Mel Mann was diagnosed with a cancer known as CML and given three years to live. That was in 1995. Today, in 2023, Mel Mann is still living and still advocating – all thanks to accelerated approval and a clinical trial. Hear the story of how this...
Mel Mann was diagnosed with a cancer known as CML and given three years to live. That was in 1995. Today, in 2023, Mel Mann is still living and still advocating – all thanks to accelerated approval and a clinical trial. Hear the story of how this Army vet, and longest-living Gleevec patient, beat the odds and stands here today.
And Terry and Bob discuss the brand new accelerated approval report that Patients Rising released. Unprecedented coverage decisions from CMS, the history of accelerated approval, and why it’s such an essential pathway for patients are all in today’s episode.
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Mel Mann 0:00
I've narrowed it down to chronic myeloid leukemia, and I was diagnosed in January 1995. And the prognosis was three years to live.
Terry Wilcox 0:14
That CML survivor Mel Mann who received his diagnosis in 1995. But nearly 30 years later, he's still living thanks to a clinical trial that gave him early access to breakthrough treatment. Today's episode features a story about perseverance and taking your health into your own hands, even when the odds are bleak. Welcome to the patients rising podcast. I'm your host, Terry Wilcox, CEO of patients rising. I'm joined by my co host, swapped his black T shirt, which is my favorite one for a pink one. In the spirit of the new Barbie movie. He's Dr. Bob Goldberg, co founder of the Center for medicine in the public interest. Now Bob, I have to tell you something really funny about the Barbie movie. So my kids got a stat while we were at cubs cat camp, that there was so much pink paint for the Barbie movie that they actually ran out of the colors to mix that Barbie pink paint. There was a shortage in the country. So not only do we have drug shortages, we have pink paint shortages.
Bob Goldberg 1:20
Well, I think there's also Barbie and Ken doll shortages too so a friend of mines daughter is having a birthday so we were gonna get her a Barbie suddenly walked into the walked into the toy store. There's so many toy stores left anymore. And there's all these I know I want to get something really nice. So now we have the 1999 Barbie disco Barbie $19 Okay, yeah, all right. You know the Queen Barbie $19 Well, what else do you have is a little bit more obscure says, Well, you have this Barbie here for $265
Terry Wilcox 1:53
There's nothing in between $20 and $265.
Bob Goldberg 1:57
I said $265 What kind of Barbie says oh, that's a divorce Barbie and it comes with all Ken's property. So that's why it's $265
Terry Wilcox 2:02
Of course, or course.
Bob Goldberg 2:12
Anyway, the movies commercials Barbie dolls pink paint. I'm a dreaming about Barbie in her dream house in my nightmares. So I'm so I'm gonna get work past this Barbie vortex to transition to the opening of the show, which is that we're here to discuss on the patient's rising podcast, the latest health policy news from Washington DC. We also talked about incredible stories like the one we're discussing today, of which I think impacts millions of Americans who live with a chronic illness.
Terry Wilcox 2:51
Well, and today Bob, as you mentioned, is an exciting one because we sit down and speak with Mel Mann, the world's longest living Gleevec survivor, a cancer drug that he still uses to this day, but he might not have ever had the chance to take it if it weren't for accelerated approval. Patients rising just published a report on accelerated approval called CMS versus the FDA, how Medicare coverage decisions limit access to FDA approved treatments. And Mel is actually in DC today, July 24, sharing his experience with accelerated approval on Capitol Hill. Instead of coming all the way to DC, like we said, we're bringing DC to you today.
Bob Goldberg 3:35
No story starts in January of 1995. When he was diagnosed with chronic myelogenous leukemia, or CML, he was given three years to live and his hope for survival depended on finding a bone marrow transplant donor. His best chance was his family. But there was no match that left the national registry but because it needed to find a donor from the same ethnic background, his chances for a match dropped to less than 1%. And here's Mel to talk about it.
Mel Mann 4:06
I was in shock with my diagnosis, and the prognosis. And I had a five year old daughter, and my wife and I were teaching her how to ride a bicycle that she got for Christmas. And that one training wheel on it. And I was wondering if I how long I would live because she was only five. And I kept counting up the years and it kept coming out to eight. So that was very disturbing. It was very like that was in shock when I was told the prognosis.
