Without this pill, lots of people would be dead

Summary

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Short Wave: "Without this pill, lots of people would be dead"

Air Date: May 11, 2026
Host: Emily Kwong
Guest: Sydney Lupkin (NPR pharmaceuticals correspondent)
Length: ~15 minutes

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Overview

This episode of Short Wave celebrates the 25th anniversary of the FDA approval of Gleevec, a pioneering cancer drug that transformed the outlook for patients with chronic myeloid leukemia (CML) and set the stage for targeted cancer therapies. Through patient stories, scientific breakthroughs, and industry cooperation, the episode explores how Gleevec changed not just one disease, but the entire approach to cancer treatment.

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Key Discussion Points & Insights

The Impact of Gleevec (00:16–03:58)

• Milestone Anniversary:
  Sydney Lupkin introduces Gleevec’s 25th anniversary since FDA approval (00:44).
• Why Gleevec Matters:
  Gleevec was among the first targeted cancer therapies, making previously fatal cancers survivable (00:54).
• Patient Story – Mel Mann:
  Diagnosed with CML in 1995, given 3 years to live; unable to find a bone marrow donor due to low match rates for Black patients. Participated in clinical trials and eventually was helped by Gleevec (01:06–03:28).
  • Quote - Mel’s daughter at a bone marrow drive:
    “Daddy, I can’t see why you can’t find a match. All the blood looks the same to me.” (02:14)
  • After starting Gleevec in 1998, Mel was running a marathon less than a year later.

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The Origin Story of Gleevec (05:04–07:12)

• Innovative Thinking:
  Oncologist Dr. Brian Drucker envisioned targeting the root cause of cancer cell growth rather than using broadly toxic chemotherapy (05:13–05:55).
  • Quote - Brian Drucker:
    “My view was there had to be a better way, and that better way had to be based on a knowledge that was driving the growth of a specific cancer.” (05:47)
• Scientific Breakthrough:
  The discovery of the Philadelphia chromosome identified a genetic cause for CML and enabled a targeted therapeutic approach (06:04–06:38).
• Professional Risks:
  Drucker left his Boston job to pursue the idea at Oregon Health & Science University due to skepticism from colleagues (06:51–07:07).

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Gleevec’s Clinical Trials and Approval (07:12–10:11)

• First Experiments:
  Upon moving to Oregon, Drucker quickly began testing compounds and identified what became Gleevec, initially discovered by scientist Nicholas Lydon (07:12–07:31).
  • Quote - Brian Drucker:
    “I wasn’t just a researcher. I also had patients... It allowed me to have the courage to be their mouthpiece and voice to lobby, to get this drug into clinical trials.” (07:31)
• Innovation in Trials:
  The clinical trial for Gleevec only included patients with CML, rather than grouping cancers together as was common at the time (08:05–08:21).
• Unprecedented Results:
  Every CML patient taking more than 300mg responded to the drug—a shocking outcome at the time (08:34–08:45).
  • Quote - Sydney Lupkin:
    “It was unheard of, Emily.” (08:45)
• Global Reach and Urgency:
  Early internet allowed patients from around the world to connect and seek out trials (08:59–09:07).
• Novartis and Data Crunching:
  Biostatistician Insa Gothman led data analysis at Novartis; she initially thought her “survival curves” were a mistake because the results were so positive (09:30–10:10).
  • Quote - Insa Gothman:
    “I must have done a programming mistake... Can’t be right.” (09:53–10:10)

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The Science and Legacy of Gleevec (10:11–12:19)

• How it Works:
  Gleevec inhibits the abnormal enzyme caused by the fused gene in the Philadelphia chromosome, turning off uncontrolled cell growth (10:11–10:22).
• Record-Setting Approval:
  Gleevec received FDA approval in just 72 days—then the fastest ever—for its remarkable efficacy (10:23).
• Collaboration and Public Investment:
  Gleevec’s story highlights public and private cooperation, with government funding helping to discover the genetic mutation (10:52–11:26).
  • Quote - Brian Drucker:
    “We have one of the most robust innovation systems in the world... early stage funding by the government of science of medical research.” (10:52)

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Ripple Effects and Modern Targeted Therapies (11:37–12:19)

• Risk and Reward:
  From scientists and companies to trial participants, everyone took risks to make Gleevec happen.
• Legacy:
  Gleevec ushered in the era of targeted cancer therapies—now more than 100 such drugs exist (12:04–12:19).
  • Quote - Brian Drucker:
    “There’s now over 100 targeted cancer drugs on the market...” (12:06)

