January 22, 2025 • 27 mins
Not just seeking treatment for every disease, but making sure the treatments we have already developed are being used in every possible way. I speak with Dr. David Fajgenbaum (Fay-gen-baum), a Professor of Translational Medicine and Human Genetics at the University of Pennsylvania School of Medicine, and President of Every Cure.
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Episode Transcript
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Dr. Moira Gunn (00:11):
Flipping the script on the idea of for every
disease, a drug. To for everydrug, all the diseases it can
treat. I'm speaking with doctorDavid Fajgenbaum, a professor in
translational medicine and humangenetics at the University of
Pennsylvania School of Medicineand the cofounder of Every Cure.
(00:34):
And now, doctor DavidFajgenbaum. Well, David, welcome
to the program.
Dr. David Fajgenbaum (00:40):
Thanks so much for having me.
Dr. Moira Gunn (00:42):
Now I learned from your website, which is
everycure.org, that there areabout 3,000 FDA approved medical
treatments which address anynumber of medical conditions.
And that's in total, 3,000 inthat area. But to quote that
website, no organization isresponsible for ensuring that
all approved medicines are usedto treat
Dr. David Fajgenbaum (01:04):
every disease they possibly can. And
that's true. That's right.
Dr. Moira Gunn (01:04):
They come through to cure a particular
disease possibly can, and that'strue.
Dr. David Fajgenbaum (01:07):
That's right.
Dr. Moira Gunn (01:08):
They come through to cure a particular
disease or address it. Tell usabout that.
Dr. David Fajgenbaum (01:12):
Sure. I mean, it it's really been
surprising for me to get intothe space and to learn this. I
mean, we think about theincredible effort and resources
that go into getting one drugapproved for one disease. I
mean, literally, 1,000,000,000of dollars and, in some cases,
more than 10 or 20 years for onedrug for one disease. And so you
would just sort of assume thatthe work would be done to figure
out all of the diseases thatcould possibly benefit from that
(01:34):
drug.
But, unfortunately, in ourcurrent system, there are
incentives in place that wouldmake a company pick one disease
over another disease. And thenonce that drug becomes generic,
there's really no incentives tofind more uses for them.
Dr. Moira Gunn (01:45):
So, once the patent goes off expiration,
anybody can make it, thenthere's no real real big
profits.
Dr. David Fajgenbaum (01:52):
That's exactly right. There's no
profit. There's no incentivewhatsoever. I mean, really, once
a drug is generic and over 80%of drugs are already generic,
there's no incentive to to findmore uses for these drugs. So
the tools that are within ourreach, the drugs that are
literally on our pharmacyshelves to help patients are the
ones that we're not studying.
They're the ones that we're notfully utilizing.
Dr. Moira Gunn (02:12):
Now let me flip the question. How many medical
conditions today are in need ofmedications?
Dr. David Fajgenbaum (02:19):
It's a huge number. We like to think
about all the progress thatwe've made as a medical
community, and and we should bereally proud. As you said, 3,000
approved drugs. Those 3,000drugs are approved for about
3,000 diseases, and that isreally amazing. But depending on
what classification system youuse, there's anywhere between
9,017,000 more diseases thatdon't have a single approved
(02:43):
therapy.
Dr. Moira Gunn (02:44):
Now these aren't just one offs. There's millions
of people who have thesediseases.
Dr. David Fajgenbaum (02:49):
That's right. It's estimated about 1 in
10 Americans have one of thesebetween 9,017,000 diseases that
don't have an approved therapy.1 in 10 of all of us listening
and and part of the show rightnow either has or will develop a
disease without a singletreatment.
Dr. Moira Gunn (03:04):
Now you come to Every Cure. You cofounded it.
It's a nonprofit. And what is ittrying to do?
Dr. David Fajgenbaum (03:11):
Sure. Every cure is on a mission to
make sure that every FDAapproved drug is utilized for
every disease possible, andwe're utilizing the world's
biomedical knowledge. All theincredible work that's been done
by pharmaceutical companies andacademic groups and and others
leverage all of that knowledgeto figure out what are the
possible uses for every approveddrug. And let's go out and let's
actually drive forward advancethe trials to prove that they
(03:33):
actually work regardless ofwhether it's profitable to do
that or regardless of whether,the disease population is large
or small, just what are thethings that we can do to help
people as quickly as possible?
Dr. Moira Gunn (03:44):
I have to say, I can imagine you're sitting
there. You have a you have acondition. There's some drugs
there, but maybe not working foryou. They're working on new
ones. I mean, it just frost youto think, you mean, we have some
drugs that might already work.
Dr. David Fajgenbaum (03:57):
Yeah. It's kinda mind blowing. Right? I
mean, and I know that we'll chatabout this later on, but as
someone who's gone throughmedical challenges, we tend to
think that the drugs that, youknow, our our doctors tell us
about are are the only drugsthat could possibly treat our
diseases. But there's this wholeliterally, pharmacy worth of
(04:18):
other drugs that have thepotential to maybe treat us or
someone we love.
Dr. Moira Gunn (04:22):
1st, you're building a database, a drug
repurposing database. What isit? What's in it? What's in that
database?
Dr. David Fajgenbaum (04:31):
Sure. So right now, it's about 70
different databases worth ofdatasets. And so, basically, the
world's knowledge of what do weknow as a as a human society
about all the drugs that areapproved? What do we know about
all the diseases that affecthumans? What do we know about
all the genes in our bodies?
What do we know about all of theways those genes interact with
one another? So imagine saying,I wanted to get, like, what the
(04:53):
world knows about everythingthat could be relevant. That is
in our, basically, database ofdatabases, all that information
in one central place.
Dr. Moira Gunn (05:02):
And, of course, they're all different formats,
different limits, differentthey're okay. So you've got a
lot of it's a tower of babble ofmedical information.
Dr. David Fajgenbaum (05:09):
That's right.
Dr. Moira Gunn (05:10):
But you'll say bring it on. If it's good data,
bring
Dr. David Fajgenbaum (05:12):
it on.
The more the data, the better.
Dr. Moira Gunn (05:14):
Now frequently, when we are prescribed a
medicine, they're going by oursymptoms. They're not really
able to go inside and see thedisease. So at what level are is
some of this data?
Dr. David Fajgenbaum (05:28):
Oh, that's a great question. So, the data
that we're collecting is onevery level from what's
happening within the cell ofsomeone with the disease all the
way through how do patientsperform or or respond to a drug
in a clinical trial, forexample. But in terms of the
recommendations that come out ofit, and and I know we'll we'll
get to that in a minute, butthat's on a disease level. So
it's basically gonna say, foryour disease, what is the rank
(05:50):
ordered list of all 3,000 drugsin terms of how likely they are
to treat you? You.
So, you know, the first couplemight be the first two drugs
that are approved for yourdisease. The next couple might
be a couple drugs that maybehave been utilized in a few
patients here and there. Andthen the other almost 3,000
drugs are gonna be where we'reutilizing artificial
intelligence to help us to comeup with a score for how likely
that drug is to treat yourdisease.
Dr. Moira Gunn (06:12):
So we got 3,000 drugs possible. And how many
databases do you have?
Dr. David Fajgenbaum (06:18):
So right now, we're at about 80, but
we're eager for people to donateadditional data to us. We we
really want to get as much dataas possible. The 80 databases
are fairly comprehensive interms of what's publicly
available. So a lot of times,when the federal government
funds research, therequirements, the data be made
publicly available so anyone candownload it. We've gotten all
those datasets.