Bob Goldberg 4:40
The grim statistics didn't determine Mel who set off on a mission to start bone marrow drives all across the country in the world from Germany to Hawaii to the Pentagon right outside of Washington DC. His face was put on posters and he took to the media to share his plea that anyone could be a match
Mel Mann 4:59
I decided it was best for me to go out. So people can see me and get a sense of urgency that it was a real person that needed this donor. It's a big process because I had to set up briefings and educate people about marrow. So I would go into the churches and talk to them first. And then we'll answer all the questions. And I will come back a few weeks later and do the drive on the military bases, I would do television interviews, and they would play that again and again, on the station and televisions.
News Report 5:32
At 38, retired Army Major Melvin man is fighting for his life. He has leukemia, and the only cure is a bone marrow transplant. Since his only sister is not a match, he has to find a stranger whose tissue is compatible with his that I've been searching the registry. And there's nobody out there right now. So I need somebody new to come on the list. And that person will be a donor,
Mel Mann 6:00
Three or four times a day when they will play the interview. And that will drive up people becoming aware of the need for donors.
Bob Goldberg 6:08
So Terry Miller shared the stance, I guess, with you that in 95, there were less than 1 million people on the national registry now there's 41 million.
Terry Wilcox 6:18
That's amazing. And it just shows the power of this push for registering. And for, you know, wanting to be the match, as they call it. Right? Isn't that the bone marrow.
Bob Goldberg 6:26
Be the match. Yeah. And it also speaks volumes to the fact that even when patients understand they may not be the beneficiaries of something, they're paying it forward. It's another reason why we need accelerated approval, people are putting their lives and their time on the line to help others.
Terry Wilcox 6:50
Well, exactly. And while Mel was able to help promote several successful drives, he could not find a match for himself. But at one of these drives, he connected with a hairy cell leukemia cancer survivor, who urged him to reach out to his doctor at MD Anderson. Then Mel flew from Atlanta to Houston, where the doctor took him on as a patient and committed to finding him a clinical trial. And just around three years after Mel's original diagnosis in 1995, his doctor told him about a promising new drug. Here's Mel.
Mel Mann 7:22
He says that what we got one drug that's close, but we have problems in the lab with the animals. But if we ever get it approved, you will be the first person to use it. And his nurse she said, Well, you just told Mr. So and so the same thing. And, you know, Terry looked at me, he said, Okay, you have been number two. And I asked him more than any more drugs. He said, There's one drug is close, but we're having problems with animals in the lab. That really didn't sound close to me. But he said it was close. So I went home. And about six months later, he called and said they had approval to use in humans. So he told me to come on out. And I came straight out. And I became the second person at MD Anderson since he used to drive. And it worked.
Terry Wilcox 8:14
Now described the moment when he realized the drug Gleevec was working for him.
Mel Mann 8:20
I knew that the clinical trial was going to be successful, because there were certain ways that they measured your blood, and one was cytogenetics. They would like do an aspiration and go in and check out your bone marrow. It take like 20 cells, examine those cells and get your percentage of how many had to Philadelphia chromosome. And that number started going down. Before the trial, the number always went up. And it was like always high like 80 to 90%. Then when they start doing the trial, it kept going down. And that's the surrogate marker. That's very important. They still use markers like that today. They're able to get more precise today, but that's one of the major markers.
Terry Wilcox 9:13
So Bob, Mel mentions of surrogate marker, you know, how are surrogate markers used with accelerated approval drugs?
Bob Goldberg 9:20
Well, surrogate markers have gotten a bad name, really by people who don't know what surrogate markers are and want to undermine the use of surrogate markers. A surrogate markers are measures of the presence of disease based upon the level of the number of cells that you have that are cancerous, the viral load in drugs like HIV, or your inflammatory levels like an autoimmune disorders. So they're very, very good markers because they are directly caused by the change in the actual biology of the disease itself. So they're extremely important.
Terry Wilcox 10:04
One last case, the surrogate marker was that the number kept going down and down and down.
Bob Goldberg 10:10
Terry Wilcox 10:12
Which is amazingly, we all know the story of cleaved packets, you know, was one of the greatest breakthroughs in cancer, right?
Bob Goldberg 10:19
Well, I guess if surrogate marker is on enough for people, the fact is 10 months after he was in the clinical trial, Mel went on to run a marathon in Alaska. And he was in school to get his master's degree and cycled over 100 miles a few short years later, in 2001, the drug received accelerated approval from the FDA. And he described it as a huge moment for the CML patient community.
