Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are rare diseases affecting the liver and bile ducts. Though each disorder has distinct causes and symptoms, both can lead to scar tissue formation, increasing the risk of liver failure and the eventual need for liver transplantation.
The limited knowledge of their pathogenesis leads to limited therapeutic options. However, the landscape of novel therapies for cholangiopathies is quickly shifting, offering new treatment opportunities for patients and healthcare providers.
Today, medical research is exploring combination therapies for PBC and PSC to protect liver cells, reduce the chance of infections like Clostridioides difficile infection, and slow or even halt disease progression. And with organizations like Science 37 making clinical study participation simpler, more patients can participate in studies that could improve quality of life and offer hope for better treatments.
Let’s look at the latest advancements and see why combination therapies could shape a brighter future for those affected by these challenging conditions.
Combination therapy means using more than one treatment at the same time to tackle a disease. In the case of PBC and PSC, it often involves medications that reduce inflammation, support bile flow, and protect liver cells from permanent damage. Because each of these liver diseases can cause scar tissue in the bile ducts, doctors hope that working on several fronts will produce better results than relying on a single treatment alone.
Combination therapies for PBC and PSC focus on using multiple treatments to target different aspects of these liver diseases. For example, ursodeoxycholic acid (UDCA), the standard therapy for PBC, is often combined with newer drugs like obeticholic acid (OCA) or bezafibrate. These combinations aim to improve bile flow, reduce inflammation, and lower alkaline phosphatase levels—a key marker of liver damage. Studies have shown that these pairings can help patients who don’t fully respond to UDCA alone.
Other approaches include combining anti-inflammatory drugs with antifibrotic agents to reduce liver inflammation and scar tissue formation, or using biologic therapies alongside standard treatments to manage more severe cases. In PSC, antibiotics like vancomycin are also being studied in combination with anti-inflammatory drugs to address bacterial imbalances and reduce symptoms. These therapies hold promise for slowing disease progression, protecting liver cells, and improving overall quality of life for patients.
PBC and PSC are part of a broader spectrum of chronic liver conditions that include nonalcoholic fatty liver disease (NAFLD), liver cancer, and others. In PBC, the immune system progressively damages the small bile ducts, while PSC often involves a more complex network of inflammation that may also affect the large bile ducts.
Standard therapies, such as bile acid supplements and immunosuppressive drugs, have shown some success in slowing these conditions. However, many patients still risk permanent damage to their liver cells, requiring additional treatments or, eventually, liver transplantation.
Researchers have discovered that combining two or more therapies may not only slow disease progression but also address complications like clostridioides difficile infection that can occur in advanced stages. This dual or triple approach aims to reduce inflammation, enhance bile flow, and protect liver cells from ongoing injury.
Groundbreaking medical research thrives on well-designed clinical study protocols, thorough blood tests, and advanced imaging tools like a CT scan. In studying PBC and PSC, investigators often track changes in alkaline phosphatase levels—an enzyme that can indicate ongoing inflammation or damage to the liver. These meticulous measurements help researchers determine how effective each therapy is, and identify the combination that offers the most benefit with the fewest side effects.
The past decade has witnessed an expanded focus on combination therapies, driven by collaborations among pharmaceutical companies, academic centers, and patient advocacy groups. Insights gleaned from each clinical study can be rapidly shared and analyzed, allowing researchers to refine their approaches and accelerate drug development. This teamwork is critical: each discovery propels the field forward, offering renewed optimism that severe complications and liver failure might be avoided.
Accessing rare disease trials has traditionally been challenging due to geographic barriers and the unique expertise needed to treat conditions like PBC and PSC. That’s where innovators like Science 37 step in. The organization harnesses digital platforms, telemedicine, and a network of specialized healthcare professionals to help patients overcome many of the common hurdles to participation—such as traveling long distances or coordinating multiple onsite visits.
Joining a Science 37 clinical study is simple and convenient, especially if you’re managing a condition like PBC or PSC. Science 37 lets you participate in groundbreaking research from your place of choice—no need to travel to a doctor’s office or clinic. Your involvement is crucial in advancing treatments for rare diseases like these.
The first step is signing up to join the Science 37 community. You’ll be asked for basic contact information and a brief medical history overview. This helps the team determine if there’s a current clinical study that’s a good fit for you. If not, they’ll stay in touch and let you know as soon as a matching study becomes available.
If a study matches your profile, you’ll move on to the pre-qualification stage. Here, you’ll answer more specific questions about your health through an online screener or by speaking with a team member over the phone. This step ensures that the study is right for you and that you meet its eligibility criteria.
Advancements in combination therapies for PBC and PSC mark a significant step forward in the fight against liver diseases that once felt unbeatable. These therapies tackle multiple aspects of the disease at the same time, aiming to prevent permanent damage and lessen the need for liver transplantation.
Organizations like Science 37 are improving trial designs and offering remote participation options, making crucial research accessible to more patients. This means effective treatments could reach those who need them faster.
There is still much work to be done, but the progress in medical research and the teamwork of experts worldwide bring hope to many. For anyone dealing with conditions that might lead to liver failure or other serious health issues, breakthroughs in combination therapies—and easier access to clinical studies—could protect the liver, keep vital liver cells functioning, and ultimately enhance quality of life.
