Speaker 1: Previously unsymptomatic. David Fegenbaum, a college quarterback deeply impacted by
Speaker 1: his mother's fight with brain cancer, was a prime example
Speaker 1: of peak health and motivation.
Speaker 2: The moment that I saw her dealing with her cancer,
Speaker 2: I just said, this is what I have to spend
Speaker 2: the rest of my life doing. And by this, it's
Speaker 2: taking care of people with horrible diseases, and it's searching
Speaker 2: for treatments so that people don't suffer.
Speaker 1: But during med school, everything quickly came crashing down when
Speaker 1: Castleman disease took him from treating patients to the brink
Speaker 1: of death in a matter of days.
Speaker 2: I was so sick that my doctors encouraged my family
Speaker 2: to say goodbye to me.
Speaker 3: I'll never forget the moment when a priest came in
Speaker 3: and read him his last rites. In this moment of sorrow,
Speaker 3: I remember I couldn't really keep it together, I couldn't
Speaker 3: hide help. Horrifying it was to see him in that state.
Speaker 1: After exhausting all the treatments available for Castleman, David's body
Speaker 1: was ravaged by chemo and he was out of viable options.
Speaker 1: That's when he decided to take matters into his own hands.
Speaker 2: I had to take some action, and it had to
Speaker 2: be a pretty crazy step, and that is to start
Speaker 2: testing one of these drugs that I was studying in
Speaker 2: the lab on myself. All the drugs I was studying
Speaker 2: the lab were all ft approved for something else, they
Speaker 2: just weren't used for Castlemen.
Speaker 1: After repurposing a medicine originally designed for kidney transplant patients
Speaker 1: to send his disease into long term remission, David was
Speaker 1: determined to prevent others from that similar dead end. His
Speaker 1: theories around expanding the approved uses for certain medicines would
Speaker 1: quickly be put to the test. Seeing Gary Gravina fight
Speaker 1: for his life against Castleman, just like David had a
Speaker 1: few short years before.
Speaker 2: Yes, his heart is technically beating. Yes he's technically breathing
Speaker 2: because of what we're doing medically, but he's not going
Speaker 2: to come back from this. And I just remember being
Speaker 2: so devastated to hear that, and then at the same
Speaker 2: time remember still running down the hall to the lab
Speaker 2: and still running the experiments because like who knows, there was.
Speaker 4: At least one night in both of my flares where
Speaker 4: it was just that close. I was in a coma.
Speaker 4: You know, everything in me was tanking, and I can
Speaker 4: just remember crying tears of frustration.
Speaker 1: How terrifying would it be to fight an unknown enemy,
Speaker 1: one you didn't recognize and didn't see coming. What if
Speaker 1: that enemy was coming from within a disease that even
Speaker 1: doctors couldn't identify. Nearly half of all Americans suffer from
Speaker 1: some chronic illness, and many struggle for an accurate diagnosis.
Speaker 1: These are their stories, Lauren Bribe Pacheco, and this is symptomatic.
Speaker 1: Gary Gravina built a life working with his hands, first
Speaker 1: as a marine and then as a carpenter for over
Speaker 1: twenty seven years. He felt fulfilled when he could craft something,
Speaker 1: even if it was just a song on his guitar.
Speaker 1: Before Gary would face a fight for his life, he
Speaker 1: and his wife Stacy had been hit with their first
Speaker 1: seemingly insurmountable hurdle.
Speaker 4: Let's see about nine months after we all moved in together,
Speaker 4: my steps on Jacob presented with a seizure and had
Speaker 4: a diagnostic journey that the end result was that he
Speaker 4: had brain cancer, and so we were heavily engaged in
Speaker 4: fighting that for just over a year. They had given
Speaker 4: him six months to maybe a year to live as
Speaker 4: the original prognosis, which being Jake. He beat that by
Speaker 4: a week.
Speaker 1: Wow.
Speaker 4: It was huge. It was traumatic, and there was all
Speaker 4: this medical acquaintance that we wish we never had. And
Speaker 4: when the doctor said there was nothing more they could do,
Speaker 4: we were preparing hospice and we had the hospital bed
Speaker 4: being sent here.
Speaker 1: Oh Gary, I'm so sorry.
Speaker 4: Yeah, thank you.
Speaker 1: So you had already been through the ringer before you
Speaker 1: even realized you had symptoms.
Speaker 4: Yeah, something like that happened, and you think, wow, that
Speaker 4: was the most awful thing, and you kind of in
Speaker 4: the back of your head, you're thinking, that must have
Speaker 4: been our big, awful thing that we have to overcome.
Speaker 1: But it was not long until Gary started noticing his
Speaker 1: body change. Heavy fatigue made getting through the day more
Speaker 1: of a chore than usual. Looking back, now, what are
Speaker 1: some of the earliest symptoms.
