Researchers at Weill Cornell Medicine have developed an experimental treatment that uses controlled doses of carbon monoxide to significantly reduce the spread of some of the deadliest forms of cancer, raising hopes for a new approach to preventing recurrence after treatment.
The preclinical study, recently published in the journal Advanced Science, focused on pancreatic cancer and triple-negative breast cancer, two aggressive diseases known for their ability to spread to other parts of the body even after surgery and chemotherapy. Researchers said the treatment could eventually be used to target microscopic cancer cells that survive initial treatment and later form new tumors.
The therapy centers on a specially designed metal-free carbon monoxide prodrug, an inactive compound that releases low, controlled amounts of carbon monoxide once inside the body. While carbon monoxide is toxic at high concentrations, the human body naturally produces small amounts of the gas.
Researchers have spent years investigating whether those low levels can interfere with cancer progression. Previous attempts to deliver carbon monoxide as a treatment have faced significant challenges, including safety concerns associated with inhalation methods and the toxic byproducts left behind by metal-based delivery systems. The newly developed compound, known as CO-116, was designed to avoid those problems.
Scientists tested CO-116 in multiple mouse models of pancreatic and triple-negative breast cancer. The treatment significantly reduced the growth of metastatic tumors in the liver and lungs without producing signs of toxicity, weight loss or behavioral changes in the animals.
Researchers also discovered that smaller doses administered more frequently were more effective than larger doses delivered less often. The finding could play an important role in shaping future clinical trials and treatment protocols if the therapy advances to human testing.
Beyond slowing tumor growth, the study identified a possible biological explanation for the treatment’s effectiveness. The carbon monoxide prodrug reduced levels of a protein known as HRG1, which helps cancer cells absorb heme, an iron-containing molecule essential to many cellular processes.
Lowering HRG1 levels disrupted signaling pathways that cancer cells use to migrate and establish new tumors elsewhere in the body. Researchers found that increasing HRG1 levels made cancer cells more aggressive, while reducing the protein significantly slowed metastatic growth in both pancreatic and breast cancer models. The findings suggest HRG1 could become both a therapeutic target and a biomarker used to identify patients most likely to benefit from treatment.
While researchers caution that additional testing is needed before the therapy can move into human trials, they believe CO-116 and related compounds could eventually serve as adjuvant treatments administered after surgery or chemotherapy to reduce the risk of cancer returning.
Researchers said future studies will focus on long-term safety, optimal dosing schedules and whether the treatment’s anti-metastatic effects continue after therapy ends. If successful, the approach could offer a new weapon against metastasis, the process responsible for most cancer-related deaths.
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