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.
Parkinson’s Disease Stopped in Animal Model: Molecular “Tweezers” Break Up Toxic Aggregations of Proteins
In an article from ScienceDaily, scientists have found a way to stop Parkinson’s disease in animals. Scientists at UCLA have successfully prevented a protein, a-synuclein, from “binding together and forming clumps, stopped their toxicity, and also break up existing clumps.” This is a huge breakthrough because a-synuclein is a common protein in all patients with Parkinson’s disease. In addition, when this protein binds in clumps, becomes toxic, and eventually kills brain neurons; it is thought to be a pathway to Parkinson’s disease.
Scientists have developed a molecular tweezer, known as CLRO1, that is able to prevent a-synuclein from becoming toxic and causing brain damage. An additional upside of the molecular tweezer is that it does not show any negative side effects to normal functioning brain cells. Testing for this research has been done in zebrafish with posititve outcomes, in which studies are now being done in mice models with hopes of leading to human clinical trials.
There has been a between a fatal neurological disease in Tibetan Terrier dog and a heredity of Parkinson’s disease in humans. The findings can help lead to a treatment in early on-set Parkinson’s disease. Scientists studied a Tibetan Terrier named Topper. He had to be euthanize after behavioral changes and on set seizures. Researchers from the University of Missouri, took the DNA a from the dog to identify gene mutations that triggered the neuronal ceroid-lipofuscinosis in Topper. Hopefully their finds can help to deplete this genetic mutation from the species of canine and also help to discover treatments for Parkinson’s disease.
Wow! who knew that man’s best friend was more than just a lovable creacher. Now, Toto can save the life of Dorothy( Wizard of OZ).
An article in Lancet Neurology, claims that scientists have devised a way to potentially treat Parkinson’s patients using gene therapy. The trial involved drilling a hole into the skulls of Parkinson’s patients and introducing a virus which carries billions of copies of a gene that was designed to make the brain produce more GABA. The study shows mild statistical success: 23% improved who got the treatment, while 13% improved who got a fake surgery and treatment. While one of the patients testified that he could not move before the treatment and now he is playing jazz and travelling, the success could be attributed to the similar effects of deep brain stimulation (also a Parkinson’s treatment). Additionally, the study was funded by the biotech company that invented the procedure. It was also only performed on 37 people. More research is clearly needed.
This article discusses a clinical trial in which patients with Parkinson’s Disease who were injected with the GAD (glutamic acid decarboxylase) gene improved in their motor functioning. The GAD gene was injected directly into their brain and the enzyme produces GABA, a chemical involved in motor coordination that is low in individuals with PD. This was a double-blind clinical trial that involved 22 individuals who received the gene therapy, and 23 individuals whom did not (control). Improvements in motor functioning were assessed on all participants using the UPDRS (Unified Parkinson’s Disease Rating Scale) up to six months after treatment.
With further clinical trials, this form of gene therapy will hopefully improve the lives of individuals with Parkinson’s disease who do not benefit from drugs.
“The major common genetic variants for Parkinson’s have been found,” said Nick Wood, a professor at the Institute of Neurology at University College London, one of the researchers who led the study. “We haven’t put together all the pieces of the puzzle yet, but we’re not that far off,” he said.
Scientists analyzed genetic samples from more than 12,000 people with the disease and more than 21,000 of the general population of Europe and the U.S. The highest number of mutations were in the eleven genes linked to Parkinson’s disease are two and half times more likely to develop the disease then people with less mutations.
Scientists say identifying Parkinson’s disease could help explain what sparks the disease and have hope that it will lead to new treatments.
“There is good reason for optimism that these advances will be translated into direct benefits for our patients,” wrote Christine Klein and Andreas Ziegler of the University of Lubeck in Germany.—