By E. Anders Kolb, M.D.
Welcome to the latest edition of my Behind the Breakthroughs blog series, where I speak with experts across the blood cancer ecosystem who are driving, supporting, or benefiting from game-changing research. These discussions are designed to pull back the curtain on the long arc of discovery, to show what those breakthroughs truly mean for patients and their families.
This month, I’m excited to share highlights from my conversation with two remarkable voices representing the impact of precision medicine—Brian Druker, MD and Mel Mann, MBA, M.Ed., who spoke recently to Blood Cancer United staff.
Meet Brian Druker, MD
Dr. Druker is a pioneering clinician-scientist whose work transformed cancer treatment. His role in the development of imatinib—now known as Gleevec—helped launch the era of precision medicine. As the first therapy designed to specifically target cancer cells while largely sparing healthy ones, Gleevec dramatically changed the trajectory of chronic myeloid leukemia (CML). Where a diagnosis once meant a life expectancy of just three to five years, patients today can often manage CML as a chronic condition.
The impact of that breakthrough is profound. Since Gleevec’s FDA approval 25 years ago in 2001, it has contributed to more than a million years of life gained for people living with CML and changed the way we think about treating cancer generally. Blood Cancer United is proud to have provided Dr. Druker with a grant early on in his research 30 years ago and supported the foundational science that led to his discovery.
Today, precision therapies are the norm in cancer treatment and in treating other diseases as well. Dr. Druker set a powerful example of what’s possible when science, persistence, and purpose come together to benefit patients.
Meet Mel Mann, MBA, M.Ed.
Mel Mann is living proof of that impact.
At age 37, Army Major Mel Mann had a lot of life ahead of him. That changed in an instant when he was diagnosed with CML, and told he’d likely live only three more years. Mel enrolled in a clinical trial at MD Anderson Cancer Center in Houston, where he was the second person there treated with imatinib. The daily oral treatment has kept Mel in remission for three decades. Those added years represent far more than time. They are moments with family, friendships sustained, and possibilities once thought out of reach.
Together, Dr. Druker and Mel Mann offer two perspectives on the same breakthrough—one from the lab and clinic, and one from experience.
My conversation with them explored that journey—from the early days of discovery to the real-world impact still unfolding today.
Targeted therapy changes the course of cancer treatment
E. Anders Kolb, MD: Dr. Druker, take us back over 30 years ago when you were working on this drug. What on earth made you think you could turn a cell off with a drug?
Brian Druker, MD: There had already been 30 years of basic science studying the things that drive the growth of cancer: from the discovery of a genetic abnormality called the Philadelphia chromosome in 1973 by Janet Rowley, MD (one of my true heroes); to the work of George Q. Daley, MD, who's now the Dean of Harvard Medical School, and Rick Van Etten, MD, who's now the Cancer Center Director at UC Irvine, who showed that if you put an activated enzyme called tyrosine kinase into mice, they got chronic myeloid leukemia. I looked at that, and it felt obvious: This causes leukemia. If we shut it down, we might be able to treat this leukemia more effectively.
At that time, in 1996, funding rates from the NIH National Cancer Institute were in the single digits, much like they are today. And my lab was hanging on by a thread. I was on the wards one day, and I saw this flyer from The Leukemia & Lymphoma Society (now Blood Cancer United) announcing its inaugural translational research grants. It said: “We believe that researchers could have drugs in the lab that are ready to move into the clinic, and we want to provide you with some funding.” I got that first grant from your organization. It was a lifeline to my lab, and it allowed us to get into clinical trials in 1998. And the rest was history.
Learnings for other cancers
EAK: When you think back to those early days and the lessons learned, how have those learnings guided your career? And how are you applying them today in your role as lead investigator for the Beat AML master clinical trial, as we work to bring that same progress to one of the most aggressive blood cancers?
BD: We always start in our lab by collecting patient samples, trying to understand what is driving the growth of their cancer. And that was the impetus behind the Beat AML trial, to figure out all the molecular abnormalities driving AML and match patients with more effective therapies.
