By E. Anders Kolb, M.D.
When you hear the word breakthrough, you might imagine a single eureka moment—a discovery that suddenly changes everything. But in blood cancer research, and across medicine more broadly, breakthroughs don’t happen overnight. True breakthroughs are the result of years—sometimes decades—of careful study, persistent scientists, and steady investment to move from the lab to the clinic, and ultimately to patients who need better treatment options.
That’s why I’m launching this new blog series, Behind the Breakthroughs. In each installment, I’ll sit down with a researcher, clinician scientist, or expert in pharma and drug development to explore how progress really happens. These conversations are meant to pull back the curtain on the long arc of discovery—and highlight why consistent support for research matters at every step.
Today I’m pleased to kick off the series with Carol Gallagher, PharmD, someone who has seen drug development from just about every angle. Over three-plus decades, Carol has been involved in the development of five approved therapies, advised major pharmaceutical companies and academic institutions, and served on numerous boards—including Blood Cancer United’s national board. In our conversation, Carol brings a rare combination of scientific depth, business acumen, and hard-earned perspective.
Meet Carol Gallagher
E. Anders Kolb, MD: How’d you get started in this field, and how has your career evolved?
Carol Gallagher, PharmD: I knew I wanted to do hands-on science, but I was also interested in the business of science. I wanted to understand how you translate basic biological research into a medicine to benefit patients. My career has involved working on a number of drugs that made it to approval and interacting with teams trying to advance innovations.
I started out at Eli Lilly and then Amgen, which had just launched a drug called Neupogen, an important supportive therapy in cancer. I worked at several pharmaceutical companies, big and small, including IDEC, the inventor of Rituxan, a monoclonal antibody that treats lymphoma and chronic lymphocytic leukemia (CLL). There I worked on further developing Rituxan for certain auto immune diseases. It’s a good example of how foundational immunology can lead to multiple therapeutic opportunities. I eventually became CEO of a small biotech company, where we developed a drug for CLL and indolent non-Hodgkin lymphoma that was approved in 2014.
I also spent ten years at a venture capital firm, looking for opportunities to invest in biotech companies, and then sitting on the boards of those companies to help them make their way. I semi-retired a few years ago, but I still do board work with early-stage companies trying to develop new therapies. The cherry on top has been joining the board of Blood Cancer United. It’s a way for me to give back and pass along some of the things I’ve learned throughout my career.
The Long Arc of Discovery
EAK: How does progress toward a drug evolve over time? What are the different touchpoints that make innovation possible?
CG: Developing a transformative treatment is a building block process that can usually take decades. Each new discovery builds on previous discoveries. We stand on the shoulders of the scientists and researchers who came before us.
Each new discovery builds on previous discoveries. We stand on the shoulders of the scientists and researchers who came before us.
I like to use the story of Gleevec as an example of how a transformative drug goes from basic research to development to clinical trials and finally to FDA approval. Approved in 2001, Gleevec was the first targeted therapy for a specific genetic mutation in chronic myeloid leukemia (CML).
But the foundational research for that drug dates back to 1979. That’s when Tony Hunter, Ph.D., a scientist who’s still doing work at the Salk Institute, published a paper that described the basic biochemistry of how tyrosine kinases (the chemical target that Gleevec inhibits) work in the cell processes. At that time, we didn’t know what that discovery might mean for medicine. We needed to understand that biochemistry before we could even imagine what might be possible.
Nineteen years later in 1998, Gleevec went into clinical trials led by Brian Druker, MD. Dr. Druker’s work would not have been possible without Dr. Hunter’s basic research, and others who built on it. It took time to learn that if we could knock down that single mutation with a drug, we could potentially get a lot of clinical benefit for people with CML. It was 2001 before Gleevec got approved by the FDA—22 years from Dr. Hunter’s foundational research. And that’s considered fast. Many drugs can take 30 years or more to go from basic research to approval.
EAK: As you know, Blood Cancer United was instrumental in advancing Gleevec, both through funding to Dr. Druker and the research that came before. When I was early in my career, the therapies to control CML were interferon and hydroxyurea. Both were only transiently effective and quite toxic. Transplant was a curative option available to a small subset of patients. Today, if you are diagnosed with CML, your chances of a relatively normal life are outstanding. This is a great testament to why early and sustained funding for research is so important.
CG: And it’s a good illustration of how targeted therapies like Gleevec help us become more selective in our treatments so that we can avoid some of the side effects of earlier therapies. But to get there we really had to understand that molecular biology, which goes back to the basic science that Dr. Hunter and other people in academia do.
Validating the potential of a great idea
EAK: I’ve heard you and others familiar with the venture capital world talk about the “valley of death” that can exist between a great idea and a commercial drug. Once you have a promising idea, what happens along the way? What are the things that might prevent drugs from ever getting out of that valley of death and to the public?
CG: OK, so we have the basic science, and a disease setting in which it could be helpful. Now, a pharmaceutical or small biotech company decides the science is far enough along to start a discovery program to figure out how to “drug” that science. That involves a lot of trial and error and asking questions like: How will the medicine work to enable that basic science? What’s the right potency? How do we get the drug to the right place in the body? Can we give it orally?
It can take several years and millions of dollars to go from foundational science to a drug candidate that we can then put into animal studies. If it looks safe enough, potent enough, and effective enough in animals, the next step is testing it in humans in clinical trials.
Clinical trials are an important step to validating a potential innovation, but that means now we actually have to make the drug, which can cost several million dollars more, which can be prohibitive for a small biotech company. Thank goodness for donors, partners, and investors who help fund the trials, the investigators who run them, and the patients who are willing to enter them.
