This story originally appeared on Brigham Clinical Research News.

Zander Berlinski was just 14 when he was diagnosed with primary sclerosing cholangitis (PSC), a rare, chronic liver disease. For more than a decade, Berlinski’s life was largely unaffected by PSC. But that all changed in 2020, when the young New Yorker’s conditioned worsened to the point where he needed a liver transplant.

PSC disrupts normal liver function and can lead to cirrhosis and eventually liver failure. There are currently no effective treatments or a cure, and people with PSC often need a liver transplant. After waiting several months on the transplant list with no available transplant opportunity, Berlinski underwent a liver transplant when his best friend offered to be a living donor.

Throughout this process, Berlinski, now 31, searched for an institution that might want tissue samples from his original liver to advance research into PSC. That search led him to the Brigham and the Resnek Family Center for PSC Research.

Directed by gastroenterologist Joshua Korzenik, MD, the Resnek Center brings together scientists from across the Brigham, throughout the Mass General Brigham system and around the world to better understand and treat PSC and related conditions like ulcerative colitis (about 80 percent of people with PSC also have ulcerative colitis). The center spans the entire research continuum — from bench science to translational studies to clinical trials — and has become an international hub for PSC research.

“Through all my research, it became clear that the Resnek Center was the leader in the field, so I reached out to Dr. Korzenik,” Berlinski said.

All Roads Lead to the Brigham

Coincidentally, it was a similar search — spearheaded just a few years earlier by Boston philanthropists Frank and Barbara Resnek — that ultimately led to the creation of the Resnek Center.

Just like Berlinski, the Resneks are acutely aware of the impact PSC has on patients and families: Their grandson is one of approximately 30,000 people in the U.S. living with this disease. Committed to bringing better treatments and outcomes to all those affected, the Resneks began a worldwide search for institutions best positioned to accelerate PSC research.

And just like Berlinski, their search led them to the Brigham and Korzenik.

Impressed by Korzenik and his sense of urgency, the couple gave more than $20 million to launch the Resnek Family Center for PSC Research in 2019. And this spring, they made yet another transformative gift of $25 million to continue fueling the center’s research. In total, the Resneks have given more than $45 million to PSC research at the Brigham — the world’s largest investment in the disease.

“Up to this point, PSC was a neglected disease,” said Korzenik, who holds the Resnek Family Distinguished Chair in Gastroenterology and Hepatology. “The Resneks changed that, enabling us to focus on PSC in a comprehensive way. In just four years, we’ve made remarkable strides in better understanding the disease, moving potential therapies into clinical trials and partnering with researchers around the globe to move the entire field forward.”

A Culture of Collaboration

Collaboration is a cornerstone of the Resnek Center. Since its inception, Korzenik and his team have built an expansive network of collaborators — dozens of scientists and clinicians across multiple disciplines and institutions, whose varied expertise allows the center to tackle PSC, the cause of which is currently unknown, from myriad angles.

The center is examining mitochondrial dysfunction to decipher the underlying mechanisms of PSC, studying the microbiome and its role in the disease, working to uncover biomarkers to better diagnose, and treat PSC and exploring novel approaches to drug delivery. It’s also spearheading several clinical trials, such as studies that repurpose already approved medications, test entirely new therapies, and evaluate nutritional approaches, including the first-ever clinical trial of a dietary intervention for PSC.

“We’re using cutting-edge techniques to investigate the pathophysiology of the disease, and at the same time we’re moving forward with potential clinical treatments,” said Korzenik, who credits the Resneks’ generosity with allowing the team to pursue multiple studies and directions simultaneously, rather than step by step.

The Resneks’ latest gift will allow Korzenik to expand his team and recruit scientists with the expertise to tap into new areas of inquiry, such as immunology, cell repair, and fibrosis (a hallmark of PSC is scarring of the bile ducts). Ideally, these findings will not only benefit patients with PSC, but also translate to helping patients with other conditions closely linked to the disease, such as ulcerative colitis.

It will also allow the center to further expand and strengthen its collaborations and move the entire field forward.

“We’re really excited to help catalyze PSC research around the world, grow our network of collaborators, and work together to accelerate advances in this disease,” Korzenik said.

A Patient-Centered Approach

The ultimate goal of the center is to quickly bring treatments to patients with PSC, and this patient-centered mission imbues all of its work.

For instance, patients can face numerous hurdles to participating in clinical trials, particularly if they don’t live close to the trial site. To overcome this challenge, the center structured one of its trials to allow for research nurses to enroll patients and administer the therapy in the patients’ homes, making participation easier and more equitable. Korzenik also collaborates closely with PSC Partners Seeking a Cure, a national patient advocacy group, to ensure the center’s research agenda is informed by the patient perspective.

“We’re not only working to help people in the here and now, but also help those in the future — who won’t know of our work but will benefit from our efforts to create a world where PSC and other diseases are better understood and easily treated,” said Korzenik.

It’s a message that resonates with Berlinski.

“The center is not only driven to find a treatment for PSC — which would alter countless lives — but it’s also providing hope to people affected by what was previously an area of research the medical field had not focused on significantly,” he said.