Shepherding Leading-Edge Clinical Research
Unlocking the answers to treatments and a cure for rare diseases depends on clinical trials. Dr. Erika Fullwood Augustine, a highly regarded clinician, scientist, mentor and leader, is moving clinical and translational research strategy and operations to a new standard of scale. She partners across the Institute to develop, grow, structure and manage the Clinical Trials Unit, and serves as the primary liaison with the Johns Hopkins School of Medicine Institute for Clinical and Translational Research. In her role, she works closely with the Office of Human Research Administration on clinical trials oversight and regulatory compliance.
A pioneer in mentoring in clinical research, in 2017 she launched a pathways development program for child neurologists early in their training who aspire to become physician-scientists. This collaboration between the national Child Neurologist Career Development Program (housed at Kennedy Krieger) and the Child Neurology Society fosters the next generation of physician-scientists.
Finding Answers and Hope for Rare Diseases
Solving the puzzle of rare neurological diseases, like Batten disease, for example, will help solve others. An often-fatal group of childhood illnesses, Batten disease is a special area of expertise for Dr. Augustine.
Children with Batten disease suffer symptoms that include progressive vision loss leading to blindness, seizures, cognitive decline and dementia, problems with movement, personality and behavior changes, and shortened lifespan. There is no cure.
Dr. Augustine and her team are working towards unlocking novel, needed treatments for Batten and other rare diseases by increasing the scale and scope of Kennedy Krieger’s Clinical Trials Unit. “You’re always learning at multiple levels at any one time, so an advancement in care or in a method of research ends up applying to many other diseases,” says Dr. Augustine.
Each rare disease may only affect a relative few, but a discovery made for any one disease may translate to solutions for others. “When we learn more about the mechanisms and biology of rare diseases, the knowledge often sheds light on common diseases as well,” Dr. Augustine adds.
Embracing the Urgency for Research Discoveries
Kennedy Krieger is the world’s premier healthcare organization dedicated to improving the lives of children and young adults with care and research focused on disorders and injuries of the developing nervous system, from autism spectrum disorder and intellectual disability to Batten disease, cerebral palsy, acute flaccid myelitis and more than 200 ultra-rare diseases.
That means we are comfortable in uncharted territory. In fact, it’s where we do our best work. Kennedy Krieger has a long history of undertaking and harnessing groundbreaking scientific research, conducting world-class clinical trials and creating new treatments to change lives.
Heartbreak and frustration are evident in Dr. Augustine’s work. So are unwavering hope and determination. “There are treatments that are just within reach,” she says. “Rare diseases can be devastating conditions and they often affect children. If Kennedy Krieger could connect each child to a trial, then ideally, we could prevent loss of life. There’s really a great deal of urgency to our work.”
With endowment support for an endowed chair, Dr. Augustine can broaden the scale, outcomes and global impact of clinical trials for rare diseases across the nation and world. Funding groundbreaking research trials for children with Batten and other rare diseases—at precisely the moment when that child’s potential is blossoming—drives Dr. Augustine and her colleagues. The treatment and knowledge gleaned through clinical trials means each child can embrace their potential—and science is closer than ever to finding answers to eradicate rare diseases.
Every person, every child, has the right to high-quality treatment.” – Dr. Augustine