Dinakar Singh - A Father’s Call To Action - [Invest Like the Best, EP.428]

My guest today is Dinakar Singh. Dinakar is the founder and CEO of Axon, the family office successor to TPG Axon, which was a successful global long-short hedge fund. We wanted to share his story on Father's Day to honor the person and the dad that Dinakar is. He shares one of the most extraordinary stories at the intersection of finance and medicine that I've ever encountered.

This conversation explores the highest-stakes investment themes: timing, concentrated conviction, exceptional team building, and deploying resources toward outcomes that matter most. I want to let him tell the story of the remarkable journey he's been on to cure a disease called SMA that so deeply affected his family. So, Dinakar, I'm so excited to tell your entire story. It's one of the most unique, moving, and incredible stories I've encountered in investing, and I guess in life more generally. I was thinking about how to ask about it to kick us off, and maybe the appropriate way is just to ask you why you named your firm TPG Axon.

1. Saving Nora Became The Mission

The axon is the nerve that connects your brain to your muscle, and it happens to be the core part of what degenerates in spinal muscular atrophy, which is the disease my daughter was diagnosed with. From my perspective, an important factor in starting the firm and leaving Goldman at that time was that, on one hand, while I loved investing, I also resolved that there was a glimmer of hope to save my daughter and other kids who had her condition, but that glimmer of hope was not going to last very long, and time was not our friend.

I needed to go as quickly as possible to drive success as quickly as possible, obviously for its own sake, but more importantly, so that I could throw a variable fortune into the research we needed to do to make sure we did it as fast as possible, in time to save her. She was diagnosed in 2001. I'd just come back from Hong Kong literally a month or 2 months before. I'd become global head of the group while there. I was in Hong Kong for 4 years, setting up the Asian business and becoming global head at that time.

I came back, and we were on top of the world. Our group was extraordinarily profitable, even during difficult years for the firm. I came back, and a month later, 9/11 happened. A month later, our daughter was diagnosed with SMA, spinal muscular atrophy. She was a year and a half at that time.

We'd been shifting back and forth between Hong Kong and New York. It was our first child, and it's ironic because you actually don't know what is normal in terms of development. We thought she was developing normally, but we were at a party, and one of our friends' mothers was a very good pediatrician. She said, “Look, you want to go and have her checked out. Just go have her checked out.” That started the saga of getting test after test after test.

Eventually, literally a month after 9/11, I was sitting in my office watching the smoldering ruin of the World Trade Center, which was right in front of my window, and I got this call from the doctor basically saying that she had SMA. For a bunch of reasons, it was a shock. Technically, he called my wife. It was a 30-second call: “Sorry, she has SMA. Gotta go.” Then he hung up.

She called me in tears. We called him back, and it was maybe 60 seconds this time, but at that time, no one knew much about this disease. The gene for it had just been discovered a few years before. Until then, people thought it was just a variant of muscular dystrophy or some other muscle disease. So, in SMA, you're missing a key gene that makes a key protein.

That protein is like a fertilizer for muscles and nerves. Like fertilizer with plants, without it, they wither and die. All of us have a backup gene because it's such a critical protein. Your body has hundreds of them, you see. Some people have 1, some people have 2 or 3. That backup gene doesn't work very well. It makes a protein that falls apart because it's got a defect in it, but it has some.

Depending on how many backup genes you have and how little or how much protein you have, you might die within a few weeks, or you might die within 10 or 15 years. Our daughter was in the middle, and so the diagnosis at the time was that she'd probably live to be in her mid-teens or so, but getting weaker and suffering along the way. My parents had been scientists, as it happened, probably a source of my strange thinking in life. After school in India, I'd go hang out in their lab and do mouse dissections when I was 6 years old. It's a typical after-school activity.

I grew up in the science world and had enough scientific knowledge to be dangerous. As you stare at this, it seems super intriguing to me. In the first few months, it was just devastation. When you're told there's no treatment, there's no cure, and it's just a question of when and with how much pain, you're mentally clobbered.

We first started giving some money to some groups, and as I started digging into the research, it became clear that there wasn't as much known about this as one would think, given the gene we had just found. For example, why did that neurologist diagnose her and hang up in 60 seconds? Obviously, because he didn't know anything about this. When he went to medical school, there was no spinal muscular atrophy because no one knew that it was a separate disease, let alone one that actually affected 20,000 kids.

Technically, at that time, it was the most common genetic killer of kids, which is a pretty incredible thing when you think about it. Intrigue started setting in. At first, my wife and I started giving money to some causes. There were a few groups that were involved, and so we gave a bit of money. As we dug in more, we started talking to scientist friends at Columbia, et cetera, and became more intrigued by the possibility because some really good work had been done.

