7,000 Diseases. 4,800 Drugs. The Gap in Modern Medicine

What if the biggest problem in medicine is not discovery, but what gets funded?

In this episode of The NEXT BIG THING with Keith D. Terry, Keith sits down with Barbara Goodman, President and CEO of Cures Within Reach, to unpack one of the most overlooked challenges in healthcare: the gap between scientific possibility and what actually reaches patients.

With more than 7,000 diseases worldwide and fewer than 5,000 approved drugs, the system does not fail because of a lack of ideas. Many promising treatments never fail scientifically—they fail financially.

Barbara shares how her organization is addressing this “missing middle” by funding proof-of-concept clinical trials that unlock new uses for existing therapies and accelerate access to care.

🔍  In this episode, you will learn:

• Why many promising treatments never move forward in traditional drug development
 • How financial incentives shape what gets funded in healthcare
 • What drug repurposing is and why it is gaining momentum
 • The role of off-label use in real-world patient care
 • How AI is accelerating the identification of new treatment opportunities
 • Why some of the most impactful solutions may already exist

This conversation goes beyond healthcare. It explores how systems prioritize, how innovation actually happens, and what it takes to move ideas from possibility to real-world impact.

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YouTube: @keithdterry
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#Healthcare #DrugRepurposing #AIinHealthcare #MedicalInnovation #Biotech #Podcast

Transcript

Off-Label Use Explained

Barbara Goodman 0:00

What a lot of people don't know or understand is what off-label use is in the pharmaceutical world.

Keith D. Terry 0:08

Why don't you explain it?

Why Repurposing Beats Starting Over

Barbara Goodman 0:10

Most drugs in the United States, they get approved by the FDA and then they are used what's known as on label. So the FDA approves, approves ozembic for type 2 diabetes, for example. It's used off-label when a clinician, a doctor, prescribes that particular drug for a disease or an indication that the FDA has not said is safe and effective. It's known as off-label. The FDA allows a pharmaceutical company to create a drug label that says drug X is safe and effective for disease I. And that's what's known as on label. What most people in the United States and around the world don't realize is it's pretty well known that about 25% of drugs, of prescription drugs in the United States are actually prescribed off-label. And for cancer, for oncology treatments, frequently up to 50%, much higher than people realize. The off-label space is a fascinating one and a really important dynamic in drug repurposing. We as a nonprofit can talk about off-label use. You can promote it, we can talk about it. Publication, doctors, clinicians in the United States, it's perfectly acceptable. And free, but not always reimbursed by insurers. What's interesting, though, and understandable is the FDA does not allow pharmaceutical companies to talk about anything off label.

Keith D. Terry 1:42

That is Welcome to the podcast, The Next Big Thing. I'm your host, Keith D.Terry, a consultant, a coach, and a serial entrepreneur. The mission here is to teach, inspire, and to motivate. Most people believe that medical breakthroughs come from discovering entirely new drugs. And in many cases, that's true. But what people don't realize is how the system actually works. Modern drug development was designed to ensure safety and effectiveness, especially after the event of the thalmaldehyde, uh thalidomide, sorry, tragedy forced regulators to tighten control. And if you don't know about that story, you should go and look it up. Today, before a drug ever reaches patients, it must go through years of testing and approval through agencies like the Food and Drug Administration. And that process can take 10 to 15 years, and it definitely costs billions of dollars. So here's the reality. And in many cases, even when the science is strong, they never make it out of the research phase. Some of the most promising ideas don't fail scientifically, they fail financially. Now think about this. There are thousands of drugs already approved in use today. Drugs that have already been tested for safety and effectiveness, already proven to work in the area that they were deemed. But what if some of those same drugs could treat entirely different diseases? What if the next breakthrough was something not new, but something we already have? Because if that's true, then the question is not scientific, it's structural. Who decides what ideas move forward, what drugs move forward, which treatments get funded, and more importantly, which ones get stuck in the middle where no one steps in? That's where this conversation today becomes really important because there's an organization, Cures Within Reach, that's operating in the gap between what is possible and what actually reaches patients. My guest today is Barbara Goodman, president and CEO of Cures Within Reach. She brings nearly 30 years of experience across healthcare, life sciences with a strong background in strategy, growth, and innovation. Barbara, welcome to the next big thing. How are you?

Barbara Goodman 4:09

I'm good. Thank you so much for having me today.

Keith D. Terry 4:11

You got it. So let's let's start here. I, you know, if people don't know about Cures Within Reach, you guys are doing some really phenomenal things. And I have a question, and and and you're in a unique position. What patterns do you see of what gets funded and more importantly, what gets stuck in the middle?