Speaker 4: You can pinpoint the morning when I felt ill, like
Speaker 4: I felt like I had a flu, but I could
Speaker 4: not push through it. I could not get up and
Speaker 4: go to work. And I mean for the previous eighteen years,
Speaker 4: I think I had taken off two and a half
Speaker 4: days of sick time. I just did not miss work
Speaker 4: if I felt sick during the day, I pushed through
Speaker 4: the end of the day. I got some rest that night,
Speaker 4: and then the next morning I came in and started
Speaker 4: to feel better. But this wasn't happening. And I took
Speaker 4: one day off and I did not feel any better,
Speaker 4: and so I say, it felt like a flu, that
Speaker 4: achy feeling, that drained feeling, and the swimminess in my head.
Speaker 4: My wife started to notice that my stomach was getting larger,
Speaker 4: and I wasn't really eating because I felt so ill,
Speaker 4: and so for me to be putting on weight was weird.
Speaker 4: So Stacy said, you know, we have to get you
Speaker 4: to the doctor. The next thing I remember was being
Speaker 4: at the doctor and him listening to my lungs and saying,
Speaker 4: you need to get to the host hospital.
Speaker 1: Wow. And at what point did they realize they were
Speaker 1: dealing with something far more serious than the flow.
Speaker 4: That whole first day at the emergency room, I'm thinking, well,
Speaker 4: this is going to be something really simple and they're
Speaker 4: going to take care of it. And then when they
Speaker 4: started talking about admitting me, I'm like, come on, this
Speaker 4: is this is not something like that. I don't need
Speaker 4: to be in the hospital. This is ridiculous. I just
Speaker 4: this sense of this, this is all just a big mistake.
Speaker 4: By the end of that week, I was in a
Speaker 4: medically induced coma, and I don't think I really squared
Speaker 4: myself with that until I woke up, you know, three
Speaker 4: and a half weeks later. What was going on going
Speaker 4: into the hospital, it was this sense was frustration, like this,
Speaker 4: we just need to find the thing that this is.
Speaker 4: And this should be a simple answer. I shouldn't. I
Speaker 4: shouldn't be here.
Speaker 1: Though Gary didn't know it. As he endured the challenges
Speaker 1: of his undiagnosed Castleman disease, David was hard at work
Speaker 1: in a lab not far away. Through the creation of
Speaker 1: the Castleman Disease Collaborative Network or CDCN, David was honoring
Speaker 1: his mission to repurpose drugs that could treat Castleman disease
Speaker 1: and other immuno deficiency conditions. There are only three thousand
Speaker 1: FDA approved drugs, and ninety five percent of rare diseases
Speaker 1: have no medical approved treatment in terms of medication.
Speaker 2: That's right, there's so much here. I mean, all I've
Speaker 2: been able to think about for these last ten years
Speaker 2: is how many more drugs are sitting out there. They
Speaker 2: could be life saving for other people and what we've learned.
Speaker 2: The more I've done it, the more I've just been
Speaker 2: blown away by the fact that of those three thousand drugs,
Speaker 2: the vast majority of them are generic. Over eighty percent
Speaker 2: of them are generic, which means that they are very
Speaker 2: inexpensive and they're not profitable any longer because once it
Speaker 2: drug becomes generic, then multiple manufacturers can make that drug
Speaker 2: price plummets. And so what that means is that our
Speaker 2: medical system doesn't study over eighty percent of our drugs
Speaker 2: that are at the CBS to find new uses for
Speaker 2: them because there is no incentive within our system. And
Speaker 2: it's like, wait a minute, of the drugs, the three
Speaker 2: thousand that can help us, eighty percent of them are
Speaker 2: not being studied. That's crazy.
Speaker 1: It wasn't always smooth sailing for the CDCN. David shared
Speaker 1: his dream with Grant, his best friend from medical school,
Speaker 1: but they were up against lots of skepticism from the
Speaker 1: medical community to make it happen. Tell me just a
Speaker 1: little bit about thinking outside the box in terms of
Speaker 1: wanting to evaluate medicines that already had FDA approval but
Speaker 1: for off label impact.
Speaker 3: Yeah, and maybe before I do that, I'll tell you
Speaker 3: one more story about our sort of naive optimism. When
Speaker 3: we first realized that we were going to try to
Speaker 3: be they, we called everyone we knew to currently be they,
Speaker 3: which was all the researchers in the world that might
Speaker 3: know anything about this disease. There were only twenty of
Speaker 3: them or so. They were in France and Japan at
Speaker 3: the NIH and we invited them all to a conference
Speaker 3: that they might have been interested in attending anyway, and said,
Speaker 3: if you're already going to be there, you know, please
Speaker 3: come to this side room with David and I to
Speaker 3: discuss Castleman disease research. And I'll never forget sitting there
Speaker 3: at the head of the table with David said this
Speaker 3: is David. I'm grant, you know, and we're here to
Speaker 3: cure Castleman disease, and just seeing these researchers who had
Speaker 3: been at this for years around the table kind of
Speaker 3: chuckle and say, okay, kids, you know, good luck. You know,
Speaker 3: we'll try and be helpful. It was just one of
Speaker 3: those moments where we were young and naive enough to have
Speaker 3: the audacity to think that we could do anything.