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Cost, Ongoing Use, and Hope (12:19–13:48)

• Price Tag:
  Gleevec was initially very expensive ($26,000/year in the early 2000s), but is now generic and much cheaper (12:19–12:33).
• Long-term Impact:
  Still widely prescribed, with many patients able to live normal lives; one child patient grew up, came off Gleevec, and started a family (12:59–13:07).
  • Quote - Brian Drucker:
    “Her parents thought that she would never live long enough to graduate from high school. And here she is with us today.” (12:59)
• Expanded Use:
  Later trials showed Gleevec works on other cancers, such as certain gastrointestinal tumors, with rapid patient recovery (13:07–13:47).
  • Memorable Moment: Drucker recalls a patient with a huge tumor, bedridden, who started playing golf days after starting Gleevec (13:27–13:47).

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Where Are They Now? (13:48–15:29)

• Insa Gothman:
  Remained at Novartis, rising to executive director in biostatistics; soon retiring (13:59–14:04).
• Mel Mann:
  Now 69, watched his daughter grow up, earned another degree, and plans to run the Boston Marathon (14:06–14:20).
  • Quote - Mel Mann:
    “I got a second chance at life.” (14:45)
• Dr. Brian Drucker:
  Still practicing, celebrating life events (weddings, births, graduations) of phase-one trial patients—some are still alive decades later (15:02–15:29).
  • Quote - Brian Drucker:
    “It is a remarkable and magic moment of reflection because some of the patients on that phase one first in human trial are still with me...” (15:02)

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Notable Quotes

• Mel Mann (CML Survivor)
  “That’s the first time that I had to face my own mortality.” (01:33)
  “I’m just thankful for the physicians who I know had to burn a lot of midnight oil to make it happen... I got a second chance at life.” (14:20, 14:45)
• Dr. Brian Drucker (Oncologist & Gleevec Pioneer)
  “My view was there had to be a better way... based on knowledge that was driving the growth of a specific cancer.” (05:47)
  “I wasn’t just a researcher. I also had patients....” (07:31)
  “It is a remarkable and magic moment of reflection...” (15:02)
• Insa Gothman (Biostatistician, Novartis)
  “I must have done a programming mistake... Can't be right.” (09:53)

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Timeline of Important Segments

| Timestamp | Segment | |----------------|-------------------------------------------------------------| | 00:16–03:58 | Mel Mann’s survival story & Gleevec’s personal impact | | 05:04–07:12 | Brian Drucker’s vision for targeted cancer therapies | | 07:12–10:11 | Clinical trials, lab breakthroughs, and Novartis’ role | | 10:11–12:19 | How Gleevec works, FDA approval, and public/private efforts | | 12:19–13:48 | Cost, generics, and Gleevec’s impact beyond CML | | 13:48–15:29 | Reflections on legacy and where key players are today |

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Takeaways

• Gleevec transformed a terminal diagnosis into a survivable condition for many CML patients.
• This drug represented one of the first successful examples of using targeted genetic knowledge to develop effective and less toxic cancer treatments.
• The path to this breakthrough involved risk-taking, innovation, immense collaboration, and personal sacrifice—from patients to researchers to industry partners.
• The ripple effect of Gleevec’s story continues today, with more than 100 targeted cancer drugs available, saving countless lives and giving families new hope.
• Individual patient stories—like those of Mel Mann and the child who outgrew her cancer—highlight the human impact of scientific progress.

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Transcript

[Commercial Announcer]

This message comes from U.S. bank. Simplify how you do business with business essentials. A powerful combination of no monthly maintenance fee, checking and card payment processing. Deposit products are offered by U.S. bank National association member FDIC.

[Emily Kwong]

You're listening to Short Wave from NPR. Hey, Shortwavers. Emily Kwong here with NPR pharmaceuticals correspondent Sydney Lupkin.

[Sydney Lupkin]

Hello. Hi, Emily. So this weekend marked a very important anniversary.

[Emily Kwong]

Was it our anniversary? We have been friends for a long time.

[Sydney Lupkin]

No. Oh, not our friend. Iversary, Emily. The anniversary of a pill that revolutionized cancer care.

[Emily Kwong]

Oh, that's way better. Not that I don't love you. What pill?