Barbara Goodman 4:28

And so I I'll start by commenting on the introduction. So thank you so much for for that. And it's it's not really a matter of if it's happening, it happens every day.

Keith D. Terry 4:38

Okay.

Barbara Goodman 4:38

This is going on every day, and many, many treatments that are currently reaching patients were approved for a different indication originally. They're uh tested and funded for a different disease, like Viagra, for example. And what many people are talking about over the last year or two is Ozembik and Manjaro both approved their uses before it was found to be useful for other issues and other healthcare situations. So for us, innovation and what's going on in the ecosystem, you're absolutely right. There are incentives by pharma to focus on their financial opportunities. They are very frequently publicly traded companies with shareholders. Government agencies as stakeholders in this need to make sure that treatments are safe and effective before they will approve them. Clinicians, doctors, physicians need to have data that knows that it's a useful, viable option to suggest to their patients. Patients trust doctors and the FDA or any regulatory body where you may live and really seek options that work for treatments that they're just, they just want solutions that work. And frequently, when there's uh intellectual property, when there's a patent, when there's a commercial market, the system works fine. It's not great, but it's sufficient. The really issues and the challenges and where Cures Within Reach steps in and has focused on for now over 20 years is helping those therapies that may have less of an option, may have less of a commercial market, or before it is known that they are going to be safe and effective in a second, third, or fourth different disease.

Keith D. Terry 6:32

Well, tell tell us a little bit more about Cures Within Reach, because I want my audience to really understand kind of the uniqueness and the beauty of what you guys are doing.

Barbara Goodman 6:40

Yeah. So you mentioned thalidomide. It's actually, coincidentally, one of the therapies that got Cures Within Reach started years and years ago. We at Cures Within Reach are known in the repurposing ecosystem as the grandfather of repurposing because of the thalidomide story and what happened. It was pulled from the market, obviously, for a lot of problems that it had. And it was a researcher named uh Dr. Raj Kumar, Vincent Raj Kumar at Mayo Clinic, who looked at thalidomide and said, this might work in multiple myeloma, which after everything that happened with thalidomide, um, and it did get approved for leprosy, they said, that's a crazy idea. Why would you, why would you test that? And it took the innovation and uh entrepreneurial spirit of our founding family to say, you know what, it may be a high risk, but it could be a high reward. And Cures Within Reach and our parent organization funded the Mayo Clinic for that late-stage preclinical prior to getting in and a human trials, funded the human trial, that first validation point, that efficacy proof of concept. It's known as a proof of concept trial, uh, phase one, phase two A types of trials, and it was successful. In that case, there was intellectual property developed, industry player participated, took it from Mayo Clinic, brought it to regulatory approval using a spin-off of sorts of that drug. And a lanolitamide is still to this day uh considered a first-line treatment option for a multiple myeloma and blood cancer. And after now 25 years, that is considered a the grandfather example of what in in our world we call drug repurposing.

Keith D. Terry 8:34

Repurposing. Good job. I mean, so let's stay there for a second, not repurposing, but structurally. What do you see as the biggest challenge right now? Where is the biggest challenge right now? Because you guys are perched at a very unique place. So how how do you what how what do you see?

Barbara Goodman 8:50

Yeah, I think like many situations, it's never just one thing. This is a very complex situation. Drug development is complex. The answer is um gonna vary based on the disease in the area. I will, I will say, I think there's a couple structural and systemic challenges. One is incentives. When when a drug that's been approved for a specific disease ends its patent life, there are very few financial incentives for anyone in the ecosystem to look at that safe, effective, clear, safe known profiles in other indications, in other diseases for other uses. There's not a lot of when there's no intellectual property, when there's no patent life, there's just an unclear incentive area to bring that forward, to get that tested. That's definitely one of the issues. The area is not short of ideas.

Keith D. Terry 9:53

Yeah, you're not kidding.

Why Off-Label Matters So Much

Barbara Goodman 9:54

Clinicians and researchers, doctors around the world have ideas. They see what's going on in their patients, they want to test things. There is a lot of funding by the federal government and others on what is known as bench research, lab research to study new drug development, looking at things in the lab. What is harder is the space to prove that things will work. And then it's reaching the larger funding available from industry, from the NIH in the United States, other regulatory bodies around the world, and large philanthropic organizations will fund these larger trials. So there's a also a challenge of a gap of funding for startups and the entrepreneurial world. It's known as the Valley of Death. It's very, very similar in this space as well, which is why Cures Within Reach focuses so much on that proof of concept, because we know there are funds available in the ecosystem once that proof of concept clinical trial shows that this drug has clear signals that it could work for a new disease or an unmet need. And it really needs a clinical, clinically validated data that a regulatory body or a regulatory agency will consider strong enough for approval, even if the goal isn't for a drug approval by a regulatory body. What a lot of people don't know or understand is what off-label use is in the pharmaceutical world.

