The findings, which were recently published in the international journal Molecular Cancer, could signal the start of a new chapter in cancer therapy.
According to a statement by the center, the team’s research focused on transforming a highly toxic anticancer compound, pyrrolino-daunorubicin, into a safer and more effective treatment option. By encapsulating the molecule in what is called a liposomal nanoformulation, the team created LiPyDau (liposomal pyrrolino-daunorubicin), a version that retained its strong anticancer properties while reducing its harmful effects on healthy tissues. The results exceeded expectations.
In animal models, LiPyDau successfully inhibited tumor growth in six distinct cancer types, including melanoma and lung cancer. Even more striking, it caused full tumor regression in a hereditary breast cancer model. The compound also proved effective against drug-resistant tumors that had shown little to no response to existing clinical agents.
With a single treatment, LiPyDau eradicated tumors derived from 4T1 mouse mammary tumor cells (one of the most treatment-resistant murine breast cancer models) within one week. Luminescent imaging confirmed the tumors’ complete disappearance, as shown in false-color visualizations taken during the experiment.
Dr. Kristóf Hegedüs, who synthesized the compound under the supervision of Dr. Gábor Mező at ELTE, said the molecule’s potential was clear early on. According to him, once they recognized how promising the molecule was, they began developing the techniques needed to produce it at the required quality and in sufficient quantities.
“This opened the way to animal studies,” added the HUN-REN RCNS researcher. Pyrrolino-daunorubicin is roughly a thousand times more cytotoxic to cancer cells than conventional agents, making it highly potent but difficult to administer safely. The researchers therefore needed a way to prevent it from harming healthy tissues.
“That was when the idea of a liposomal nanoformulation emerged. It would shield healthy cells from the active compound while enabling targeted accumulation in the tumor. That is how LiPyDau was born,” says Dr. Szilárd Tóth, who led the testing of LiPyDau in cell lines.
According to Dr. András Füredi, one of the study’s lead authors, LiPyDau stands out even among the most advanced experimental treatments. According to him, this is not merely an effective formulation; based on their experience and the published literature, it ranks among the most potent anti-tumor compounds ever tested in mice.
“We have been developing cancer therapies for 15 years, but we have never observed such a marked effect. A single dose of LiPyDau cured the experimental animals,” the researcher emphasizes.
The success of the project was built on a decade of intensive collaboration. Within HUN-REN RCNS, multiple research units played key roles, including groups led by Dr. Gergely Szakács, Dr. Zoltán Varga, Dr. Pál Szabó, and Dr. Dávid Szüts. Dr. Csaba Magyar contributed the in silico molecular modeling, which helped refine the compound’s design. The teams have worked in close cooperation for nearly 10 years.
Szakács, who led the overall research at HUN-REN RCNS, emphasized the national and international collaboration that made the discovery possible. Partners included the National Laboratory of Pharmaceutical Research and Development, the HUN-REN Center for Energy Research, Óbuda University, the University of Pécs, the National Institute of Oncology, the Medical University of Vienna, and the University of Veterinary Medicine Vienna.
The research also received support from Kineto Lab Ltd., one of Hungary’s leading oncology development companies. Together, they are already preparing the next steps toward human clinical trials of LiPyDau.
“The research was the result of exemplary national collaboration,” Szakács noted. “Our combined expertise allowed us to progress from molecule design to preclinical validation efficiently while maintaining scientific rigor at every stage.”
Despite the remarkable animal trial results, more research is needed before the compound can be tested in humans. The next phase will involve additional preclinical studies to ensure LiPyDau’s safety and to meet the regulatory requirements for human trials.
Regarding when patients might benefit from the treatment and whether such an experimental formulation could reach clinical use, Attila Kigyós, founder and CEO of Kineto Lab Ltd., expressed optimism about the project’s future.
“LiPyDau is by far the most effective anti-tumor compound we have tested in the company’s history. We must do everything we can to advance it to a stage where clinical trials are possible, or where it attracts the interest of a major pharmaceutical company to acquire it and develop it into an oncology product,” he explained. “LiPyDau stands a strong chance of becoming a Hungarian-developed cancer medicine.”
This article was first published in the Budapest Business Journal print issue of November 14, 2025.
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