Speaker 1: This passionate naivete needed to change from an inspiring concept
Speaker 1: to a reality quickly if it was going to save
Speaker 1: Gary from arreparable damage. After the first flare up, Gary
Speaker 1: was now going in and out of medically induced comas,
Speaker 1: clinging to life. He was eventually diagnosed with Castleman disease,
Speaker 1: the same disease that plagued David years earlier. However, the
Speaker 1: diagnosis didn't guarantee a clear path to effective treatment. When
Speaker 1: you first woke up and you felt and saw the
Speaker 1: physical transformations of your body coming out of that coma,
Speaker 1: how surreal was that for you? That must have been
Speaker 1: honestly like living an episode of the Twilight Zone.
Speaker 4: Yeah, coming out of a coma, at least for me,
Speaker 4: was nothing like the show it in TV and movie
Speaker 4: where you open your eyes and you look up and
Speaker 4: you see your loved one and you remember the last
Speaker 4: thing that was going on. It's a kind of a
Speaker 4: slow drift back toward reality.
Speaker 5: And then also, you know, as the head of the family,
Speaker 5: I can't imagine how heavily it weighed on you that
Speaker 5: this is happening again and you're not there to physically
Speaker 5: fight it.
Speaker 4: Yeah, that was huge because with Jacob, I was by
Speaker 4: Stacy's side as a caregiver, helping her take care of him,
Speaker 4: and then in this situation, suddenly Stacy was back in
Speaker 4: the role of caregiver, and my condition was plummeting. And
Speaker 4: part of the reason I was so frustrated and wanted
Speaker 4: to figure out the simple thing that this had to
Speaker 4: be was that I didn't want to be the reason
Speaker 4: that Stacy went.
Speaker 1: To that again, grappling with guilt. On top of the
Speaker 1: physical fight for his life. Even additional symptoms pile up,
Speaker 1: and so I.
Speaker 4: Woke up with purple hands and they felt gravelly, they
Speaker 4: felt sandy. They were numb, but also uncomfortable. You know,
Speaker 4: it was not the biggest pain that I had, but
Speaker 4: it was very uncomfortable because I've always used my hands
Speaker 4: for everything I do, and so to have them not
Speaker 4: there was really it was freaky. Once I did wake up,
Speaker 4: I had to be fed by hand because my arms
Speaker 4: were so weak and my stomach had grown so large,
Speaker 4: like I couldn't put my hands together. I didn't have
Speaker 4: enough strength to reach up that high. So Stacy was
Speaker 4: spoon feeding me my food when I came out of
Speaker 4: the coma. When people talk about having an advocate, man,
Speaker 4: I had the best doctors and nurses in the world,
Speaker 4: And I mean I'm very grateful for that, but I
Speaker 4: would be dead. I would be dead without Stacy if
Speaker 4: she hadn't been there to do what she did.
Speaker 1: Local doctors had exhausted all of the known treatments for
Speaker 1: Castleman disease and had decided to transfer Gary to the
Speaker 1: hospital of the University of Pennsylvania for more help.
Speaker 4: David Figenbaum had released a study on Castleman disease. Actually
Speaker 4: the day I went into the hospital, he published this
Speaker 4: in esh newsletter for Heematology, because I think the journal
Speaker 4: is called Blood it is. And someone at the second
Speaker 4: hospital had seen that article. One of the people said, well,
Speaker 4: this guy, he has the disease and he's fighting it.
Speaker 4: And so I ended up done at penn and I'm.
Speaker 2: Told, hey, there's a patient with your subtype of Castleman's.
Speaker 2: You know, this very rare subtype of Castlemans that's here
Speaker 2: in the ICU. Can you go up and see the patient?
Speaker 2: And at this stage, I'm working in the hospital, I'm
Speaker 2: running the center that does a lot of research, but
Speaker 2: I'm like fiercely avoiding the ICU, like I don't want
Speaker 2: to walk anywhere near the ic I got some bad
Speaker 2: memories FM when I was in the ICU.
Speaker 4: And I was nervous. It was almost I was nervous
Speaker 4: like you would be for a job application, like like
Speaker 4: I hope he lets me in, you know what I mean.
Speaker 4: It's so weird. And of course you just hear, you know,
Speaker 4: doctor researcher, and you hear the name doctor Fagenbaum, and
Speaker 4: it's a beautiful name. It just it kind of sounds
Speaker 4: like old doctor. So I'm picturing an old, gray haired
Speaker 4: guy with glasses and probably broken from the disease, but
Speaker 4: still moving on. And then this young guy comes in
Speaker 4: looking full of life, and like all the fight in
Speaker 4: him and the spirit and the keenness of his intellect
Speaker 4: and how compassionate he was was just like, wow, okay,
Speaker 4: I'll take a pass on lymphoma. I'll take what that
Speaker 4: guy has. It's the craziest thing I remember, like it
Speaker 4: was yesterday. I see a patient in the bad who
Speaker 4: is so sick. All of his organs are obviously failing,
Speaker 4: and he looks just like me when I was at
Speaker 4: my sickest, Like he's got the fluid. When you lose
Speaker 4: the muscles in your face, you sort of look like,
Speaker 4: you know, you have these dense next to your eyes.
