NYSCF Hosts Panel Discussion on Parkinson’s Disease Research and Treatment

Years ago, a physician in her sixties started to develop a tremor. She sought treatment from Dr. Susan Bressman, who diagnosed her with Parkinson’s disease. The patient’s grandfather had Parkinson’s, and it seemed likely that the disease was the result of a genetic mutation passed down from her mother’s side of the family.

“Back then, people didn’t want to know if they had a Parkinson’s mutation, ” Dr. Bressman, who is a Professor of Neurology and Mirken Chair at the Icahn School of Medicine at Mount Sinai, told the audience at a discussion on Parkinson’s disease research and treatments hosted at the NYSCF Research Institute this week. “There weren’t any treatments that targeted patients with specific mutations, and patients would just worry about whether or not to tell their children that they might be at risk too.”

But Dr. Bressman’s patient wanted to know if her genetics played a role in her disease, so she took the test. Her results came back positive for a mutation in a Parkinson’s-associated gene called LRRK2. The patient then told her children about her diagnosis, that it was genetic, and that they too were at risk. None of them decided to get tested – perhaps, as Dr. Bressman speculated, because nothing could have been done about it at the time.

“Now the tide has shifted: we’ve gone from having information we didn’t know what to do with to having actionable items,” Dr. Bressman explained. “If a patient comes to me with a genetic form of Parkinson’s, we can get them into one of several clinical trials that target their genetic form of the disease. And if their kids test positive for the mutation, we can try to get them into trials that can maybe even halt the disease before it begins. It’s an exciting time.”

Attendees also heard from Dr. Gist Croft, who recently joined NYSCF as a Senior Principal Investigator in Parkinson’s disease and Neurodegeneration. Dr. Croft underscored the need for studying Parkinson’s disease in human cells rather than animal models, pointing out the powerful stem cell technologies NYSCF has developed that makes this possible.

“Mouse brains are one hundred times smaller than human brains with one thousand times fewer neurons. Additionally, mice don’t get Parkinson’s—we have to induce it experimentally,” Dr. Croft explained. “We need to study the disease in human cells to understand how it affects human patients. With the NYSCF Global Stem Cell Array, we can generate stem cells from our diverse cohort of Parkinson’s patients, turn them into the neurons affected by Parkinson’s, and use these cells to study the disease’s pathology and develop treatments.”

NYSCF Chief Medical Officer Dr. Melissa Nirenberg then joined Drs. Bressman and Croft to chair a panel discussion and address audience questions. The panel discussed the diversity of symptoms that appear in Parkinson’s patients, where treatments are headed, and how the relationship between the gut and the brain (referred to as the ‘gut-brain axis’) is an important new research area being addressed by a collaboration between NYSCF and Tufts University announced this week.

“Patients with Parkinson’s often report gastrointestinal issues, and for a long time this was overlooked,” explained Dr. Nirenberg. “But recent research has shown that the microbiome in our gut interacts with our immune system and our brain in ways that may influence neurodegenerative diseases. Our collaboration with Tufts will use 3D stem cell models to explore this relationship.”

The panelists conveyed their hope for the future of Parkinson’s treatments: better understanding the various factors that contribute to the disease, and how they vary across different patients, will help researchers develop more targeted, effective approaches — moving beyond current symptom-based treatments to disease-modifying treatments that can slow, stop, cure, and even prevent the disease from occurring.

Learn more about Parkinson’s disease research at NYSCF here.