Gleevec was the first targeted cancer therapy, but today there are literally thousands of targeted agents—like menin inhibitors; IDH inhibitors; RAS inhibitors for pancreatic cancer, but also for people with RAS mutations in myelodysplastic syndrome and AML that we’re going to get to use.
So, the whole idea behind precision oncology targeted therapy is to match the patient with the right drug that’s going to work for them most effectively.
And we're seeing remarkable results. We recently presented on an LSD1 inhibitor with venetoclax and Azacitidine with nearly 100% response rate in AML. That's how far we've come. And these are projects that Blood Cancer United is funding. And you continue to fund breakthroughs.
Helping patients live longer better lives
EAK: I love to hear how precision medicine has set the stage for new and future discoveries. But science is only half the story. The other half is the life it made possible.
Mel, when you were diagnosed with CML, there weren't any good options. Then, you tried Gleevec and gained so much more time. When you look back, not knowing that you were at the dawn of this new era of precision medicine, what does it mean to you and to patients today?
Mel Mann: Well, for me, it means hope. Because at the time, CML was fatal. At diagnosis I’d been told I only had three years, and I was already past that time. So, the clinical trial offered a bridge between despair and hope.
And, today, we not only have Gleevec, but we have second and third generations of targeted therapies. So, if one thing doesn't work, something else will. And we have great doctors continuing to do the research that just speeds up progress for patients.
BD: Mel, it's just so great to see how well you're doing, and to realize that you are the longest surviving patient from the Gleevec clinical trials back in August 1998.
And this is really what it's about: impacting patients' lives and seeing what they do with the time they thought they might not have. Mel has run marathons, and celebrated many more birthdays, anniversaries, and graduations. He’s seen his daughter become a doctor. And just to hear those stories is the greatest possible reward for the work we do.
Why clinical trials matter
EAK: Mel, you talked about that bridge between despair and hope. You enrolled on a precision medicine clinical trial when there weren't second, third, fourth generation drugs. Take us back to that moment.
MM: Well, there had been rumors before the trial started that there was a medicine that was going to go where the bad cells are and turn off that switch without hurting the good cells. That was mind-blowing because the other available therapies at that point were bone marrow transplant and daily injections of interferon, which were going to fail eventually. This option came without all the side effects, like nausea and losing your hair, so I was just happy to do it. I felt very blessed to get into the trial.
For me, it was an opportunity to get tomorrow's medicine today. There were no promises, but the trial offered a chance to live. Of course, there was some uncertainty because that’s part of being in a clinical trial. But I tell people, “You know what your options are. You’ve got things that aren’t working and then you’ve got a chance to try something that could be a gamechanger.“ I’m Exhibit A for what that could look like. It put my cancer in remission, and I’ve been in remission ever since.
"It was an opportunity to get tomorrow's medicine today."
Mel Mann
What “more time” really means
EAK: At Blood Cancer United we have this bold goal to enable patients to gain more than one million years of life by 2040. We want to give patients and their families and friends more time together. Mel, what does more time mean for you?
MM: Well, my daughter was five when I was diagnosed, and she grew up and became a physician, and I got to see all of that. I didn't miss that show. And I could have. I could have missed her graduating from elementary school. At the time I was diagnosed, I had been married to my wife, Cecilia, for 10 years and now we’re married 42 years. So, I got a whole lot of extra time with her.
But it's not all about those big occasions. Sometimes I'm driving down the road, and it’s raining. I appreciate those days, too. I have a different relationship with time now, and I just appreciate the joy of the moment. I don't rush through it.
Barriers to accessing care
EAK: We know that despite scientific discovery, there are still obstacles that can keep breakthroughs from reaching patients who need them.
Mel, you've been a volunteer advocate and talked to many patients. What do you see as some of the biggest barriers for patients getting access to new therapies?
MM: Well, first they must know about them. Then they must be asked to join the trial. There’s also the financial part. Blood Cancer United has done a great job advocating in Congress and state governments for policy changes that help more patients access and afford the treatments they need.
EAK: Dr. Druker, is there one bottleneck that you'd like to remove most?
