Parkinson’s disease treated with gene therapy

CBS News and the Huffington Post both recently reported that a British woman suffering from Parkinson’s disease has been successfully treated with gene therapy. Gene therapy involves injecting normal functioning genes into cells where the dysfunctional genes reside with the hope of treating the disorder associated with the dysfunctional genes. Gene therapy has had many failures in the past, making it a very unpopular and highly discredited form of disease treatment. However, the therapy has proven effective in treating Sheila Roy, a woman who has suffered from Parkinson’s disease for the past 17 years. Doctors injected ProSavin into Ms. Roy’s brain, delivering genes that code for three enzymes which aid in allowing nerve endings in the brain to produce the neurotransmitter dopamine. Dopamine is lacking in people with Parkinson’s disease because the disease destroys nerve endings which produce the neurotransmitter. After less than a year of treatment, Sheila is now able to perform such activities as writing, something she has been unable to do for 15 years. She should not need any more ProSavin injections but will need to undergo regular brain scans to monitor how much dopamine her nerve endings are producing. While she is not fully cured of her Parkinson’s, Sheila has more control over her movements than ever before, proving that gene therapy can be an effective form of treatment for patients with Parkinson’s disease. According to the Parkinson’s Disease Foundation, one million Americans suffer from Parkinson’s with 60,000 diagnosed every year. Worldwide the number of people with Parkinson’s is estimated to be 6 to 7 million. Gene therapy treatment could help patients reduce their daily medication dosage, allow them to get more sleep at night, and give them a better quality of daily life overall. This case proves that gene therapy cannot yet be excluded as a possible form of disease treatment.