Local Doctor Leads Study Of Gene Therapy Treatment For Parkinson’s Patients

PITTSBURGH (KDKA) – When part of the brain is no longer working properly, would it be possible to put something in to boost function?

Neurosurgeon Dr. Mark Richardson is trying to find out.

“What we’re trying to do with this study is to replace an enzyme that’s lost as cells degenerate in Parkinson’s disease,” Dr. Richardson said. “The enzyme helps the brain make dopamine.”

The brain chemical dopamine is important to keeping movements smooth. The problem in Parkinson’s disease is the lack of dopamine because of worn out brain cells, and you end up with shaking, stiffness, and slowness of movement.

People can take medicine for Parkinson’s disease, but there can be symptom fluctuations and at higher doses, side effects.

“Typically in Parkinson’s disease, these symptoms kind of go up and down like this, and they can be masked very well by medication, but unfortunately what tends to happen for all of these patients is progression to more of a roller coaster ride of ups and downs during the day,” Dr. Richardson said.

Dr. Richardson is leading part of a study, first funded by the Michael J. Fox Foundation and now by a biotherapy company, to see whether inserting a gene into a specific part of the brain will be the on switch for more dopamine production.

“The idea of brain surgery for a chronic disease is very different than continuing to take medication,” Dr. Richardson said.

The gene is delivered into the brain through the skull by a thin tube and carried by a virus

“The idea of a virus probably sounds very scary to some people. But, this virus cannot reproduce,” Dr. Richardson said. “It can insert itself into a cell, and it can only do one thing there. It can release the gene to allow this enzyme to be made.”

Dr. Richardson and the lead investigator in San Francisco are looking for 20 patients to participate. They will be followed for three years, and their need for medication will be evaluated and compared before and after.

To qualify you have to be between 40 and 70 and on certain medicines for Parkinson’s disease for at least three years with increasing fluctuations in movement.

Dr. Richardson hopes gene therapy leads to smoother days and fewer symptoms.

“If we can show this is a small group of patients, the trial will be expanded,” Dr. Richardson said. “With a little bit of luck, within the next decade, we will see a gene therapy accepted as a proven and viable treatment option.”

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