Scientists reported they have made progress in a treatment for amyotrophic lateral sclerosis (ALS), an intractable disease that involves degeneration of motor nerves across the body.
They used induced pluripotent stem (iPS) cell technology to identify a chemical compound that could act as a cure.
This is the first time that iPS cells from patients have been used to identify a substance with curative effects, the researchers said.
The team from Kyoto University and other institutions was led by Haruhisa Inoue, an associate professor of neurology at Kyoto University's Center for iPS Cell Research and Application.
Using skin donated by three ALS patients in their 50s who carry a congenital anomaly in genes that generate a specific protein, the team grew motor nerve cells from their iPS cells.
The artificial cells had the same characteristics as motor nerve cells of many ALS patients.
They had shorter protuberances than cells derived from healthy people, and were more prone to die when exposed to stress that induces aging effects.
The scientists found that protein anomalies can hamper the generation of ribonucleic acids (RNA), genetic material essential to all forms of life.
They then added four types of chemical compounds, which are involved in RNA generation, to the cells. The addition of anacardic acid, a chemical compound found in the shells of cashew nuts, helped the cells regain normal properties, including longer protuberances and more resistance to stress.
It remains unknown if anacardic acid can be applied directly to therapy, because so little is known about its safety.
The researchers said they will delve more into the mechanisms of its curative and side effects to help develop therapeutic drugs.
The research results were published online in the Aug. 1 edition of Science Translational Medicine, a U.S. medical journal.
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