News website