In your body, if you're missing a gene and it's completely gone, replacing it—true gene therapy—is still really, really hard to do. On the other hand, here you had this backup gene that was defective, but having a backup copy in place that you could try to crank up and fix to make a bit better is a much easier challenge than having to replace something that simply went missing in the first place. That struck me as being pretty interesting from a drug-development perspective as well.

There was some work being done. You could create mice that had SMA, which is also a pretty big, powerful feat. You had an intriguing thing to tackle. You had some tools that were pretty nifty in these cell models and mouse models, so you could test things in them to see whether they had an impact or not.

This is getting a little nitty-gritty, but for a lot of drugs, one of the biggest problems is what they call the therapeutic window. With almost anything in your body, too much or too little is pretty bad for you. I joke with people: take water as an example. Water has a very wide therapeutic window, but if you drink enough of it, you'll actually get kidney damage and die.

Most other things are much, much narrower, and so there are many drugs that might work, but we just don't know exactly what the precise amount is and where to get it. As a result, they end up having toxicity problems or undershooting, et cetera. With SMA, the difference between some of these kids with multiple copies versus not, someone who is a carrier versus having a disease, or someone like you, presumably, who doesn't have it at all, represents such a massive, wide range of this protein in the body that it's pretty clear that you almost can't have too much.

There's no real worry about that. That really was a pretty intriguing thing for developing a drug as well. Again, not to get too much in the weeds, but as I read into these things, it really seemed like there was at least something to be tried here. As it happened, there was an interesting coincidence: the NIH had, for the first time ever, begun to focus on rare disease. The world is very different today, but rare disease was not something people focused on back then.

No one cared. There had really only ever been 1 rare-disease drug, and companies were focused on big things at that time: statins, diabetes drugs, and oncology.

No one wanted to work on or look at rare disease. NIH was also funding all these big diseases, but they came up with a novel program at that time to have the first-ever grants awarded for translational research. They picked SMA because it was this cool, nifty, new disease and said, “We think this is the best target. We’d like to have a small program where we can provide some grants to work not just on basic science, but on actual drug discovery.” Again, today this seems completely obvious. Back then, that was a really novel thing.

Unfortunately, there were a bunch of changes. The head of NIH stepped down, and the head of NINDS, the National Institute of Neurological Disorders and Stroke, stepped down, so you had acting directors in both places. I would joke to people that when you have acting directors, that means there is no action on stuff. Nothing was really happening.

There was pitiful grant money from NIH for this disease, even though other diseases of similar size and with a lot less potential probability of success were getting tons of funding. There is just a latency bias in things. If you think about this, if you’re a student in medical school and a disease is well known, you might want to focus on it afterward, so you do more research. If it’s a disease no one’s ever heard of until recently, there aren’t many people working on it, so there aren’t many people applying for grants. When you apply for grants, there aren’t many people who understand it well enough to know whether it’s a good grant or not. There’s a lot of stuff that just takes a long time for the cycle to work.

2. Parallel Processing Beats Delay

As we stared at this and said, “Look, there’s a chance here, but we might be waiting a couple of decades or so for this to work. By that time, she’s going to be gone,” it occurred to me—and I tell people this now—that the second-worst thing that can happen to a parent is to see their child suffer and die. The single worst thing, though, is to see your child suffer and die, only to find out that you could have done something about it, but it wasn’t done in time.

Once we got the idea in our head that there was a glimmer of hope here, it really almost deranges you, if you will, because then any second you’re spending doing anything that isn’t max speed is a wasted moment. That led to, “We’re going to take a shot at this. I’m not going to take a shot only to find out we went slowly. This has to be max turbo. Pull out all the stops. Let’s go for it.”

Part of that was money. It struck me that all these things take time. You could fund some scientists, get some basic research, and then go to the next one, to the next one, to the next one, and that will eventually work, but it’ll take forever. It struck me that we needed to do things in parallel processing, not serial processing. If there are 5 interesting ideas, let’s do all 5 at the same time and see which ones work, not do 1 at a time and go A, B, C, D, E, F, G.

Basically, what we drew up was a pretty aggressive vision. Early on, it was giving money to people like Columbia, et cetera, and we did. We set up Motor Neuron Institute there with someone who wanted to fund ALS as well, and they’re quite similar diseases in terms of some of the impact.

It occurred to us that academia, while very important, was never going to develop a drug, and so we really had to create a virtual company. The thought then was to hire some scientists, get some amazing advisors together to really help be our brain trust, and then sit down every so often with everyone and say, “What do we need to do to move this ball down the field?” Then go and do it, whether we pay someone at a company, give a grant, or whatever. Give money, get it done, move on every front at the same time, and then keep coming back and saying, “Okay, what did we learn? What didn’t we learn? Let’s go on to the next thing.”