[Sydney Lupkin]

So the drug is called Gleevec. And May 10 marks 25 years since the Food and Drug Administration first approved it.

[Emily Kwong]

Okay, so Gleevec. Why is this pill such a big deal?

[Sydney Lupkin]

Well, it's considered one of the first targeted cancer therapies. And what it really did was make some cancers that were once fatal, not so fatal anymore. Survivable. Wow.

[Emily Kwong]

That is a big deal.

[Sydney Lupkin]

Yeah. And I talked about it with this guy in Atlanta. His name is Mel Mann. And he told me that in 1995 he was dealing with some back pain and fatigue, but it wasn't really clear why. Then after an MRI showed what was going on with his bone marrow, he was diagnosed with chronic myeloid leukemia. Leukemia, A kind of blood cancer. He was 37 at the time and had a five year old daughter at home. The doctors told him he had three years to live.

[Mel Mann]

Yeah, I was shocked because, you know, that's the first time that I had to face my own mortality.

[Sydney Lupkin]

But Emily, Mel is actually still here.

[Emily Kwong]

That is amazing. After being given only three years to live. So how. How did that happen?

[Sydney Lupkin]

So after his diagnosis in January 1995, Mel starts doing bone marrow drives, hoping to a match and get a life saving bone marrow transplant. Yeah, but at the time, if you were black, you had such a low chance of finding a match that he knew it was kind of an impossibility.

[Mel Mann]

And I would take my daughter with me. So she understood that I needed a match to live. In fact, one of the drives, she could have been more than probably still five. She says, daddy, I can't see why you can't find a match. All the blood looks the same to me.

[Sydney Lupkin]

But after all this searching, Mel added thousands of people to the registry. But he didn't find a match for himself.

[Mel Mann]

Immediately, you're gonna think of your family. And I thought, you know how she's gonna be when at the end of three years and it was eight years, and I was trying to bargain for more.

[Sydney Lupkin]

So he started participating in clinical trials for experimental treatments still in development. They would work for a few months and then stop. He outlived his expiration but wasn't doing well.

[Mel Mann]

By then, I was like, really tired. You know, I was asleep for eight hours, drank two cups of coffee, wake up and felt like I never went to bed and become like, really lost a lot of weight. So I asked the doctor were there any more drugs?

[Sydney Lupkin]

And there was another one, Gleevec, though of course it was still being studied and didn't have that name yet. Mel started the drug in August of 1998. By the next June, Emily, he was running a marathon in Anchorage, Alaska.

[Mel Mann]

And I did it in a pretty decent time, too.

[Emily Kwong]

Today on the show Gleevec and how it ushered in a new era for cancer care. You're listening to Short Wave Science podcast from npr. This message comes from Mint Mobile. If you're tired of spending hundreds on big wireless bills, bogus fees and free perks, Mint Mobile is for you. Shop [e-mail] Switch taxes and fees extra. See Mint Mobile for details.

[Commercial Announcer]

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[Emily Kwong]

we are talking about a revolutionary cancer drug. Developing a new drug, of course, is not easy. So what is the origin story of this one?

[Sydney Lupkin]

GLEEVEC yeah, so it kind of starts with a setback. Oncologist Brian Drucker had this idea that at the time was really new in cancer medicine. He thought, what if instead of just trying to kill cancer cells and hoping that the healthy Cells were mostly spared. You could target the reason that cancer cells Were growing out of control in the first place.

[Brian Drucker]

What I saw with chemotherapy Is that we are giving patients Pretty toxic drugs. We didn't quite know if they'd work. And my view was there had to be a better way. And that better way had to be based on a knowledge that was driving the growth of a specific cancer.

[Emily Kwong]

I mean, it makes total sense and is a pretty widely accepted idea now. But you're saying back then, no one had ever tried this kind of targeted approach before?

[Sydney Lupkin]

We didn't because we didn't know what to target. There was a lot we didn't understand about cancers. And we learned it in chronic myeloid leukemia, which became one of the first cancers to be linked to a genetic abnormality, the lidelphia chromosome. Scientists knew that people with this chromosome Had a fused gene. That gene results in an abnormal enzyme that regulates One of the on, off switches for certain cell growth. This one gets stuck in the on position, Leading to uncontrolled white blood cell growth. Chronic myeloid leukemia. Brian was trying to turn that switch off, so he wanted to find a drug that would inhibit the abnormal enzyme.

[Emily Kwong]

That's brilliant. Yeah.

