Inspirational Leadership: Dr. Ronald Sokol

“A leader’s first responsibility is to set the vision and inspire the team to carry it out.”

— Ronald J. Sokol, MD, Professor and Vice Chair of Pediatrics, University of Colorado School of Medicine

BILIARY ATRESIA IS a rare, incurable and potentially fatal disease causing blockage of the bile ducts and affecting only infants. Even with surgical intervention to reroute the intestine to drain bile from the liver, 80 percent of all children diagnosed with biliary atresia require a liver transplant before they reach adulthood. These dire statistics are not lost on Dr. Ronald J. Sokol, professor and vice chair of pediatrics at the University of Colorado School of Medicine and Children’s Hospital Colorado, whose life work revolves around the research and treatment of childhood liver diseases and other pediatric disorders.

Sokol is a man who wears many hats, but embraces his multiple callings with confidence. Among his numerous endeavors, he serves as director and principal investigator of the Colorado Clinical and Translational Sciences Institute at University of Colorado Denver, funded by the National Institutes of Health. He is also the chair of the Steering Committee of the NIH-supported Childhood Liver Disease Research and Education Network (ChiLDREN), a collaborative team of doctors, nurses, scientists and research coordinators at 16 medical facilities and patient support organizations focused on improving the lives of children and families dealing with rare liver diseases. ChiLDREN also receives funding from the Cystic Fibrosis Foundation, the Alpha One Foundation and the Alagille Syndrome Alliance. A review of his career accomplishments shows Sokol has much to be proud of, but what fuels his passion these days is his role as the lead investigator for a NIH-funded study on biliary atresia using intravenous immune globulin (IVIG). “Over the last five years, a focus has been the potential impact of antiinflammatory therapy in biliary atresia patients,” says Sokol. “IVIG has been used to successfully treat a number of other autoimmune diseases, and it is proposed that it may have similar positive anti-inflammatory effects when used post-surgery in biliary atresia patients. This study has the potential to break new ground in terms of the long-term prognosis of these patients and might delay or even eliminate the need for a future liver transplant.”

Sokol says ChiLDREN just completed a clinical trial that tested another drug’s ability to reduce inflammation in post-surgery biliary atresia patients, noting the IVIG trial will be much more targeted. This early phase clinical trial aims to enroll 29 patients over a 12-month to 18-month period. All patients will participate in the testing, with no placebos. The groundwork for the study began several years ago, and Sokol leads efforts to secure funding, which led to a partnership with FFF Enterprises, in Temecula, Calif. “A colleague had worked with FFF and encouraged us to reach out to them since they are the country’s largest distributor of IVIG,” says Sokol. “I met Patrick Schmidt, FFF’s chief executive officer, earlier this year when he paid a visit to Children’s Hospital Colorado in Denver. While here, he had the opportunity to meet a family whose infant was recently diagnosed with biliary atresia. I think the experience was very moving for him, and fueled his decision to provide funding for this important study.”

A Team Approach to Leadership

As a physician and researcher, Sokol is known for his commitment to leading by example. He is quick to stress that, by definition, good leadership is only as strong as the team behind it. “A leader’s first responsibility is to set the vision and inspire the team to carry it out,” he says. “Especially when you face obstacles, you need to empower people so that they feel excited and rewarded for their efforts. I currently hold several leadership roles, and I try to always be mindful of those principles.”

Sokol adds that he draws his motivation first and foremost from his role model. Dr. Arnold Silverman was one of the first pediatric gastroenterologists in the nation, and Sokol says it was Dr. Silverman’s style of practicing medicine that inspired him toward professional excellence. Still, with so many balls in the air, Sokol says finding a balance and managing work flow can be a challenge. “I received good advice from a former chair of mine,” says Sokol. “He said the method he uses to get everything done is simply tackling one thing at a time. Of course, in these days of multitasking that may not be as easy as it sounds, but it’s a philosophy that still helps me when things get hectic.”

Working in any pediatric specialty puts you in touch with families at a very vulnerable time in their lives. The nature of Sokol’s work, especially in the IVIG study, puts him in direct contact with the youngest and most vulnerable of patients: infants. “Although biliary atresia is rare, my heart goes out to the children and families suffering with this awful disease — I could not imagine what it is like to have a newborn diagnosed with it,” he says. “The families inspire me every day to use my resources and abilities to try and improve the outcomes for these patients.”

Leading Research, Now and in the Future

Sokol has a history of leading research teams to identify scientific breakthroughs. A number of years ago, children with liver disease commonly developed ataxia, a nervous system disorder causing them to lose balance, strength and mobility. Sokol was tasked with finding out the cause of the puzzling condition. At the time, there was some indication that a vitamin E deficiency might be linked, and after completing a study and working with the U.S. Food and Drug Administration, it was determined that treating patients with a novel liquid form of vitamin E could actually reverse the debilitating symptoms. “This was a very gratifying accomplishment,” says Sokol. “Vitamin E supplementation with this novel preparation is now the standard treatment in the U.S. for children presenting with cholestatic liver diseases to prevent vitamin E deficiency and ataxia symptoms.”

As he looks to the future, Sokol plans to focus his attention on gaining new insights and leading change in the area of treatment protocols for childhood liver diseases. On a personal note, he is committed to ensuring the ChiLDREN research network that he chairs remains funded, and is also looking forward to providing future investigators with the tools and inspiration needed to blaze new trails within this challenging field. “We actually have a program in our network that is focused on training and supporting the next generation of clinical researchers,” explains Sokol. “We want to guarantee that we have a pipeline in place that will foster significant medical breakthroughs for generations of future families.”