Keith D. Terry 11:36

Why don't you explain it?

Barbara Goodman 11:39

Most drugs in the United States, they get approved by the FDA, and then they are used what's known as on label. So the FDA approves, approves ozembic for type 2 diabetes. It's used off-label when a clinician, a doctor, prescribes that particular drug for a disease or an indication that the FDA has not said is safe and effective. It's known as off-label. The FDA allows a pharmaceutical company to create a drug label that says drug X is safe and effective for disease Y. And that's what's known as on label. What most people in the United States and around the world don't realize is it's pretty well known that about 25% of drugs, of prescription drugs in the United States are actually prescribed off-label. And for cancer, for oncology treatments, frequently up to 50%, much higher than people realize. The off-label space is a fascinating one and a really important dynamic in drug repurposing. We as a nonprofit can talk about off-label use. You can promote it, we can talk about it, publications, doctors, clinicians in the United States, it's perfectly acceptable. And freak, but not always reimbursed by insurers. What's interesting, though, and understandable is the FDA does not allow pharmaceutical companies to talk about anything off-label. That is prohibited.

Keith D. Terry 13:12

Yes, it is.

Barbara Goodman 13:13

We can talk about it as a nonprofit, and clinicians and doctors can publish about it and they can talk about case reports. It's just pharmaceutical companies can't promote, advertise, or recommend anything that is not on the label of the drug.

Keith D. Terry 13:30

And just for clarity for me, your company can do that because you're a non-for-profit, or is there or is there some other I mean, am I restricted from talking about drugs off-label?

Barbara Goodman 13:41

Trevor Burrus, Jr. The the organizations that are prohibited from talking about off-label use are the companies who own the market authorization from the FDA.

How To Prioritize Patient Need

Keith D. Terry 13:53

So I think pretty interesting is, you know, I did a little research here. There are 7,000 diseases. And of course I got this off your website, but I did some more research. And there are there's seven, I want people to understand this. There's 7,000 diseases worldwide. And there are only 4,800 drugs approved for use. And if you consider what you just said, that means that 4,800 drugs are treating a disease category of nine of 7,000 diseases. But then I went a step further and I asked, you know, how what percentage is it is it curing? And some of the research that I came back said it was hitting only 10%, which bodes well about what you're saying. That if you know 90% of the people walking around here with diseases or afflictions and they have nothing available to treat them. So you are you are in a really, really good spot. And so with that said, let's talk a little bit about prioritization. You know, it's a big problem. You got pharma, and I do get the incentives. And when you say incentives, I think you're talking financial returns. And if I'm not clarify it for me. But when we start talking about prioritization, I can only imagine coming from the healthcare space for full disclosure, folks listening to me now. I get this. But for those folks that don't understand, from your vantage point, what do you see as the prioritization of what gets funded and what doesn't in a disease category space? And even yours, because you guys have to prioritize what you fund and what you don't.

Barbara Goodman 15:27

Yeah, so we look at incentives and impact across several areas.

Keith D. Terry 15:34

Okay.

Barbara Goodman 15:35

There's commercial opportunity and commercial impact, but there's also patient impact. Right? So pharmaceutical company absolutely has, especially publicly traded, they have shareholder fiduciary fiscal requirements to make strategic financial decisions that are good for the shareholders as well as the patients. I could tell you, pharma companies, the people at those organizations, and and you used to be one of them, want nothing but impacting patients. It's why people are in healthcare, is to make a difference in people's lives. At the same time, the company has a financial responsibility. So they are looking at and prioritizing strategic decisions on where they should be funding certain therapeutic opportunities. We do the same thing, but we have additional levels because we don't have shareholders and we don't have a profit and a bottom line to impact. So we look at both opportunity from the patient's view. So what is the patient need? What is the unmet need? What is the problem that the market is not solving? I think people often look at a disease and say, oh, there's a drug for that. So that problem is solved. People forget, especially in cancer, that drugs approved for those diseases may often only solve 20% of the cohort or the patients with that disease, or it might work for five years and then it stops working for patients, or the disease progresses. And you need different options. And patients need a kind of a breadth of options, and the clinicians want to provide those. When treatment A stops working, you need treatment B, C, and D lined up on the shelf. So not only are there 7,000 diseases, today people would actually say it's more than 10,000 because rare diseases are being discovered every day. Wow. And it is frequently considered higher. For example, think about breast cancer.