Speaker 2: Everything. It was like I'm looking at I'm like, oh
Speaker 2: my gosh, he he just looks so much like me.
Speaker 2: And then b I look and I see his wife Stacy,
Speaker 2: and I like see in her eyes this deep concern,
Speaker 2: this care. You can just see right away that like,
Speaker 2: you know, this is her everything right now in the
Speaker 2: hospital bed and she's losing him, and it's like you
Speaker 2: just feel it when you see her. And I look
Speaker 2: at this window and I'm like, I recognize this view.
Speaker 1: You've said that when David first visited you that you
Speaker 1: immediately clutched at the hope that you could get there,
Speaker 1: that you could get where he is. Did that tap
Speaker 1: into your like marine determination spirit?
Speaker 4: Yeah, that, and in particular that he was leading a
Speaker 4: research effort and said that I could contribute to it,
Speaker 4: that I could not only fight my way out of
Speaker 4: the bed, but help him the whole disease was huge.
Speaker 4: To turn from this helpless two hundred and sixty pound
Speaker 4: bag of fluid into somebody who was fighting was a
Speaker 4: huge turning point for me up until that point. It
Speaker 4: was something that I was looking for, and that was
Speaker 4: part of my frustration, like why can't we take care
Speaker 4: of this? Why can't we hit this back, and so
Speaker 4: that was the turning point where he said, you know,
Speaker 4: will you contribute samples to the research and that was
Speaker 4: no brainer I was. I was like, hell, yes.
Speaker 2: I remember telling him that you know, we're going to
Speaker 2: beat this, and I really believe it because i'd been
Speaker 2: in his bed, although a minute or two later I
Speaker 2: learned that literally had been in his bed because when
Speaker 2: I walked out, one of the nurses came over and
Speaker 2: gave me a hug, and I actually didn't know who
Speaker 2: she was or you know, what was going on, and
Speaker 2: she said, I was your nurse when you were here
Speaker 2: a few years ago, which I didn't realize. Was again,
Speaker 2: when you're a nice you oftentimes you know you're just
Speaker 2: out of it, and so I didn't realize that. And
Speaker 2: she said, and this was your hospital room, and I
Speaker 2: was like, oh my gosh. That's why I recognized that
Speaker 2: view out the window because that's the window I'd been
Speaker 2: looking at for weeks and weeks and weeks, and I
Speaker 2: just dreamed. I was like, one day I could get
Speaker 2: out of here.
Speaker 1: I get the hairs on my arms, stand up on
Speaker 1: that It's amazing. In the same exact shoes and room
Speaker 1: as David once was. The challenge was now to rethink
Speaker 1: Gary's treatment. Under the care of David and the CDCN,
Speaker 1: who were busily working behind the scenes on repurposed treatment
Speaker 1: options for rare diseases, there was a renewed sense of hope.
Speaker 2: Well, first you got a Castleman's drug that we thought
Speaker 2: would work, and that drug seemed to work a little bit,
Speaker 2: and then he relapsed, and then we ended up giving
Speaker 2: him a bunch of chemotherapy that sort of seemed to
Speaker 2: work and get him out of this thing. But the
Speaker 2: question became, how do we keep it from coming back.
Speaker 2: We were getting blood samples on Gary basically every day,
Speaker 2: and as soon as we get the blood sample, we'd
Speaker 2: run down to the lab. We'd run something called flow sytometry,
Speaker 2: and we were getting results back and looking at those
Speaker 2: results and then saying, you know, is there a different
Speaker 2: drug we should be using based on what we're seeing
Speaker 2: in the lab. And it was like this race against time.
Speaker 1: It was clear to everyone that Gary's time was running out.
Speaker 1: His body was too weak to push through continued chemotherapy.
Speaker 1: David and the CDCN needed to produce results immediately if
Speaker 1: they were going to save Gary's life.
Speaker 2: I get so excited every time we save a life
Speaker 2: and we find a drug that can help someone, and
Speaker 2: that's it's just incredible. But even more incredible, or at
Speaker 2: least even greater of a feeling than how thankful we
Speaker 2: are when it works, is just how devastated we are
Speaker 2: when these drugs don't work. And it's both of those
Speaker 2: emotions that just drive us to keep working. You get
Speaker 2: a carrot and a stick, and it's just like you're
Speaker 2: just constantly chasing, and you get constantly motivated by both
Speaker 2: the people who are here because of our work and
Speaker 2: also the people who aren't here despite our work.
Speaker 1: Gary, was there a time time that you felt like
Speaker 1: it was pointless to keep fighting? Were you in that
Speaker 1: much exhaustion and pain at any point to think I
Speaker 1: can't do this? And what was it that pulled you
Speaker 1: out of that? So?
Speaker 4: I would visualize them as scenarios, But what I kept
Speaker 4: feeling was myself approaching some sort of a boundary or
Speaker 4: a doorway or that sort of thing. And it wasn't
Speaker 4: like a blessed realm where your loved ones come and
Speaker 4: say now is not your time? Or you know, there
Speaker 4: were no singing or anything like that. I would always
Speaker 4: see my wife and my kids, and I would turn
Speaker 4: away from that and turn turn toward them with my will.
