Klein is satisfied with the treatment – even if, as the first patient received a dose well below the 11 patients to be treated. Moreover, none of these 12 patients received the full treatment. For security reasons, only part of their brain has been treated.“It ‘promising at this stage, but like everything else, the proof is in the pudding,” Freed WebMD. “Everything will depend on their clinical trial.”
The new treatment will not be practical until Freed and his colleagues are developing a cell line producing a large amount of fetal brain cells in the laboratory. That said, it’s just a matter of time.
It has been three years since Nathan Klein was the first to get an experimental gene therapy for Parkinson’s disease infused into his brain.
These encouraging results prove nothing, for notes. This will depend on a large clinical trial in which participants are aware of what treatment they receive, and that some participants do not receive active treatment (placebo). A test of this kind is being planned.
“My interest is to go beyond the use of fetal tissue and the use of tissue in the laboratory,” said Freed. “We could have that in patients over the next 2-5 years.”
Therapy created by During and Kaplitt attacks the gene of a chemical messenger, called GAD, a harmless virus. After injection in the STN through a fine needle, engineered the virus enters the brain cells and causes them to send signals TAG. It ‘a signal that tells the brain to calm down.
One year after treatment, nine of 12 patients showed an average improvement of 37% on a measure of the severity of Parkinson’s disease. Five patients were between 40% and an improvement of 65%.
Only 3% of these transplanted cells survive. But those that survive, Freed told the conference of Neuroscience, take root and grow – and continue to grow. If at least 40,000 cells survive, which means that patients no longer have to take the L-dopa in Parkinson’s largest.
The degeneration of the brain in Parkinson’s disease excites the brain called the subthalamic nucleus (STN). This leads to abnormal movements, tremor, rigidity and difficulty walking that make life difficult for Parkinson’s patients.
“These results are only dealing with one side of the brain, and, finally, allow us to face is:” During WebMD. “We hope our results will be equal to or greater than those observed with deep brain stimulation, even if we just match the effectiveness of deep brain stimulation, gene therapy could be more simple. No hardware, fewer adverse events and we have data suggesting that protects against disease progression., so that over time you will see a plus. ”
“Before the operation, I was a quivering mass of flesh,” Klein, 58, tells WebMD. “With my medication, I’m like 80% or 90% more. I’m at a point in time, but I knew I had Parkinson’s disease, you could not say.”
Released should know. Twenty years ago, he and others began to get interesting results when fetal brain cells implanted in the brains of people with Parkinson’s disease. These cells are transplanted to replace the dopamine-producing cells die in Parkinson’s disease.
During the reported results of a presentation at the 36th Annual Meeting of the Society for Neuroscience, held October 14 to 18 in Atlanta. While Michael G Kaplitt, MD, Ph.D., co-founder of Neurologix Inc., the company developing gene therapy. A pacemaker for the brain
“Nothing happened during the first week. And then nothing happened for the second week. And then, one month and two months, three months later, I thought I was a bit ‘better, nothing good,” said Klein . “It ‘was like watching grass grow. But six months later I started feeling much better.”
Until then, it’s just another promising treatment, says researcher Curt R. Parkinson Freed, MD, Director of the Division of Clinical Pharmacology and Toxicology at the University of Colorado Health Science Center.
“We are encouraged, the results seem to be maintained over time,” Durant said. “When we look at the other side of the brain, the side that does not treat, we always see a worsening of the disease. It is a progressive disease. If we had not done the treatment, we expect these patients to be significantly worse.” Really works ?
While Klein said that the problems of movement has about 40% more after treatment. Not all patients have done well. But those who received the higher dose tended to get the most improvement.
However, this treatment seems to work half as deep brain stimulation, the best treatment for Parkinson’s disease. It is safe, reports therapy co-developer Matthew J. Durant, MD, DSC, New York-Presbyterian Hospital and Weill Cornell Medical Center.