Power in a Tiny Package

Nanomedicine could provide cure for some childhood diseases

After years of disappointing battles against such illnesses as sickle cell disease and cystic fibrosis, Dr. Gang Bao, the director of the Center for Pediatric Nanomedicine, sees a small instrument of hope.

Very small.  Really small. Say, about a millionth of the size of a ladybug. But when it comes to nanotechnology, the tiny dimensions are the very reason why it could be such a big success.

Nanotechnology – which has already been implemented in adult medicine – is now moving rapidly into the pediatric realm. And researchers believe it has the capacity to correct such childhood illnesses as sickle cell disease, cystic fibrosis, pediatric heart disease, HIV and cancer.

“I don’t think any of us knew that nanomedicine would become so big,” said Dr. Bao, who is also a Professor of the joint Georgia Tech-Emory Biomedical Engineering department. “And that its potential would be so great.”

In the case of sickle cell disease and cystic fibrosis, researchers are exploring whether it might be possible to simply cut out an identified mutation  – in effect, develop “nanoscissors” to slice out the bad, but leave the good.

Obviously, this has challenges: Researchers have to make sure they don’t cut too much and that they don’t cut in the wrong place. And they’d have to make sure they don’t inadvertently cause another mutation.

And since the work is done “in-vitro” - the classic term for test tube experiments - researchers have to somehow get the repaired cells back in the body – in the right place – before they die in the lab.

Dr. Bao admits these are tough obstacles – but he also believes these can be overcome. “I’m pretty confident” that researchers can find a way, he said.

His colleague, Dr. Nael McCarty, director of the Emory+Children’s Center for Cystic Fibrosis Research, shares Dr. Bao’s enthusiasm. Unlike other therapies, nanotechnology eliminates a critical barrier to finding a cure for CF: The patient’s immune system.

“This approach bypasses the immune system,” he said. “And that, I believe, makes all the difference about whether this could be a successful gene therapy.”

Beyond repairing a patient’s genetic mutations, nanomedicine could also help researchers identify and destroy other diseases’ building blocks.  In cancer, for example, nanotechnology could locate early stage tumors and – in a stunning new theory – find and destroy cancer stem cells.

That would make nanotechnology a powerful weapon of molecular precision – a medical rifle that would do far less damage to healthy cells than the shotguns of surgery, chemotherapy and radiation.

That’s a lot of hope resting on such small particles. But both Dr. Bao and Dr. McCarty believe that is why nanomedicine is critical for pediatric patients.

“As scientists, we want to do cool stuff and make big discoveries,” said Dr. McCarty, also a senior cystic fibrosis scientist with Emory University’s School of Medicine. “But we also never want to forget what a difference it could make to our patients. I truly believe this could change lives.”