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Researchers from the University of California‑Davis have reported encouraging results from a phase II clinical trial of a lab‑made antibody called nebokitug. This monoclonal antibody targets a protein linked to inflammation and scarring in the liver. In the study, the drug was safe and showed signs of slowing disease progression in people with primary sclerosing cholangitis (PSC), a rare chronic liver disease with no effective treatment other than liver transplantation. The trial offers hope for patients who currently rely on symptom management and surgical procedures to relieve blocked bile ducts.
Background
Primary sclerosing cholangitis is a long‑term liver disease in which the bile ducts – tubes that carry bile from the liver to the gallbladder and intestine – become inflamed and scarred. As the ducts narrow, bile backs up into the liver and gradually damages liver cells. PSC affects about one in 10,000 people and often occurs alongside inflammatory bowel diseases such as ulcerative colitis. The exact cause is unknown, but most experts believe it is an immune‑mediated condition triggered by a combination of genetic factors, gut microbiome changes and environmental influences. Men are more commonly affected than women, and the disease is most often diagnosed in middle age.
Key facts
- Symptoms develop gradually: Many people with PSC have no symptoms at first. As bile backs up, they may experience extreme tiredness, itching, belly pain, fever and jaundice (yellowing of the skin and eyes). Episodes of infection in the bile ducts are common.
- Associated with inflammatory bowel disease (IBD): Around 70–80 % of PSC patients also have IBD, most often ulcerative colitis. Doctors therefore recommend regular screening for both conditions.
- Risk factors: PSC is more prevalent among men, people with Northern European ancestry and those with certain genetic variations. The disease typically appears between 30 and 50 years of age.
- Complications: Long‑term inflammation and scarring can lead to cirrhosis, liver failure and cancers of the bile ducts, gallbladder or colon. Regular monitoring is essential for early detection of malignancies.
- Treatment options: At present, care focuses on managing symptoms and complications. Procedures may temporarily open narrowed ducts, but liver transplant is the only curative option for advanced disease. Drugs such as ursodeoxycholic acid have limited benefit. New therapies like nebokitug aim to reduce inflammation and fibrosis by blocking the CCL24 protein that promotes scarring.
Significance
The promising results from the nebokitug trial mark a pivotal moment for PSC patients. By targeting the underlying inflammatory pathway rather than simply relieving symptoms, this treatment could slow disease progression and delay the need for transplantation. Early diagnosis and management of associated bowel diseases remain crucial, but advances in immunotherapy offer hope that PSC may soon have an approved medical treatment.
Conclusion
Primary sclerosing cholangitis remains a serious and poorly understood illness. Although rare, it has devastating consequences for those affected. Continued research into the immune mechanisms driving PSC, along with clinical trials of targeted therapies, is essential. For patients today, vigilant monitoring and timely referral for liver transplantation remain the mainstay of care.
Source: NDTV
