In a significant step forward for cancer therapy, researchers at Northwestern University have redesigned the molecular structure of a well-known chemotherapy drug, greatly increasing its solubility, effectiveness, and safety.

For this study, the scientists created the drug entirely from scratch as a spherical nucleic acid (SNA), a nanoscale structure that incorporates the drug into DNA strands surrounding tiny spheres. This innovative design transforms a compound that normally dissolves poorly and works weakly into a highly potent, precisely targeted treatment that spares healthy cells from damage.

When tested in a small animal model of acute myeloid leukemia (AML), an aggressive and hard-to-treat blood cancer, the SNA-based version showed remarkable results. It entered leukemia cells 12.5 times more efficiently, destroyed them up to 20,000 times more effectively, and slowed cancer progression by a factor of 59, all without causing noticeable side effects.

According to the researchers, this achievement highlights the growing promise of structural nanomedicine, an emerging area of research where scientists carefully design both the structure and composition of nanomedicines to control how they behave inside the body. With seven SNA-based therapies already in clinical trials, this approach could pave the way for advanced vaccines and new treatments for cancer, infectious diseases, neurodegenerative disorders, and autoimmune conditions.