CHICAGO — A big breakthrough in cancer research and it’s all due to a tiny structure. The science was developed in Chicago. They took an old drug and made it better. The restructured drug appears to destroy a common blood cancer in animal models, and it’s poised to reach patients next year.
Chad A. Mirkin, PhD is the Director of the International Institute for Nanotechnology at Northwestern University.
“It really is exciting,” he said. “We figured out how to take two technologies and merge them to create a whole new class of drugs.”
That process started with a commonly used chemotherapy agent 5-Fluorouracil, known as 5FU. The drug is used to treat a variety of cancers, including acute myeloid leukemia, a form of blood cancer.
“One of the problems with that drug is it is horribly insoluble and so it is very difficult to put it into a form where you can deliver it effectively,” Mirkin said. “Second of all, it goes to many different types of cells.”
In other words, like many chemotherapy agents, 5FU impacts healthy cells as it seeks out cancer. That means patients often experience harsh side effects.
It’s the type of problem Mirkin and his team at Northwestern University have tackled for years – how to restructure drugs to make them more effective using their novel invention – spherical nucleic acid, a tiny structure they created to deliver therapies to hard-to-reach cells in the body.
“They have this unusual property that they are rapidly taken up by cells. And it turns out they are rapidly taken up by myeloid cells,” he said.
In the case of AML, the team incorporated 5FU into the DNA of the spherical structure.
“You restructure it on the nanoscale from a linear to a globular form, spherical form, and it completely changes its properties,” Mirkin said. “It changes where it goes, how soluble it is what it can enter, and ultimately what it can kill in this particular case.”
In nano scale, the restructured drug not only avoided most healthy cells, it made it to the leukemia cells more efficiently, where it penetrated and destroyed them.
“I think it’s one of the real tenants of nanotechnology and that’s everything old becomes new when restructured at the nanoscale,” Mirkin said.
Researchers Tim Kim and Taokun Kuo make components of the spherical nucleic acid. In mouse models, the nano-structure helped increase the original drug’s power by 20,000-fold.
“So we’re actually tricking the cancer cells, making it a nanoscale trojan horse,” Kuo said.
“You look at how the animals fare after injected and what you see is the tumors melt away and animals survival go 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 several other nano-structured medicines in clinical trials, including cancer vaccines.
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