What we don't have are theproprietary private datasets
(06:41):
from companies like Elsevier andClarivate and Wolters Kluwer
where they charge subscriptionsfor access. We're hopeful to
partner with all of them. And ifanyone from from those companies
is listening, we would love towork with you.
Dr. Moira Gunn (06:51):
Bring it on. Send us a password.
Dr. David Fajgenbaum (06:52):
Yeah. Bring it on. Yeah. Exactly.
Donate the data to us.
You know, we're a nonprofitorganization literally just on a
mission to save as many lives aspossible in the shortest period
of time. And the way to do thatis not to develop a new drug
from scratch. It's to use thetools already within reach.
Dr. Moira Gunn (07:07):
And I have to say, when we're talking about
these datasets, frequently,we're talking about clinical
trial datasets, which are great.They have a lot of information
in them. And these are thousandsof people over time, lots of
data, all kinds of data, youknow, from MRIs to blood test,
amazing kind of information thatyou haven't even tapped in yet,
(07:30):
but are relevant possibly to oneor another drug or in one or
another condition. This is anenormous, enormous task to just
look at the size of the data andthe complexity of linking it. I
mean, there's some human inthere that has to kinda do that
translation so the AI can get inand do its work.
(07:51):
How do you do you just have awhole staff of people that are
called inkers?
Dr. David Fajgenbaum (07:56):
I don't know how you do this.
There's actually a whole field that's
called harm data harmonization.And so we work with an amazing
researcher, Melissa Heindel,who's at the University of
Colorado, where she has a teamof about 20 data harmonizers,
and they literally look betweenall these datasets and try to
harmonize the data so that itcan be linked. You know, are we
using the same unit for this?Are we calling, is it called
(08:20):
Castleman disease or is itCastleman's disease? You know,
ways to basically make surewe're all using the same
language, the same vocabulary.
And so they're one of our closepartners, and, we're we're
thankful that they're doing theharmonization so that way we can
make the connections.
Dr. Moira Gunn (08:34):
Okay. So do the math. 3000 drugs, 80 datasets of
all kinds of things, and you'relooking for more. Lots and lots
more. 20,000 diseases.
You know? You're not doing thison an Excel spreadsheet.
Dr. David Fajgenbaum (08:46):
No. Yeah. It's been it's been described by
a few folks in this phase inthis space. One recently
described it, as, not a moonshot, but like a Mars shot. It's
like further than the moon.
And and another person describedit as as the Manhattan Project.
And so, yeah, it's a hugeeffort. But to me, as a
physician, as a scientist, as apatient, as an advocate myself,
(09:09):
I can't imagine how we couldhave gone this long without ever
doing this. You know, as someonewho has benefited from drugs and
also developed drugs forpatients, again, I just can't
believe that our society hasn'tcome together to say, we're
gonna take on this Mars shot.You know, we're gonna take on
this Manhattan Project to makesure that every drug is fully
utilized.
And so after spending about 10years, working within this space
(09:34):
and trying to figure out who'sdoing this and learning that no
one's doing it, we finallydecided that we needed to do it.
Dr. Moira Gunn (09:39):
You're listening to Tech Nation. I'm Moira Gunn.
My guest today is doctor DavidFaganbaum, an associate
professor in translationalmedicine and human genetics at
the University of PennsylvaniaSchool of Medicine. He's also
the cofounder and president ofthe nonprofit Every Cure. His
2021 book is Chasing My Cure, ADoctor's Race to Turn Hope Into
(10:02):
Action.
Well, I said I was gonna ask youabout this at the end, but I
gotta ask you about it now.
Dr. David Fajgenbaum (10:07):
We're always asking, you know, if
people are actually walkingtheir talk. You did the
Dr. Moira Gunn (10:07):
walk. If people are actually walking their talk.
You did the walk, and now you'vemade it your talk.
Dr. David Fajgenbaum (10:16):
So we
just talked about your talk.
Dr. Moira Gunn (10:19):
Tell us how you got here, and it's it's almost a
miracle you are. Tell us aboutthat.
Dr. David Fajgenbaum (10:24):
Sure. Well, in 2010, I was a 3rd year
medical student here at theUniversity of Pennsylvania. I
was treating patients. I, waswas healthy, and, I on a
mission. I wanted to become adoctor in memory of my mom.
She had passed away from cancera few years before. And, out of
nowhere, I became criticallyill. The same hospital I was
(10:46):
treating patients, all of asudden I was in ICU. All of my
organs were shutting down. I wason life support requiring daily
transfusions just to keep mealive, all with no diagnosis.
That lasted for months that Iwas in the ICU. I was eventually
diagnosed with a rare diseasecalled Castleman disease, where
there were no approvedtherapies. And I I spent a total
(11:07):
of about 6 months hospitalized.I had my last rights read to me.
I, you know, just struggled withthis condition, received a lot
of chemotherapy, that was usedfor my disease.
And chemo kept saving my life,but then I kept relapsing. And,
you know, about 2 years intothis journey, I was in the
middle of med school and I was,you know, dealing with this
illness. I realized that Icouldn't just hope that some
(11:29):
researcher somewhere would finda drug for me. I mean, my doctor
explained to me that he was outof options. He said, there are
no more drugs to try for you.
This is it. We've triedeverything. And so I I know I
couldn't just wait for someoneelse to find something that if I
wanted any chance of survival, Iwould need to get involved in
research myself. And so Icreated an organization called
the Castle Disease CollaborativeNetwork, and I got to work. I
started performing experimentson my own blood samples.
(11:51):
I started going through all thestudies out there. We talk about
the world's biomedicalknowledge. I I probably read
through the whole world'sbiomedical knowledge trying to
find a drug to save my life.And, over the course of about a
year, I ended up discovering anold drug called sirolimus. It's
been around for decades.
It had never been used for mydisease, and I thought it might
work. And, so I began testing iton myself. And actually, this
(12:13):
month marks 10 years that I'vebeen in remission on this drug
that wasn't made for my disease.It wasn't supposed to be given
me. I'm not supposed to be here,but because of that drug, I'm
alive.
And as you said earlier, nowit's my mission to find as many
of these drugs as possible tosave patients as quickly as
possible.
Dr. Moira Gunn (12:29):
Yeah. When they say, well, we don't believe you
can do it, you say, well, sitdown.
Dr. David Fajgenbaum (12:33):
Exactly. I gotta sit down. Sorry. Exhibit
a. Right?
I'm like, yeah. Exact you know,you're right. Because there are
a lot of people who are like,you know, this is, you know,
this is too ambitious andaudacious. You know, drug
companies spend 1,000,000,000 ofdollars to develop drugs. You
know?
How is your nonprofit going togonna make a huge difference?
And, yeah, exactly the answeris, well, we already have, and
(12:54):
now we just wanna do it atscale.
Dr. Moira Gunn (12:56):
Okay. So if I was in a typical pharmaceutical
or biopharmaceutical company andwe were choosing drugs, we have
we have a number of candidates.You're sitting there going,
okay. Well, we're we've got allof these scores. And you
recently figured out how many ofthese scores you did.
Now let's take us from therebecause from there, we're gonna
(13:18):
go down the plan. What whatyou're planning on doing?
Dr. David Fajgenbaum (13:21):
Sure. So, yeah, I think there's 2 places
to go, from those importantcomments you made. I mean, first
off, when a drug companydevelops a new drug, on average,
somewhere between 30 40 diseasesare considered for that single
drug. So drug companies, youknow, have a promising product
and they start saying, you know,is it this disease or that
disease? As many as 30 to 40 areconsidered, and that drug
(13:42):
company has to make really toughdecisions to pick the 5 or so
that it'll actually study intrials.
