Discovering effective treatments for neurodegenerative diseases through innovative, patient-focused research
Currently, neurodegenerative diseases have minimal therapies. Therefore, it is critical for researchers and clinicians to consider innovative ways to address the needs of patients and caregivers. The Parkinson’s Institute and Clinical Center (the PI) is pioneering neurodegenerative research and has made groundbreaking discoveries in Parkinson's disease studies. The PI is the only independent nonprofit institute in the U.S. that performs laboratory and clinical research, conducts clinical trials, and provides world-class patient care for Parkinson’s disease in one center. From early stage studies focused on uncovering the genetic causes of various neurodegenerative diseases, to testing Phase 3 clinical therapies, research at the PI broadly explores dual-purpose strategies that directly change lives. The work of the Institute begins with the patient, which has led to their continued success in transformative Parkinson’s disease research. The PI’s innovative model shows how patients with neurodegenerative diseases can be empowered, as well as revealing the exciting possibilities of their translational research; and solving the mystery of Parkinson’s disease can lead to understanding and transforming the landscape for other neurodegenerative diseases, such as Alzheimer’s and Amyotrophic Lateral Sclerosis.
Currently, there are many basic science initiatives for Parkinson’s diseases. However, aside from publications, there’s often no place for these important discoveries to go. The PI is addressing this problem through their scientifically-driven model that pursues translational science for patient treatment. For the past 30 years, the PI’s teams of epidemiologists, physicians, scientists, and therapists have been at the forefront of the most important clinical and scientific discoveries in the field of Parkinson’s research. The PI has provided a springboard for the best physicians and scientists who continue to be on the cutting edge of Parkinson’s research and treatment. The organization continues to have a huge impact on the Parkinson’s community; their credibility is a direct result of their members and achievements, which are made possible by their patients and philanthropic supporters.
The Institute has contributed greatly to Parkinson’s disease data, and they continue to seek out ways to translate this information into therapies that will directly impact their patients and others around the world. The majority of the PI’s research projects are focused on bringing therapeutic sciences to their patients and—through their clinical trials—to patients worldwide.
Current research includes:
- Recruiting Patients For Novel Clinical Studies - The PI has managed more than 125 clinical research trials, medically evaluating more than 90 different drugs and therapies for Parkinson’s disease. This has resulted in the PI’s direct involvement in the evaluation of virtually every Parkinson’s drug currently available on the market. To complete these clinical studies, however, the PI must recruit Parkinson’s disease patients and their family members to enroll. They do this through engaging patients through education and outreach events, where they share the ways participation makes a significant difference for Parkinson’s research and impacts patients around the world. The PI can typically recruit 25 participants per outreach event. Further incentivizing patients to enroll are the close connections they form with the doctors performing those studies; they’re able to see what they’re doing and build valuable doctor-patient trust. By performing more clinical studies, the PI can identify improved clinical measures to help advance new treatments, such as identifying potential targets that can be used to create new, effective drugs.
- Delivering Care Through Telemedicine - Since the PI’s inception, patients have traveled from 38 states and many countries around the globe to receive unmatched care. However, many patients around the nation want to visit the clinic and get professional advice, but cannot due to the distance. The PI is exploring better ways to deliver care to Parkinson’s disease patients around the nation. They are looking at ways to utilize telemedicine, enabling patients to interact with clinicians from the Institute from their home. Through direct phone calls or Skype sessions with doctors, patients can to stay up-to-date on their health. Implementing innovative diagnostic techniques, the patient and doctor can both decide if a visit to the institute is necessary. Advocating for patients worldwide, the PI is able to serve the larger Parkinson’s community through this program. Currently, the PI is performing studies that evaluate how beneficial this method is; from there, they aim to share that information with insurance and medicare partners to garner support.
