What does it take to solve a mysterious orphan disease? It demands a clear mission, inspiring leaders, talent, teamwork, unstoppable energy, and visionary investment.
These engines are in motion at Brigham and Women’s Hospital, where Barbara and Frank Resnek have pledged $20 million, including a recent $15 million commitment, to launch a first-of-its-kind research center for primary sclerosing cholangitis (PSC). A rare, chronic bile duct disease that mainly affects young men, PSC disrupts normal liver function and slowly causes cirrhosis and liver failure. The causes of PSC are still unknown. Currently, it is only treatable with a liver transplant.
“We are operating with a sense of urgency, hope, and tremendous excitement about the momentum we can create. Because of the Resneks’ support, we can accelerate the timeline for these breakthroughs to come in the next years—not decades.” —Joshua Korzenik, MD
Now, thanks to this extraordinary gift, the Resnek Family Center for PSC Research is undertaking one of the world’s broadest efforts to find a treatment that prevents these life-threatening complications. Joshua Korzenik, MD, director of the Crohn’s and Colitis Center at the Brigham and leader in PSC care and research, is heading up the center’s clinical investigators and bench scientists. Their combined expertise is in gastroenterology, neuroscience, immunology, pathology, and translational medicine.
The team aims to improve understanding and diagnosis of PSC, build an expansive collection of tissue samples to analyze the disease, and identify existing treatments that could work for PSC. They will also focus on developing new therapies specifically for the disease and will share their findings with the broader medical community.
“We are operating with a sense of urgency, hope, and tremendous excitement about the momentum we can create,” says Korzenik.
Korzenik says one crucial lead is the link between PSC and ulcerative colitis—up to 80% of patients with PSC have this form of inflammatory bowel disease. This avenue of study will help understand the cause of PSC and may also benefit patients with ulcerative colitis.
Though the center is new, its research is well underway because of the Resneks’ previous support. Frank and Barbara pledged $5 million in 2017 after months of careful research and consideration. They were inspired by Korzenik and his team’s determination to get a viable treatment to patients as soon as possible.
Korzenik adds, “Thanks to the Resneks’ incredible generosity, we can accelerate the timeline for these breakthroughs to come in the next years—not decades.”