1 will get an approval, andmaybe they'll pursue 1 or 2
additional drugs to add to thelabel to make that drug approved
for between 1 and 3 things. Butremember, if you go back to the
very beginning, now there's 20to 30, maybe even 40 other ideas
that haven't been pursued atall. There are great ideas. Some
really smart people in the drugcompany considered them. And so
(14:05):
so that just sort of tells youthe vast potential here is, you
know, the untapped potential ofof every individual drug.
Now multiply that times 3,000.But then in terms of your
question, so we're saying, youknow, let's not start by just
saying that sirolimus, the drugthat literally saved my life,
that it is you know, it's a it'sa proof for 3 diseases. It's
used off label for a few morediseases. Let's not start with
(14:26):
the mentality that sirolimus isbeing used or could be used for
a handful of diseases. Let'sstart out by just saying, let's
look across every disease andlet's look at the potential
mechanisms of sirolimus blockssomething called mTOR.
It's a key communication line ofthe immune system. Let's look to
see what diseases mTOR isimportant in. Let's look at all
of them. Who knows if they'veever been studied before? And
(14:47):
let's start making connectionsthat way.
And so if you look at all 3,000approved drugs and you use the
categorization system, I talkedabout harmonization earlier. If
you categorize diseases into onegroup, or or in one way, there
are about 12,000 diseases. Andso when we did that most
recently, we generated36,000,000 scores, all 3,000
drugs against all 12,000diseases, so 36,000,000
(15:08):
possibilities.
Dr. Moira Gunn (15:10):
Okay.
Dr. David Fajgenbaum (15:12):
I think I'll start tomorrow. I'm very
tired today. 36,000,000.
I know.
Dr. Moira Gunn (15:17):
That's like, okay. Every everybody take a
drug.
Dr. David Fajgenbaum (15:20):
We everybody take 1. We only need
36,000,000 volunteers. You know,it's like, okay. Okay.
Dr. Moira Gunn (15:25):
So you're like, alright. Now
Dr. David Fajgenbaum (15:27):
we gotta narrow this down. What's the
next step?
Dr. Moira Gunn (15:29):
Where are you trying to go with that?
Dr. David Fajgenbaum (15:31):
So now when you got 36,000,000 scores,
it's from 0 to 1. So the scoresthat are zeros are where we
think that you know, forexample, a toenail fungus drug
to treat pancreatic cancer.Right? No mechanistic reason for
why that toenail fungus drug isgonna treat, pancreatic cancer.
Those are gonna get zeros.
We're they're we're certainlygonna, you know, throw them out.
Right?
Dr. Moira Gunn (15:50):
Now if I if I can just interrupt you or ask
you for a second here, when yousay mechanism, is mechanistic,
you mean how it the drug worksin your system and how cancer
works that those 2 don't match.
Dr. David Fajgenbaum (16:02):
That's right. Yeah. That's exactly
right. So a great mechanisticlink would be so in Castleman
disease, the disease I mentionedthat I have, we've discovered
that that communication linemTOR is important in Castleman.
It's too high.
There's too much mTOR. Well,sirolimus works by inhibiting
mTOR, so there's a mechanisticlink. The disease has too much
of something and the drugreduces that thing. So that's a
(16:23):
nice mechanistic link. You know,too much and then the drug
reduces it.
And I used a random example ofpancreatic cancer and antifungal
medication. There's no evidencethat fungi play any role in
pancreatic cancer, so there's areally poor link between an
antifungal and a and a and apancreatic cancer, for example.
So a lot of low scores. But, asyou look across every drug
(16:44):
versus every disease, you startfinding these matches where
there's knowledge that existswithin the world that maybe you
or I haven't ever come acrossthat study. But if you actually
unleash AI across everything theworld has ever studied, you
start finding these lengths thatactually, no pancreatic cancer
doesn't have any role of fungi,but this one protein in that's
(17:04):
involved in the in in thedevelopment of this cancer could
be inhibited by a a drug thatincreases hair growth.
Who knows? Right? And so youstart making a connection
between a hair growth drug andpancreatic cancer because
there's a mechanistic link.There's a protein that's
important to the cancer, andthere's, and that drug does
something to that protein thatthat could be helpful for the
(17:25):
disease. And so now you've gotall 36,000,000 scores.
You got a lot of zeros, butyou've got a lot of point ones
and point twos. And you startgetting interested when you see
the point sevens, the pointeights, the point nines. And so
that's where we start digging inor the point sevens, eights, and
nines because, basically, our AIalgorithm is telling us, hey. If
it's a point 7 or higher, youshould really look into that
because there might be somethinghere. The world's knowledge is
(17:45):
telling me that there's aninteresting pattern
here.
Dr. Moira Gunn (17:47):
So then you say, out of 36,000,000, how many
should we look at first?
Dr. David Fajgenbaum (17:52):
Yeah. So we we we talk about it in terms
of the the top 10% as a startingplace, which ends up being about
the sevens the point sevens,point eights, point nines. So
now you're you're somewherearound 3 to 4000000. And then
you start applying filters tothat number. And so you might
say, I don't wanna look at anyof those diseases where there's
already an approved drug.
So let's take out all of thehits where there's already an
(18:14):
approved drug because we wannago after the diseases where no
one is being helped. Right? Solet's just let's just take out
all the ones with an approveddrug. The next thing you could
say is, I wanna take out all theexpensive drugs because I know
that if I found a repurposingmatch and it's an inexpensive
drug, I know it'll be easier toget it to patients. So let's
just wipe out all the drugs thatcost more than a $100,000 a
year.
So now you're gonna start, youknow, filtering down, lowering
(18:35):
your number. You could say, Iwanna just include drugs that
are really, really safe. Becauseif I'm gonna repurpose a drug, I
don't wanna ever cause any harm.I mean, with every cure, we're
here to cure people. We're nothere to hurt anyone.
So so let's just wipe out allthe drugs that have, you know,
not great safety profiles. Andso you can imagine you start
applying these filters and youget down to a more manageable
number. And then with thatmanageable number, humans then
(18:57):
start going through and say,wow. Arginine for sickle cell
disease. That's a really safedrug.
It's really inexpensive. Sicklecell is a horrible disease. I
like that hit, and that actuallyis one of our top hits right
now. And so, you know, youbasically and then you start
having humans go through and youreally say, I like this and then
is this better than that? Andthen the fun really starts
happening where where you havethe opportunity to to, you know,
(19:18):
decide which ones do you wannatake forward.
Dr. Moira Gunn (19:20):
One thing we have to understand is that you
have to take these drugs. Eventhough they've been approved,
you've got to take them throughclinical trials for the diseases
and the conditions that you'retalking about. That still costs
money and takes time.
Dr. David Fajgenbaum (19:34):
That's right. And you're you're exactly
right. You still have to proveout that the drug works. So even
if there's this really clearmechanistic link where this drug
almost certainly is gonna workfor this disease, you still
gotta prove it. You know, yougotta do either a a rigorous
clinical trial or in some rarecases, if the drug's already
being given to patients let'ssay let's say some doctor at
Penn is giving all of herpatients arginine for sickle
(19:57):
cell disease, and she's treated200 patients of sickle cell
disease, and they all are doinggreat on arginine.
