Kyoto – A drug discovered through a method using induced pluripotent stem cells, or iPS cells, from sufferers of amyotrophic lateral sclerosis (ALS) was effective in stopping the progression of the neurological disease in five out of nine patients in a clinical trial, a research team at Kyoto University said Thursday.
While drugs that slow down the progression of amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, have been used in the past, this is the first time that a chronic myeloid leukemia drug called bosutinib has been found to stop its progression, according to the team.
Haruhisa Inoue, a professor of neurology at Kyoto University leading the team, said that larger clinical trials will be needed to determine if the drug can be put to practical use, but “We are now looking at the possibility of being able to control ALS with the power of science.”
The team reproduced the disease by culturing iPS cells derived from the skin of patients into motor neurons. They then tested a series of drug compounds on the cells to find that bosutinib was effective in slowing down the progression of ALS.
The drug was administered for around three months to nine patients who were in the early stages of the disease and showing signs of deterioration. Progression of the disease stopped in five patients during the treatment period, while four patients continued to deteriorate at the same pace as before.
A comparison of the blood samples from both sets of patients showed that they had differing amounts of a protein unique to nerve cells before the drug was administered. Researchers plan to conduct clinical trials with larger groups while adjusting the dosage and other conditions in the future.
There are about 9,000 people in Japan who suffer from ALS. The disease causes motor neurons to progressively die, resulting in paralysis, with many patients requiring a ventilator to stay alive. Its exact cause is unknown, and no fundamental treatments have been established.
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