Researcher clinicians help bridge the gap between scientific discovery and medical care – Part 2

By Charlene N. Rivera-Bonet | Waisman Science Writer

Part 1

There is no one “typical” type of researcher clinician. There are differences in training, percentage of focus on research vs clinic, and type of research done. Many also carry teaching and administrative responsibilities on top of their already demanding positions.

Kathleen Kastner, MD
Kathleen Kastner, MD

“I do see myself primarily as a clinician,” says Kathleen Kastner, MD, developmental behavioral pediatrician. “But having a scholarly approach to thinking about questions that come up in the context of clinic and trying to be thoughtful about answering those in an academically rigorous way to help improve clinical care for the patients that I’m seeing in clinic.”

Kastner is part of the Newborn Follow-up and Autism and Developmental Disabilities Clinics, where she sees patients for initial diagnosis, evaluation, and monitors the development of babies born with a high risk of developmental delays or disabilities. Kastner is involved in research related to autism spectrum disorder, as well as developmental outcomes of babies who have experienced a traumatic brain injury.

In addition to her clinical and research responsibilities, Kastner is in charge of rotations for pediatric medical residents in developmental pediatrics, which is another way she works to improve clinical care. “I try to understand the current training experience related to developmental care for our learners, and then develop curriculum and content to continue to improve that educational experience, and help providers be better prepared to care for individuals with disabilities,” Kastner says.

Bernadette Gillick
Bernadette Gillick, PhD, MSPT, PT

Bernadette Gillick, PhD, MSPT, PT, associate professor of developmental pediatrics and rehabilitation medicine decided to pursue her doctoral degree after 17 years of clinical practice as a physical therapist. During her clinical work she became aware the opportunities that still existed to advance rehabilitation using novel technologies. “That’s when I went back and got a PhD. And thought, ‘we can do more to advance recovery and I’m going to figure out how to be part of that equation to try to help’,” Gillick says.

After earning her PhD and over a decade in academia, Gillick joined the Waisman Center in 2021 and to continue her examination of brain recovery after early brain injury, development over time, and the potential development of cerebral palsy after injury. “In terms of clinical research, I work with the very patients who I used to treat [as a physical therapist],” Gillick says. Her line of research uses neuroimaging and non-invasive brain stimulation upon brain injury. This brain stimulation as an intervention, coupled with rehabilitation, Gillick says, has shown significant improvements in movement and function.

“If I can be a part of the journey with people as they’re working through this, that’s the biggest reward. When they turn around and say, ‘thank you for being a part of this [recovery process]’,” Gillick expresses, it is an honor.

Clinicians in bench work

David Gamm, MD, PhD
David Gamm, MD, PhD (Photo © Andy Manis)

Clinician researchers use many different tools to advance their research, including stem cells and animal models. David Gamm, MD, PhD, is a pediatric ophthalmologist, surgeon, professor of ophthalmology and visual sciences, and director of the McPherson Eye Research Institute, who uses stem cells to study degenerative diseases of the retina, including what he describes as one of the most devastating group of diseases of the eye, retinitis pigmentosa.

Retinitis pigmentosa causes the cells in the retina to degenerate, leading to vision loss. At the moment, there is no cure for it, but Gamm has dedicated more than 20 years of research to the advancement of gene and drug therapies that might be able to help people that are in the earlier stages of the disease. His lab was the first to develop retinal organoid structures from stem cells, which allows them to both study disease mechanisms and develop treatments. Being a clinician, Gamm says, gives him “first-hand knowledge of how these various diseases progress, and what their effect on patients and families are. And that helps in terms of writing grants [to fund the studies] and talking to companies [that support his research].” Through his clinical practice, Gamm has much experience and opportunity to explain complex medical terms and scenarios. These communication skills translate to the research community as well as they allow him to accessibly describe his research to the community and donors.

Pelin Cengiz, MD, is a pediatric intensivist and a professor in pediatrics who treats and researches neonatal brian injury. “I started observing patient populations with the same degree of injury, but different outcomes. And I started asking ‘why is there a difference in the outcome?’,” Cengiz says.

Pelin Cengiz, MD

In her research, Cengiz focuses on finding a novel therapy for neonatal encephalopathy after hypoxia ischemia (or disrupted neurological functions after a experiencing a lack of oxygen and blood flow to the brain) using mouse models. Even when using animal models, she is able to apply what she learns through research at the clinic. For example, if she observes that a drug used in humans has negative effects on an injured animal, she seeks alternative ones for her patients. “Even though what I am working on may not primarily affect my patients in my lifetime, it may just create a foundation for the young investigators to build up on to achieve the same goal of providing the best care for children with brain injury,” Cengiz says.

Cengiz closely collaborates with Peter Ferrazzano, MD, a pediatric critical care physician who directs the Waisman Pediatric Brain Care Clinic and is a professor of pediatrics and the division chief for pediatric critical care medicine at the School of Medicine and Public Health. Ferrazzano uses MRI in clinical studies and pre-clinical animal models of traumatic brain injury and stroke to identify biomarkers of injury and therapeutic effects. By finding these biomarkers, he hopes to identify patients most likely to benefit from specific interventions.

Peter Ferrazzano, MD at the clinic.
Peter Ferrazzano, MD at the clinic.

His research efforts are based on the observation that acquired brain injury in kids was one of the biggest sources of long-term disability, but there aren’t many good treatments for it. “I love what I do in the ICU. It’s a really challenging and high stakes clinical environment, and you can do a lot of good with the patients that that you take care of in the ICU,” Ferrazzano says. “But my research activity gives me the opportunity to potentially impact kids that I never have the chance to take care of directly.”

Being a scientist, Ferrazzano points out, allows him to employ a scientific approach to problems in the clinic. Furthermore, collaborations within and outside of Waisman elevate his clinical work.

Collaborative work in nature

Collaborations within the Waisman Center have been a significant draw and anchor for this group of researcher clinicians to come and stay at the center, and has been an incredible boost to their research. Many of these researcher clinicians routinely collaborate with each other and have multiple projects together. “I think having such a great interdisciplinary group of folks that I’m working with really facilitates both the clinical and academic work that I’m involved in, helps all of us be able to think of things from different perspectives and to have a more holistic and well-rounded view of any particular situation or question that’s coming up,” Kastner says. This sentiment is echoed by all the physician scientists mentioned here.

This work, however, doesn’t come without challenges. When asked what was the most challenging part of being a physician scientist, everyone said “time.” Balancing both research and clinical work, plus administrative and teaching responsibilities for some, can be demanding. Yet, “the benefits far outweigh any other issues. And that is that you have that connection with patients and families and you really understand the disease aspects of things,” Gamm says.

Researcher clinicians are the embodiment of the Waisman Center, which has both a clinical space and research facilities under one roof to not only determine the cause and consequences of intellectual disabilities and neurodegenerative diseases but develop and provide interventions and treatments for patients and their families.

This story includes many, but not all researcher clinicians at the Waisman Center.

Click here for Part 1 of this story.

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