Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease that leads to progressive loss of muscle function and ultimately death. There is currently no cure for ALS, and treatments are focused on alleviating symptoms and providing supportive care. However, recently, researchers from Japan have discovered that the Parkinson’s disease drug ropinirole can delay the progression of ALS in early clinical trials.
The study was published in the journal Cell Stem Cell, where the team showed that ropinirole was safe to use in patients with sporadic ALS and potentially had a therapeutic effect, delaying the disease progression by an average of 27.9 weeks. However, the study was conducted on a small cohort of 20 patients, and more trials are needed to confirm the effectiveness of the drug.
Scientists have been searching for treatments to cure ALS for years. This neurodegenerative disease is caused by the gradual degeneration and death of motor neurons that control muscle movement. As a result, muscles gradually weaken, leading to difficulty in walking, talking, and even breathing. Eventually, patients become paralyzed and rely on artificial ventilation to survive.
Current treatments, such as riluzole and edaravone, aim to slow down the progression of the disease. However, none of these treatments can cure the disease.
The study found that patients who received ropinirole during both phases of the trial were more physically active than the placebo group. They also showed slower rates of decline in mobility, muscle strength, and lung function, and they were more likely to survive. However, patients who started taking ropinirole halfway through the trial did not experience these improvements, which suggests that ropinirole treatment may only be effective if treatment is started earlier and administered over a longer duration.
More interestingly, the researchers found that the motor neurons grown from ALS patients had distinct differences in structure, gene expression, and metabolite concentrations compared to healthy motor neurons. But, ropinirole treatment reduced these differences. Furthermore, the researchers identified 29 genes related to cholesterol synthesis that tended to be upregulated in motor neurons from ALS patients, but ropinirole treatment suppressed their gene expressions over time. The team also identified lipid peroxide as a good surrogate marker for estimating the effect of ropinirole both in vitro and clinically.
Through the study, the researchers found that motor neurons from ALS patients showed a very striking correlation between a patient’s clinical response and the response of their motor neurons in vitro. This correlation suggests that this method could be used clinically to predict how effective the drug would be for a given patient.
ALS is a complex disease that involves multiple factors, including both genetic and environmental factors. Scientists believe that different patients may respond differently to drugs like ropinirole due to genetic differences. The team hopes to pinpoint these genetic differences in future studies.
In conclusion, the study has shown promising results in early clinical trials. Ropinirole has shown to be safe to use in ALS patients and potentially delaying the disease progression. However, more trials are needed to confirm its effectiveness. The study has also shed light on the importance of growing and testing motor neurons from patient-derived induced pluripotent stem cells, which could be used clinically to predict how effective the drug would be for a given patient.