Keith D. Terry 17:43

Yeah.

Barbara Goodman 17:43

We now know a lot more about breast cancer. There's 10 different types of breast cancer. And some of them are, you know, genetic related, and there's type two positive and triple negative and her septin and all of these types. So is that one disease or is that a whole host of diseases? Because different drugs are needed for each one of those. At the end of the day, how do you prioritize and how do we as a nonprofit look at this? Yeah. We always keep the patient in mind. And what we frequently say is patients don't care if a treatment is on or off label. Yes. And they don't care if it's on or off patent. They want a treatment that solves their problem. They want a treatment that works. Clinicians want to be able to provide a therapeutic treatment option to their patients that they can trust has good data behind it and that is going to be successful or has a really good shot. And that's really where we keep our eye on the ball is whether it's going to make a dollar or if it's going to be a generic, right? There are a lot of generic drug companies around the world.

Proof Of Concept Trials That De-Risk

Keith D. Terry 18:50

They are. So I'm very interested because I do know the drug development process. And when I'm sitting here talking to the CEO of Cures Within Reach, are your studies in phase one, phase two? Is it is it mostly still in the RD phase, or have you moved? How do you, how do you process that? Help me to understand where you guys are.

Barbara Goodman 19:40

So as a nonprofit, we have the ability to fund any of those areas, preclinical, phase one, into phase two, three. That said, we have found the biggest need, the biggest bottleneck and opportunity is in phase one and phase two A clinical trials, about 90% of the funds we deploy and the funds that we're investing in our clinical trials, it's 90% is phase one, phase two A. That's known as proof of concept trials. And when you're looking at an already approved drug, and I will say Cures Within Reach not only studies and approves drugs, but we will fund drugs, devices, Gnostics, nutraceuticals. If we're looking at, um, if we're looking at uh phase one or phase two A, it's known as the proof of concept. When you're looking at an already approved drug, it has a safety profile. Oftentimes you're looking at dosing, a dose determination study. Is it safe in a very, very different indication? Is it a different dose? Is it a different combination? Is it additive to standard of care? Is it in place of? Is it a combination of multiple drugs? Where you need for those, you often need five, 10, 25 patients in some diseases, maybe more, where you're getting an early signal. The results are, yeah, this actually could work. The goal is de-risking. That's really where we see our role is de-risking with the clinical data that once that's completed, the investigators, because we're not doing the clinical trials ourselves, we're funding experts in the field, clinicians and clinical researchers, doing the work, seeing these patients. They're coming up with the idea and they're doing these clinical trials that then once they have that proof of concept data, that phase one, phase two A, we know there are funders like the NIH and industry who will then fund the larger confirmatory study, could be a phase two B, could be a phase three. What's interesting though, Keith, is because of off-label use, a phase one or a phase two that we could fund results in a publication that immediately makes patient impact much broader than just 20 patients in the Original clinical trial, patients, clinicians, doctors anywhere can read about this successful phase 2A trial and start using it off label while or during that larger confirmatory study. In an ultra-rare disease, if this is, you know, 200 patients around the world and it works in three patients, you're gonna get a very quick uptick with a really good publication or presentation at a key conference. You can start using off-label. And that's where off-label use is really critical in the world.

Keith D. Terry 22:35

And now I get it. You know, you just uh I don't know, you you just made me understand, you know, intuitively I I understood it. But in my head, and I I want to ask this next question, I think a lot of people believe that um breakthroughs come with new drug entities. And you've just proven right there, proof of concept in an entirely different disease category. There's 7,000 diseases, and you're absolutely right. I care very little about a patent, what the law says, help me to stay healthy. So um, you know, why do you think people believe that new why do you think the industry focuses on that? Is that because of the power of the of the pharma dollar, or is it some other constructural issue?

The Mad Libs Funding Model

Barbara Goodman 23:20

Yeah, I think it depends on what your view of innovation is, right? Innovation can be the new patented drug that may be a blockbuster, but others would say innovation comes from creating magic from something that can be faster to a patient, cheaper to a patient, and more successful. It's it's it's more likely to be successful in repurposing than devouts and then de novo new drug development. So we think it's incredibly innovative to look at repurposing because it's faster, it's cheaper, and it has a more immediate impact if you're looking at pediatric population, rare disease population, underserved people who live in countries that don't have access to the de novo drug the minute that that gets approved. There are millions of people who don't have access to the healthcare in the United States, and repurposing is really those options.