BD: Right now, funding is the big bottleneck. My faculty members are spending way too much time writing grants. It's almost like holding a winning lottery ticket to get funding. We definitely need a more sustainable source of funding.
We're at a time where we can accelerate progress like nobody could imagine just a few decades ago. But we're struggling to get enough funding. Research funding absolutely needs to be lobbied for. And this isn't a partisan issue. Everybody in their family has had a brush with cancer.
This is an area where Blood Cancer United has always been ahead of the curve in figuring out where these blocks are and addressing them through advocacy and other ways, like the Therapy Acceleration Program (TAP) , that helps biotechs get funding to get promising drugs out of labs and into the clinic.
Because many of the blood cancers are relatively rare diseases, drug companies aren’t likely to invest. And that's where funding from Blood Cancer United can be incredibly helpful in getting that proof of concept. Because once you've got that, you've de-risked the drug, so that it can get developed more quickly. That is a huge area where Blood Cancer United has stepped in to help.
"We're at a time where we can accelerate progress like nobody could imagine just a few decades ago. But we're struggling to get enough funding. Research funding absolutely needs to be lobbied for. And this isn't a partisan issue. Everybody in their family has had a brush with cancer."
Brian Druker, MD
Hope for the future
EAK: Dr. Druker, if we fast forward ten years, what's the next Gleevec-like leap that you're most hopeful for in blood cancer?
BD: Well, there are still some big gaps in survival in AML, and we have some significant work to do there. But we're learning about some early indicators in people who are at risk of developing AML or other blood cancers that we might be able to stop in their tracks before it happens. And in smoldering myeloma, which we know will turn into myeloma, treatments are being developed to prevent that from happening. These are the things that are coming along in the next 10 to 15 years. The hope is that we might even be able to prevent people from getting AML or other blood cancers. And that’s where we really want to be.
This conversation is for general education and discussion of research, innovation, and patients' needs. Blood Cancer United does not endorse any specific company, product, treatment, or clinical trial, and treatment decisions should be made between patients and their care teams.
- Do you need help finding a clinical trial? Contact our Clinical Trial Support Center.
- Want to help fund research and support services that help patients like Mel? Donate here.
- If you missed my first Behind the Breakthroughs blog, you can read it here.
About the author
E. Anders Kolb, MD, a 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 200 articles in peer-reviewed journals and received numerous awards.
About Brian Druker, MD
Brian Druker, MD., CEO of the Knight Cancer Institute, revolutionized the treatment of cancer through research that resulted in the first drug to target the molecular defect of a cancer while leaving healthy cells unharmed. Marketed under the name Gleevec®, his discovery turned a once-fatal cancer, chronic myeloid leukemia, into a manageable condition.
Treatment with Gleevec received FDA approval in record time, was featured on the cover of Time magazine, and established Druker as a pioneer in the field of precision medicine. Most important, his discovery became a new proof of principle for targeted therapies, spurring the development of more than 50 similar precision therapies for other cancers.
Dr. Druker has been recognized with numerous awards, including the Sjöberg Prize from the Royal Swedish Academy of Sciences, the Warren Alpert Prize from Harvard Medical School, the Lasker-DeBakey Award for Clinical Medical Research, the Japan Prize in Healthcare and Medical Technology, the Tang Prize in Biopharmaceutical Science, and the Prince Mahidol Award for outstanding achievements in medicine and public health worldwide. He has been elected to the National Academy of Medicine, the National Academy of Sciences and the American Academy of Arts and Sciences.
Read more about Dr. Druker here.
About Mel Mann
Mel Mann is a retired Army Major who was diagnosed with chronic myeloid leukemia (CML) at the age of 37. At the time, CML was a grim diagnosis, and Mel was expected to live just three years. But that’s not what happened.
Mel enrolled in a clinical trial at MD Anderson Cancer Center in Houston, where he was the second person there treated with imatinib (now marketed as Gleevec). The daily oral treatment has kept Mel in remission for three decades. He calls himself the world’s longest living Gleevec survivor.
Mel has been featured in countless media outlets, speaks nationwide about his experience, and is a volunteer advocate and patient ambassador at many healthcare organizations, including Blood Cancer United.
