including the testimony of Nathan Klein, the first patient to receive gene therapy. But these results as evidence of early treatment in 12 patients. It will take more clinical studies to demonstrate the treatment actually helps patients.No one has thrown their Parkinson’s. But patients who had less, even in ‘off’ state between doses of medication.
‘If you or a family member has Parkinson’s disease, and are interested to participate in research, gene therapy is a treatment that will now be directed to the subject,’ Kieburtz tells WebMD. ‘Gene therapy is on the fringe of integration. And ‘good for patients and their families to know the fate that now.’
‘There was no toxicity, no matter how you look,’ Kaplitt tells WebMD. ‘When we started, there were those who suggested that we could do great harm to patients. However, at higher doses we injected 35 billion of these particles in the brain, even a single patient had a fever.’
The result: The improvements were seen from the treated part of the body controlled by the brain. Improvement began three months after treatment and lasted for a year in most patients.
Gene therapy involves the injection of a genetically modified virus deep in the brain – in a region called the subthalamic nucleus or STN. For the PD, the STN becomes wildly overactive. The virus enters the cells of the brain and leads them to a chemical signal that soothes the STN.
‘The world is changing for patients with neurodegenerative diseases,’ Kaplitt says ‘If you have a disease at an early stage or even moderates in the course of their illness and other things that keep the promise down the line -. And not only for generations future, but for them in their illness. ‘
These are produced when fats and oils are chemically reduced. If you are listed at the top of the list of ingredients, the formulation will probably be difficult to tolerate.
‘Some patients are more dramatic, some less. After a year, 10 of 12 patients were better than they were in the [first],’ says Kaplitt.
Parkinson disease affects both sides of the brain.
But for reasons of safety, has insisted that regulators Kaplitt and colleagues to test gene therapy on one side.