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Parkinson's disease drug proves effective against ALS in Japan research using iPS cells

This Feb. 2, 2019 file photo shows professor Hideyuki Okano, left, giving a presentation on the progress of his research before ALS patients and others. (Mainichi/Suzuko Araki)

TOKYO -- A Parkinson's disease drug has been confirmed effective in treating amyotrophic lateral sclerosis (ALS), a disease causing gradual loss of muscle strength throughout the body, according to a Japanese research team's findings announced May 20.

    In a clinical trial using induced pluripotent stem (iPS) cells derived from patients, the team led by Hideyuki Okano, a professor of physiology at Keio University, was able to slow down the disease's progression. The researchers say it could become a new treatment option for ALS, for which there are few effective treatments, and they aim to apply as soon as possible for its approval as a therapeutic drug.

    By using iPS cells made from cells donated by patients with intractable diseases, it is possible to recreate disease conditions in test tubes and investigate the efficacy of various drug candidates. For this reason, iPS cells are promising not only for use in regenerative medicine but also in the development of new drugs. The team says that this is the first time in the world that a drug discovery trial using iPS cells has been able to confirm the efficacy of a therapeutic drug.

    ALS is an intractable disease that causes gradual loss of motor nerve cells transmitting commands from the brain to muscles throughout the body; as it progresses, even breathing becomes difficult. About 10,000 ALS patients live in Japan; the disease has no permanent cure.

    The Parkinson's disease tablets confirmed effective in the clinical trial were ropinirole hydrochloride extended-release tablets (marketed as ReQuip CR Tablets). The team has been conducting research using iPS cells to understand the cause of the disease and develop new drugs.

    Using motor nerve cells generated from patient-derived iPS cells, they reproduced the disease conditions of ALS and added 1,232 types of existing drugs to the cells, through which they found that ropinirole hydrochloride was effective in suppressing cell death.

    In the cell experiments, they reportedly confirmed the drug was two to three times more potent than existing ALS drugs in improving disease conditions, and claim it is believed to be effective in about 70% of ALS patients.

    The clinical trial was conducted on 20 patients whose disease had developed within five years and who were still able to independently do activities such as walking because their symptoms had yet to reach an advanced stage. Thirteen of the patients took ropinirole hydrochloride tablets for a year, seven took a placebo for the first six months and ropinirole hydrochloride tablets for the remaining six months; the drugs' safety and efficacy were then compared.

    The results showed that the 13 patients who took ropinirole hydrochloride tablets for a year showed less decline in activity and multiple muscle strength six months after the start of medication than the remaining seven who only took the tablets for six months. They also showed less decline in motor function across the entire period, delaying the disease's progression by about seven months.

    The study also found early medication was important, and taking the drug for longer periods of time was more effective. Its safety was also confirmed.

    Professor Okano said, "We were able to see a good response in the treatment of ALS. I would like to work toward the goal of making ALS no longer a lethal disease as soon as possible."

    (Japanese original by Ayumu Iwasaki, Science & Environment News Department)

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