We may not need to do a largerandomized controlled trial. We
could package that data up,basically do a large what's
called a case series where youdescribe, hey. The use of
arginine, this cheap, generic,inexpensive drug widely
available, is helping all thesekids who are suffering from
(20:17):
sickle cell disease, and thatmight actually be enough to
persuade an insurance company tocover because it's such an
inexpensive drug. Now if it's anexpensive drug, you sure as heck
better do a clinical trialbecause to persuade an insurance
company to pay for an expensivedrug to be used in a way that it
wasn't initially intended, youbetter really prove it out with
the trial.
Dr. Moira Gunn (20:35):
And we've all heard the term off label.
Dr. David Fajgenbaum (20:37):
Yes.
Dr. Moira Gunn (20:38):
So that doctor that you were just describing
Dr. David Fajgenbaum (20:41):
Yes.
Dr. Moira Gunn (20:41):
Was likely prescribing it off label. Yes.
So we have some indicators.
Dr. David Fajgenbaum (20:48):
Yeah. Exactly. Let's talk about that a
bit. So, you know, when a druggets FDA approval, it gets
approved for a specific diseasethat goes on the label. So
siltuximab got approved forCastleman.
If you look at the label forsiltuximab, it says Castleman's
on it. To use that drug offlabel and what's amazing is
every doctor in the US canprescribe any drug for any
disease that they want. There'sno rules. Once once it's
(21:09):
approved for something, I can Ican prescribe that for anything
I want? The big question iswhether the insurance company
will pay for it.
Of course, if it's aninexpensive drug, it's much
easier to persuade an insurancecompany. If it's an expensive
drug, it's harder to persuadethem to cover, off label use.
Dr. Moira Gunn (21:23):
Now we're we're gonna go down. We're gonna
figure this out. I know you havesome goals for how many you're
gonna try to do in the next howmany years? You're thinking a 5
year plan.
Dr. David Fajgenbaum (21:33):
That's right.
Dr. Moira Gunn (21:33):
Of course, 5 year plan keeps moving. As as
time goes on, the 5 year goesout. Yeah. And, you're hoping to
do 25 of these clinical trials.What do you think?
Dr. David Fajgenbaum (21:43):
In the next 5 years, we would like to,
advance 25 treatments for newdiseases that weren't previously
being treated. And just to giveyou a perspective, if we're able
to achieve that really ambitiousgoal, that would put us on par
with you name big drug companyin terms of their productivity
(22:04):
on a per on a, you know, 5 yearbasis. So Eli Lilly, Johnson and
Johnson, Pfizer, Merck, about 5or so new approvals a year. We
wanna do that. We wanna do thatwith, you know, less than 1 1
thousandth of the budget.
And the way we're gonna do thatwith less than 1 1 thousandth of
the budget is because we're justgonna go after drugs that are
(22:25):
already on the market. They'realready FDA approved. They don't
need any of the priorpreclinical studies. They don't
need nearly as many clinicaltrial patients because it's
already been approved for onething. And we wanna focus on the
diseases that have beenneglected where, you know, no
one has gone after that diseaseand using the drugs that are
neglected as well becausethey're inexpensive and they're
generic.
And so for all these reasons,we're able to mobilize resources
(22:48):
in ways that, the drug companiesjust aren't able to based on the
way the system is set up. But,man, you know, are there a lot
of patients waiting for thesesolutions?
Dr. Moira Gunn (22:57):
Now you would be mistaken if you thought that
every cure was just a lovelylovely, you know, building with
a nice parking lot and greeneryaround and, and all the work is
going on inside the building.Every cure is a pretty small
outfit with a lot ofcollaboration, big and small.
(23:18):
Everyone can collaborate from anindividual up to a huge
pharmaceutical company, up to aninstitute, up to a university,
up to a researcher with a labbench. Let's talk about all the
various ways, you know, justsort of quickly is, like, how
can various entities,organizations, and individual
(23:39):
humans actually participate?
Dr. David Fajgenbaum (23:42):
Oh, I love this question. Yeah. We I mean,
we we called it every CUREbecause we wanna be as inclusive
as possible. It's all of us. Youknow?
We at one point, we thoughtabout calling it the CURE
Collective or the CURECollaborative because it's
really about bringing all of ustogether. And so, yeah, what are
a few ways the different,listeners could be involved? So
first off, every single patient,every per that means every
(24:04):
person in the United States andbeyond that has ever received a
drug off label or repurposed, soa drug for something that it
wasn't intended for. And by theway, 20% of all prescriptions
that are written every day arefor off label uses. So so
probably everyone listeningright now has received a drug
off label.
But, truly, everyone who, whohas an idea or has maybe
(24:25):
benefited from an off label drugor they have an idea about a
repurposed drug, we want you toshare those with us. Right now,
it's at everycure.org. In thefuture, we'll have likely have a
separate portal specifically.But if you go to everycure.org,
you can share with us about howyou might think that, folic acid
might be a great drug for yourlupus, or I I I don't know what
(24:46):
it might be. But but you sharethose with us.
We want you to share those withus because we wanna be that
entity that just drives greatideas forward. So so anyone can
share ideas with us, whetheryou're a patient, you're a
physician, and you've given itto your patients, you're a
researcher, and you have ideas,share those ideas with us. We we
want you to do that. If you area health care organization, so
(25:06):
let's say you have access todata, genomic data, proteomic
data, drugs screen data, thatcould be useful for us, that
could go into that knowledgegraph, please share it with us.
You know, if you are a companythat has data like that and you
license it out typically, I hopeyou'll consider donating it to
us.
We're a nonprofit organization,and we would love to highlight
the fact that your companydonated your data to us for
(25:29):
free. We could utilize that. Andthen and maybe, one of the most
important categories is ifyou're involved in clinical
trials, so you're a contractresearch organization, or you,
serve as a site for clinicaltrials, we would love to work
with you because we the the mostexpensive part of this whole
equation, as you mentionedearlier, is the clinical trials.
(25:49):
And so if we can partner withgroups that can do clinical
trials efficiently, can maybeprovide discounted pricing to
our organization, that's gonnahelp us to do more clinical
trials for less dollars. And soas you can tell, pretty much
anyone and everyone couldcontribute to us, and we hope
you all will.
Dr. Moira Gunn (26:04):
And, of course, this is a nonprofit. Donations
are always welcome.
Dr. David Fajgenbaum (26:09):
That's correct.
Dr. Moira Gunn (26:11):
So just please know that. Please know that.
Dr. David Fajgenbaum: Everycure.org. That's (26:14):
undefined
Dr. Moira Gunn (26:15):
right. Everycure.org. I wanna thank you
so much, David, for coming in.And you know we'll do we'll do
anything we can for you, and,please come back. You know,
you're always welcome on TechNation.
Dr. David Fajgenbaum (26:26):
Well, thank you so much for having me
and just highlighting thisincredible opportunity, that I I
never, could have imagined thepotential out there to help a
lot of people. And, of course,as I laid in my hospital bed 13
years ago, I never thought thatI'd be talking with you, today
and share about this this, youknow, where we are. And and I
really hope, as as I look to thefuture that, you know, 5 years
(26:50):
from now, we can look back andand we can see the 25 diseases,
that we're treating now withrepurposed drugs and just, so
excited about the theopportunity to help a lot of
people and hope that everyonelistening, will join on this
mission.