[Brian Drucker]

The problem was not a lot of people believe that was going to work.

[Sydney Lupkin]

He said that his institution thought it was an interesting idea For a grant or something, but he ultimately had to leave his job in Boston and move across the country to oregon health and science university to really actually pursue it.

[Emily Kwong]

Yeah, he's like, this is big science. I gotta take a risk here. Was it worth it?

[Sydney Lupkin]

Well, he says that within six weeks of arriving, he was testing five compounds from the drug company that would become novartis in his lab. One of them was gleevec. It was a compound discovered by scientist Nicholas lyden, and it worked, at least in the lab.

[Brian Drucker]

The reality is, I wasn't just a researcher. I also had patients. Many, many patients who believed in me, Thought that this was a potential way forward for them. And it allowed me to have enough courage to be their. Their mouthpiece and voice to lobby, to get this drug into clinical trials.

[Emily Kwong]

So it sounds like he was in a race against time, really, to try to save these patients lives.

[Sydney Lupkin]

Yeah, exactly. And these trials were also unique because they only included patients with this specific, specific kind of leukemia.

[Emily Kwong]

Wait, so was that not common in cancer drug trials? At the.

[Insa Gothman]

No.

[Sydney Lupkin]

Before this trial, cancer drug studies Would include patients with all kinds of cancer. And they just sort of hope that the drug worked for some of them.

[Emily Kwong]

They would just Lump them all together,

[Sydney Lupkin]

and for a few, it would become the standard of care. It would work. But for most people, the drug wouldn't work.

[Emily Kwong]

That's so interesting. But I guess Brian's trial, he. He wanted to focus just on this type of leukemia because his pill was designed to target that cancer and the thing causing the cancer.

[Sydney Lupkin]

Exactly.

[Insa Gothman]

Yeah.

[Sydney Lupkin]

Within six months, every cml, which is the abbreviation for chronic myeloid leukemia, Patient taking more than 300 milligrams responded to the treatment.

[Emily Kwong]

Every patient.

[Sydney Lupkin]

It was unheard of, Emily. And because it was 1999, it was also the early days of blogging and Internet chat room, so the patients were talking to each other.

[Brian Drucker]

So all of a sudden, I was getting contacted by patients from around the world who wanted to enroll in this clinical trial.

[Sydney Lupkin]

They knew that they had to scale up trials and get the drug application to regulators and, like, fast. And that's where the folks at Novartis come in.

[Emily Kwong]

That's the drug company, Right?

[Sydney Lupkin]

Right. So the next step would be for a drug company to take the drug to the FDA for approval. To do that, you've got to look at the data and prepare a whole package. Entered insa gothman, who was right out of school and got hired as a biostatistician.

[Insa Gothman]

And it was apparent back then that this is a game changer and that we needed people with energy who could make this drug development even faster and bringing this to authorities as fast as we could.

[Sydney Lupkin]

She became the statistical lead on the project, Working long hours.

[Emily Kwong]

What was she doing?

[Sydney Lupkin]

So one of her jobs was to look at survival curves.

[Insa Gothman]

And when I was first running them for gleevec, I was like, okay, I must have done a programming mistake. Something is wrong. Because the curves were just. I mean, they were all doing fantastic, and they were all alive. And I'm like, no, no, no. Something is wrong. This can't be. Can't be right.

[Emily Kwong]

But it was right.

[Sydney Lupkin]

Yeah. So gleevec was able to switch the enzyme to the off position, Shutting down that cell overgrowth, which let patients white blood cell counts return to normal.

[Emily Kwong]

This is so inspiring.

[Sydney Lupkin]

And when gleevec was approved on May 10, 2001, it was the fastest approval at the time. 72 days. And everyone from the NIH to the FDA to the HHS Secretary to the company executives wanted to take a bow. Amit sarpotwari is an assistant professor of population medicine at Harvard medical school. And he says he. It was an example of the public and private sectors working together at their best to bring forward meaningful medical advancements.

[Brian Drucker]

We have one of the most robust innovation systems in the world. And that system in pharmaceuticals really relies on early stage funding by the government of science of medical research.

[Sydney Lupkin]

And, emily, none of this would have happened without people knowing about the genetic mutation that causes this cancer in the first place. The philadelphia chromosome. That discovery was funded by the national cancer institute, which is part of the NIH.

[Emily Kwong]

So this, even though it got approved in 72 days, that's because of years of work ahead of it and layers of cooperation from so many different groups.

