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Amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig's disease, can be treated with a drug already used in Parkinson's disease, a research team led by a professor at Keio University, discovered through induced pluripotent stem cell technology.
Hideyuki Okano, from the Department of Physiology at the University's Graduate School of Medicine, announced the discovery at a symposium on Regenerative Medicine Techniques.
The Okano team discovered the effectiveness of the ALS drug using "drug discovery and development" methods, which include experiments on iPS cells.
Okano said at a meeting in Tokyo on Oct. 13, that the new approach could probably help stop the progression of ALS, or even provide a cure.
ALS is an incurable disease in which nerve damage causes the patient to gradually lose control of his muscles, including those of his breathing, and gradually become completely paralyzed. It is estimated that there are more than 9,000 ALS patients in Japan.
There are medications that can slow the progression of the disease, but effective treatment remains elusive to this day.
The team mimicked the condition of patients with ALS using iPS cells based on cells taken from ALS patients for whom the disease affects the family. They tried about 1,230 types of drugs, which led to the discovery that ropinirole hydrochloride, a drug for Parkinson's disease, had an effect.
They conducted the same experiment using cell-based iPS cells harvested from ALS patients with no parent with ALS.
The results revealed that ropinirole hydrochloride was also effective for 16 of the 22 types of sporadic ALS.
The cause of ALS is unknown, but it is thought that a specific protein that accumulates in the nerve cells of patients and other factors is at the origin of the disease.
It has been confirmed that ropinirole hydrochloride is effective in slowing the accumulation of protein and in conserving more cells.
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