Dr. Moira Gunn (27:03):
My guest today is University of Pennsylvania
School of Medicine professor,doctor David Fajgenbaum. He's
the cofounder and president ofEvery Cure. More information is
available on the web ateverycure.org. That's
everycure.org. His 2021 book,Chasing My Cure, A Doctor's Race
(27:24):
to Turn Hope Into Action, ispublished by Ballantine.
Flipping the script on the idea of for every
disease, a drug. To for everydrug, all the diseases it can
treat. I'm speaking with doctorDavid Fajgenbaum, a professor in
translational medicine and humangenetics at the University of
Pennsylvania School of Medicineand the cofounder of Every Cure.
(00:34):
And now, doctor DavidFajgenbaum. Well, David, welcome
to the program.
Dr. David Fajgenbaum (00:40):
Thanks so much for having me.
Dr. Moira Gunn (00:42):
Now I learned from your website, which is
everycure.org, that there areabout 3,000 FDA approved medical
treatments which address anynumber of medical conditions.
And that's in total, 3,000 inthat area. But to quote that
website, no organization isresponsible for ensuring that
all approved medicines are usedto treat
Dr. David Fajgenbaum (01:04):
every disease they possibly can. And
that's true. That's right.
Dr. Moira Gunn (01:04):
They come through to cure a particular
disease possibly can, and that'strue.
Dr. David Fajgenbaum (01:07):
That's right.
Dr. Moira Gunn (01:08):
They come through to cure a particular
disease or address it. Tell usabout that.
Dr. David Fajgenbaum (01:12):
Sure. I mean, it it's really been
surprising for me to get intothe space and to learn this. I
mean, we think about theincredible effort and resources
that go into getting one drugapproved for one disease. I
mean, literally, 1,000,000,000of dollars and, in some cases,
more than 10 or 20 years for onedrug for one disease. And so you
would just sort of assume thatthe work would be done to figure
out all of the diseases thatcould possibly benefit from that
(01:34):
drug.
But, unfortunately, in ourcurrent system, there are
incentives in place that wouldmake a company pick one disease
over another disease. And thenonce that drug becomes generic,
there's really no incentives tofind more uses for them.
Dr. Moira Gunn (01:45):
So, once the patent goes off expiration,
anybody can make it, thenthere's no real real big
profits.
Dr. David Fajgenbaum (01:52):
That's exactly right. There's no
profit. There's no incentivewhatsoever. I mean, really, once
a drug is generic and over 80%of drugs are already generic,
there's no incentive to to findmore uses for these drugs. So
the tools that are within ourreach, the drugs that are
literally on our pharmacyshelves to help patients are the
ones that we're not studying.
They're the ones that we're notfully utilizing.
Dr. Moira Gunn (02:12):
Now let me flip the question. How many medical
conditions today are in need ofmedications?
Dr. David Fajgenbaum (02:19):
It's a huge number. We like to think
about all the progress thatwe've made as a medical
community, and and we should bereally proud. As you said, 3,000
approved drugs. Those 3,000drugs are approved for about
3,000 diseases, and that isreally amazing. But depending on
what classification system youuse, there's anywhere between
9,017,000 more diseases thatdon't have a single approved
(02:43):
therapy.
Dr. Moira Gunn (02:44):
Now these aren't just one offs. There's millions
of people who have thesediseases.
Dr. David Fajgenbaum (02:49):
That's right. It's estimated about 1 in
10 Americans have one of thesebetween 9,017,000 diseases that
don't have an approved therapy.1 in 10 of all of us listening
and and part of the show rightnow either has or will develop a
disease without a singletreatment.
Dr. Moira Gunn (03:04):
Now you come to Every Cure. You cofounded it.
It's a nonprofit. And what is ittrying to do?
Dr. David Fajgenbaum (03:11):
Sure. Every cure is on a mission to
make sure that every FDAapproved drug is utilized for
every disease possible, andwe're utilizing the world's
biomedical knowledge. All theincredible work that's been done
by pharmaceutical companies andacademic groups and and others
leverage all of that knowledgeto figure out what are the
possible uses for every approveddrug. And let's go out and let's
actually drive forward advancethe trials to prove that they
(03:33):
actually work regardless ofwhether it's profitable to do
that or regardless of whether,the disease population is large
or small, just what are thethings that we can do to help
people as quickly as possible?
Dr. Moira Gunn (03:44):
I have to say, I can imagine you're sitting
there. You have a you have acondition. There's some drugs
there, but maybe not working foryou. They're working on new
ones. I mean, it just frost youto think, you mean, we have some
drugs that might already work.
Dr. David Fajgenbaum (03:57):
Yeah. It's kinda mind blowing. Right? I
mean, and I know that we'll chatabout this later on, but as
someone who's gone throughmedical challenges, we tend to
think that the drugs that, youknow, our our doctors tell us
about are are the only drugsthat could possibly treat our
diseases. But there's this wholeliterally, pharmacy worth of
(04:18):
other drugs that have thepotential to maybe treat us or
someone we love.
Dr. Moira Gunn (04:22):
1st, you're building a database, a drug
repurposing database. What isit? What's in it? What's in that
database?
Dr. David Fajgenbaum (04:31):
Sure. So right now, it's about 70
different databases worth ofdatasets. And so, basically, the
world's knowledge of what do weknow as a as a human society
about all the drugs that areapproved? What do we know about
all the diseases that affecthumans? What do we know about
all the genes in our bodies?
What do we know about all of theways those genes interact with
one another? So imagine saying,I wanted to get, like, what the
(04:53):
world knows about everythingthat could be relevant. That is
in our, basically, database ofdatabases, all that information
in one central place.
Dr. Moira Gunn (05:02):
And, of course, they're all different formats,
different limits, differentthey're okay. So you've got a
lot of it's a tower of babble ofmedical information.
Dr. David Fajgenbaum (05:09):
That's right.
Dr. Moira Gunn (05:10):
But you'll say bring it on. If it's good data,
bring
Dr. David Fajgenbaum (05:12):
it on.
The more the data, the better.
Dr. Moira Gunn (05:14):
Now frequently, when we are prescribed a
medicine, they're going by oursymptoms. They're not really
able to go inside and see thedisease. So at what level are is
some of this data?
Dr. David Fajgenbaum (05:28):
Oh, that's a great question. So, the data
that we're collecting is onevery level from what's
happening within the cell ofsomeone with the disease all the
way through how do patientsperform or or respond to a drug
in a clinical trial, forexample. But in terms of the
recommendations that come out ofit, and and I know we'll we'll
get to that in a minute, butthat's on a disease level. So
it's basically gonna say, foryour disease, what is the rank
(05:50):
ordered list of all 3,000 drugsin terms of how likely they are
to treat you? You.
So, you know, the first couplemight be the first two drugs
that are approved for yourdisease. The next couple might
be a couple drugs that maybehave been utilized in a few
patients here and there. Andthen the other almost 3,000
drugs are gonna be where we'reutilizing artificial
intelligence to help us to comeup with a score for how likely
that drug is to treat yourdisease.
Dr. Moira Gunn (06:12):
So we got 3,000 drugs possible. And how many
databases do you have?
Dr. David Fajgenbaum (06:18):
So right now, we're at about 80, but
we're eager for people to donateadditional data to us. We we
really want to get as much dataas possible. The 80 databases
are fairly comprehensive interms of what's publicly
available. So a lot of times,when the federal government
funds research, therequirements, the data be made
publicly available so anyone candownload it. We've gotten all
those datasets.
What we don't have are theproprietary private datasets
(06:41):
from companies like Elsevier andClarivate and Wolters Kluwer
where they charge subscriptionsfor access. We're hopeful to
partner with all of them. And ifanyone from from those companies
is listening, we would love towork with you.