[Sydney Lupkin]

Cooperation and taking on risk. Remember, brian drucker moved across the country. The drug company that would become novartis Took on the financial risk of doing the expensive clinical trials. The patients took a risk trying a new drug. And then a whole bunch of people moved to heaven and earth to speed up the approval time. All of this changed medicine.

[Brian Drucker]

You can think of it as really ushering in this era that we have

[Mel Mann]

now

[Brian Drucker]

targeted therapies, Targeted cancer drugs on the market. There's now over 100 targeted cancer drugs on the market, which, in many cases, have meaningfully improved Overall survival and quality of life.

[Sydney Lupkin]

Though I should add that gleevec was also considered Incredibly expensive When it launched at $26,000 a year. And that meant even more money Back in the early 2000s. Yeah, the drug is now generic and much cheaper.

[Emily Kwong]

So gleevec is still being used today?

[Sydney Lupkin]

Yep, it's still used. Brian still prescribes gleevec and the second, third, and fourth generation drugs that came after. And he's still able to tell patients that they'll be able to live a normal life. He told me about one patient who had leukemia as a child and started taking gleevec. Eventually, she was able to come off the drug with no recurrence of her cancer. And she now has two kids.

[Brian Drucker]

Her parents thought that she would never live long enough to graduate from high school. And here she is with us today.

[Sydney Lupkin]

And the researchers kept studying gleevec, and they found out it actually works for other cancers. For example, it can also treat A certain kind of gastrointestinal tumor by shutting off its out of control growth. Dr. Drucker, Brian remembers that he had this one patient who had one of these tumors that he said Made his stomach look like he was nine months pregnant. And he was uncomfortable. He couldn't roll over in bed. He was miserable. And within days of starting gleevec, Brian got a call. The patient was on the other end of the line. And he said he felt so great that he was playing a round of

[Emily Kwong]

golf, because gleevec was able to, like, turn off the cell growth.

[Insa Gothman]

Uh huh.

[Brian Drucker]

Wow.

[Emily Kwong]

Sydney, in marking the anniversary of Gleevec this week, where is everyone at now? All the people who helped make this possible, like that Swiss biostatistician, Insa.

[Sydney Lupkin]

She stayed at Novartis and became the executive director in biostatistics. And she's about to retire.

[Emily Kwong]

And what about Mel the dad?

[Sydney Lupkin]

Mel the dad? Given three years to live in 1995. He's now 69 years old.

[Emily Kwong]

Wow.

[Sydney Lupkin]

Yeah. He got to watch his daughter grow up. He even went back to school and got a second bachelor's degree. And he wants to run the Boston Marathon next. Heck, yeah.

[Mel Mann]

I'm just thankful for the physicians who I know had to burn a lot of midnight oil to make it happen, and the researchers and people who were involved in the whole process, the people at the sites, the people who crunched the numbers, and just so many people that I have to be thankful for. So it means a lot. I got a second chance at life.

[Emily Kwong]

Yeah. And what about Brian Drucker, the oncologist who really is responsible for a lot of this and figured out Gleevec?

[Sydney Lupkin]

Yeah, he developed it. He's gotten to watch people given a death sentence actually go on to live normal lives. And some of them celebrated Gleevec's anniversary with him this past weekend.

[Brian Drucker]

It is a remarkable and magic moment of reflection because some of the patients on that phase one first in human trial are still with me. And they've seen weddings, births, graduations, grandchildren, all the things that they never thought that they could imagine would happen.

[Emily Kwong]

Sydney, thank you so much for sharing this story and the science behind it.

[Sydney Lupkin]

Thank you for having me.

[Emily Kwong]

If you liked this episode, share it with a friend because it really helps the show out. Also, check out the episode we did with Sydney on accelerated drug approvals and the development of the GLP1 pills. This episode was produced by Hannah Chin. It was edited by Rebecca Ramirez. The fact checker was Tyler Jones. Jimmy Keeley was the audio engineer. I'm Emily Kwong. Thank you for listening to Short Wave from npr.

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[Emily Kwong]

Download today, Dr. Eric Topol says health influencers make big claims about longevity, but he's offering us a reality check.

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[Brian Drucker]

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[Emily Kwong]

Straight talk about how to grow old and stay healthy. That's on the TED Radio Hour podcast. Listen on the NPR app or wherever you get your podcasts.