Keith D. Terry 24:20

And I appreciate that because I was going to ask you to define what repurpose means. My next question is this explain to me how your model actually works. Are people approaching you? Are you approaching the disease categories?

Speaker 3 24:33

How the how do you guys So the best way that I have found to explain our model is a game that I used to play when I was young called Mad Libs. Did you ever play Mad Libs when you were young?

Speaker 5 24:45

Once.

Speaker 3 24:45

Once. So we really have a customized approach where we talk to and work with donors, whether they are family offices, individual donors, large philanthropic organizations, corporate foundations, anyone who's looking to make an impact, a fast track impact in patients' lives through the lens of already approved treatment options. So we, and I'll give you some real examples, we have a large philanthropic foundation based in Silicon Valley, whose goal is impacting high disease burden in low and lower middle income countries. So what used to be known as the developing world, it's low and lower middle income countries. We want generic drugs accessible to these patient populations and funding clinical trials by clinicians based in these countries, known as LMICs, to impact patients. It's it's funding the clinical trials there, it's their ideas, improving the clinical trial ecosystem in LMICs with already approved drugs. So we've partnered with that philanthropic organization to fund clinical trials and LMICs. We work with a family who has a disease impacting their family. It's a high net worth family, and their approach is not to solve their family member's disease, but they know you need to build a treatment pipeline to impact patients with different types of diseases at different levels and course and timelines of the disease, whether it's stage one or stage four, for example. And we're partnering with that family in a disease category. We have funded preclinical, we have funded phase one, we have funded phase two A, because our goal there is to build a pipeline of already approved treatments that are working for a disease that has no options in that case. We have a family foundation, our founding family foundation. We still partner with them on blood cancer, mentioned thalidomide and multiple myeloma. Matriarch of the family passed away from multiple myeloma, and they still have an interest in creating treatment options in the blood cancer space. What's really interesting that we haven't yet talked about is our partnership with organizations that are really looking to accelerate how AI is going to be used in drug development. We've partnered with several philanthropic foundations who are interested in looking at purposing as a fast-track way to bring AI into drug development, fast tracking these into the clinic and making an impact in patients with AI as a way to speed impact in any disease. So the AI work are with other philanthropic organizations where we can actually pool together funds from disparate organizations when there's aligned interest. So a pediatric donor who funds we work with them can be partnered with a cancer donor, and maybe we pool them with a donor who's interested in funding Texas-based research institutions or California-based research institutions. And that's why it's a mad libs approach. So we talk to our donors, what's your area of interest? What do you want to make an impact in? And how can we align those goals? And then we have calls for proposals in specific areas, but we also work to customize that approach. So investigators, clinicians submit to us their ideas, but we're always on the lookout for the next big thing.

Defining Success Beyond FDA Approval

Keith D. Terry 28:32

Yeah. Yeah. I appreciate that little plug there. When I was in industry and we were bringing a product to market, success was getting the FDA to give you the green light to sell it. Um and I know it's different for you, but how so how do you guys view view success? Because I would assume that it's also impacting fundraising.

Speaker 3 28:52

Right. Yeah. So we have a short-term and a long-term view of success. The short-term and how we best measure our success is unlocking additional investment by other players, other stakeholders in the ecosystem to fund the next stage of our trial and really unlocking through our de-risking approach. So if we fund a phase one, phase two A, and the NIH says, now we have the data that works. Here's $3 million for the larger phase 2B or phase three trial. Our $100,000 leveraged $3 million. It's a very real example because it happened, it happened recently. So it's measuring the catalytic impact that our de-risking funding has unlocked. That's a short-term measure of success, right? Longer term, we're looking to solve patients who don't have treatment options. We're looking to provide treatment to pay with to patients who don't have any to create treatment options and build the portfolio of drugs, devices, diagnostics for patients on or off label, right? We want to put ourselves out of business eventually. And then there's drugs that solve other people's, all the people's issues that we have. So that's a long-term goal, right? Patient impact is always going to be our long-term lens. But we do measure our success short-term in the catalytic impact that our funds create.

Keith D. Terry 30:22

And your funders are individuals, companies. Can you elaborate on that a little bit?

