First ever study for Parkinson’s in human’s successful

The first ever clinical trial conducted on Parkinson struck people provided a ray of hope to the diseased as the new gene therapy is safe and effective in reducing the motor symptoms of the disease, as stated by scientists in the 23 June issue of the British medical journal, Lancet.

Michael Kaplitt, a professor and director of movement disorders surgery at NewYork-Presbyterian Hospital/Weill Cornell Medical Center in New York, led the study involving 11 males and 1 female suffering with advanced Parkinson's symptoms and had lived with the disease for an average of eight to 10 years. The technique is also known to have been effective in rats, as reported in Science five years ago.

The scientists injected a gene, tucked in a disabled cold virus, in one side of the brain (as a precautionary measure because if something went wrong at least half of the brain would work and also for after treatment comparison) in order to calm activity in the hyperactive sub thalamic nucleus and other brain parts. In the technique, a surgeon makes a quarter-sized hole in the brain, inserts a tube which has the width of a human hair and injects a drop of fluid holding 3.5 billion to 35 billion genes that are contained within a harmless virus, known as a viral vector. The genes are called glutamic acid decarboylase. The GAD genes produce GABA, a neurotransmitter that helps quiet overactive neuronal firing in the brain.

After about three months of the treatment the symptoms like tremors, stiffness and trouble walking started improving by an average of 25% and lasted for up to a year and even four years in some. According to the motor function test called the Unified Parkinson's Disease Rating Scale (UPDRS), five patients showed a recovery of 40-65%.

The study funded by Neurologix intended to test the safety of the gene also discovered unanticipated results of the improvement of symptoms without any side effects. It led a pathway for further research in this field, according to scientists.

"We still have quite a bit more testing to do," said Dr. Kaplitt. Still, "the initial results are extremely encouraging."

Scientists including Deutch mentioned the need for new methods of treatment. Drugs often used for treatment are effective but are known to cause a lot of side effects. Two anti-Parkinson drugs, named Permax and Dostinex were found to be at greater risk of developing the same kind of heart damage that led to the removal of the diet drug combination "fen-phen”. The drugs, Pergolide, developed by Eli Lilly & Co. and sold under the brand name Permax and Cabergoline, developed by Pfizer Inc. and sold under the brand Dostinex were found to carry serious risks of damaging heart valves.

The U.S. Food and Drug Administration had announced approval of Neupro, a skin patch designed to treat symptoms of early Parkinson's disease, containing the drug rotigotine in May.

In an earlier study, Spanish scientists treated Parkinson's disease symptoms in monkeys by implanting dopamine generators into the animals' brain cells.

About 1.5 million people in the United States have Parkinson's, with the disease afflicting mostly people over the age of 65. The disease is usually seen in up to 15% of those aged 65–74, and almost 30% of those aged 75–84 occurring mostly in men.

First noted by British physician James Parkinson in the early 1800s, Parkinson’s disease is an incurable progressive movement disorder that occurs when the muscle movement controlling cells of the brain stop functioning for unknown reasons (neurons that make a chemical called dopamine which sends signals for movement coordination die or do not work properly). The person’s ability to control his movement initiation, speed and smoothness is affected. They may have trouble walking, talking or doing simple tasks. They may also have problems such as depression, sleep problems or trouble chewing, swallowing or speaking.

Also called Paralysis agitans or Shaking palsy, the disease is caused when brain cells called substantia nigra that produce dopamine, one of the movement control centers of the brain, die off. Exposure to pesticides and herbicide is strongly linked with disease risk.

Symptoms of PD may include

• Trembling of hands, arms, legs, jaw and face
• Stiffness of the arms, legs and trunk
• Slowness of movement
• Poor balance and coordination

A study conducted in April showed startling results that smoking cigarettes and drinking coffee may protect people from Parkinson's disease but scientists and doctors have not suggested people do so.