Dr. Paola Leone is leading research in one of the most complex medical fields of our time—the human brain.
As a Rowan University School of Osteopathic Medicine professor and director of the Cell & Gene Therapy Center, Leone studies pathophysiologic mechanisms underlying neurodegenerative diseases while training future generations of doctors, researchers and scientists.
Inheriting her father’s work ethic, Leone knew at a young age what she wanted to do and took it upon herself to accomplish it, going against her family’s initial wishes for her future. She embraced her own independence and leadership and recalls being a very intense and curious child.
“I was always exceptionally interested in unanswered questions—from medicine to biology to astronomy,” said Leone. “My interest in the neurosciences came early on in high school when I realized it was the most challenging field in medicine in terms of therapies.”
“We still don’t know much about how thoughts are formed or what really drives our passion as humans. There are so many unanswered questions about the brain and how it translates into who we are. That’s what I knew I wanted to find out.”
After earning her Ph.D. in neuroscience from the University of Padua in Italy, Leone began her career in academia studying drug addiction and gene therapy for Parkinson’s disease. She has researched all over the world, including Italy, Canada, and New Zealand. After spending time teaching at Thomas Jefferson University, she joined the University of Medicine and Dentistry of New Jersey (UMDNJ) as a faculty member and transitioned with the beginnings of RowanSOM, spending the last 18 years focusing on neurodegenerative diseases—developing treatments and meticulously working to slow down, stop and ultimately cure tragic disabilities to the brain. Leone specifically focuses on Canavan disease, but is also researching traumatic brain injury as well as Alzheimer’s disease.
Canavan disease is a fatal, neurological disorder that begins in infancy. Leone remembers the moment she chose to make the disease her point of study.
“Over 20 years ago when I was a researcher at Yale, a family came in with their sick child,” she said. “The doctors diagnosed her with Canavan disease and it was so, so rare. I knew then that it would be my primary focus. Her father was an MD who understood that research and funds would be necessary to fight it, and it soon became a passion project to me.”
Because Canavan disease is so rare, Leone knows almost every patient that is diagnosed. “We’ve learned everything there is to learn about the disease,” she said. “Clinical trials, historical national data—we know it all. We work to improve clinical end points with collaborators. We’ve designed a novel gene therapy that has been successful in support of those with this disease. We’ve worked very hard.”
Although Leone has made monumental progress over the past two-decades, she notes that making a clinical trial happen is no easy feat. She works long days, usually seven days a week-on life saving programs for children that need to be tested first. That comes with many challenges, including acquiring FDA support, pre-clinical status for neurodegenerative disease, finalizing administrative and legal details and pushing through huge delays, sometimes waiting as long as six months to have her proposals reviewed.
“The process and nature of my work in combination with the transition from UMDNJ to RowanSOM was hard,” added Leone. “But I knew this was the area where I wanted to stay and continue my research and teaching. The beginning wasn’t easy, but eventually things fell into place and we were able to be very active in and out of the lab.”
While her work on neurodegenerative diseases has been rapidly rising during her time at RowanSOM, Leone notes that the opportunity Rowan has given her to teach and build relationships with her students has been “serendipitous.”
“I enjoy teaching very much,” Leone said. “This is very demanding work and my students are incredibly motivated. I’ve had close to 50 students every year for 10 years and I love writing each and every one of them a letter of recommendation.”
Leone credits the environment at Rowan for her success not only as a researcher but as a professor and mentor to her students.
“There are many aspects in my line of work in which supportive leadership and a supportive environment are necessary,” said Leone. “It’s important to have time to devote to my students and build relationships so that I can help them as much as I can. And without that environment I wouldn’t be able to spend time on my own academic research goals to develop treatments for these awful, incurable diseases.”