Speaker 4: The first flare was I am not going anywhere near there.
Speaker 4: I want to find the way toward what I knew.
Speaker 4: I wanted to find my way toward awareness. I was
Speaker 4: so frustrated that we know what this is. Why am
Speaker 4: I still here? Why am I stuck in the state.
Speaker 4: None of us liked the lows. I wish I could
Speaker 4: give all these lows back. But the lows in some
Speaker 4: ways can really create this sort of like potential energy
Speaker 4: or like this like slingshot effect, and then it's that
Speaker 4: horrible moment that results in action, and that action results
Speaker 4: in something that we celebrate.
Speaker 1: We'll be right back with Symptomatic a Medical Mystery Podcast.
Speaker 1: Now back to Symptomatic a Medical Mystery Podcast. Gary Gravina,
Speaker 1: a former carpenter and marine, went from his usual routine
Speaker 1: to being placed in a medically induced coma within a week.
Speaker 1: Having supported his wife through the loss of his stepson,
Speaker 1: Gary was determined to prevent her from experiencing that pain again.
Speaker 1: But after the treatment for his Castleman disease had failed,
Speaker 1: Gary's last hope was the research on alternative treatments from
Speaker 1: doctor David Fagenbaum. Initially, though I know that they had
Speaker 1: tried a medicine that had worked on other Castleman's patients
Speaker 1: that didn't work on you. Was there in those initial
Speaker 1: attempts to find what for you could be the silver bullet?
Speaker 1: Was it devastating or was it more just like bring
Speaker 1: on the next No?
Speaker 4: And when I think about David having five flares, there
Speaker 4: was a difference in my energy. I was on celtuxamab
Speaker 4: to get out of the first flare, and I thought
Speaker 4: that was going to do it. And I think after
Speaker 4: my third treatment, my blood all looked like it was
Speaker 4: trending in the right direction. And then like a week later,
Speaker 4: I was going into my second flare and it was
Speaker 4: just not working anymore. It had quit, and I thought,
Speaker 4: oh no, not again, Like can this just be some
Speaker 4: other thing that's going on a side effect? But it
Speaker 4: had that same feeling, that same I cannot lift myself
Speaker 4: to push through this. The day before at physical therapy,
Speaker 4: I was pushing and pushing and getting better and better
Speaker 4: results every day, and there were stuff I just couldn't
Speaker 4: do that day, and the physical therapist said, you know,
Speaker 4: make sure you reach out to your doctor and let
Speaker 4: them know that you've had this little turning point.
Speaker 1: Despite some signs of relief and a brief return to routine,
Speaker 1: Castleman continually returned, sending Gary back to the ICU and
Speaker 1: David back to the drawing board.
Speaker 4: Even during my second player being comatose again. That fueled
Speaker 4: the anger that we know what this is? Why am
Speaker 4: I stuck in the state again? And you're in the
Speaker 4: ice your room, and you know all the things are
Speaker 4: connected to you, and you're just kind of you feel
Speaker 4: apart from everything, and you start to wonder why am
Speaker 4: I being singled out? Like am I being punished?
Speaker 3: What did I do?
Speaker 4: I must have done something to deserve this, And I
Speaker 4: can just remember crying tears of frustration.
Speaker 5: David.
Speaker 1: It has to be kind of like solving a puzzle
Speaker 1: and playing chess at the same time.
Speaker 2: Yes, that's exactly right. And I think that for me.
Speaker 2: You know, before I got into this world, when I
Speaker 2: heard a doctor say we've tried everything, there's nothing more
Speaker 2: that we can do. I sort of assumed that meant that,
Speaker 2: like there were no more chess pieces on the chess
Speaker 2: board and there were no more puzzle pieces. But I
Speaker 2: think what I've subsequently learned is that when a doctor
Speaker 2: says we've tried everything, many times I think they are
Speaker 2: right and that there isn't actually something that could help
Speaker 2: that patient. But many times I also believe, and I've
Speaker 2: seen and witnessed that actually there is something that we
Speaker 2: can do. There is that chess piece that's on the board,
Speaker 2: but we the medical community, just haven't unlocked the potential
Speaker 2: of it.
Speaker 1: Grant breaks down why rare diseases are often referred to
Speaker 1: as orphans.
Speaker 3: An orphan disease is very much what it sounds like.
Speaker 3: It's a disease that very few people get, defined by
Speaker 3: less than two hundred thousand people having it, and it
Speaker 3: actually represents the majority of the diseases that are out there,
Speaker 3: depending on how you count diseases. But let's say if
Speaker 3: there's twelve thousand, the vast majority, let's say almost nine
Speaker 3: thousand of those are rare, and most of those don't
Speaker 3: have an approved treatment, not a single approved treatment. Ninety
Speaker 3: five percent of rare diseasees don't have a single afday
Speaker 3: approved treatment.
Speaker 1: Wow, because there's not the money in it, because the
Speaker 1: numbers aren't driving it.