Dr. Moira Gunn (06:51):
Bring it on. Send us a password.
Dr. David Fajgenbaum (06:52):
Yeah. Bring it on. Yeah. Exactly.
Donate the data to us.
You know, we're a nonprofitorganization literally just on a
mission to save as many lives aspossible in the shortest period
of time. And the way to do thatis not to develop a new drug
from scratch. It's to use thetools already within reach.
Dr. Moira Gunn (07:07):
And I have to say, when we're talking about
these datasets, frequently,we're talking about clinical
trial datasets, which are great.They have a lot of information
in them. And these are thousandsof people over time, lots of
data, all kinds of data, youknow, from MRIs to blood test,
amazing kind of information thatyou haven't even tapped in yet,
(07:30):
but are relevant possibly to oneor another drug or in one or
another condition. This is anenormous, enormous task to just
look at the size of the data andthe complexity of linking it. I
mean, there's some human inthere that has to kinda do that
translation so the AI can get inand do its work.
(07:51):
How do you do you just have awhole staff of people that are
called inkers?
Dr. David Fajgenbaum (07:56):
I don't know how you do this.
There's actually a whole field that's
called harm data harmonization.And so we work with an amazing
researcher, Melissa Heindel,who's at the University of
Colorado, where she has a teamof about 20 data harmonizers,
and they literally look betweenall these datasets and try to
harmonize the data so that itcan be linked. You know, are we
using the same unit for this?Are we calling, is it called
(08:20):
Castleman disease or is itCastleman's disease? You know,
ways to basically make surewe're all using the same
language, the same vocabulary.
And so they're one of our closepartners, and, we're we're
thankful that they're doing theharmonization so that way we can
make the connections.
Dr. Moira Gunn (08:34):
Okay. So do the math. 3000 drugs, 80 datasets of
all kinds of things, and you'relooking for more. Lots and lots
more. 20,000 diseases.
You know? You're not doing thison an Excel spreadsheet.
Dr. David Fajgenbaum (08:46):
No. Yeah. It's been it's been described by
a few folks in this phase inthis space. One recently
described it, as, not a moonshot, but like a Mars shot. It's
like further than the moon.
And and another person describedit as as the Manhattan Project.
And so, yeah, it's a hugeeffort. But to me, as a
physician, as a scientist, as apatient, as an advocate myself,
(09:09):
I can't imagine how we couldhave gone this long without ever
doing this. You know, as someonewho has benefited from drugs and
also developed drugs forpatients, again, I just can't
believe that our society hasn'tcome together to say, we're
gonna take on this Mars shot.You know, we're gonna take on
this Manhattan Project to makesure that every drug is fully
utilized.
And so after spending about 10years, working within this space
(09:34):
and trying to figure out who'sdoing this and learning that no
one's doing it, we finallydecided that we needed to do it.
Dr. Moira Gunn (09:39):
You're listening to Tech Nation. I'm Moira Gunn.
My guest today is doctor DavidFaganbaum, an associate
professor in translationalmedicine and human genetics at
the University of PennsylvaniaSchool of Medicine. He's also
the cofounder and president ofthe nonprofit Every Cure. His
2021 book is Chasing My Cure, ADoctor's Race to Turn Hope Into
(10:02):
Action.
Well, I said I was gonna ask youabout this at the end, but I
gotta ask you about it now.
Dr. David Fajgenbaum (10:07):
We're always asking, you know, if
people are actually walkingtheir talk. You did the
Dr. Moira Gunn (10:07):
walk. If people are actually walking their talk.
You did the walk, and now you'vemade it your talk.
Dr. David Fajgenbaum (10:16):
So we
just talked about your talk.
Dr. Moira Gunn (10:19):
Tell us how you got here, and it's it's almost a
miracle you are. Tell us aboutthat.
Dr. David Fajgenbaum (10:24):
Sure. Well, in 2010, I was a 3rd year
medical student here at theUniversity of Pennsylvania. I
was treating patients. I, waswas healthy, and, I on a
mission. I wanted to become adoctor in memory of my mom.
She had passed away from cancera few years before. And, out of
nowhere, I became criticallyill. The same hospital I was
(10:46):
treating patients, all of asudden I was in ICU. All of my
organs were shutting down. I wason life support requiring daily
transfusions just to keep mealive, all with no diagnosis.
That lasted for months that Iwas in the ICU. I was eventually
diagnosed with a rare diseasecalled Castleman disease, where
there were no approvedtherapies. And I I spent a total
(11:07):
of about 6 months hospitalized.I had my last rights read to me.
I, you know, just struggled withthis condition, received a lot
of chemotherapy, that was usedfor my disease.
And chemo kept saving my life,but then I kept relapsing. And,
you know, about 2 years intothis journey, I was in the
middle of med school and I was,you know, dealing with this
illness. I realized that Icouldn't just hope that some
(11:29):
researcher somewhere would finda drug for me. I mean, my doctor
explained to me that he was outof options. He said, there are
no more drugs to try for you.
This is it. We've triedeverything. And so I I know I
couldn't just wait for someoneelse to find something that if I
wanted any chance of survival, Iwould need to get involved in
research myself. And so Icreated an organization called
the Castle Disease CollaborativeNetwork, and I got to work. I
started performing experimentson my own blood samples.
(11:51):
I started going through all thestudies out there. We talk about
the world's biomedicalknowledge. I I probably read
through the whole world'sbiomedical knowledge trying to
find a drug to save my life.And, over the course of about a
year, I ended up discovering anold drug called sirolimus. It's
been around for decades.
It had never been used for mydisease, and I thought it might
work. And, so I began testing iton myself. And actually, this
(12:13):
month marks 10 years that I'vebeen in remission on this drug
that wasn't made for my disease.It wasn't supposed to be given
me. I'm not supposed to be here,but because of that drug, I'm
alive.
And as you said earlier, nowit's my mission to find as many
of these drugs as possible tosave patients as quickly as
possible.
Dr. Moira Gunn (12:29):
Yeah. When they say, well, we don't believe you
can do it, you say, well, sitdown.
Dr. David Fajgenbaum (12:33):
Exactly. I gotta sit down. Sorry. Exhibit
a. Right?
I'm like, yeah. Exact you know,you're right. Because there are
a lot of people who are like,you know, this is, you know,
this is too ambitious andaudacious. You know, drug
companies spend 1,000,000,000 ofdollars to develop drugs. You
know?
How is your nonprofit going togonna make a huge difference?
And, yeah, exactly the answeris, well, we already have, and
(12:54):
now we just wanna do it atscale.
Dr. Moira Gunn (12:56):
Okay. So if I was in a typical pharmaceutical
or biopharmaceutical company andwe were choosing drugs, we have
we have a number of candidates.You're sitting there going,
okay. Well, we're we've got allof these scores. And you
recently figured out how many ofthese scores you did.
Now let's take us from therebecause from there, we're gonna
(13:18):
go down the plan. What whatyou're planning on doing?
Dr. David Fajgenbaum (13:21):
Sure. So, yeah, I think there's 2 places
to go, from those importantcomments you made. I mean, first
off, when a drug companydevelops a new drug, on average,
somewhere between 30 40 diseasesare considered for that single
drug. So drug companies, youknow, have a promising product
and they start saying, you know,is it this disease or that
disease? As many as 30 to 40 areconsidered, and that drug
(13:42):
company has to make really toughdecisions to pick the 5 or so
that it'll actually study intrials.
