I Survived Leukemia Because of a Clinical Trial. Will Future Patients Be So Lucky?

MEDPAGE TODAY ^ | April 15, 2025 | Robert L. Dilley, MD, PhD

[nickcarraway]

Sustained research funding is essential to progress

I don't usually talk about my cancer diagnosis. But as a physician-scientist who survived leukemia at 18 years old and now cares for people facing their own cancer diagnoses, I feel compelled to share my story -- because it speaks directly to what's at stake in medicine today.

The clinical trial that saved my life wouldn't have existed without robust investment in biomedical research. And as debates over federal research funding continue, I think about whether future patients will have access to the same life-saving opportunities I did.

It was the summer of 2007. I had just graduated from high school and was getting ready to start college when I began experiencing vague flu-like symptoms -- low-grade fevers, fatigue, nothing too alarming. My doctor ordered routine bloodwork, mostly out of caution. That same evening, I was admitted to the Children's Hospital of Philadelphia. The diagnosis: acute myeloid leukemia (AML), a rare and aggressive blood cancer that affects approximately eight in a million childrenopens in a new tab or window in the U.S. each year.

I was terrified. But I was also incredibly lucky.

Because I was treated at a major academic center, I had access to a clinical trial testing an experimental therapy -- gemtuzumab ozogamicin, an antibody-drug conjugate (ADC), given in combination with standard chemotherapy for pediatric AML. The trialopens in a new tab or window was led by the Children's Oncology Group and funded by the National Cancer Instituteopens in a new tab or window through its National Clinical Trials Network.

That treatment saved my life.

I started college a year later with a new sense of purpose. I wanted to become an oncologist -- to join the very system that had given me a second chance. Over the past two decades, that goal has shaped my life: medical school, a PhD in cancer biology, residency in internal medicine, and now subspecialty training in hematology and oncology.

Today, I sit across from patients facing the same fear I once did. I counsel them about treatments that exist only because of long-term investment in biomedical research. And I worry that we're taking that system for granted.

Research doesn't happen by accident. Clinical trials don't appear overnight. Every treatment we use at the bedside starts with years -- often decades -- of basic science, drug development, trial design, and infrastructure. That system depends on funding -- and right now, that funding is under threat.

Federal investment in the NIH and FDA -- the cornerstones of American biomedical research -- is facing increasing uncertainty. Yet, the return on that investment has been extraordinary: new therapies, better diagnostics, and improved patient outcomes across virtually every field of medicine.

When I was treated, gemtuzumab ozogamicin was the first FDA-approved ADC. Today, more than a dozen ADCs are approved for various cancers, with many more in development. That kind of progress doesn't happen without sustained research funding.

But most people -- understandably -- only see the final product: the chemotherapy infusion, the pill bottle, the scan result. They don't see the decades of science behind it.

That's why those of us who have lived through the system -- as patients, physicians, and researchers -- need to speak up.

We need to remind policymakers and the public that biomedical research isn't just a noble idea. It's life-saving infrastructure. It's as essential to patient care as any hospital or medication.

When I sit with a newly diagnosed patient today, I think often about what terrified the 18-year-old version of myself. I think about the clinical trial that saved me. And I wonder: Will we continue to invest in science so that the next patient has the same chance I did?

I hope so. But hope isn't enough. It takes action, investment, and advocacy.

Biomedical research saved my life. Now it's up to all of us -- clinicians, researchers, policymakers, and patients -- to make sure it can save many more.

Robert L. Dilley, MD, PhD,opens in a new tab or window is a clinical fellow in hematology/oncology at the Dana-Farber Cancer Institute and Mass General Brigham in Boston.

[nickcarraway]

True. But remember, it didn't have more victims in the United States, because of one woman, Frances Oldham Kelsey. She refused to authorize it in the U.S. because of concerns about the lack of evidence of safety.

But if you read about it, current employees in the 2000s in the FDA are steamrolled by their bosses to approve drugs anyway.

[HollyB]

I did not know that about Frances Kelsey.
We need more like her these days. But, they’d probably fire them.

While undergoing a donor stem cell transplant for AML, I agreed to take part in a clinical trial for a CMV vaccine. It was testing that particular cmv vax on bone marrow/stem cell transplant patients.
This was before COVID and I trusted my dr and figured I was so sick anyway, so what the hell. I actually completed the series of shots.

It never got released. Too many side effects and a couple people died. It may have been due to the transplant. I don’t know if how I felt was due to the shots or just because of what I was going thru.

I also took part in another trial. It was a maintenance chemo I took for 3 additional years post transplant. It had not yet been officially approved for my situation, but was being prescribed via the trial. I’m sure it’s been approved since then as I’ve heard of others taking it.

[b4me]

why is the “research” neglecting food, lifestyle choices, non invasive or made up drugs with their usually harmful side effects?

oh because that might put a bunch of them out of business.

most disease is a lack of some thing the body needs to be balanced and fight it off. I know the body isn’t lacking chemo drugs and such.

so long as our medical community focuses on a body part or specific disease and not treat the body as a whole, many more people will die needlessly like so many already have.

[norwaypinesavage]

The good doctor is conflating conducting effective research, with simply spending lots of money. Most of the government agencies along with the NGOs have become money pits where little effective research is conducted, and much of the money is distributed among cronies, relatives, and friends. A government bureaucracy is simply not an effective way of spending money wisely.

When a government agency is given a budget, and then that budget is increased significantly each year without any meaningful reviews of effectiveness, at some point most of the money gets wasted.