Q&A: How Research on Mpox is Preparing Scientists for Future Pandemics

The 2022 global mpox outbreak in the United States resulted in over 20,000 cases and nearly sixty deaths. San Francisco was among the hardest-hit areas. Currently, mpox continues to be recognized as a public health emergency by the World Health Organization.

During the outbreak, healthcare providers used investigational treatments like tecovirimat (TPOXX), a drug effective against smallpox, which is a close relative of mpox. Physicians prescribed it to manage symptoms and potentially reduce disease severity, although its effectiveness specifically against mpox was under investigation.

In response, UCSF swiftly initiated a clinical trial within two months to evaluate TPOXX for mpox. An interview with Dr. Annie Luetkemeyer from UCSF's Advancing Clinical Trials Globally (ACTG) provides insights into the virus, the trial results, and implications for future pandemic preparedness.

Transmission of Mpox
Mpox spreads primarily through contact with infected skin lesions, and sexual transmission was notably significant during the 2022 outbreak. Two main types of the virus exist: Clade 1, mainly in Central and East Africa, and Clade 2, predominant in West Africa. The variant responsible for the outbreak was a form of Clade 2 that spread from Europe to the U.S.

Symptoms and Severity
Mpox can cause painful blisters, rashes, and discomfort, often resolving on their own. However, severe cases can occur, especially in children, pregnant women, and immunocompromised individuals. These groups are at higher risk of serious outcomes and death. Symptomatic treatment focuses on alleviating pain and preventing infected lesions from worsening.

Challenges in Outbreak Clinical Trials
Dr. Luetkemeyer highlighted the difficulty of conducting clinical trials during outbreaks but noted that existing research on smallpox treatments enabled rapid response for mpox. The UCSF trial's goal was to assess safety and efficacy of TPOXX and include vulnerable groups like pregnant women and children, who are often excluded from initial studies.

Study Findings
The trial found TPOXX to be safe and well tolerated. However, it did not accelerate recovery, possibly due to dosing or administration issues or the drug's limited efficacy against mpox. Similar findings from studies in the Democratic Republic of Congo, involving different virus strains, supported these results.

Including Vulnerable Populations in Trials
The trial was designed to ethically include pregnant women and children by providing open-label treatment to higher-risk individuals. This approach ensures equitable access to potential treatments and gathers valuable safety data.

Future Directions for Mpox Treatments
Research is ongoing into other antiviral options, like brincidofovir, which shows promise in laboratory tests. Monoclonal antibodies are also being explored as targeted therapies, mirroring strategies used during the COVID-19 pandemic.

Role of Clinical Trials in Emergencies
Dr. Luetkemeyer emphasized that well-designed clinical trials are crucial during outbreaks. They allow researchers to determine whether investigational treatments are truly effective, guiding public health responses and future preparedness. The mpox trial provided clear evidence that TPOXX does not improve recovery times, preventing unnecessary widespread use and encouraging exploration of other strategies.

This research underscores the importance of swift, inclusive, and scientifically rigorous trial designs during health emergencies to improve responses and outcomes for all populations.

Source: https://medicalxpress.com/news/2025-06-qa-mpox-scientists-pandemic.html