Speaker 3: Yeah, and you know, the pharmaceutical industry. It's a business,
Speaker 3: and they are shared holders, and there's fiduciary obligations to
Speaker 3: those shareholders to make money and return their capital. So
Speaker 3: I don't blame them for not focusing on diseases that
Speaker 3: don't make money. That's how the free market works. But
Speaker 3: that means that there's a lot of diseases that ultimately
Speaker 3: get neglected and there's no incentive to focus on them,
Speaker 3: and so that really bothered us.
Speaker 1: Gary David talks about a point where he was told
Speaker 1: by one of the nurses that you weren't gonna make it.
Speaker 4: Yeah, and I think there was at least one night
Speaker 4: in both of my flares where it was just that close.
Speaker 4: But I was in a coma and he was there
Speaker 4: with Stacy during the second flare, and I was just,
Speaker 4: you know, everything in me was tanking.
Speaker 2: He was on a ventilator, he was receiving what are
Speaker 2: called pressers, which is basically, when your heart's failing, you
Speaker 2: give drugs to keep the heart beating as best as
Speaker 2: it can, even though it's like basically dying. Without the pressers,
Speaker 2: you die. But just trying to say, you know, we're
Speaker 2: we're still looking for drugs for you, Gary, like I'm
Speaker 2: gonna take your sample to the lab. We're going to
Speaker 2: keep fighting for you. And as I walked out of
Speaker 2: his room, I remember his nurse pulling me aside and saying, David,
Speaker 2: this is it. You know, I'm an ice, you nurse,
Speaker 2: this is what I do. I'm seeing the changes in
Speaker 2: his hands and his feet that occur when they're is
Speaker 2: no coming back, Like, yes, his heart is technically beating. Yes,
Speaker 2: he's technically breathing because of what we're doing medically, but
Speaker 2: he's not going to come back from this. And I
Speaker 2: just remember being so so devastated to hear that, and
Speaker 2: then at the same time remember still running down the
Speaker 2: hall to the lab and still running the experiments because
Speaker 2: like who knows.
Speaker 4: And he was telling Stacy, there is hope, don't give
Speaker 4: up hope. And as he was leaving, this ICU nurse
Speaker 4: who had actually been there when he was there, said,
Speaker 4: you should not have told her that you're giving her
Speaker 4: false hope. He's not going to make it through the night.
Speaker 4: And my understanding is she went in and told Stacy
Speaker 4: he's that bad. You need to be ready for that.
Speaker 2: And we still ran the fless atometry and a sample,
Speaker 2: and we still thought about work for the next drug
Speaker 2: be but I.
Speaker 4: Mean it to them, meant.
Speaker 1: Gary not only survived the night, but made it through
Speaker 1: the entire flare up. David and the CDCN found it
Speaker 1: treatment regimen that put his Castleman disease into remission, one
Speaker 1: that was originally developed and approved to treat lymphoma. As
Speaker 1: the weeks passed and turned into months, they realized this
Speaker 1: repurposed to medicine had given Gary a second chance at life. So, David,
Speaker 1: if you can think of what if you look back,
Speaker 1: is probably the highest happiest moment in terms of Gary.
Speaker 2: There are a few. I think seeing him get into
Speaker 2: a wheelchair and get rolled out of his hospital room
Speaker 2: was just amazing. You know, shortly before that we didn't
Speaker 2: know if he was going to make it, and then
Speaker 2: now I get to watch him, you know, get wheeled
Speaker 2: down the hall.
Speaker 4: If I don't feel a flare coming on, I have today,
Speaker 4: and today is beautiful. It's good. It makes me enjoy
Speaker 4: moments more, and it makes me more ready to accept
Speaker 4: other setbacks because a different sense of scale and proportion
Speaker 4: when problems arise, I see what can continue. I see
Speaker 4: a way to fight through those things a little better
Speaker 4: knowing that, you know, if I wake up tomorrow, that
Speaker 4: could be the last thing I remember for a long
Speaker 4: time before I start that fight all over again.
Speaker 1: Grant and David maintain their collaboration through the early days
Speaker 1: of their divergent career paths, continually coming back to the
Speaker 1: idea of scaling the cdcn's research. Impacted by Gary's experience
Speaker 1: and the rise of AI data aggregation, they saw new
Speaker 1: potential for revolutionizing their approach to repurposing medicines.
Speaker 2: And so Grant and I spent a lot of time
Speaker 2: thinking about could we start an initiative, an effort to
Speaker 2: try to unlock more uses for fdapproved drugs. In about
Speaker 2: two years ago is when we launched this nonprofit called
Speaker 2: every Year, and we are on a mission to unlock.
Speaker 3: The life saving potential of every FDA approved medication to
Speaker 3: treat every disease that it possibly can. There's nothing more
Speaker 3: motivating that seeing a patient that is now able to
Speaker 3: be with their family and live their life to their
Speaker 3: fullest because of the science that you did. It's an
Speaker 3: amazing thing that you get to participate in. And so
Speaker 3: that's always the sort of the guiding light, the north star.
Speaker 1: So David how did Gary's case play a big part
Speaker 1: of the evolution of every cure?
