Of Mice and Medicine

To develop new drugs that can battle cancer and reduce disease, Dr. Pablo Lapuerta is unlocking genetic solutions

A major medical revolution is happening in Texas with the help of 5,000 mice. The rodents reside in The Woodlands, Texas, inside a lab owned by Lexicon Pharmaceuticals, Inc. With the goal of discovering new ways to treat human disease, one gene is removed from each mouse, allowing clinical scientists such as Dr. Pablo Lapuerta, executive vice president of clinical development and chief medical officer, to observe the mice and their reactions to drugs. The genetics revolution is changing medicine, and Lapuerta is harnessing its potential to find new drugs to treat irritable bowel syndrome, diabetes, and cancer.

What got you interested in pharmaceuticals?
I come from a family of physicians and always wanted to be a doctor, but I also love how this industry combines science, people, and the enormous potential of pharmaceuticals in terms of collaborations for cures. Before working at Lexicon, I met a CEO who made a real impact on me. We talked about how the next major advancement in AIDS wouldn’t come from one university laboratory but from a huge collaboration with a company investing hundreds of millions of dollars. I wanted to be part of something like that.

And now you are, leading clinical trials at Lexicon Pharmaceuticals.
It’s a dream come true. We work with top medical professionals at leading centers who recruit patients at hospitals or clinics and send us data to analyze with experts in statistics. We then work with global regulatory agencies to ensure our new medicines are safe and effective.

What are the most significant, current advancements in medicine?
Clinical trials are getting bigger and more complicated. With electronics and technology we can share better, more accurate data faster than ever before. Our work is also more international in scope. It is important for professionals to have deep, cultural awareness in this field. Speaking several languages is a tremendous asset. I speak Spanish and French. The real innovations, however, are in genetics, because we are starting to understand the role of genes in the treatment of multiple diseases. A team of scientists recently identified several new genes that have a role in Alzheimer’s disease. This realization was the result of the efforts of neurologists working in many countries around the world, which is amazing. We can accept data generated in one country and analyze it in another. This facility will almost certainly lead to new treatments.

How exactly is genetics leading your work at Lexicon?
The key at Lexicon is our ability to remove just one gene from our mice. We subject them to tests and discover which are resistant or prone to certain diseases. These experiments help us come up with new ways to treat people.

Through drug development?
Yes. A mouse resistant to diabetes lacks a protein involved in absorbing sugar from the stomach, so we’ve developed a drug that inhibits that protein in humans. We’ve tested it in hundreds of people and found that it lowers glucose and blood pressure. Patients are losing weight and getting healthy. Advancements in information technology are also changing our procedures. In a recent diabetes study, we received information about glucose directly from patients through wireless devices on a daily basis. This made the clinical trial safer and more effective.

What else are you working on?
We’re in phase two of a study on a drug to treat irritable bowel syndrome, and we are trying to treat carcinoid syndrome, which is caused by a metastatic cancer that secretes serotonin.

When you observe these mice, are you looking for something specific or are you connecting the dots for a cure?
We apply our observations to our knowledge of medicine. We had a mouse that produced no serotonin in the stomach. It was missing the right enzyme for production. We were impressed to find that the mouse was completely healthy. From that observation, we were able to identify the issue in carcinoid syndrome and embark on a clinical program to give human patients a molecule to slow down serotonin production from the cancer. It seems to be working and we have taken it into phase three.

What are the main issues in your industry from a Latino perspective?
I’m glad to be working on diabetes because this issue impacts the Latino community at a high rate. Diabetics often require insulin, but I hope our pill can reduce and simplify that, which would have enormous value in the Latino community.

Where is the industry headed in the next three to five years?
More technology will be available. We’re also treating an aging population. We’re going to see more diabetes, more hypertension, and more obesity. We used to be reluctant to have older people in clinical trials, but now we want them. We know we can treat them safely, and we actually need them, so we can find ways to treat the issues that will face us as a community in the coming years.