Tiny structure, big breakthrough: Drug targets blood cancer with precision
CHICAGO — A major breakthrough in cancer treatment is emerging from Chicago labs, thanks to a tiny, cutting-edge structure. Scientists at Northwestern University have taken a long-used chemotherapy drug and redesigned it at the nanoscale, creating a treatment that, in animal models, appears to wipe out a common form of blood cancer—and could reach patients as soon as next year.
Chad A. Mirkin, PhD, director of the International Institute for Nanotechnology at Northwestern, called the work “really exciting.”
“We figured out how to take two technologies and merge them to create a whole new class of drugs,” Mirkin said.
The process began with 5-Fluorouracil, or 5FU, a chemotherapy agent commonly used to treat cancers including acute myeloid leukemia (AML), a type of blood cancer.
“One of the problems with that drug is it is horribly insoluble, making it difficult to deliver effectively,” Mirkin explained. “It also goes to many different types of cells.”
Like many traditional chemotherapy drugs, 5FU can harm healthy cells while targeting cancer, often causing severe side effects.
Mirkin and his team have spent years trying to solve this problem using their invention: spherical nucleic acids, tiny structures designed to carry therapies to hard-to-reach cells.
“These structures are rapidly taken up by cells—and it turns out, especially by myeloid cells,” he said.
For AML, the team incorporated 5FU into the DNA of the spherical nucleic acid.
“When you restructure it at the nanoscale—from a linear form to a globular, spherical form—it completely changes its properties,” Mirkin said. “It alters where the drug goes, how soluble it is, what it can enter, and ultimately what it can kill.”
In mouse models, the restructured drug avoided most healthy cells while efficiently reaching leukemia cells, penetrating and destroying them.
“I think one of the real tenets of nanotechnology is that everything old becomes new when restructured at the nanoscale,” Mirkin said.
Researchers Tim Kim and Taokun Kuo, who helped create the spherical nucleic acids, said the nano-structured drug increased the power of 5FU by 20,000-fold in mice.
“So we’re actually tricking the cancer cells, making it a nanoscale Trojan horse,” Kuo said.
Observations in the animals were striking. “You look at how the animals fare after injection, and the tumors melt away while survival goes way up,” Mirkin said. “It’s early days, but that really bodes well for taking this into the clinic and hopefully seeing similar effects in humans.”
The lab already has multiple other nano-structured medicines in clinical trials, including cancer vaccines, showing the potential of this innovative approach.
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