Medical forecast: Gene therapy, health information technology boom

What will 2016 bring in the way of medical advances?

As president and CEO of Philadelphia's University City Science Center, an incubator of medical research, Stephen Tang has an uncommon vantage point on that question.

He predicts gene therapy, an experimental technique that uses genes to treat or prevent disease, and health information technology will boom this year. He spoke to us recently about the center and what lies ahead.


Tell us more about the Science Center.

We are a nonprofit, shareholder-owned organization. Our shareholder base is a Who's Who of academic and medical research institutes in the tristate area.

When it was founded in 1963, the original intent was urban renewal on Market Street between 34th and 39th Streets. The goal was to reclaim and repurpose 17 acres of land. Over our 52 years, the science center has run many research labs. But of late, over the last 20 years, we have been in the business of gathering innovators and entrepreneurs through the region. Now, we are probably most well-known for business incubation. At any time, there are 20 or so groups in our facilities on campus.

The point is that the mechanics of taking an idea out of a university and into a commercial setting require you to move off the campus. Typically, the campus labs are federally funded, and you can't do commercialization work in federally funded labs. That is the impetus for this.

The short answer is: We are a convener. We are a promoter. We are in some cases a funder for early-stage ideas into commercialization. Our role is essentially to be the intermediary between the academic world and the industry.


And what you see for 2016 is a boom in gene therapy and health IT?

The overall theme that impacts the application of gene therapy and digital health information is that patients are becoming the center of health care. The traditional health-care system has been designed by the providers and payers of health care. Now, more and more, patients are taking control of their own health care. More information is available. They can go direct to the source. They don't have to go to their doctor to get credible information about their own health.


Let's talk about gene therapy. What do you see happening in that realm?

The industry in many ways is very high risk, very high reward. Essentially, what they are trying to do is reprogram individual patients' cells to the point where the cells begin to fight diseases on their own. When you can link an individual gene to a disease, and you can reprogram a cell, then you have a chance of essentially training those cells to fight disease.

The difficulty is that the immune system is very sensitive to foreign bodies. If your cells start to look like outsiders or invaders, that creates complications. Your immune system might react to it. The industry had to overcome the death of an individual who was receiving gene therapy in clinical trials (at Penn) in 1999.

But the reason there's so much interest in this is that you can essentially create an individual therapy for an individual patient, and you can do it once. Essentially, what that means is you receive therapy once in your life, and you're cured.

Traditionally, the drug industry is looking to serve large populations of patients. But what if you turn that model on its head and say, no, we're going to be developing this just for you? With gene therapy, you're trying to develop therapies for maybe one in a million patients. But how do you get paid for doing that? This is a dilemma in health care.

I think we are close to having critical mass in this highly skilled area. There are, right now, five companies associated with the science center that are all developing different aspects of gene therapy - Spark Therapeutics, AUM Lifetech, REGENXBIO, PhenGen Biotech, and Adaptimmune.

These are all companies that are here on the campus, and they are developing different aspects of gene therapy. This is unprecedented. The importance of this industry to the region is that if we are successful in addressing the challenges, we can easily distinguish ourselves as the place to be - for both researchers and patients.


What about health IT - what you call digital health?

The reason this is important in Philadelphia is that we have such great institutions for both research and clinical applications - Penn, Drexel, Hahnemann, CHOP, and many others - that provide us with an opportunity to really transform health care. Some of the work we're doing in digital health is designed to create applications for patients to take more control of their health care. Some of that is helping patients find the right information. But it's not only about information. It's about compliance - reminding you when to take your medicine, for example. It's about developing devices to help with rehabilitation. Some even involve smartphones for diagnostic tests - the so-called lab-on-a-chip technology, where you can do blood analysis using the computing power of your cellphone.

But we also have better technology for the providers and payers. They can take the vast amount of records and health information and look for ways to analyze that data, and find better ways of managing the cost and delivery of quality health care.

Let's take an example of an orthopedic procedure where you're doing knee replacements. The more information you have about all the patients who have knee replacements, the better chance you have of avoiding complications (in individuals) because you've looked at their demographic information - their height, their weight, etc. - to determine who would be at risk by doing the procedure. You can then make decisions about whether the patient should have the procedure, how long the patient should be in the hospital, what the after-hospital care looks like. The difficulty is that all this information is in silos. The idea is to get access to all this information and aggregate it in such a way that you can actually analyze it.

Overall, what would you say to patients who are hoping for better ways of treating what ails them?

The world is going to be better.