Cancer therapy breakthrough: New chemotherapy agent proves 20,000 times more effective and free of side effects in animal trials
Chemotherapy remains a standard treatment for many types of cancer, but it’s far from perfect. This approach relies on cytotoxins to kill rapidly dividing cells, yet it lacks precision. One of the most commonly used drugs, 5-fluorouracil (5-Fu), targets not only cancer cells but also healthy tissue, often causing severe side effects such as nausea, fatigue, or cardiac complications. Its poor solubility in bodily fluids further limits its effectiveness.
A team at Northwestern University led by Professor Chad A. Mirkin, Director of the International Institute for Nanotechnology, may have found a solution to these challenges. The researchers redesigned the molecular structure of the chemotherapy compound, making it 20,000 times more effective against leukemia cells in animal studies – without causing any side effects. Their findings were published on October 29 in ACS Nano.
The key: Spherical nucleic acids
The advanced compound is built on spherical nucleic acids (SNAs) – tiny nanostructures consisting of a particle core surrounded by tightly packed strands of DNA or RNA. In this design, the chemotherapy compound 5-Fu is chemically embedded within the DNA shell. The concept is simple yet powerful: cancer cells recognize and absorb the spherical particles through specific surface receptors. Once inside, enzymes break down the DNA shell, releasing the drug precisely where it’s needed. While this was the theory, the results now suggest it works just as well in practice.
A short video from Northwestern University illustrates how the nanostructure enters a cancer cell and releases the drug from within:
In animal studies on acute myeloid leukemia (AML), the results were striking. The SNA-based treatment entered cancer cells 12.5 times more efficiently and was about 20,000 times more effective at killing them than the standard drug. Disease progression slowed by a factor of 59, and leukemia cells nearly disappeared from the blood and spleen of treated animals. Meanwhile, healthy tissue and organs remained unaffected, with no side effects observed.
A milestone in cancer research
The researchers describe their work as a milestone in structural nanomedicine – a new approach that leverages both the chemical composition and spatial design of drugs to precisely control how they behave in the body. As a result, the findings represent a major step forward in cancer treatment. Looking ahead, the same strategy could potentially be applied to combat infections, neurodegenerative disorders, or autoimmune diseases.
Currently, seven SNA-based therapies are undergoing clinical trials. As for the newly developed leukemia treatment, the next steps involve more extensive animal studies, followed by clinical testing in humans. However, it will likely take several years before the drug is ready for the market.
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