If we see response rates greater than 30% in those early clinical trials, we have evidence of benefit and are likely to have a drug that can one day get approved. When we start to see these responses, it is very gratifying and everyone on the team is highly motivated to get this new medicine to as many patients as possible, as soon as possible.
There are so many amazing stories of patients who were out of treatment options, and they go on the clinical trial, and they respond. And they live months or years longer.
But unfortunately, basic research doesn’t always translate well to humans, for many reasons. It might be safety. Maybe we need a more potent drug. Maybe the mechanism just didn’t turn out how we thought. In the early part of my career, I worked on a drug that did well in pre-clinical testing, but we just couldn’t get enough clinical benefit—the response rate wasn’t enough to see tumors shrink. Our hypothesis sounded good but didn’t actually work out. So, the program had to go away. And of course that was quite disappointing.
And sometimes a promising drug may not make it through clinical trials because of lack of funding, or because a smaller company may not have the range of expertise on staff that’s required to navigate all of the different challenges in drug development. That can be the valley of death right there.
The importance of sustained funding and drug development expertise
EAK: I want to make sure that our readers can fully appreciate this. When we’re talking about drug development, we’re talking about anywhere from a $20 to $70 million dollar investment in an experiment that may or may not lead to drug approval. I’m sure that is mind-boggling to many. But without that investment, you’ll never get the learnings that can lead to a potentially groundbreaking drug. So steady funding support is critical. And when you get to the clinical trial stage, it’s also vital that biotech companies have thoughtful leadership, a good development plan, and experts in their corner informing them. Drugs can die from insufficient funding or insufficient expertise.
CG: One of the things about Blood Cancer United that’s so amazing to me is that we’re there at every step. We’re there to fund a range of early science through academic grant giving to find ideas with the best chance of success. And we advance clinical trials and accelerate progress through our sponsored master trials—Beat AML and PedAL—and through the Therapy Acceleration Program (TAP), our venture philanthropy initiative. Through TAP, we can connect biotech companies with a network of experts to help them make it through the valley of death.
Accelerating progress through venture philanthropy
EAK: You’re on the committee that oversees and guides TAP. What drew you to TAP’s model and how does it work?
CG: I’ve seen close up the innovation that can come from small, focused teams in early-stage biotech and the venture capital funding model that enables this innovation. In my long career, I’ve also observed how Blood Cancer United has brought together networks of researchers and clinical trialists with early stage biotechs.
TAP invests in biotech companies developing potentially groundbreaking therapies and helps accelerate those treatments. TAP provides more than just dollars—we bring scientific expertise and years of leadership to develop a powerful network that researchers can tap into.
TAP has invested $115 million to date to accelerate clinical development of transformative blood cancer drugs, resulting in six approved therapies. Any financial returns are put toward funding the next innovations. So, if you’re a donor investing in our TAP fund, your philanthropic dollar is being recycled over and over again to fuel the next breakthrough for patients.
EAK: How do you evaluate which ideas need acceleration through TAP funding?
CG: As TAP looks for opportunities to invest, it always starts with the science. We look for a solid hypothesis. Then, how doable is the clinical trial–in both dollars and time? Is there a patient population that’s going to benefit, and can we find and enroll them? In ultra rare cancers, there may only be a few thousand patients a year being treated, so how do we find even 50 to get into the clinical trial? We spend a lot of time talking to investigators who lead the clinical trials and asking these questions.
Lastly, does the company have the right team with experience in drug development, designing clinical trials, and working with the FDA? It’s all very complex and the team is very important.
What’s great is that we can help companies through our TAP network. We make sure they are working with the right people and getting the right advice. I always say, drug development takes a whole village with our patients at the center.
Providing hope for today and the future
EAK: What excites you most about where blood cancer research is headed?
CG: Every day we learn more about blood cells and bone marrow and the immune system, which helps us better understand potential targets for treatment with new and maybe even existing drugs. We also have more treatment options in our toolbox— from small molecule drugs that are given orally, to antibody immunotherapies, to cell therapy where a patient’s own immune system is given the tools it needs to recognize and kill cancer cells. It’s an exciting time to think about further innovations for patients so that we can keep extending their lives and helping make the quality of their time better.
About Carol Gallagher, PharmD
Carol Gallagher, PharmD has over thirty-five years of experience in the Biopharmaceutical industry in both business and drug-development roles, as a CEO, and as a venture capital investor. She has been involved in the development of five approved therapies. She has also served as an advisor to large pharma and academic institutions. Currently, she serves as chairman or as a director on several biopharma boards. In addition to Blood Cancer United, her philanthropic interests include serving on the board of trustees at the Salk Institute and as a board member of Life Science Cares San Diego. She previously served on the board of Sharp Healthcare which is an integrated healthcare provider in San Diego. Dr. Gallagher attended Vanderbilt University and received BS and Doctor of Pharmacy degrees from the University of Kentucky College of Pharmacy.
About the author
E. Anders Kolb, M.D., a world-renowned pediatric hematologist oncologist and researcher, is president and chief executive officer of Blood Cancer United, formerly The Leukemia & Lymphoma Society (LLS), a global leader in the fight against cancer. Dr. Kolb leads the mission and operations of this $500 million cancer patient advocacy agency with offices serving patients and families across North America.
Dr. Kolb has devoted his life's work to caring for children with pediatric blood cancer and conducting research to find cures. Before joining the organization, he spent 15 years at Nemours Children's Health, where he built the Blood and Bone Marrow Transplant Program and most recently served as chief of the division of hematology and oncology, director of the Moseley Foundation Institute for Cancer and Blood Disorders, and vice chairman for research in the department of pediatrics at the Sidney Kimmel Medical College at Thomas Jefferson University.
Dr. Kolb has authored or co-authored more than 150 articles in peer-reviewed journals and received numerous awards.
