Parkinson’s disease occurs when the brain gradually stops producing the nerve-controlling chemical dopamine. Over time symptoms such as tremors, slow movement and stiffness get worse.
ProSavin, the new treatment, uses a “stripped-down” virus to transport dopamine-making genes into the brain. It is injected into a region called the striatum that helps control movement. Once the virus gets into the brain cells, it reprogrammes them to gradually start producing their own dopamine.
The study is at an early stage and has only been used to treat a small number of patients. More studies, involving hundreds of patients, are likely to be needed to confirm that the treatment is safe and effective. So, even if the trials progress as well as scientists hope, it is likely to be at least five years before this becomes a routine clinical treatment.
But the principle of creating medicine factories inside patients’ bodies is extremely exciting.
The side effects from current drug treatments often result from the high doses needed and their indiscriminate effects on the body. By injecting genes into particular cells – where the chemical is needed most – the doses are lower and the effects are localised.
Areas to think about:
- What other diseases may benefit from a similar approach?
- What are some of the potential risks of gene therapy?