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Hannah Song, PhD
Contributing Editor, ISCT Telegraft
NIH Clinical Center
United States

Dr. Edwin Horwitz, MD, PhD, has the distinction of being one of ISCT’s most impactful leaders. He was a founding member of the MSC Scientific Committee, an ISCT Treasurer, and the ISCT President from 2010-2012. He is a Professor of Pediatrics and the Marcus Professor of Cellular Therapy at the Emory University School of Medicine, and he currently serves as a member on ISCT’s Board of Directors and the Editor-in-Chief of ISCT’s journal, Cytotherapy. 

Read on to find out about his incredible contributions, what gives him optimism as the field progresses, and his words of advice for the next generation of leaders. 

Tell us about how you got into cell and gene therapy initially, and your personal contributions to field? 

During medical school, a professor once tried to convince me to go into pathology by suggesting, “Wouldn’t you like to have a disease named after you?” And I said, “Well, no actually – but I’d like to have a cure named after me!” That was the light-bulb moment that I needed to study therapeutics. Cell and gene therapy seemed like the right fit.

I told myself that when I start my career, I want people to think about a disease or treatment differently when I end my career. I want to change the way we think about things. And infusing mesenchymal stromal cells (MSCs) into kids with Osteogenesis Imperfecta (OI) has accomplished that. We now consider MSCs as a bona fide therapy for OI, and there’s two companies trying to commercialize MSCs for OI which I think is fabulous – because this is an orphan disease and the market is small.

I also helped coin the term “mesenchymal stromal cells.” Everyone called them stem cells before that, but I didn’t believe they’re stem cells, and even to this day it’s controversial. In 2005 I wrote an opinion piece in Cytotherapy – and it’s caught on quite a bit. I was also the first to infuse allogeneic MSCs into a patient, and the first person to infuse genetically modified MSCs, so I think I’ve accomplished what I set out to do. I’m very gratified with my career, very grateful for the opportunities I’ve had.

Take us back to 2010-2012 when you led ISCT as president. What was going on in the society at the time, and what do you consider your most impactful contributions?

Two things, really. At the time there was a great need but very little being done to foster academic-industry partnerships. You know, cell therapy is expensive, so you have to partner with industry to bring an idea to fruition; there’s simply no other way to do it. So, I said, let’s bring industry into ISCT. There needed to be some place to bring everybody together. That was one of the big initiatives I wanted to accomplish. We’re both highly motivated to work on the same problem – for industry, that’s the way their business will flourish, and for me, an academic, that’s the problem that I am currently trying to solve. At the end we have the same goal, but we need each other to be successful. I really believe that.

Second, what drew me to ISCT to begin with was the international nature, you can make friends all over the world. I’m a globalist; I think we’re all in this together. At the time the society really wasn’t international, it was US, Canada, and a small group of Europeans. It wasn’t a powerful international society. I said, we’re going to change this, and we’re going to make it truly global. Nobody’s special, everybody works together as a team, and everybody’s important. We increased our membership in places like Europe, South America, Asia, and Australia, achieving my vision of being a truly international society.

What are some of the biggest hurdles you see right now in the field of cell and gene therapy?

I hate to say this, but it’s just that money is getting very, very tight. I don’t think big pharma can drive the field right now, I think it’s got to be small biotech startups that will move the field when they hit it big. And where do biotech startups come from? Typically, academia. So, the pipeline has to be academia to biotech to big pharma – and to do that you need money for biotech and academia to do their work. That is a huge problem.

I don’t think funding agencies understand how expensive it is to get cell and gene therapies off the ground. A lot of funding agencies want to fund the clinical trial, but not all the things you need to get to be able to open the clinical trial. We call the IND-enabling studies the “valley of funding death” because no one wants to pay for it, but you’ve got to do it. So, it really comes down to investment in research. I think there should be far more investment in science, all kinds of science.

What gives you the most optimism about the future of the field?

The die-hards in the field are absolutely committed to it. How many times has the field of cell and gene therapy been pronounced dead? But the die-hards, the people who really believed in it, kept going. That’s why I think we can get there. Of course there will be attrition, and not everything will work out, but there will be a group that will keep moving forward. I really believe this will define medicine over the next 50 years.

We need the people who are motivated to get into the clinic fast – and not afraid to fail. If you’re afraid, you’re never going to do it, because let’s face it when you do something for the first time you never know. You can have the best animal models in world, but you really don’t know what will happen in humans until you test it in humans.

For example, I think that CAR-T cells for solid tumors will start working as well as for liquid tumors – a lot of very smart people are working on it, and they’re determined to get it done. A post-doc when I was a graduate student once told me, “We can do anything – the question is are you smart enough to figure out how to do it.” I really believe that. 

Think of it this way – every bone marrow transplant center is now a bone marrow AND cell therapy center. Everyone claims to be in cell therapy. Twenty years ago, that wasn’t the case. At that time if you told someone “I study cell therapy,” people would ask “what’s that?” Now everyone wants to be a cell therapist, because it’s cool. We’ve beaten the stigma and over time we will have a major foothold in the industry of therapeutics.

How does ISCT bring together the "die-hards"?

One of the problems is that we’re far apart from each other. We need a mechanism to come together – and that breeds friendships and collaborations. That’s what ISCT does. The science is fantastic, and you learn so much, but one of the greatest things about these meetings is that it brings together people from all over the world. We need a forum to bring us all together – manufacturing, regulatory, industry, academics, clinicians – all in one space to learn from each other. 

What would you say to the next generation of leaders?

Never, never, never give up. That’s Winston Churchill, actually. But I mean that sincerely. 

Second, get involved with people who will help you grow. You don’t want to be in an environment where you’re the expert, because frankly at this stage you are not. Once you’ve been around ten or fifteen years and you want to go someplace where you can be the expert – that’s great. But when you’re young and getting started, you need people who are accomplished and want to help you. You need to find a mentor who knows what they’re doing, who is willing to share their knowledge, and is willing to help you grow.

What are you looking forward to at the ISCT 2026 Scientific Annual  Meeting?

It’s the same thing I’m excited about every meeting - seeing my friends. The science is always good, the presentations are going to be fantastic, but I go to see my friends from all over the world in one place.

—As told to Hannah Song, PhD, and Nazanin Tatari, PhD