Barbara Goodman’s Path To Nonprofits

Barbara Goodman 30:29

Yeah. Yes, to all of those. We have uh philanthropic partners that are individual donors, family offices, foundations, small foundations, very large foundations, corporate foundations, philanthropic partners. What they're all looking for is impact in specific categories like pediatrics, impacting AI, impacting geographically or disease-specific. So we are incredibly broad because we're disease agnostic and geography agnostic. At the same time, we're incredibly narrow because we are only looking at already approved drugs, devices, diagnostics. And so our donors are anyone who is looking to make an impact in those areas in a fast track approach, faster, lower risk, higher reward type of opportunities that it's just faster to impact because they're already approved.

Keith D. Terry 31:31

What led you to do this work? I want to hear your story.

Barbara Goodman 31:35

Yeah. So I never would have guessed that I was going to be doing this when I got my MBA, however many years ago that was. Right, right. Never. And I think that that's the lesson I would share you can create a plan, but let life happen. I started out doing strategy and growth for for-profit companies and worked for several for-profit companies in strategy, corporate development positions, things like that. And then I started doing that work for a hospital. And that was my first nonprofit experience. And I realized that the nonprofit side of healthcare was much more fulfilling personally and professionally than the for-profit side of healthcare. And so I took at that time over 10, 12 years of for-profit experience and decided to just stay in the nonprofit side of healthcare. And I've been in the nonprofit side of healthcare now a very long time, much more than my for-profit time. And it's so fulfilling to work in healthcare, making an impact in patients' lives. But I'm not the scientific brain. I'm not the PhD or the MD brain. But having an impact in patients' lives from that business angle, people often forget that nonprofits is their nonprofits are still businesses, right? You still have to have a budget. You still have to spend money thoughtfully. It's still an organization. So I knew of this organization, Cures Within Reach, for a while before I joined it. The mission is so obvious. We should be doing more with what we already have on the shelf. How can we unlock the impact that these approved drugs should be having and joining the organization to grow it and branch off and make it as successful as possible? And suddenly I've been at the organization eight years now.

Keith D. Terry 33:35

So let's stay there for a second. So you've been on the non, you've been on the for-profit side, the nonprofit side. How has being on both sides shaped how you think about innovation?

Speaker 3 33:46

Well, it's, as I said, for cures within reach, it's the patient voice and putting the patient first. It's many nonprofits and my time working at a hospital. You got to make, and even today can make strategic decisions that aren't always the blockbuster drug, right? Or the best decision, but it's the most impactful, or it's the right decision, or um, a decision that is important or risky, that deserves, you know, ultra-rare is a great example when you have 200 patients or a thousand patients in the world, they deserve treatment options too.

Keith D. Terry 34:24

I I want to now shift towards AI. Do you think it will quicken your your pace or deeper your focus?

Barbara Goodman 34:35

So AI and drug development is a fascinating space. We are in an ecosystem. We're just getting started, right? We're just figuring this out. What is fascinating about AI when it meets drug development is you can have the most amazing models, the most credible data, but you still have to put it in a human patient. It still needs to be tested. And the FDA or any clinician isn't going to look at an AI output, an AI prediction or a hypothesis without knowing that there's more than just that prediction, right? It has to be validated biologically and it has to be validated in a human clinical trial. We are not yet in 2026 at a place to only look at virtual trials and only look at digital twin trials, right? We're not there yet. We need to make sure that the AI models out there are more robust, have less data gaps, and we can trust and then reproduce, trust and verify that the predictions that these models are coming out with are actually working in patients. And so we see really, really exciting time. I talk about this from the we're at the early adopter stage. Clinical researchers are just starting to use these models in repurposing and just getting comfortable with it. What we know is the AI models, first of all, there are many, many more than people realized in repurposing alone, not just drug development, but AI models built for repurposing of drugs. There are dozens and dozens of these, not just a handful. There's way more than everyone realized. And everyone knows that there are gaps in the data that is building these models. Just this week, Keith, the FDA reminded people that 30% of the clinical trials that the FDA approves can happen in humans don't get their data published on the results of those trials. That means 30% of the clinical trials don't have the data that says, did it work? Did it not work? Was it a little successful? Was it not successful? There are clear gaps in data that need to be filled and need to be tested and verified. And so bringing AI, it's an exciting opportunity to accelerate ideas that are coming and helping clinicians prioritize if an AI model says, you know, all these approved drugs, these three are the most likely. You've just accelerated and sped up that process because now we can just focus on those three because you can validate the biological, is that output well, maybe that's a new funding source for you, is the AI companies that exist. We we agree.

Keith D. Terry 37:40

Okay. And so it leads me to this next question is is pharma friend or foe?

Barbara Goodman 37:46

And that's a great question. I truly believe you cannot do this with industry with pharma against on the other side of the t on the other side uh in in opposition.