Speaker 2: You know, my book's called Chasing My Cure, because of
Speaker 2: course this all started out well, even before Chasing My Cure,
Speaker 2: it was obviously witnessing my mom's battle, but this all
Speaker 2: really started with Chasing My Cure. And I've said before
Speaker 2: and I really believe it, we should have called it
Speaker 2: Chasing our Cure, because, as you know, and as you've
Speaker 2: heard us discussed, there were a lot of people part
Speaker 2: of us. It was actually not me, it was a
Speaker 2: lot of us, But it started out that way. But
Speaker 2: then for every patient like Gary, for every patient like
Speaker 2: Kyla who's getting ready to go off to college because
Speaker 2: we discovered a drug to save her life just before
Speaker 2: she would have passed, and patients like Joseph who right
Speaker 2: now is recovering after we found a drug to save
Speaker 2: his life just a few months ago. Each one of
Speaker 2: those patients has just led us to say, we are
Speaker 2: so happy we did this for them, But how many
Speaker 2: more they're out there?
Speaker 3: And what began to dawn on us throughout the years,
Speaker 3: because my career went off into the world of machine
Speaker 3: learning and AI, was that the way to unlock this
Speaker 3: problem that we have been able to start as a
Speaker 3: business is potentially to use artificial intelligence and structured as
Speaker 3: a nonprofit, and that was really what unlocked our next
Speaker 3: sort of journey, which was become every cure grant.
Speaker 1: It's brilliant, it really is. A lot of people are
Speaker 1: frightened by AI, but I can't think of a more
Speaker 1: wonderful way to utilize it.
Speaker 3: This technology could be utilized in maybe a different context
Speaker 3: that maybe instead of focusing on one drug and one
Speaker 3: disease at a time for a company, maybe we could
Speaker 3: widen the lens. Maybe we could look across all diseases
Speaker 3: and all drugs and find the interconnectivity between them to
Speaker 3: figure out which drugs should work for which diseases.
Speaker 1: With all the conversations and fear around AI and its
Speaker 1: evolution upending certain industries, it's heartening to see a powerful
Speaker 1: and potentially transformative application to save people's lives and offer
Speaker 1: different treatment options for rare diseases.
Speaker 2: Over the last few years, even just the last two years,
Speaker 2: there has been so many advances so that whereas what
Speaker 2: we used to do in my lab, where we would
Speaker 2: study Castlin disease for example, or related diseases, you know,
Speaker 2: we spend years working on a proteomic study or sequencing study,
Speaker 2: for example, years to come up with an insight. We're
Speaker 2: really proud of the progress we've made. But now utilizing
Speaker 2: artificial intelligence, we can actually apply AI to the world's
Speaker 2: biomedical knowledge.
Speaker 3: And we would talk all the time about what is
Speaker 3: the potential here, what is the potential to scale what's
Speaker 3: happening in lab by doing it in the data and
Speaker 3: then doing it beyond just one drug and one disease
Speaker 3: at a time, And we would check in on this
Speaker 3: every few years. Twenty sixteen, it wasn't really possible to
Speaker 3: think as big as we were thinking. The data just
Speaker 3: wasn't there, And it really wasn't until the last few
Speaker 3: years that it started to dawn on us that maybe
Speaker 3: something truly was possible here. So ultimately, what we do
Speaker 3: is we compile every kind of data that exists in
Speaker 3: the world that might be able to link one biomedical
Speaker 3: concept to another. So does a drug hit a target,
Speaker 3: where is that target on this protein, what gene generates
Speaker 3: that protein, what pathway does that protein sit in, what
Speaker 3: cell type, what organ system.
Speaker 2: Of course, the predictions are not going to be perfect,
Speaker 2: and we're not going to say, oh, just because it's
Speaker 2: number one on our list, it's going to work for everyone.
Speaker 2: We still need to do clinical trials to validate it,
Speaker 2: but the ability to focus in on all the possibilities
Speaker 2: to the ones that look most promising is just something
Speaker 2: that's never been possible before in human history.
Speaker 1: Grant, what are you most proud of in terms of
Speaker 1: every cure?
Speaker 3: I think I'm most proud of the fact that we
Speaker 3: have not let this go. And then I'm so proud
Speaker 3: of the people that come and join our team. These
Speaker 3: are some of the smartest people in the world that
Speaker 3: could have higher paying jobs, that could be in Silicon
Speaker 3: Valley doing other things, and they are compelled by this mission.
Speaker 3: They're compelled by the ability to relieve suffering and save lives,
Speaker 3: and they're compelled by the opportunity to create an engine
Speaker 3: that should outlast us and will save lives in perpetuity,
Speaker 3: whether we're working at every cure or not. In the future,
Speaker 3: we want to create something that lasts in something that
Speaker 3: has major impact.
Speaker 2: Over fifteen drug where we've identified in or advanced a
Speaker 2: repurpose drug for a disease they weren't intended for. And
Speaker 2: I'm so proud of that.
Speaker 1: Through the work at the CDCN and every cure over
Speaker 1: the past ten years, David and Grant have tirelessly worked
Speaker 1: to prevent others from enduring the suffering of orphan diseases.
