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GENE THERAPY TREATMENT GIVES 'ENCOURAGING' RESULTS AFTER VIRUS INJECTED INTO BRAIN RESEARCHERS may have made a breakthrough in treating Parkinson's disease with a gene therapy
that is injected directly into the brain of sufferers. Fifteen patients (12 in France and three in the UK) received injections of a modified virus that stimulated production of dopamine, a
natural substance that is known to help control movement. French team leader Prof Stéphane Palfi, a neurosurgeon at Henri-Mondor Hospital in Créteil and a researcher at CEA/Inserm, said that
early results showed that the treatment was "well tolerated" and produced "encouraging results, especially at higher doses". In addition, the enhanced production of
dopamine did not lead to any abnormal movements - a problem that is a severe side-effect of oral treatments with dopamine. However, Prof Palfi warned that progress would be slow as each
stage of progress had to be evaluated. Parkinson's is marked by loss of balance and severe tremors and the cause is linked to problems in the brain where neurones create dopamine.
Treating the disease with oral dopamine, L Dopa, has been used for nearly 50 years and has good early results but causes severe side-effects. Abnormal movements, including sufferers hitting
themselves and others, become increasingly common and are as disturbing as the original problem. The new treatment, which is also being tested at Addenbrooke’s Hospital in Cambridge, is made
by British company Oxford BioMedica and uses a modified virus carrying three genes to stimulate dopamine production. The aim is to produce dopamine in a continuous and measured way.
Patients received different doses of the treatment and Prof Palfi reported to the Congress of the European Society of Gene and Cell Therapy in Versailles that those on the higher dosage were
responding well. Oxford BioMedica says that the ProSavin gene therapy gives up to 61% improvement in motor control.