Grant for Precision Medicine Study Looks at How Microbiomes Affect Parkinson’s Disease Risk

Using a gut-to-brain disease-seeding process, a multi-institutional team of researchers seeks to learn more about Parkinson’s disease-linked fibrils that form in nerve cells in the intestines

Michael G. Kaplitt, MD, PhD, vice-chair for research in the Department of Neurological Surgery at Weill Cornell Medicine and a neurosurgeon at NewYork-Presbyterian/Weill Cornell Medical Center, leads a new microbiome-related Parkinson’s disease research study.
Michael G. Kaplitt, MD, PhD (above), leads a Weill Cornell Medicine microbiome-related Parkinson’s disease research project. For this grant-funded project, a multi-institutional team is studying the fibrils that spread from the intestines to the brain and which lead to disease progression. (Photo: Stephanie Diani via Weill Cornell Medicine)

As hospital leaders follow developments in precision medicine research in considering services their hospitals can offer, they will be interested to learn about new research at Cornell. Funded by a $8.9 million grant, this new study examines the microbiome, or the bacterial organisms specific to an individual’s gut, and how specific microbiomes impact the development of Parkinson’s disease.

“Interaction between the body and the brain is a very exciting and important area of research, and it is increasingly clear that, at least in some cases, Parkinson’s disease may begin in the gut, with the disease spreading through nerve connections to the brain and eventually throughout the brain,” said project lead Michael G. Kaplitt, MD, PhD, in Weill Cornell Medicine news release. Kaplitt is vice-chair for research in the Department of Neurological Surgery at Weill Cornell Medicine and a neurosurgeon at New York-Presbyterian/Weill Cornell Medical Center.

Using a mouse model gut-to-brain disease-seeding process, the researchers seek to learn more about disease-linked fibrils that form in nerve cells in the intestines, according to Weill Cornell Medicine. These fibrils eventually spread through the brain via the vagus nerve and are seen in a characteristic pattern of “abnormal clumps of protein within brain cells in affected brain regions.”

Developing Scientific Models to Study Progression of Parkinson’s

Years ago, pioneers combined pathology and biochemistry to discover abnormal proteins in brain cells, noted in this case alpha-synuclein. Through the establishment of a brain bank, stored brains from patients with diseases such as Alzheimer’s and Parkinson’s, as well as from people without degenerative brain diseases, have been used for comparison and study into what proteins are involved in diseases such as Parkinson’s and Alzheimer’s.

For this new grant-funded Parkinson’s research study, the Weill Cornell Medicine team brings extensive experience in gene therapy. “Bringing our nearly 30 years of experience with gene therapy to this project will allow us to not only understand how this gut-to-brain transmission happens, but also potentially intervene genetically to improve brain function and stop the spread of disease,” Kaplitt explained.

The Weill Cornell Medicine Parkinson’s research project also includes two other co-investigators, including:

The international group of researchers will provide multiple resources that enhance the quality of research that can be performed.

The project is made possible through a nearly $9 million award from the Aligning Science Across Parkinson’s (ASAP) initiative and its implementation partner The Michael J. Fox Foundation for Parkinson’s Research.

“There is a growing amount of evidence of the gut’s role in Parkinson’s disease, and we are developing excellent scientific models to study the disease’s progression from the start,” added Dawson as part of Weill Cornell Medicine’s announcement. “Our multidisciplinary approach holds tremendous promise in identifying innovative ways to treat Parkinson’s disease.”

Options for Developing a Hospital Precision Medicine Program

While precision medicine typically invokes the concept of providing treatments based on a patient’s unique genetic traits, this new research highlights the versatility of hospital opportunities in precision medicine. Fueling the multi-institutional research is that a person’s microbiomes are unique and play an important, but largely unknown, role in their overall health.

“Bringing together this international team of the most forward-thinking, most innovative researchers in Parkinson’s disease holds so much promise for patients,” stated Philip E. Stieg, MD, PhD. “We all look forward to the discoveries that come from this research, which has the potential to create effective new treatments for this debilitating disease.”

The Weill Cornell Medicine microbiome Parkinson’s research initiative demonstrates that personalized medicine includes more than just genetics. By considering other approaches to precision medicine, such as the microbiome connection to overall health, hospital leaders will discover they have a wide range of options for developing precision medicine programs of their own.

—Caleb Williams

Related Information:

Aligning Science Across Parkinson’s (ASAP) Initiative

Michael J. Fox Foundation for Parkinson’s Research

$9M Grant Funds Study of Gut-Brain Connection in Parkinson’s Disease

John Q. Trojanowski Dies at 75; Changed Understanding of Brain Diseases

Per Svenningsson, MD, PhD

Michael G. Kaplitt, MD, PhD

Philip E. Stieg, MD, PhD

Ted Dawson, MD, PhD

Interviews With Precision Medicine Movers

Setting The Foundation For A Best-In-Class Precision Medicine Program: Four Steps To Guide Hospital And Health System Leaders