Now, all that takes a lot of money. My father would joke that, for this straight-arrow kid, I developed the world’s most expensive drug habit. I was a partner at Goldman, and, look, at some point I would have left anyway. In reality, at some point, if you love investing, you want to do it in its own pure form. But it certainly occurred to me at that point that, given all this, it was time to get going—1, because of course I’m passionate about investing and I wanted to build a great firm, but 2, because the value of doing that right now, and the dollars that could come from it right now, could save my daughter in a way that if I waited 5 years, 3 years, or 2 years, it wouldn’t.

The Axon name, going back to the long answer to the very short question, was really just there to remind me of what the mission was. It was always amusing because people would wonder, and I’d explain, “Well, you know, the axon is the thing that connects muscles, nerves, and the brain, so it’s connecting thought to action.” They thought that was a cool analogy from a general perspective.

Every now and then, you’d get a scientist or a former company guy who’d come in, see our logo—which is actually the shape of an axon—and get the fact that this was actually about medical research and stuff, which was always kind of cute. It’s a bit of an Easter egg that we sort of hid in there.

But, look, that was what we did. Functionally, my day job was really there to pay for my night job, if you will. We got teams together. TPG was my partner in starting up Axon. They were remarkably helpful. Through my relationships and through some of their network, we were able to connect with some of the absolute rock stars in drug development—people who were not just smart scientists, but actually biotech guys who really understood this stuff.

For all of them, I think what was extraordinary was how much of their time and energy they were willing to commit. Obviously, we weren’t paying anyone, but we would get a dozen and a half of the smartest drug-development people in the entire world coming together for a couple of weekends a year, taking time away from their families and their work, and sitting with us and brainstorming about our plan of action.

Part of it was because, as crazy as it sounds, there had not been a single meaningful drug for a neurological disease developed in decades. Crazy, right? The idea that there was a chance to do something here was really cool because neurology was kind of a graveyard. I think everyone seemed to have this sense of excitement that, okay, there was actually an interesting plan here. Maybe it would work, maybe it wouldn’t, but there was someone willing to write the check and fund this, so it wasn’t about begging for money. I think people were excited by the idea of joining in to figure out whether we could actually solve the disease.

3. The Drug Hunt Begins

One of the first stops was that there were some scientists who had done some work suggesting that some existing drugs could actually modify the disease in mice and things like that. We basically created all these mouse models and cell models of different types to test different versions. Each had pros and cons, and we set up facilities to essentially give them away for free.

What we said to everyone around the world was, “Guys, send in your drug libraries, your compound libraries. Test them for free. We’ll pay for it. You keep the IP. We just want you to know if there’s something interesting here.” Again, because no one cared about rare disease, let alone a pediatric one, it took years before any company, even for free, was willing to do that, which is crazy.

Finally, we got Novartis to bite, and they ended up finding a drug that had failed for toxicity reasons for anorexia or something back in the ’70s that was off-the-charts good. Suddenly, it was, “Oh, this is interesting.”

To give an example of just how different the world is now versus then, early on I’d use relationships to drag some of our scientists and advisors to meet with heads of research at different companies and try to pitch them. I’d say, “Look, guys, I know this is a small disease, but there’s a chance to do something. It won’t cost a whole lot to find out. You don’t have to spend 40% on marketing, all that kind of stuff.”

One of my slides showed that if you charged $5,000 to $15,000 a year, you could actually end up with a drug worth hundreds of millions of dollars in profit. It wouldn’t just be a charity project; you could actually make a little bit of money from it.

The head of one of the largest biotech companies actually laughed at me in the meeting and said, “Look, Tancrede, the most expensive drug in the world is Avastin,” which was an oncology drug. “It’s $50,000 a year. No one’s ever going to pay that kind of money for a small rare disease for children.”

It became a running joke in the end because this person, Alessandro Rock, was an incredible advisor and helper to us. He joined our scientific advisory board and really helped us immensely. In the end, the first drug that we helped develop went through Cold Spring Harbor Laboratory and then a company called Ionis. We then had them partner with Biogen.

Biogen essentially bought into the drug and became the first to market with an SMA drug. When they did, they charged, I think, $730,000 a year for it. I think it’s one of the top 3 drugs, pulling in $4 billion or $5 billion in revenue. Amazingly, today the SMA drug market, I think, is high single-digit billions, and it’s a top-3 drug for Roche, Novartis, and Biogen.

But the notion that a pediatric neurology drug would be anything of value back then was a hard sell. We basically had to put it in a box with a bow on it and give it to people for free because otherwise they’d never touch it. That’s really what we did, and we spent a fortune on models testing everything.