Mel Mann 10:47
Everybody knew and very wanted to everybody who had CML because your clock is ticking down. And I'm probably now at four years and eight months. But some of these people who were diagnosed with me at the same time, if they weren't on that trial, and they, they were not alive, most likely, because they only had three years to live. If you look at it, I was diagnosed in January 1995. Started the drug in August 1998, I was given three years. So it was CML, three years is a very important time span. And then it took another after I was diagnosed with another three years for it to be approved by the FDA. And that was accelerated. So now we're already at like six and a half years. And then if it was normal of the traditional approval, studies shown that 85% of cancer drugs would take another three years. So that would be nine years, and there was no way I was gonna survive nine years. So it's good time to go on the trial. But all these other patients that are on the trial, that were able to take advantage of that drug. So they were I mean, at least three years, or for people were able to get that drug.
Terry Wilcox 11:58
Bob, I really think that timeline, he indicates about how much longer it would have taken if the CML community hadn't had access to Gleevec earlier through accelerated approval, which is why it's so important. Because there's so many treatments like this, and we heard last week, we've heard on many other shows of patients just you know, so many patients and families are waiting. And especially if you're in the rare disease space, or you have a life threatening condition like a cancer. And often cancers are rare diseases, many of them, yep, 25% of them, 25% of them. So now 28 years later, now continues to advocate for patients. He's passionate about clinical trial recruitment, health equity and bone marrow drives. So a big thank you to Mel for sharing your inspiring story with us today. You can visit the show notes to learn more about his journey and connect with him online.
Terry Wilcox 13:00
This episode of the patients rising podcast is brought to you by the patient's rising helpline, this free service is a tremendous way to make sure all patients, including you listening right now can get the resources you need. We have navigated through hundreds of individual situations with patients from finding medical transportation to sifting through and understanding insurance coverage, the list goes on. If you or someone you know has a health care question, challenge or issue, we are standing by and ready to help to get in touch, leave us a voicemail or send us an email using the link in the show notes.
Terry Wilcox 13:39
Now we mentioned earlier that patients rising has a brand new report out on accelerated approval. And this report analyzes the patient impact of growing coverage restrictions for FDA accelerated approval treatments. Now we have told you right here on this podcast about the unprecedented decisions of late from CMS. And this report gets into the history of accelerated approval and why it's such an important pathway for patients like Mel and others.
Bob Goldberg 14:06
Well, you know, my feelings about this, Terry? It's unconscionable. I think the report is fantastic. One of the first thorough assessments of the impact of accelerated approval and sort of leaves me wondering, you know, can people it's CMS sleep at night?
Terry Wilcox 14:26
Exactly. I mean, the timeliness of the report that patients rising is put out with the leadership of McKay Jemison, who is our Acting Executive Director for patients rising now has been amazing. He's really put a lot into this and focused on the needs of why it is important right now. And that's basically with all this going on with the accelerated approval pathway. And CMS has decision to carte blanche say they're not going to cover an entire class of drugs unless A, B and C are met, which was unprecedented you they usually would do things like this on a case by case basis, I suppose, and not a class of drugs, which is sort of what makes this so important. You know, the report is informed by policy experts, physicians or a patient's patients who have benefited from accelerated approval drugs. And it's going to assess the impact of new CMS guardrails and, yeah, you know, roadblocks, I call them
Bob Goldberg 15:28
Roadblocks. And I think it also has implications for the whole drug pricing, negotiation stuff, because if they're going to ding accelerated approval as being lesser than, what does that mean, in terms of the quote unquote, you know, the value is perceived by the, the arbiters at CMS. And so I think this is a hugely, hugely important issue. on a happier note, we'd be remiss if we didn't mention that this week is the 33rd anniversary of the Americans with Disabilities Act being signed into law. And that took place on July 26 1990, by President George HW Bush. And you can learn more about the act, as well as the activities taking place this week by using the link in the show notes. So Happy anniversary, ADA.
Terry Wilcox 16:19
Yes, happy anniversary, ADA. And thank you for listening to today's episode of the patients rising podcast. Don't forget to pass this episode along to a fellow advocate.
Bob Goldberg 16:29
And we have more episodes on the way that you're not gonna want to miss. So make sure to follow the podcast on your favorite podcast app.
Terry Wilcox 16:38
We'll be right back here on Monday with another new episode. Until then, for Bob and everyone a patient's rising. I'm Terry Wilcox, stay healthy.