Title:Cutting-Edge Alzheimer’s and Parkinson’s Research

Lights, Camera, Brain Surgery

Nearly 20 years ago, when Ray Farkas began showing symptoms of Parkinson’s disease— a declining tennis game, shaking limbs, balance problems—the TV producer and documentary filmmaker researched his options. Having seen his own father become debilitated and die from the disease, he opted for a (then) new procedure at MedStar Georgetown University Hospital called deep brain stimulation (DBS), surgery involving the precise placement of something akin to a pace maker in the brain. A natural storyteller, he also decided that the operation would make great television, and could raise awareness about the procedure to help others suffering from Parkinson’s.

The successful operation took place in 2003, as the patient-documentarian (who was awake for the procedure) narrated the experience on film, with five cameras in the operating room capturing every angle. It was a complicated production, but through the process he became close with his medical team.

Along with this unconventional approach to his own brain surgery, Farkas brought a sense of humor, and even a theme song, to what he liked to call his documentary-movie-musical: “I Need This Operation Like I Need a Hole in the Head.”

After the surgery he regained his mobility, and continued to work with the MedStar Georgetown neurology team to monitor his care. He became a national advocate for DBS, appearing on Oprah and in PeopleMagazine to share his incredible story. He died of colon cancer in 2008.

Advancing Science and Hope

When his daughter Julie Farkas learned of a new Parkinson’s study underway at Georgetown University, led by Fernando Pagan, M.D., her father’s neurologist, and scientist Charbel Moussa, MBBS, Ph.D., she and her husband Seth Goldman chose to continue her father’s legacy by supporting the research.

Dr. Charbel Moussa and Dr. Fernando Pagan bridge cutting-edge laboratory research with state-of-the-art patient care. 

Julie Farkas views the gift as a natural way to support the personal connection and gratitude her family feels for the neurology team, who helped her father take advantage of cutting-edge science, and who cared for him with dignity and warmth.

“…that neurology team helped him live the kind of life he wanted to live almost until the end.”

“We’re so grateful to MedStar Georgetown as an institution and the neurology department in particular for giving my father his life back,” she says. “Before the surgery, he’d gotten to a point in his Parkinson’s where he would suddenly freeze up. He became self-conscious, did not want to go outside; he almost stopped working. After his surgery he returned to the vibrant, fun-loving dad I knew. I’m appreciative that he found the surgeon willing to treat his Parkinson’s in the early stage, and the ongoing care he got from Dr. Pagan—we couldn’t have asked for anything better. My father died at 71, premature for sure, from the cancer. But that neurology team helped him live the kind of life he wanted to live almost until the end.”

Connecting Research and Patient Care

The phase II clinical trial for Parkinson’s is one of two Georgetown University studies examining new applications of nilotinib, a cancer drug FDA-approved to treat leukemia. The second study examines nilotinib in patients with mild-to-moderate Alzheimer’s. Both trials opened in 2017.

Moussa is credited with discovering the drug’s potential for treating certain neurodegenerative diseases. The effect of nilotinib appears to involve a process known as autophagy, a mechanism that clears away neurotoxic proteins associated with Parkinson’s and Alzheimer’s diseases.

“This understanding represents the newest frontier of neurotherapeutic research and changes the paradigm on how we think about neurodegenerative diseases,” explains Moussa.

“Philanthropy propelled our research for some of the most debilitating diseases.”

By bridging cutting-edge laboratory research with state-of-the- art clinical patient care, Georgetown is uniquely positioned to translate discoveries like this one into potential new treatments for individuals affected by neurodegenerative disease.

“I am pleased to offer this study to my patients,”adds Pagan. “It demonstrates the importance of teaming Parkinson’s care with academic research. Only through clinical trials will we be able to move the field forward so that we can offer better treatments to our patients in the future.”

From the beginning, philanthropy has made the study possible. Two $25,000 seed grants in 2011 helped Moussa expand his research, which led to the nilotinib discovery.

“Philanthropy propelled our research for some of the most debilitating diseases,”Moussa says.

Julie Farkas is grateful to join her father’s creative, courageous, hard-fought effort to help move the science forward.

“If there’s something I can do to help others not suffer like my family did, and help others benefit from advancing the science, so they can live longer with a higher quality of life, I want to do that,”shares Farkas. “That’s what DBS did for my father. It’s wonderful to give to an institution and know it’s going to great use.”

Georgetown University holds an issued U.S. patent on the use of nilotinib for the treatment of certain neurodegenerative diseases and has other pending patent applications in U.S. and foreign jurisdictions. Dr. Charbel Moussa is the named inventor of the intellectual property protected. Earlier this year, the university licensed the intellectual property to a company for further development and commercialization.

Donor Julie Farkas gets a hug from her dad at Dulles Airport in 1989. After he passed away, she continued to support the Georgetown medical team who had treated him.