It was clear after testing everything that some of the existing FDA-approved drugs would have some impact, but none would be good enough, and you’d have toxicity issues, so it didn’t work. So we then pursued all 3 shots on goal that we thought were relevant. Cold Spring Harbor Labs had a great scientist who had what’s called an antisense oligo, a biologic—one of the first biologics. Essentially, the viral vector takes the protein in the body, and it was designed to patch that defect in that backup gene and help that backup gene become a fully functioning regular gene, which would be magic, presto.

That became the first drug, the one that Biogen ended up buying in. Second was good old-fashioned small molecules, syringes and pills. It was a bit like what Novartis, for example, had found: something that worked. We basically paid a company called PTC Therapeutics lots and lots of money to go and test everything under the sun they could imagine with different screens, and ended up finding one that really, really worked.

And so we basically just paid to develop that, and then actually got Roche in to go and take it from there. That’s actually the drug my daughter’s on now. In between was gene therapy, which a company called AveXis did, and then Novartis bought it for $8 billion. And I guess it’s really the first gene therapy drug ever approved.

Here, we didn’t support it as directly from a financial and other perspective, though our board members were active investors, and we helped them with the network and the library and the clinical trial networks. Our focus, just being selfish to some degree, was that gene therapy is great, but only works for very little kids. Essentially, in gene therapy, you’re taking viruses and bringing protein into your body, and when you’re large, that amount of virus will kill you, basically. The toxicity becomes an issue.

But we wanted all 3 shots on goal to be scored. Look, we got lucky. Today, we have all 3 as approved drugs. So the first was that Biogen drug, the second was the gene therapy with AveXis, and the third is Roche, which is just a syringe you take every day. That was incredible luck, I suppose.

4. Accelerating FDA Approval

But part of what we did from a time perspective, because we were desperately worried about the clock running out, was try and make sure that we could have everything in place so that when these drugs were ready, they could go 90 miles an hour. And so, for example, we went to the FDA through lots of relationships on the political side, both to get NIH moving, by the way, in the early days, and to help later on. We got to know, if you will, all the relevant senators and congressmen that mattered when it came to drug development, NIH, and things like that.

And look, our view was just that a parent with tears on their face doesn’t get very far. But we would say, “Look, we want to be really supportive of you. There’s a chance here to actually achieve a success, and that’s why you should focus on this, not just because it’s sad, but because it might be fixable.” And I think that became a powerful combination. It took some work.

We, for example, got 50 top scientists who are Nobel Prize winners to sign a letter talking about how there was a really good chance of success here. We then started literally taking out full-page color ads in some of the political newspapers in Washington—Roll Call, Daily Herald, that sort of stuff—so that all the staffers would see this very striking, full-color ad every day on this stuff. Lots of stuff like that, to really go and essentially create a real sense of momentum on the side, because it did occur to us that while, at the end of the day, developing the drug mattered, getting companies involved would matter, and everyone would move faster if they thought that the other guy was there too. And with the FDA as an example, that really helped.

They worked with us, and we said, “Look, guys, we need this to move fast. So what can we do? And by the way, kids are dying. We can’t afford to run a placebo trial; it doesn’t make any sense.” And so we worked with them and got their approval to essentially run a natural history study, meaning while the drugs were being developed, we basically paid for people’s care for a handful of years so that we could have a log of what the natural progression of the disease was and use that as our placebo.

And so when it came time for the trials, the trials didn’t have to be placebo-blind trials, essentially because the FDA was willing to accept the fact that it would compare the results from the trial to what the natural history study results were for kids normally with SMA. That saved a lot of lives and a lot of money because otherwise, half the kids would have died in those trials, including potentially our daughter.

With all the persistence and with a little help from our friends as well, the first SMA drug, I think, was then, and maybe still now, the fastest approval by the FDA ever in the history of any drug.

Wow.

Which is pretty cool. The good news, bad news, of course, is that my daughter ended up being on the first drug since she was 12, so she has been stable since then and has ended up being just the rock star of the family. She went to Yale, won 3 of the 11 awards for most outstanding student, was class speaker—you name it. And during COVID, amazingly, she went on Hinge and met up with this unbelievably amazing, 6-foot-4, fantastic Princeton kid. They fell in love, and they got married last fall. She’s now getting her PhD at Cornell. So I guess we ended up with our doctor in the family.

But just watching her thrive has been remarkable. That said, the clock was the clock. Every year, she got weaker until the drugs came along. In SMA, the progression typically would be that kids get weaker, stuff starts falling apart, like your spine, and you need spinal fusions. That means your spine is constricted, you can’t grow, and that creates lung damage. Then you start getting pneumonia and all sorts of stuff.