Speaker 1: David and Gary were both on the brink of death,
Speaker 1: told there was nothing left to do, just to find
Speaker 1: out that a medicine that already existed could be the cure.
Speaker 1: Despite having every reason to give up, they persevered, Gary,
Speaker 1: what do you want people to take away from your
Speaker 1: experience and what got you through it?
Speaker 4: Well, that's a toughye. So through the CDCN, I try
Speaker 4: and act as an ambassador to other people who are
Speaker 4: facing Castle and disease and just let them note that
Speaker 4: they're not alone. It was right after my last round
Speaker 4: of chemo and the second FLARE that I attended the
Speaker 4: first Patient Loved One summit. I was in a room
Speaker 4: full of sixty people who had Castle of disease or
Speaker 4: who had a loved one who had Castle and disease.
Speaker 4: And so when I talked about that sense of having
Speaker 4: felt singled out and being punished, that was the polar opposite.
Speaker 4: That was a huge turning point to be around other
Speaker 4: people who they're describing things, and I'm like, yeah, I
Speaker 4: get that. So that was a big thing for me
Speaker 4: to really learn to enjoy connecting with people. And you
Speaker 4: just have that sense of other people being with you.
Speaker 4: You fight better when you have somebody by.
Speaker 1: Your side, especially someone like doctor David Fagenbaum. I have
Speaker 1: to tell you it is such a comfort to me
Speaker 1: to know that people like you exist. And I am
Speaker 1: deeply indebted to your mother, thank you, and to your father,
Speaker 1: your sisters, to Caitlin, because anybody who has gotten you
Speaker 1: through what you've gone through to make it possible for
Speaker 1: me to be sitting and talking to you today honestly
Speaker 1: in their debt and the world is too. I don't
Speaker 1: say that lightly. What do you want people to take
Speaker 1: away from your story, from your personal challenges?
Speaker 2: I think the things that immediately come to mind are
Speaker 2: that no matter how tough things get, no matter how
Speaker 2: low the odds are, no matter how difficult things are,
Speaker 2: I think just realizing that there may be a path
Speaker 2: out of this. At the same time, I think it's
Speaker 2: also important emphasize that the path out isn't necessarily going
Speaker 2: to be delivered on your door, and that for me,
Speaker 2: the reason I'm alive is not because I got really
Speaker 2: lucky multiple times. It's because I had the right circumstances
Speaker 2: around me, the amazing family, the amazing support, and I
Speaker 2: started taking action. And that doesn't mean that anyone who's
Speaker 2: dealing with illness or death needs to start experimenting on
Speaker 2: their own blood samples or starting a grief organization. But
Speaker 2: it does mean that you need to take action in
Speaker 2: ways that makes sense for you. And so that might
Speaker 2: be getting involved with the disease organization for the disease
Speaker 2: that you have. It might be driving in your car
Speaker 2: to the world's expert because, like you know, your local
Speaker 2: doctor isn't doing what you need. I do think it
Speaker 2: takes action.
Speaker 1: If you'd like to find out more about the work
Speaker 1: Grant and David and the rest of the team at
Speaker 1: Every Cure Doing, visit Everycure dot org. They emphasize the
Speaker 1: importance of raising awareness and donations as the best way
Speaker 1: to support their ongoing efforts.
Speaker 4: My name is Gary Gravina, I'm David Fagenbaum, and I
Speaker 4: fought Casselman disease for nine months.
Speaker 2: And after nearly dying five times in three years from
Speaker 2: a deadly disease called Castleman.
Speaker 4: Disease, David Fagenbaum and the CDCN came up with a
Speaker 4: regimen that has held me in remission. I've now been
Speaker 4: alive for over ten years, for almost eight years now.
Speaker 2: Thanks to a drug that I discovered that I had
Speaker 2: been sitting on my pharmacy shelf all along, and now
Speaker 2: I'm on a mission to try to find as many
Speaker 2: more uses of existing drugs as possible to save as
Speaker 2: many lives as possible.
Speaker 1: Next week on Symptomatic, we dive into the modern landscape
Speaker 1: of prostate cancer and care. We'll explore emerging therapies, new research,
Speaker 1: and some of the biggest hurdles faced by both patients
Speaker 1: and healthcare professionals when trying to fight this deadly disease.
Speaker 1: We're also excited to announce that Symptomatic will return for
Speaker 1: a full season later this year. Thank you for trusting
Speaker 1: us to share your stories. If you've experienced a mysterious
Speaker 1: diagnostic journey or have a story of interest you want
Speaker 1: to share, reach out to us at Symptomatic at iHeartMedia
Speaker 1: dot com and keep an eye on your feed Until then,
Speaker 1: Stay well. Symptomatic Medical Mystery Podcast is a production of
Speaker 1: Ruby Studio from iHeartMedia. Our show is hosted by me
Speaker 1: Lauren breg Pacheco. Executive producers are Matt Romano and myself.
Speaker 1: Our EP of post production is James Foster. Our producers
Speaker 1: are Sierra Kaiser and John Irwin. And this episode was
Speaker 1: researched by Diana Davis