Keith D. Terry 37:59

So let's slow down for a second. Are you are is it because people on your side and their side believe that we're they're inherently at odds? I want people to see the problem.

Barbara Goodman 38:08

Yeah, I think oftentimes it's a people view it as an us versus them, or pharma is big and they're not funding this and they should be funding this. I personally believe, and Cures is very supportive of expanding the table, make the table bigger. Don't make uh less of a table. But what's really, really important in this, in the space of drug repurposing, when a company, when a pharmaceutical company goes to the FDA and gets their de novo drug or repurpose drug, right, gets it on label, gets approved, they are the market authorization holder. They are the original holder and they have rights. And what's really, really important is all of the repurposing in the world can happen in support when a pharma company is supportive of it. They are, at the end of the day, people who care about patients and want to make impact in patients. Frequently, the clinical trials that we're funding have the researchers, the doctors doing these trials, are talking to pharma who produces the drug, whether it's a generic or a patented drug, on or off patent, they're having conversations where the pharmaceutical company can donate drug to the trial that we fund. Oftentimes they're watching it and they're saying, oh, well, if you get this proof of concept, we will then fund the larger confirmatory trial, or we're going to donate drug for that trial. Um, pharma has to be at the table and are a very important player because in the United States, we're actually quite lucky because off-label use of drugs is common and accepted. Yes. But not every country in the world is like that.

Keith D. Terry 39:52

So I didn't know that.

Barbara Goodman 39:55

So there are many countries in Europe where off-label use is only accepted and reimbursed on a case-by-case basis. So it is a fascinating. We're actually very lucky in the United States that off-label is so common. It's allowed in other countries. Every country is different, right? Every regulatory body is different. So it really you need and want pharma on your side because often they can open the door to get something on label with and by collaboration from nonprofits, from patient groups, from disease groups. There's a lot of efforts going on around the world on that topic specifically is how do you get things on label when there's no financial incentive for a pharmaceutical company to go to a regulatory agency and put it on label?

Keith D. Terry 40:46

Two comments. First one is I thought that the debate or the pushback from pharma would be because they were trying to protect their intellectual property. And the second comment I have is, and you can comment on the first one, but the second one, I know about repurposing because I work for a company called Pathogenesis, which repurposed tobermycin for patients who had cystic fibrosis. And it was one of the most effective drugs at extending people with cystic fibrosis life. I mean, outside of the United States, the average life was nine or 12. Inside the United States, the average lifespan was 29. And it's a remarkable difference because of what you just said off label use.

Barbara Goodman 41:26

Yeah. And I think, you know, what people forget is, and especially uh, you know, to be fair to pharma, we call it drug repurposing and medical repurposing in the nonprofit and the patient side and the researcher side. Repurposing happens every day at Pharma, to what you just said. But it's not called repurposing. It's called life cycle management, it's called label extension and indication expansion. A very popular drug called Humura has multiple indications, blockbuster drug. That's repurposing. I already mentioned Viagra. It's repurposing. So Pharma is doing this every day. It happens every day. Cures Within Reach is focused when there's less financial incentives, higher risk, smaller patient population, or when it is off patent or soon to be off patent, and there's less likelihood, but still a need to get that de-risked and uh closer to patient impact. So pharma's doing this every day, but they're not focusing on the higher risk, the smaller patient populations, underserved patients in low and lower middle income countries. Pediatrics often is considered off-label. It'll be approved for an adult population and not for a pediatric population. So repurposing is a really important piece of the ecosystem. It's just not talked about. It's incredibly innovative. It's just not talked about.

Keith D. Terry 42:56

Help me to understand that on the on the diagnostic side. You just said or device side. Talk a little about how you guys impact that.

Barbara Goodman 43:05

So I'll give you two examples of successful repurposing on the diagnostic or the device side. So, first, there's a uh medical device that got approved several years ago in the mental health space that because of the Veterans Administration and uh retired military, every VA hospital in the country had to buy this very expensive piece of medical equipment because it got approved for one use. What it caused is all of these clinical researchers at VA hospitals, veterans administrations, hospitals around the country to say, how else can we use this very expensive device? How else can it be used? And we within a couple years got several submissions for second, third, and fourth uses of this medical device for PTSD, for Parkinson's, for different uses. Other than the original use. It's a great example of technology and innovation meeting patients where they are. And in this case, it's veterans, right? So that's one example. Another example is a fascinating example where it's using drugs as a diagnostic tool, which may not make sense, except in this case, it's not a disease, but a healthcare situation when you have a limb trauma that is crushing your nerves.

