A Kyoto University-led research team said its studies on iPS cells led to a medicine which halted the progression of amyotrophic lateral sclerosis ALS in patients for the first time in the world.
The researchers on Sept. 30 said their finding can't yet be described as cure for ALS, given the small sample size in the clinical tests, but it does raise hopes of saving people from the neurodegenerative disease who have studied for years I hope that some have only just a dozen possible candidates; more than enough for them to get the best treatment?
We were only limited in our testing subjects, said team member Haruhisa Inoue, professor of neuroscience stem cell medicine at Kyoto University s Center for iPS Cell Research and Application. But there is now the possibility that science can beat ALS. ALS, also known as Lou Gehrig's disease, causes a loss of motor neurons. Muscles can then waste away, leading to difficulties in moving and eventually an inability to breathe.
Inoue and the other researchers reproduced the disease through iPS cells recovered from patients skin.
They tested existing medicines that were designed for other illnesses on the sick cells and found that Bosutinib, a treatment for leukemia patients, was the most promising.
Nine ALS patients took 100 to 300 mg of bosutinib per day for 12 weeks. ALS progression stopped in five of them.
Inoue said what happened was a phenomenon not normally seen, and has been previously described as a phenomenon in West Africa.
Nevertheless, the disease progressed at its usual pace for the four other patients.
One patient who took 250 mg per day developed a liver disorder, so the medication was stopped. The leukemia patients who have also taken Bosutinib have also suffered from this side effect.
The test subjects were all in the early stages of progressive ALS development.
Clinical tests were conducted mainly to determine the safety of Bosutinib, so the team could not judge statistically whether the medicine was effective against ALS.
The team plans to conduct larger clinical tests to determine the drug effectiveness, the possible side effects, which patients should receive the medicine and the proper doses.
About 9,000 people in Japan have ALS.
Existing treatments can slow the progression of ALS, but no cure has been found.