The prospect of treating Parkinson's by gene therapy

British scientists believe that the gene therapy they just developed may open up hope to cure the agonist syndrome - Parkinson's disease, which is affecting 4.1 million patients globally and is expected to increase. Sheila Roy, one of 15 Parkinson's patients in the world who participated in a new therapy test, could write for the first time in 15 years.

Mrs. Roy had Parkinson's disease when she was 40 years old and had been fighting for the disease for 17 years. Parkinson's disease occurs when the brain slowly stops producing dopamine, an essential substance in controlling movement. Lack of dopamine can lead to symptoms such as shaking hands and feet, slow movements, poor balance . leading to Parkinson's. In addition to physiological problems, Parkinson's patients also suffer from psychological disorders and depression, which greatly affect their lives.

Thanks to gene therapy, Sheila Roy can write, draw like before

New therapy called ProSavin, developed by Oxford BioMedica, uses a weakened virus that brings dopamine-producing genes to the brain. It is implanted in the striatum - the brain's motor nerve center. When the virus enters the brain cell, it reprograms the cells, causing them to start producing dopamine. This testing process is being carried out at Addenbrooke's Hospital in Cambridge (England) and Henri Mondor Hospital in Paris (France). Dr. Philip Buttery of Cambridge Brain Recovery Center said that despite being at an early stage, this method brought many positive results. 'It brings about an overall beneficial effect, helps the patient's life more pleasant, can allow reducing the dose of the drug and in some patients, the quality of sleep and life are improved' , Dr. Buttery said.

The method of suppressing Parkinson's with drugs often causes many side effects for the body due to high doses. Meanwhile, introducing genes into brain cells can be done at low doses and its effects are more concentrated. Ms. Roy and five other patients in the UK were treated with new gene therapy at high doses. After 3 months, the test results showed that their motor function improved by 29%. Mrs. Roy said: 'ProSavin helped me regain confidence, allowing me to move better and open up hope. I can do everyday tasks and for the first time in 15 years, I can write words' . She still has to take medicine and doctors still monitor the level of dopamine production in her brain.

Because it is only at an early stage, ProSavin therapy is only used to treat a few patients. In order to confirm its safety and effectiveness, scientists need to carry out many studies with the participation of hundreds of people. If the trial process goes well, ProSavin can become a clinical treatment in hospitals for at least another 5 years.