Keith D. Terry 44:33

Yeah.

The Future FDA Meets AI Validation

Barbara Goodman 44:33

If you you do not want this happening, but you have some traumatic injury, you go to the ER, and it looks like you may need to lose an arm or a leg, and it may need to be amputated. The orthopedic surgeon doesn't know with certainty if the nerves in that limb are crushed or if those nerves have been severed. And your prognosis and your treatment plan is very different. And it's not clear there isn't a diagnostic test for measuring those nerve endings. A doctor and the team that we ended up funding used a multiple sclerosis generic drug, gave it to the patient, and it used it almost like a flickering light bulb. Is that nerve possibly crushed, not severed? And if there was a flickering light bulb, it meant it was crushed and just needed time to heal. Using a drug as a diagnostic is incredibly innovative, right? It has huge military impact. It has impact in the orthopedic trauma stage. It needed that proof of concept to say, yep, that actually could work, right? So that's using drugs as a diagnostic, but there are other examples that um and successful entrepreneurial exits where the medical device got approved for one thing in, for example, real example in the ER. And then an anesthesiologist saw what it was using, what it was used for in the ER and said, you know, we could probably use this over here in electrophysiology. And a clinical trial was done in the UK, it worked. The startup then, it was a pre, it was a uh early young company, and it went all the way to approval, and that company got acquired.

Keith D. Terry 46:23

Okay. Well, if you look out five, 10 years from now, what kind of world do you see when it comes to drug development and and and delivery of treatments?

Barbara Goodman 46:33

So I think AI is really going to gain change and speed to impact. Um, I can't speak to the de novo side of AI since that's not what I look at. I can tell you on the repurposing side, AI is going to accelerate and reprioritize opportunities that we'll all need validating. It's going to be incredibly exciting to see what the AI models can suggest. They need validation. And what's so important is a feedback loop back to make the AI models smarter, since there are so many gaps in data, unfortunately. And I think that's the biggest need is filling those data gaps and thinking about data and more robust data. Not everything gets published. A lot of negative trials and a lot of trials don't get published, and no one is known, no one knows the results. And that means the AI models don't know the results either, right?

Keith D. Terry 47:35

Well, that is a very, very, very good point. I mean, there's research happening all the time. And boy, if you could have access to that data, that is quite insightful.

Barbara Goodman 47:45

And that's really the the critical.

Keith D. Terry 47:47

You think that would happen?

Barbara Goodman 47:49

Open source data should allow that. But the other thing that's going on is we're really at the early adopter, fast follower time point with AI and drug development. We're nowhere near fast follower yet. We're really in early adopters. People who are testing it out, getting comfortable with it, are willing to ask those questions. As we get closer to clinicians everywhere using AI to speed options in repurposing to their patients, if you have a thoughtful, validated model that's learning all at ghost. I mean, that's really huge potential. And it's really exciting.

Keith D. Terry 48:29

Do you see the FDA relaxing its rules and it not taking 10 to 15 years to get something approved?

Barbara Goodman 48:36

I don't think they need to relax their rules. They need to adapt to preclinical data that starts from AI and then gets validated, right? Or what we talk about is a surprising prediction from AI that gets validated. You don't always know the mechanism of action. If it works, do we really need to know exactly why it works, or do we just know it works? I think that's gonna, I don't think the FDA or any regulatory body and really not just the FDA, but institutional review boards of every institution running clinical trials, they shouldn't lessen their safety and risk profile. They just need to, I think it's going to evolve to better understand what the models can and cannot do and what needs validating and what allows comfort in the reproducibility of those predictions.

Keith D. Terry 49:30

If someone wanted to get involved in what you guys do and help move things forward, how do they uh do that?

Barbara Goodman 49:36

Well, they can just reach out, whether they're a patient who wants to join a review committee and and help us select trials and their disease to donors who want to make an impact, to clinical researchers who have ideas looking for funding. It takes an ecosystem. And as I've said, we want to expand the table. So they should just reach out to Cures Within Reach. Contact information is on our website, and I'd be happy to send them to the right team if they start with me.

Keith D. Terry 50:06

Thanks for listening to the next big thing. I'm your host, Keith D. Terry. If you've enjoyed this episode and you'd like to support this podcast, please share it with others. Post about it on social media or leave a rating and a review. To catch all the latest from me, you can follow me on my YouTube channel at Keith D.Terry. If you want to recommend a guest, please email me at infoterry performance group.com. This has been produced by your host and Jake Productions. Uh