And so you end up—I mean, in the old days—with enormous deformities in children. And like many degenerative diseases—ALS, Parkinson’s, even Alzheimer’s—no one dies of SMA. It just makes you really weak, and then something gets you, whether it’s asphyxiation, choking, or pneumonia, more often than not. Val Kilmer just died yesterday of pneumonia, but pneumonia was really just a side effect of throat cancer.

In her case, our goal was to manically keep her as strong as possible until the drugs could come along. We had physical therapists coming to see her every day. We built a pool in our apartment building for her because you need warm water, and it’s a special therapy pool. But that was important because when you’re a person who’s weak, part of the problem is that it’s circular. You’re weak, you can’t exercise and stretch your muscles and bones, so they keep getting weaker and weaker on top of the original problem in the first place, and that creates this devastating cycle.

And so here, between diet, exercise, and therapy, the goal was to go and keep her as strong as possible to hold off that damage from all these things. It worked and it didn’t work. She did end up needing spinal fusion and having rods put in her back, et cetera. But fortunately, those were late enough that she had already grown, and so it didn’t create the deformity you’d normally see in someone else. She had problems with her hip that blew out and things like that.

When she was turning 10 or 11, every year became a major surgery. And even aside from those major surgeries, she was in the operating room or in the hospital probably 3 times a year with severe pneumonia, at risk of dying each time. And with all these things, it is a bit Humpty Dumpty-like. Once it breaks, you can’t just put it back together again.

On one hand, if it had been a year earlier, that would’ve been all the better. But the good news is that, compared to what we expected at the onset, to see her here, a rock star and truly happy with amazing friends and a husband, is just—

So touching.

5. Regenerating Damaged Muscle

I describe to people: if you have a boat and you have a hole in it, trying to bail water out of the boat is not really going to be a useful strategy. You’re going to sink, just maybe a tiny bit slower. Once you fix that hole, and now there’s water in the boat, bailing water out of the boat could actually be a really good strategy.

Mission stage 2 now is that we’re working on a ton of drugs that we think can regenerate muscle. Nerve is harder to do, but muscle is a bit easier, and we can also help existing nerves get a little bit stronger. We have 1 already in the clinic. It’s not the best one, but we expect to have 3 or 4 things coming forward that could be really impactful. And what’s actually pretty cool about this is that the SMA drugs are SMA-specific: 20,000 kids, so it’s a lot of people, but they’re SMA-specific.

These regeneration drugs will actually help lots of people. And it’s ironic because SMA is actually becoming a poster child for this because—take muscle as an example. I assume that there would be tons of people and experts who knew all about muscle development, from muscle stem cells called satellite cells into muscles, et cetera. There may be 6 people in this country who know what they’re talking about when it comes to muscle. It’s crazy.

I assumed, well, gosh, if you’ve got A-Rod and steroids and all that kind of stuff, I mean, there must be big money in it. But the reality is that muscle has been a really tough field for a couple of interesting reasons. There are 2 types of people who have muscle problems: people with degenerative diseases like SMA, muscular dystrophy, et cetera, or, on the other side, old, weak people. Old, weak people are a disaster to go and do trials on, and you’re trying to see what a moderate impact can make when there’s such decline happening. And so clinical trials—and this is the problem with Alzheimer’s—are very, very challenging in elderly, degenerating patients.

In that first category, people with muscular disease, until SMA, none of it had been solved. It was a bit like that boat where you haven't fixed the hole in the first place.

SMA is now becoming this really fascinating area because suddenly, if you have an idea for a muscle drug, you can actually test it in SMA and see whether it does something. Now you've fixed that boat, if you will. It's quite a difference from 20 years ago, when no one cared. Now suddenly we're calling up people and saying, “Look, we think there's an idea. Your drug can really work here. Let's go test it out.” They're doing it, so fingers crossed.

I think my hope here is that we'll end up with a handful of drugs in the next handful of years that could really help bring back some strength for kids with SMA and other things as well. That would be amazing because, as amazing a life as our daughter has now, she is still fragile. When you're fragile, as you get older, things happen. If we can bring back some strength, that will be even more transformative and impactful, so it's exciting stuff.

The third time I'll say it: one of the most remarkable, inspiring, incredible stories I've ever heard of this type. I'd love just to put a bow on it by asking what the prognosis is now for a new child born with this issue, with these drugs available from a very early age, versus from the age of 12, when your daughter started on them.

A child born and getting the drug post-diagnosis will never show symptoms.

Unbelievable. 20,000 kids.

Yeah, 20,000 kids and their families. Many of the people that we've gotten to know the best over the years are people whose kids are affected by the disease, because there's a commonality and it's devastating for everyone. Parents who lose their jobs because they keep taking time off and can't afford insurance—I mean, it's just this horrible spiral.

