Pitt to study Parkinson’s

The Michael J. Fox Foundation for Parkinson’s Research recently granted RheoGene Inc. $4.2… The Michael J. Fox Foundation for Parkinson’s Research recently granted RheoGene Inc. $4.2 million towards gene therapy treatment of Parkinson’s disease.

RheoGene, owned by the University of Pittsburgh Medical Center, is working to advance cell and gene therapy approaches in hopes to regulate Parkinson’s disease and other debilitating diseases.

Dr. Mark Braughler, vice president of therapeutics at RheoGene, said that the company was developed in 2002 specifically for the purpose of developing gene regulation therapy. Braughler said that one of the challenges in gene therapy is replacing a gene.

“We’re developing a regulated gene therapy … that can be turned on and off and the level of gene expression can be customized for each patient,” Braughler said.

He said that there are about a million diagnosed cases of Parkinson’s disease in the United States and that the disease in itself is very complicated and hard to control.

Braughler said that Parkinson’s disease is caused by the loss of the ability of neurons to produce dopamine – a neurotransmitter involved with movement. The disease affects the central nervous system and the tremors that are associated with the disease are caused by this loss of dopamine.

Braughler said that currently there is no cure for Parkinson’s disease, but gene therapy could prevent its progression.

The Michael J. Fox Foundation for Parkinson’s Research was founded in 2000, two years after the actor publicly disclosed that he had been diagnosed with the disease. According to the foundation’s Web site, Fox shifted much of his energy into raising awareness about the disease and finding a cure for it.

Joyce Oberdorf, a spokeswoman for the Michael J. Fox Foundation, said that RheoGene applied for a grant under a program called LEAPS (Linked Efforts to Accelerate Parkinson’s Solutions).

She said the program is designed to accelerate research that is innovative and can quickly go into the clinic for patients.

“The review committee selected this grant as one of the top grants because it uses a novel technology that would have implications not just for Parkinson’s disease but for [many] diseases,” Oberdorf said.

The Michael J. Fox Foundation granted $17 million dollars in 2005. She said that grants received by various institutions range from $200,000 to their largest grant of more than $5 million.

Braughler said that there are two parts to the RheoGene. One is a gene component and the other is a gene switch. He said that every day the patient will take an activator drug that would turn the gene on until the time when the gene is turned off.

“At this point in time, we feel very confident that the activator drug is quite safe,” Braughler said. “One of the things we’ll be doing with the Michael J. Fox grant is to develop that activator drug.”

Braughler said they anticipate that during the next four years, they will bring this gene therapy to the point where they can begin clinical trials with it. Within the next seven to 10 years, the treatment could be made available to all Parkinson’s disease patients.