- Continuing to build the Brain Bank - Established 28 years ago at a time when many Parkinson’s disease cases were misdiagnosed, the PI’s brain bank was developed as a resource to improve clinical care and Parkinson’s disease diagnoses. Patients from the Institute would volunteer for an autopsy as they neared the end of their lives, enabling clinicians to look at their clinical records and assess their brain and body for disease. The brain bank enabled the PI to see if they were diagnosing patients correctly and showcased the ways autopsy-derived data could drive research, leading to improved diagnostics. It was also shown to be a way for clinicians to sit down with families of patients who passed away and discuss their level of risk for developing Parkinson’s disease, based on their genetics. Though the brain bank was set up for a particular purpose, the PI understood its potential for the future. For instance, after the brain bank was established, publications emerged about an autosomally dominantly inherited gene mutations that were thought to cause familial Parkinson's disease. This meant a person who had a family member with Parkinson’s disease would have a 50% chance of getting the disease. However, the PI discovered that the gene only counted for a fraction of Parkinson’s cases. They extracted and sequenced all of the DNA from the brains of former patients, and none of their patients had a mutation in that gene. The brain bank has the potential to completely change the face of the science and has become one of the main sources of brain tissue for basic science research worldwide. The PI aims to continue running the brain bank so their researchers and others around the world can test new ideas for potential advanced therapies.
Dr. Bill Langston, a world-renowned neurologist and pioneer in the study of Parkinson’s, first discovered a link between a tainted batch of “synthetic heroin” and parkinsonism while treating six young heroin users who mysteriously “froze.” It was his groundbreaking discovery that led to the birth of the Parkinson’s Institute and Clinical Center (PI) in 1985. This discovery ignited the field and lead to the generation of the most important research model in the history of Parkinson’s disease.
Since its founding, the PI has grown exponentially, becoming an important pioneer in both early stage and late stage work that brings new treatments for Parkinson’s disease. The PI is uniquely positioned to drive innovation in the area of therapeutic-based research to tackle one of the biggest unmet medical needs in neurodegenerative diseases. The Institute has enabled the discovery of treatments ranging from new medicines to deep brain stimulation, and has sparked a worldwide renaissance in laboratory and clinical research for Parkinson’s disease. As pioneers and leaders in the study and treatment of Parkinson’s, the Institute has had the privilege of training some of the world’s most influential physicians and scientists. More than 175 of today’s Parkinson’s thought-leaders have worked at PI.
Dr. Carrolee Barlow took the reins from Dr. Langston in 2014, when she joined the PI as the CEO. As a physician and a scientist, she has added valuable expertise and experience in neuroscience, clinical practice, business management, and drug development.
In her early career, Dr. Barlow intended to solely become a doctor. While in medical school, however, she soon found that, in addition to working with patients, she truly enjoyed being in the lab performing research projects. During that time, she wrote several research papers and was asked to do a research rotation at Rockefeller University. She was further inspired when she saw the huge impact research could have when it was focused on solving an acute human health problem.
She completed her residency in Internal Medicine at The New York Hospital, Cornell Medical Center. Once she finished that, she decided to pursue a Ph.D. She earned her Ph.D. in genetic engineering, at the Karolinska Medical Nobel Institute in Stockholm, Sweden using animal models to replicate disease, which was a completely novel approach at the time. The Karolinska is one of the most published research hospitals in the world. There, Dr. Barlow learned how to use research to transform healthcare. When she came back to the U.S., she undertook specialty training in endocrinology and genetics, and completed a postdoctoral fellowship in neurogenetics at the National Human Genome Research Institute.
Soon after, Dr. Barlow became a professor at the Salk Institute for Biological Studies, running a lab that used animal models to study neuro-diseases, helping to establish the field of neurogenomics. She looked at ways to use innovative technology—similar to the ways it was used in oncology—to explore the brain. Dr. Barlow then took a joint-appointment as Director of Molecular Neuroscience and Therapeutic Area Head at Merck Research Laboratories where she continued to explore therapeutic sciences and identify ways to directly impact patients. This motivated her to establish BrainCells, Inc., a company that works with prestigious scientists to translate their science into products for patients. She continued to work with companies that had promising drugs, but difficulty getting approved by the FDA or other regulatory agencies.
Dr. Barlow’s goal is to find solutions and treatments for Parkinson’s disease in the same way she has done with other diseases. Working with the FDA, she wants to find ways to transform clinical studies for neurodegenerative diseases by evaluating the science and designing them in a way makes the research a product.
“The Parkinson’s Institute is setup to make a difference,” says Dr. Barlow. “We want to reconfigure how we think of therapeutic sciences and be at the forefront of making that change.”
At the PI, Dr. Barlow uses her extensive background, experience and expertise, to ensure the team is working closely with partners who are key stakeholders and have the audacity to innovate, to think outside of the box, to look for ways to achieve more, and do it faster and more efficiently. Her ultimate goal for the PI is to transform lives and provide treatments to those living with disease.