There was a family whose father worked at Walmart. They had twins with SMA, and you just wanted to cry hearing their story. That is the funny irony with all this stuff: my parents were both scientists, and they would joke that it was such a tragedy that both their kids became partners at Goldman Sachs. In some ways, this all came full circle because having some of the science background, but also having the good fortune, good luck, and privilege of working in investing and finance, helped us go and actually put that science to work.

A further amazing irony is that Columbia was our leading center in all this, and my mom had worked on the faculty at Columbia. Roche makes the drug that my daughter's on, my father got a job at Roche, and it was a Roche scholarship that paid for my college.

Unbelievable.

Funny how it's ended up being a circular process, but it cost a lot of money. I think we spent probably $150 million of our own money on this. But in the scheme of things, the commitment that we made at the beginning to ourselves was that if there was a way, we were going to find it and, no matter what, make sure that we were able to drive through it.

Winding back to where we started in some of this, I guess that's a bit of the stubbornness. But a preferable word is determination, which I saw in my parents. In that sense, I think there's really nothing less than what I would do for my daughter.

When I step back, I've now had the luck to hear the story twice, and when you told it to me the first time, it seemed almost absurd. If you submitted a screenplay for this, you'd get laughed out of the room by the producer: you're going to start one of the fastest-growing investment firms, one of the most successful investment firms, save your daughter, save 20,000 kids, and develop a drug which, economically, is very valuable—to say which is a footnote compared to the impact it has on the lives and families.

It's almost not believable. It's so incredible. It's such an incredible story. What you didn't say the first time, which makes it even more magical, is the tie-back to your parents and their persistence. Honestly, it's just one of the most incredible, inspirational stories I've heard.

A follow-up question that I have is what you learned, maybe, that you've taught other parents about persisting through years of this kind of effort, and what advice you might give to people who have to dig deep every day for so long to accomplish the thing that they're trying to do for a loved one or for a similarly powerful reason. Because it's not hard for me to imagine the love for the child, but it's hard for me to imagine the ability, with my body, to get through it for so long, and I'm curious how you did that.

6. Turning Sorrow Into Purpose

I think Nora was 2 or 3. We were getting into nursery school. And, by the way, another saga and another crusade that I'm going to be on more aggressively going forward: getting schools to care or take her was unbelievably hard.

When she was going to private school, I think she was the only kid in a major New York City private school in a wheelchair. I kind of know this as well because I'm on lots of boards—the public library, lots of institutional boards—and have lots of influential friends and charity relationships. I couldn't find a single school that would take her. Many are perfectly accessible, but I'd get calls from friends on the board saying, “Look, Dan, okay, it's just not going to happen.”

The ostensible reason—I think one school said she wasn't bright enough. Crazy. It turned out to be maybe not bright enough for them, but bright enough to get straight A's at Yale—summa cum laude and all that kind of stuff. They didn't know what to make of her. We had one school actually ask to see her medical records and talk to her doctor before they'd consider.

Oh, my God.

I mean, guys, you know that's absolutely fucking illegal. But you had to do it because it might be legal, but unless I tried—I mean, when she would start in school, we would send write-ups to other parents basically saying, “Here's what SMA is and here's what to make of it.”

But in any event, for nursery school she went to All Souls, which is the Episcopalian church school. Initially, they didn't want to take her, and they said, “Look, there's great cerebral palsy schools you could send her to.” I'm like, “Yeah, that'd be great, except she doesn't have cerebral palsy, guys. This is a totally different disease.”

Eventually, I think they felt badly and took her, and she had a great experience. But you're supposed to volunteer to give sermons as members of the congregation, and so I did. I'll read you quickly the speech I gave.

Please.

Most of you have probably never heard of SMA. We certainly hadn't, and were horrified by what we learned. It is the most common genetic killer of infants, and it's untreatable, incurable, and eventually fatal. It's described as a children's version of ALS or a genetic version of polio. Simply put, motor neurons die and muscles break down, leading to eventually fatal complications.

In most cases, SMA kills within the first 2 years of life. In more or less severe cases, such as our daughter's, the progression can take many years, so as the mind grows, the body steadily weakens and eventually betrays it. Obviously, we were devastated and shattered. Over the past year, our hearts have broken every day as we've watched her become irresistibly adorable and learn to sing, tell jokes, and talk endlessly, but also lose the ability to stand up, the ability to crawl, let alone walk, and the ability to even sit up by herself.

And yet, 3 realizations have helped us slowly face the future. First, we've truly learned and appreciated the importance of friends and community. The comfort and love of friends has been a source of incredible strength for us, and meeting other parents with issues and learning from them and the incredible challenges many of them deal with has been an incredible source of inspiration for us.

