New drug quadruples three-year survival rates for mesothelioma in international trial

A drug that starves tumors of a key nutrient has quadrupled three-year survival rates for mesothelioma — one of the deadliest cancers in the world — marking the first successful new treatment combination in 20 years. Led by Queen Mary University of London and published in JAMA Oncology, the international trial gives fresh hope to thousands of patients diagnosed each year with this asbestos-linked disease.

At a glance

• Mesothelioma survival rates: Patients who received pegargiminase alongside chemotherapy survived an average of 9.3 months, compared with 7.7 months for those on chemotherapy alone — and three-year survival rates quadrupled.
• Arginine starvation therapy: The drug ADI-PEG20 (pegargiminase) works by depleting arginine — an amino acid — in the bloodstream, cutting off the food supply to tumor cells that cannot manufacture their own.
• ATOMIC-meso trial: The phase 3 trial enrolled 249 patients across 43 centers in the U.K., U.S., Australia, Italy, and Taiwan between 2017 C.E. and 2021 C.E., with no new safety concerns reported.

A cancer with almost no good options

Mesothelioma is aggressive, fast-moving, and almost always fatal. It develops primarily in the lining of the lungs and is caused mainly by occupational asbestos exposure — meaning many of its victims are older workers who had no idea they were being harmed decades earlier.

Survival rates have historically been among the worst of any cancer. Most patients are given months, not years. For two decades, no new treatment combination had meaningfully moved those numbers.

That context makes this trial’s results striking. Among patients with pleural mesothelioma — the most common form, affecting the lung lining — those who received pegargiminase alongside standard chemotherapy were significantly more likely to be alive three years later. The trial authors described the quadrupling of 36-month survival as statistically significant.

Twenty years in the making

The drug’s development traces back to a single laboratory discovery by Professor Peter Szlosarek at Queen Mary University of London: mesothelioma cells lack a protein called ASS1, which normal cells use to produce arginine. Without ASS1, tumor cells are entirely dependent on arginine from the bloodstream to grow.

That vulnerability became the target. ADI-PEG20 depletes circulating arginine, depriving tumor cells of the nutrient they cannot make themselves while leaving healthy cells — which can synthesize their own — relatively unaffected.

“It’s truly wonderful to see the research into the arginine starvation of cancer cells come to fruition,” Szlosarek said. The trial was funded by Cancer Research UK alongside biotechnology company Polaris Group.

What the numbers mean for patients

One trial participant, an 80-year-old man who asked to remain anonymous, had been given four months to live. He was diagnosed after being exposed to asbestos at a factory in the 1970s C.E. — and later won compensation from his former employer. Five years after joining the trial, he is still alive.

“I have five grandchildren and two great-grandchildren now — I wouldn’t want to miss all that,” he said.

Stories like his illustrate the difference between median survival statistics and what treatment breakthroughs can mean for individual lives. The median improvement — 1.6 months overall — sounds modest, but the tail of long-term survivors is where the real shift appears. Quadrupling three-year survival is a different order of magnitude than shaving weeks off a median.

Mesothelioma UK chief executive Liz Darlison called the trial “another much-needed treatment option and, above all, hope to those living with mesothelioma.” Dr. Tayyaba Jiwani of Cancer Research UK emphasized that the results demonstrate “the power of discovery research” — the value of understanding tumor biology deeply before attempting to intervene.

The road ahead

Pegargiminase is not yet a standard treatment. Regulatory review and health system approval processes — particularly through bodies like NICE in the U.K. and the FDA in the U.S. — will determine how quickly and equitably patients can access it. Cost and availability will matter enormously, especially for patients in lower-income countries where asbestos is still widely used and mesothelioma rates are rising.

Access to clinical innovation has historically been uneven, and mesothelioma disproportionately affects working-class communities with histories of industrial labor. Whether this drug reaches those communities quickly will be as important as the science itself.

Still, after two decades without a meaningful advance, the JAMA Oncology publication of the ATOMIC-meso results represents a genuine turning point. The biology was there. The patience was there. Now, for the first time in a generation, so is the evidence.

Read more

For more on this story, see: The Guardian

For more from Good News for Humankind, see:

• Alzheimer’s risk cut in half by drug in landmark prevention trial
• U.K. cancer death rates down to their lowest level on record
• The Good News for Humankind archive on global health

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