Nanotech makes cancer drug 20,000x stronger, without side effects

Researchers at Northwestern University have made a significant advancement in cancer treatment by redesigning the molecular structure of a widely used chemotherapy drug, dramatically enhancing its potency and solubility.

This new form of the drug, based on spherical nucleic acids, embeds the drug directly into DNA strands that coat tiny spheres, enabling it to enter leukemia cells 12.5 times more efficiently and destroy them up to 20,000 times more effectively, without causing detectable side effects.

These findings, published in ACS Nano, indicate that this innovative approach could transform 5-fluorouracil, or 5-Fu, a long-standing chemotherapy drug known for its limited effectiveness and harsh side effects.

Traditionally, 5-Fu has been criticized for its poor solubility, which results in less than 1% dissolving in biological fluids, thereby preventing effective absorption in the body. This study reveals that the drug's ineffectiveness was not due to the drug itself but rather its inability to dissolve properly.

By utilizing spherical nucleic acids, globular nanoparticles surrounded by dense shells of DNA or RNA, the researchers have created a drug that cancer cells naturally absorb. Chad Mirkin, who led the research, explained that myeloid cells, which are overexpressed in leukemia, have scavenger receptors that easily recognize and pull in these nanostructures.

Once inside the cells, enzymes break down the DNA shell, releasing the chemotherapy payload directly into the cancer cells. This method has shown a remarkable ability to nearly eliminate leukemia cells in animal models while significantly extending survival times.

The targeted nature of this therapy means that healthy tissues remain unharmed, addressing a major drawback of traditional chemotherapy, which indiscriminately affects both cancerous and healthy cells.

Mirkin emphasized the need for such precision in treatment, stating that current chemotherapeutics often kill everything they encounter. Moving forward, the research team plans to conduct tests on larger groups of small animal models before advancing to larger animals and eventually human clinical trials, pending further funding.

This research was supported by the National Cancer Institute and the National Institute of Diabetes and Digestive and Kidney Diseases, with additional backing from the Robert H. Lurie Comprehensive Cancer Center at Northwestern University.

This breakthrough signifies a potential revolution in cancer treatment, offering hope for more effective therapies with fewer side effects.