Repurposing

Experimental Cancer Drug to Help Fight Parkinson's Disease

Sergey Kolenov, N+1

Scientists have discovered that the anti-cancer drug tasquinimod, which has not yet reached pharmacies, restores the work of genes that are turned off in the early stages of Parkinson's disease. So far, experiments have been conducted only in the laboratory on cell culture, but scientists intend to determine how the drug acts on patients.

At the first stage of the study, scientists took samples of skin cells from patients with a rare hereditary form of Parkinson's disease and turned them into stem cells. Then neurons were grown from them and their development was compared with the neurons of healthy people. The analysis showed that the first deviations associated with Parkinson's disease are associated with the loss of activity of a number of important genes. It would be impossible to track these changes on living patients.

The authors suggested that the drug tasquinimod, created to fight prostate cancer and not yet on the market, can return disconnected genes to their original state by interacting with proteins inside cells.

Tests on neurons of patients with a genetic form of parkinsonism confirmed this idea.

The drug was then tested on cell cultures obtained from patients with a more common non-genetic form of the disease. Initial results were mixed: tasquinimod was effective only if the pattern of inactive genes coincided with that for the genetic form of Parkinson's disease.

The team plans to continue research to learn how to identify patients suffering from Parkinson's disease who will be able to benefit from treatment with tasquinimod. According to the researchers, their work highlights an important trend in repurposing drugs for new uses. This is a cheaper and faster approach than developing drugs from scratch.

In Japan, stem cells were used for the first time to treat Parkinson's disease. An experimental operation was performed on a 50-year-old patient who will remain under observation for the next few years. In addition to him, six more volunteers will take part in the tests.

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