Local Student Wins Prize For 'Humanized' Mouse

A local graduate student has won a prestigious science prize for a technology she created that could lead to faster and safer drug testing. Alice Chen is a doctoral candidate in a joint program in biomedical engineering at Harvard and MIT. On Wednesday, she was named the winner of this year's Lemelson-MIT Student Prize for developing a humanized mouse with a tissue-engineered liver. Wondering what exactly that means? Well, Chen explains her award-winning science in layman's terms to WBUR's All Things Considered host Sacha Pfeiffer.

Sacha Pfeiffer: You've won this prize because you've created what's being described as a "humanized mouse" with a human liver, essentially. What is this humanized mouse?

Alice Chen: It's a laboratory animal that has a tissue-engineered human liver implanted inside of it, which makes the mouse act much more like a human patient would in the way that it metabolizes drugs as well as responds to certain liver diseases.

So in the first stage, or in an early stage, of testing a drug, the drug is often tried out on an animal. But the way the animal reacts might not be quite how human reacts, and that's problematic.

That's exactly right. And the drug is metabolized primarily by the liver — the liver is the organ that metabolizes most drug compounds — and it turns out that animal livers and human livers are quite different. When we test drugs on animals, sometimes the animals do predict the way humans will react.

Such as what types of side effects a human might have?

Right. What side effects, what metabolites are formed, how different drugs that are prescribed in combination might affect a patient in the clinic and beyond. What happens now in the way drugs are developed, unfortunately — even though we do the best we can in trying to identify potential dangers — is that we see in clinical trials some unpredicted drug dangers emerge, and we also see it in hundreds of thousands of hospitalizations that occur.

You see unpredicted dangers in human clinical trials that didn't show up when you were testing the drug on the mouse?

That's absolutely right, which obviously is a problem. It's incredibly dangerous and it's also debilitating to the drug development pipeline, meaning that drugs that really need to get efficiently to the patients that need them aren't getting there because the drug is halted in its manufacturing.

But the reality of the drug testing process is that most drugs are tried first on animals.

They absolutely need to be tried on animals so that things like how to dose in patients, and how other organs might be affected, can also be tested. But the human liver is very special, and the mouse cannot always easily mimic the way the human liver will break down a drug.

How far away do you think this new technology — meaning this new mouse — is from being used in actual drug development? Because it's not there yet.

Right, it's not there. But what's great is that I think these large pharmaceutical companies, and also some start-up companies, are really looking into humanized mouse models to better recapitulate human diseases and drug metabolism. The standard method of doing that right now is through genetically engineered mice. So there's a huge push to doing this in order to create new tools for studying human disease and treating disease. There's a knowledge that we really need better tools. But these genetically engineered mice really can be improved by these tissue-engineered approaches because they're simpler and they're easily implemented in mice; it's a simple surgical implantation of this tissue-engineered liver.

Does the mouse survive even after the surgery?

The mouse survives nearly 100 percent of the time. And, again, these are animal studies that are being done already, but implanting this simple device makes the animal study just more efficient and more effective.

Alice, you're 29 years old, you're expecting your Ph.D. this month from MIT, and as part of this prize you've just won you're going to get $30,000. Do you know what you'll do with that money?

Yes, I have a good sense of what I'll do with that money. I will be investing it into a company that I co-founded with fellow graduates from MIT. The company is called Sienna Labs. It's a different technology, but it's similarly a technology that is bioengineered to improve the efficacy of medicine, and specifically laser medicine. So we're very excited about our rapid path towards commercialization in this next year or two, and I'd really like to put the money to that and make sure that's successful.