We've realized that there are no good diseases, but there are many, many strong people, and their courage is truly infectious, far more than any disease. More than ever, we've come to realize how much deeper our reserves of strength are as part of a community than as individuals.

Second, through the strength, comfort, and inspiration of our friends, we've gained some perspective and been able to focus on finding the joy in the life that we have. We slowly realized that if we just hold our heads in our hands, we're accomplishing nothing for our daughter or ourselves, for that matter. At first, it's hard not to be consumed by the shattered dreams and lost notions of what could have been.

Yet over time, we've come to focus on the many good things that can be and realize that there is joy in every day. If anything, we're now much more focused on appreciating the little things in life. Today, despite all the issues, our daughter is as happy and joyful as any child we know, and we are as lucky as any parent we know to have a child as sweet and adorable as she is.

So we've learned from our daughter that while the sources of joy and happiness might be different than what we previously imagined, that doesn't mean that the magnitude of the joy is any less.

And third, we realized the importance of channeling the inevitable anger and sorrow into productive channels so as to perhaps have some good, some purpose, and some meaning in all of this. I'm joined with other parents of children with SMA in efforts to improve care and further research so that this disease may one day be eradicated. In doing so, we found a productive outlet for our sorrow and our anger, and also a real reason for hope.

And finally, let me just say how grateful we've been to be part of such a wonderful community. The strength and comfort offered by many of you has helped us focus on life, not on sorrow. After all, in life there are no do-overs, there are no retractions, and there are no appeals.

You either find joy in the life you have or live a life without joy. And while the circumstances of your life might not be up to you, whether you choose to find joy and purpose in them is ultimately very much up to you. So that was the speech.

Wow. Jesus.

I think it captures the process that one goes through. For every parent, it's part sorrow and part rage. Depending on the moment, sometimes it's more rage than sorrow. You just want to go and break everything. But I do think that you eventually figure out that while it's very consuming, it's not going to get you very far under any circumstance.

Knowing other people and talking to people who have gone through things like this is hugely helpful because it helps you recognize that there's a journey in all this stuff. Every parent I know whose child has an issue was unbelievably emotionally supportive to us. Almost every couple of weeks, I'll get a call from someone saying, “Look, I've got a friend or someone who has a disease. They want to make an impact on it. They've heard your story. Can you help?”

To me, that's the greatest joy I have: speaking to other parents and helping them think about what they can do, what can be done, what can't be done, and so on. I joined the board of Jackson Labs. They essentially invented mouse models and are still the leader in that. It's an incredible institution. No drug has ever been developed without using their mice.

I joke that it's the most important biotech institute that no one has heard of. Early on, we had gone to them to work on building mouse models and making them free, so people could call them up and they'd send the mice over. I'm working with them on setting up a rare disease institute. We're going to have a squad of people to do a bit of what we did in SMA.

I hear all these heartbreaking stories, and they're the same things that I had 20 years ago. People who want to make a difference don't just want to make random donations that they think will be like throwing water on sand. They don't know how. In our case, we had a lot of resources, connections, and money, and could essentially reinvent or invent the wheel.

For other people, it's a waste of money and a waste of time, both of which are incredibly scarce when you're a parent with a child who's suffering. Our goal is to have what I call the Ghostbusters of rare disease: if you have a disease, you call us, and we'll get some scientists to work on it and help you put together a plan. We don't care whether it's with us or not. We want to be the brain trust for people who want to think about game plans for trying to solve a disease that affects someone they care about.

How do you hope the world of biotech evolves or changes, given your incredible ground-level experience—not only knowing how the system works and the extreme steps it took for you to speed up timelines, but also knowing that the potential impact can be so massive? There are 20,000 kids with no evidence and no symptoms in their life because of your work and everyone you worked with and their work. How do you hope, with all that experience and knowledge, that the system itself evolves?

7. Reforming The Biotech System

There have been some good things and some bad things, I guess, leading to what I hope will happen next. On the good side, you don't have to bash someone over the head to get them to understand that rare disease can be lucrative for them. There haven't been that many, but when people have seen the money that's been made on this stuff, people care.

The challenge remains that if you're a company working in oncology, even if there isn't a drug tomorrow, you're going to be working in oncology for the next 100 years, and that information can be useful in other things. But the challenge with rare disease remains that a lot of these are pretty bespoke.

The difference from 20 years ago is that you don't need to convince someone that it can eventually be very profitable. But you still have to convince them that there's a good chance they'll be able to cure it, because that early-stage investment is tricky. There needs to be a much more thoughtful effort working with NIH, FDA, and disease groups to find ways to have a much more thoughtful, practical early stage, because scientists writing papers and grants is really not a very effective way to get this done.

There's still too big a gap between a good scientific idea and when a biotech company is actually going to spend the money and do it. You don't have to get it to the 10-yard line anymore, but you still have to get it to midfield. I think more novel structures would actually be great.

