He's alive thanks to a repurposed drug. Now his dream is to find cures for millions of people from among drugs we already have.
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You discovered something that helped you, having tried many different interventions. But you finally came upon something that made a difference. You've been in remission for about 8 years now. Is that right?, also called rapamycin, is a drug that was never intended for Castleman's and had never been used before for Castleman's. Sirolimus is a potent inhibitor of mTOR.
I drove down to Washington, DC, and I took the results to a doctor of mine at National Institutes of Health and showed them to him. I said,"What do you think? I've got this deadly disease. I keep relapsing. There's no way I'm going to make it to my wedding day unless we try something new. I'm out of options." And he said,"Sure, let's give rapamycin a try."One effect is, of course, that it can help with mitochondrial dysfunction.
That approach has been groundbreaking. It's been amazing to get patients to participate in the research questions we're asking, to get researchers who had never heard of Castleman disease, but they're the best people in the world at single-cell RNA sequencing to work on our project. It's been a great example of how when you bring the right people together and you have a clear mission and vision, you can achieve so much more than you ever could have on your own.
The reverse of that is that there are patients suffering when there's a drug sitting on the pharmacy shelf that could have saved their lives. The more I saw this and, frankly, the longer I've lived on this repurposed drug, the more compelled I felt to say that we need to do something systematically. We can't just do it with Castleman disease and then POEMS syndrome, one rare disease at a time. We really need to do this at scale.
He said,"What's your dream?" And I told him. I said,"My dream is to create an initiative to do this systematically, to unlock the full potential of every drug for every disease possible." And he said,"Well, if you ever do that, know that you've got my full support. I'd love for you to announce it at the" He kept calling me every few months to check in to see if I was going to do this.
I've worked, thankfully, very closely with Carl June and John Wherry and others to think about the tail end of cellular immunotherapy, because if you can't control the cytokine storm when you give someone immunotherapy and they die from the treatment, that's a travesty. Right? So let's get them these life-saving therapies, but let's make sure that in the process of treating them with a lifesaving therapy, we don't cause more harm than good.
So, where do you go now? You're starting to have an impact globally. Are you going to move outside to the galaxy? What's the next step, David?I'm just so thankful to be here. I mean, literally to be on your podcast with you — what an honor for me. But frankly, just to be alive... I feel like I have this incredible responsibility. I'm alive because of a drug that no one paid attention to, that was just sitting there all these years.
Finally, what's so exciting about this approach is the turnaround time. If I were to develop a new drug from scratch, I would need at least 10 years and somewhere on the order of $100 million — maybe closer to $1 billion — to develop that one drug for a first disease. We can repurpose drugs and discover a new use for a drug and within weeks have a patient on that drug. Of course, the risk-benefit profile has to make sense.
What is the cost of the drug to the healthcare system? You can start to factor in things around impact that drug companies, frankly, aren't able to think about because it's not part of their business model. Is it likely to help? That's where you have to build a great team and bring together a lot of great scientists.
We've got an approved drug, it's on the market; let's ramp up R&D; let's find all the additional uses for it.Now that the drug has its first approval, let's start ramping down R&D and let's go find another drug.
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