1 will get an approval, andmaybe they'll pursue 1 or 2
additional drugs to add to thelabel to make that drug approved
for between 1 and 3 things. Butremember, if you go back to the
very beginning, now there's 20to 30, maybe even 40 other ideas
that haven't been pursued atall. There are great ideas. Some
really smart people in the drugcompany considered them. And so
(14:05):
so that just sort of tells youthe vast potential here is, you
know, the untapped potential ofof every individual drug.
Now multiply that times 3,000.But then in terms of your
question, so we're saying, youknow, let's not start by just
saying that sirolimus, the drugthat literally saved my life,
that it is you know, it's a it'sa proof for 3 diseases. It's
used off label for a few morediseases. Let's not start with
(14:26):
the mentality that sirolimus isbeing used or could be used for
a handful of diseases. Let'sstart out by just saying, let's
look across every disease andlet's look at the potential
mechanisms of sirolimus blockssomething called mTOR.
It's a key communication line ofthe immune system. Let's look to
see what diseases mTOR isimportant in. Let's look at all
of them. Who knows if they'veever been studied before? And
(14:47):
let's start making connectionsthat way.
And so if you look at all 3,000approved drugs and you use the
categorization system, I talkedabout harmonization earlier. If
you categorize diseases into onegroup, or or in one way, there
are about 12,000 diseases. Andso when we did that most
recently, we generated36,000,000 scores, all 3,000
drugs against all 12,000diseases, so 36,000,000
(15:08):
possibilities.
Dr. Moira Gunn (15:10):
Okay.
Dr. David Fajgenbaum (15:12):
I think I'll start tomorrow. I'm very
tired today. 36,000,000.
I know.
Dr. Moira Gunn (15:17):
That's like, okay. Every everybody take a
drug.
Dr. David Fajgenbaum (15:20):
We everybody take 1. We only need
36,000,000 volunteers. You know,it's like, okay. Okay.
Dr. Moira Gunn (15:25):
So you're like, alright. Now
Dr. David Fajgenbaum (15:27):
we gotta narrow this down. What's the
next step?
Dr. Moira Gunn (15:29):
Where are you trying to go with that?
Dr. David Fajgenbaum (15:31):
So now when you got 36,000,000 scores,
it's from 0 to 1. So the scoresthat are zeros are where we
think that you know, forexample, a toenail fungus drug
to treat pancreatic cancer.Right? No mechanistic reason for
why that toenail fungus drug isgonna treat, pancreatic cancer.
Those are gonna get zeros.
We're they're we're certainlygonna, you know, throw them out.
Right?
Dr. Moira Gunn (15:50):
Now if I if I can just interrupt you or ask
you for a second here, when yousay mechanism, is mechanistic,
you mean how it the drug worksin your system and how cancer
works that those 2 don't match.
Dr. David Fajgenbaum (16:02):
That's right. Yeah. That's exactly
right. So a great mechanisticlink would be so in Castleman
disease, the disease I mentionedthat I have, we've discovered
that that communication linemTOR is important in Castleman.
It's too high.
There's too much mTOR. Well,sirolimus works by inhibiting
mTOR, so there's a mechanisticlink. The disease has too much
of something and the drugreduces that thing. So that's a
(16:23):
nice mechanistic link. You know,too much and then the drug
reduces it.
And I used a random example ofpancreatic cancer and antifungal
medication. There's no evidencethat fungi play any role in
pancreatic cancer, so there's areally poor link between an
antifungal and a and a and apancreatic cancer, for example.
So a lot of low scores. But, asyou look across every drug
(16:44):
versus every disease, you startfinding these matches where
there's knowledge that existswithin the world that maybe you
or I haven't ever come acrossthat study. But if you actually
unleash AI across everything theworld has ever studied, you
start finding these lengths thatactually, no pancreatic cancer
doesn't have any role of fungi,but this one protein in that's
(17:04):
involved in the in in thedevelopment of this cancer could
be inhibited by a a drug thatincreases hair growth.
Who knows? Right? And so youstart making a connection
between a hair growth drug andpancreatic cancer because
there's a mechanistic link.There's a protein that's
important to the cancer, andthere's, and that drug does
something to that protein thatthat could be helpful for the
(17:25):
disease. And so now you've gotall 36,000,000 scores.
You got a lot of zeros, butyou've got a lot of point ones
and point twos. And you startgetting interested when you see
the point sevens, the pointeights, the point nines. And so
that's where we start digging inor the point sevens, eights, and
nines because, basically, our AIalgorithm is telling us, hey. If
it's a point 7 or higher, youshould really look into that
because there might be somethinghere. The world's knowledge is
(17:45):
telling me that there's aninteresting pattern
here.
Dr. Moira Gunn (17:47):
So then you say, out of 36,000,000, how many
should we look at first?
Dr. David Fajgenbaum (17:52):
Yeah. So we we we talk about it in terms
of the the top 10% as a startingplace, which ends up being about
the sevens the point sevens,point eights, point nines. So
now you're you're somewherearound 3 to 4000000. And then
you start applying filters tothat number. And so you might
say, I don't wanna look at anyof those diseases where there's
already an approved drug.
So let's take out all of thehits where there's already an
(18:14):
approved drug because we wannago after the diseases where no
one is being helped. Right? Solet's just let's just take out
all the ones with an approveddrug. The next thing you could
say is, I wanna take out all theexpensive drugs because I know
that if I found a repurposingmatch and it's an inexpensive
drug, I know it'll be easier toget it to patients. So let's
just wipe out all the drugs thatcost more than a $100,000 a
year.
So now you're gonna start, youknow, filtering down, lowering
(18:35):
your number. You could say, Iwanna just include drugs that
are really, really safe. Becauseif I'm gonna repurpose a drug, I
don't wanna ever cause any harm.I mean, with every cure, we're
here to cure people. We're nothere to hurt anyone.
So so let's just wipe out allthe drugs that have, you know,
not great safety profiles. Andso you can imagine you start
applying these filters and youget down to a more manageable
number. And then with thatmanageable number, humans then
(18:57):
start going through and say,wow. Arginine for sickle cell
disease. That's a really safedrug.
It's really inexpensive. Sicklecell is a horrible disease. I
like that hit, and that actuallyis one of our top hits right
now. And so, you know, youbasically and then you start
having humans go through and youreally say, I like this and then
is this better than that? Andthen the fun really starts
happening where where you havethe opportunity to to, you know,
(19:18):
decide which ones do you wannatake forward.
Dr. Moira Gunn (19:20):
One thing we have to understand is that you
have to take these drugs. Eventhough they've been approved,
you've got to take them throughclinical trials for the diseases
and the conditions that you'retalking about. That still costs
money and takes time.
Dr. David Fajgenbaum (19:34):
That's right. And you're you're exactly
right. You still have to proveout that the drug works. So even
if there's this really clearmechanistic link where this drug
almost certainly is gonna workfor this disease, you still
gotta prove it. You know, yougotta do either a a rigorous
clinical trial or in some rarecases, if the drug's already
being given to patients let'ssay let's say some doctor at
Penn is giving all of herpatients arginine for sickle
(19:57):
cell disease, and she's treated200 patients of sickle cell
disease, and they all are doinggreat on arginine.