To get back to that NIH translational research program from way back when, while it didn't end up making that much of a difference in SMA because we just ended up throwing the money at it and doing it ourselves, it's a pretty cool idea. If one could do that again, it's that Ghostbusters idea that I'm doing at Jackson Labs.

The problem is that we found, in this world, that companies mean well, but they're not trying to solve a disease. They're trying to attach value to intellectual property that they have. If you solve SMA with something that they don't have intellectual property on, it isn't very useful. Scientists are in the business of getting grants, and you don't get grants for, let's say, parallel processing or ruling things out.

You don't get a grant by saying, “I don't think this is going to work, but I just want to prove it doesn't work so I can then move on.” That doesn't get you grant money. But a lot of those things, and the tools and things like that, are very much the nitty-gritty that actually gets you through that early stage where you say, “Whoa, I've got something.”

Whether it's places like Jackson Labs or the NIH, having these amped-up translational research efforts where people can set aside a little bit of money would help. That early stage doesn't cost that much, either. It's later on that you really start going down money rabbit holes.

For a relatively small amount of money, you could actually get people a good chunk down the field, to where companies would care more. When you think about it, obviously the flavor of the day is to tear government down. Government did a pretty good job in some of this stuff, but certainly we can make government smarter.

Whether it's government or institutions like Jackson Labs, there is a role here to be played that could be pretty thoughtful. A few diseases could be picked at a time, and a modest amount of money could be put in or coordinated so that you actually end up having a game plan. What a lot of people need is a game plan, as opposed to having money handed out randomly.

You also need a third party that's viewed as somewhat neutral, because academics are great people, but they don't like collaborating that much. Companies really don't like collaborating with each other because it's an intellectual-property problem. Companies and universities are now having more difficulty collaborating because universities want intellectual property, having seen the money you can get from royalties and things like that.

Having a third party that's sort of Switzerland and can be an honest broker on this stuff is actually pretty important. That's my hope: finding a way to have a more thoughtful process so that people don't have to figure out how to see if there's something there.

On the back end, it's good that people care about rare disease, but the cost of this stuff is insane. What companies are charging is just nuts. I wish, when we had put all the money in, we had put in restrictions on what companies could eventually charge, because $1 million to $2 million a year is bonkers.

I'll give you a funny example of it. I remember talking to the people at Columbia when the first drug was coming out, and it was actually a working-capital problem. They had to bring their CFO in. Since Columbia was a major center, they could fill in Harvard or Stanford or whatever as well. The SMA drug cost $800,000 a year—the first one. To actually buy the doses to treat the kids would have cost tens of millions of dollars that they didn't have in their budget.

It's crazy. It's a real issue. I still get calls, and we get calls all the time, from people in other countries. As an example, there's a Goldman Sachs analyst from Uzbekistan, and they called asking for help because their family knows someone close to them whose daughter was diagnosed. If she gets treatment, she'll be saved.

But in Uzbekistan, there's no place to get treated. You can't just come to the United States and get treated because of all sorts of legal and other issues and loopholes. Of course, then you've got to come up with the money to do it as well. It costs a couple million bucks. You can't just go to London and do it because, again, the whole regulatory framework is crazy.

There are still kids dying of SMA, even in cases where treatment exists. In this case, for example, the family had gone around through supermarkets and stuff—collecting cans and putting up signs in town—and raised $400,000 to get her treated. They couldn't find someone to take the money or find a practical way to treat her.

She was going to die or suffer unnecessarily because of crazy regulatory and bureaucratic issues between countries. We have to figure out something on the cost. Maybe it's with these drugs having some amount of money set aside when there are these windfall profits, to go and help with charity care or things like that. But the notion that we develop these drugs and there are kids dying because they can't afford them is pretty insane.

It’s hard for me to ask my traditional closing question in this particular case because of the story you’ve just told, and knowing that contained in the story is so much kindness in so many directions that I’m sure it’ll be hard to know where to begin. But my question that I ask everybody is for the kindest thing that anyone’s ever done for them.

After my daughter was diagnosed, people just giving me hugs was about the best feeling on Earth. There’s nothing more powerful than just compassion from friends or strangers, just to feel like you’re all human together. I think in life, at our worst moments, just getting some of people’s strength and energy imbued in you a little bit gives you the ability to go and stand up and move forward the next time.

I’m sure I speak for literally everyone listening when I say thank you for telling this story. You’ve imbued us, certainly me, with that same strength and determination. It’s incredible what you’ve done already, and I’m sure what you will continue to do. I love hearing about your daughter’s current state and her wonderful marriage. What a way to spend my day. Thank you so much for your time.

Of course. Thanks, Tom.