We may not need to do a largerandomized controlled trial. We
could package that data up,basically do a large what's
called a case series where youdescribe, hey. The use of
arginine, this cheap, generic,inexpensive drug widely
available, is helping all thesekids who are suffering from
(20:17):
sickle cell disease, and thatmight actually be enough to
persuade an insurance company tocover because it's such an
inexpensive drug. Now if it's anexpensive drug, you sure as heck
better do a clinical trialbecause to persuade an insurance
company to pay for an expensivedrug to be used in a way that it
wasn't initially intended, youbetter really prove it out with
the trial.
Dr. Moira Gunn (20:35):
And we've all heard the term off label.
Dr. David Fajgenbaum (20:37):
Yes.
Dr. Moira Gunn (20:38):
So that doctor that you were just describing
Dr. David Fajgenbaum (20:41):
Yes.
Dr. Moira Gunn (20:41):
Was likely prescribing it off label. Yes.
So we have some indicators.
Dr. David Fajgenbaum (20:48):
Yeah. Exactly. Let's talk about that a
bit. So, you know, when a druggets FDA approval, it gets
approved for a specific diseasethat goes on the label. So
siltuximab got approved forCastleman.
If you look at the label forsiltuximab, it says Castleman's
on it. To use that drug offlabel and what's amazing is
every doctor in the US canprescribe any drug for any
disease that they want. There'sno rules. Once once it's
(21:09):
approved for something, I can Ican prescribe that for anything
I want? The big question iswhether the insurance company
will pay for it.
Of course, if it's aninexpensive drug, it's much
easier to persuade an insurancecompany. If it's an expensive
drug, it's harder to persuadethem to cover, off label use.
Dr. Moira Gunn (21:23):
Now we're we're gonna go down. We're gonna
figure this out. I know you havesome goals for how many you're
gonna try to do in the next howmany years? You're thinking a 5
year plan.
Dr. David Fajgenbaum (21:33):
That's right.
Dr. Moira Gunn (21:33):
Of course, 5 year plan keeps moving. As as
time goes on, the 5 year goesout. Yeah. And, you're hoping to
do 25 of these clinical trials.What do you think?
Dr. David Fajgenbaum (21:43):
In the next 5 years, we would like to,
advance 25 treatments for newdiseases that weren't previously
being treated. And just to giveyou a perspective, if we're able
to achieve that really ambitiousgoal, that would put us on par
with you name big drug companyin terms of their productivity
(22:04):
on a per on a, you know, 5 yearbasis. So Eli Lilly, Johnson and
Johnson, Pfizer, Merck, about 5or so new approvals a year. We
wanna do that. We wanna do thatwith, you know, less than 1 1
thousandth of the budget.
And the way we're gonna do thatwith less than 1 1 thousandth of
the budget is because we're justgonna go after drugs that are
(22:25):
already on the market. They'realready FDA approved. They don't
need any of the priorpreclinical studies. They don't
need nearly as many clinicaltrial patients because it's
already been approved for onething. And we wanna focus on the
diseases that have beenneglected where, you know, no
one has gone after that diseaseand using the drugs that are
neglected as well becausethey're inexpensive and they're
generic.
And so for all these reasons,we're able to mobilize resources
(22:48):
in ways that, the drug companiesjust aren't able to based on the
way the system is set up. But,man, you know, are there a lot
of patients waiting for thesesolutions?
Dr. Moira Gunn (22:57):
Now you would be mistaken if you thought that
every cure was just a lovelylovely, you know, building with
a nice parking lot and greeneryaround and, and all the work is
going on inside the building.Every cure is a pretty small
outfit with a lot ofcollaboration, big and small.
(23:18):
Everyone can collaborate from anindividual up to a huge
pharmaceutical company, up to aninstitute, up to a university,
up to a researcher with a labbench. Let's talk about all the
various ways, you know, justsort of quickly is, like, how
can various entities,organizations, and individual
(23:39):
humans actually participate?
Dr. David Fajgenbaum (23:42):
Oh, I love this question. Yeah. We I mean,
we we called it every CUREbecause we wanna be as inclusive
as possible. It's all of us. Youknow?
We at one point, we thoughtabout calling it the CURE
Collective or the CURECollaborative because it's
really about bringing all of ustogether. And so, yeah, what are
a few ways the different,listeners could be involved? So
first off, every single patient,every per that means every
(24:04):
person in the United States andbeyond that has ever received a
drug off label or repurposed, soa drug for something that it
wasn't intended for. And by theway, 20% of all prescriptions
that are written every day arefor off label uses. So so
probably everyone listeningright now has received a drug
off label.
But, truly, everyone who, whohas an idea or has maybe
(24:25):
benefited from an off label drugor they have an idea about a
repurposed drug, we want you toshare those with us. Right now,
it's at everycure.org. In thefuture, we'll have likely have a
separate portal specifically.But if you go to everycure.org,
you can share with us about howyou might think that, folic acid
might be a great drug for yourlupus, or I I I don't know what
(24:46):
it might be. But but you sharethose with us.
We want you to share those withus because we wanna be that
entity that just drives greatideas forward. So so anyone can
share ideas with us, whetheryou're a patient, you're a
physician, and you've given itto your patients, you're a
researcher, and you have ideas,share those ideas with us. We we
want you to do that. If you area health care organization, so
(25:06):
let's say you have access todata, genomic data, proteomic
data, drugs screen data, thatcould be useful for us, that
could go into that knowledgegraph, please share it with us.
You know, if you are a companythat has data like that and you
license it out typically, I hopeyou'll consider donating it to
us.
We're a nonprofit organization,and we would love to highlight
the fact that your companydonated your data to us for
(25:29):
free. We could utilize that. Andthen and maybe, one of the most
important categories is ifyou're involved in clinical
trials, so you're a contractresearch organization, or you,
serve as a site for clinicaltrials, we would love to work
with you because we the the mostexpensive part of this whole
equation, as you mentionedearlier, is the clinical trials.
(25:49):
And so if we can partner withgroups that can do clinical
trials efficiently, can maybeprovide discounted pricing to
our organization, that's gonnahelp us to do more clinical
trials for less dollars. And soas you can tell, pretty much
anyone and everyone couldcontribute to us, and we hope
you all will.
Dr. Moira Gunn (26:04):
And, of course, this is a nonprofit. Donations
are always welcome.
Dr. David Fajgenbaum (26:09):
That's correct.
Dr. Moira Gunn (26:11):
So just please know that. Please know that.
Dr. David Fajgenbaum: Everycure.org. That's (26:14):
undefined
Dr. Moira Gunn (26:15):
right. Everycure.org. I wanna thank you
so much, David, for coming in.And you know we'll do we'll do
anything we can for you, and,please come back. You know,
you're always welcome on TechNation.
Dr. David Fajgenbaum (26:26):
Well, thank you so much for having me
and just highlighting thisincredible opportunity, that I I
never, could have imagined thepotential out there to help a
lot of people. And, of course,as I laid in my hospital bed 13
years ago, I never thought thatI'd be talking with you, today
and share about this this, youknow, where we are. And and I
really hope, as as I look to thefuture that, you know, 5 years
(26:50):
from now, we can look back andand we can see the 25 diseases,
that we're treating now withrepurposed drugs and just, so
excited about the theopportunity to help a lot of
people and hope that everyonelistening, will join on this
mission.
Dr. Moira Gunn (27:03):
My guest today is University of Pennsylvania
School of Medicine professor,doctor David Fajgenbaum. He's
the cofounder and president ofEvery Cure. More information is
available on the web ateverycure.org. That's
everycure.org. His 2021 book,Chasing My Cure, A Doctor's Race
(27:24):
to Turn Hope Into Action, ispublished by Ballantine.
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