Martine Rothblatt, Inventor Health Advances and Getting Past Impossible

Right, well it's just sort of a journey of gender exploration, what we call today being transgender, and generally allowing your internal gyroscope of where you feel your gender identity is to express itself, notwithstanding society's efforts to shove everybody into either a male or female box.

I mean, generally I think that there are definitely some real boundaries in life that people have to respect. I look at those mostly as like the laws of physics, but the laws of society which are created. But within those boundaries of either the laws of society or the laws of physics, there are many borders that we make artificially for ourselves. And I think it's beautiful when people transcend those borders, such as transmitting energy through the air. That was a border that everybody said couldn't be passed, and we just saw before us that that was not a boundary but just a border that you could walk over and transcend.

So it was really a fascination with satellite Earth stations and wondering why the satellite dishes that we see sometimes at cable TV headquarters are so large. And I learned that it was because the satellites that were transmitting to them had such weak power. There's no place to plug in a satellite, there's no nuclear power sources for satellites. And I began wondering if we could make the satellites more and more powerful and focus the energy more and more on a specific part of the Earth, such as the United States. Then in turn, the satellite dishes could be made smaller and smaller. And ultimately you can imagine just taking a flat section of a big satellite dish, it would be almost flat because your section got smaller and smaller, eventually would be flat, it could fit conformally in the roof of a car, and ta-da, SiriusXM.

The content part crossed my mind, especially when I began trying to persuade other people to invest in this project. One of the first things that people brought up was, well, who's going to financially support this? And my response was that people would pay for this content because the choices on the FM and radio band were quite limited. If you were really into, for example, Howard Stern as perhaps the iconic example, if you were in a city that had Howard Stern, great. But if you were not, there was no Howard Stern. And by collecting people's desire to listen to that content, people were willing to pay 14 bucks a month. And now we have millions of listeners of Howard throughout North America. And that's actually the largest group of people that come up to me and thank me for SiriusXM.

Exactly. Rebecca, she came down with pulmonary arterial hypertension. It's a life-threatening disease between the lungs and the heart. She's our youngest daughter, fourth kid, so it was quite unexpected. Everybody else was healthy and she was healthy up until then. My first thought was I think that doctors are supposed to take care of this. But the doctors told us that she would need a lung transplant within three to four years, and even that lung transplant was just a short purchase of time until she rejected it. I thought that surely there must be a pharmaceutical company working on something, and I went and did research and I found out that nobody was working on anything because the number of patients with this condition was so few. At that time, just 3,000 patients in the whole United States, that no pharmaceutical company could make a business on the medicine for just 3,000 patients. So the next thing I thought of was, well, why don't I use some of our money from SiriusXM to create a medical research foundation and we'll give grants to scientists and surely scientists will come up with a cure? I mean, that's what scientists do. So I did that for a couple of years and really progress was getting nowhere. The people came up with kind of very beautifully written proposals, but two years later, Genesis was mostly in the intensive care ward. That's the name of our daughter. And the medicines that were promised were actually not even one month closer. They were still as far away as the day when I gave them their first grants. I don't fault the scientists. Moving biology around is not easy. But one of the scientists was very honest with me, Dr. Barst from Columbia Presbyterian Hospital. She said, 'Martin, everybody in our little field loves your generosity, but it's not going to produce a medicine in time for your daughter. There is a medicine that was developed at Glaxo Wellcome, but they kept it experimental and they won't further develop it because the number of patients is too small and they have a policy of only going after blockbuster diseases, ones that will produce a billion dollars or more in revenues. Surely you with all of your satellite communications expertise, you could go to Glaxo and get that medicine out of them and develop it for your daughter.' It's like, right. I thought she was crazy, to tell you the truth. I had never even taken biology in college. The last biology course I took was in 10th grade. But I had no choice. My soulmate Bina and I, Genesis's mother, we'd be crying. Genesis herself said, 'Can't you do something for me?' So I basically just threw myself headlong into biology. I got basic college biology textbooks, and then medical textbooks, and then journal articles. And I would read back and forth. Whenever I didn't understand anything, I went back to the college level, then went up to the journal level. I went down to Glaxo, I begged for them to license me this medicine. At first they said no, at second they said no. But one of my favorite adages is that people always say no before they say yes. So I kept bugging them. And I built up a credible team of people. I got a Nobel laureate, Sir John Vane, to join my scientific advisory board. And finally they agreed to license me the medicine for $25,000 and 10% of revenues. They thought this was just 'get Rothblatt off our floor.' They thought there was absolutely no prospect of this medicine ever being developed into a drug. And it's so funny because we have now paid them hundreds of millions of dollars in royalties of their 10%. Sales are over a billion dollars a year. The number of patients is now over 30,000, so 10 times more patients. All of those are people who just would not be alive without these medicines. And I must add, as a coder, best of all is the inspiration for all of this, our daughter Genesis. She just turned 30 and she's in charge of telepresence and digital signage at all of our companies, helps everybody work together to develop better and better treatments.

We faced a fundamental business dilemma at the beginning. Glaxo wasn't, in a sense, right based on what people paid for medicines in the 1990s. You could not support the cost of drug development for a drug that treated 3,000 patients. But fortunately, I did have a legal background too from UCLA Law School. So I looked into my own Blue Cross Blue Shield contract and I saw that they said that they wouldn't pay for cosmetic surgery, they wouldn't pay for experimental medicines, but there was no price cap on what they would pay for something that was, quote unquote, medically necessary treatment. So I just really did the math. I said, okay, what net present value do I need to satisfy investors that they'll get a good return on their investment? How many patients are there? Divide the number of patients into that amount of money, what must be the cost of the drug? It turned out it had to cost $100,000 a year. At that time, there was no medicine costing $100,000 a year. And I think many people in our little biotechnology industries should maybe thank me for the fact that now there are a couple dozen medicines that cost more than $100,000 a year. But fortunately, that means that there are thousands of people with orphan diseases that would have never got their diseases treated. And biotech companies that treat orphan diseases are now amongst the most profitable and successful segment of the pharma industry.

I think it would be pretty much equivalent. People are always disbelieving until something is created for them. And there's this great saying by Arthur C. Clarke, the famous science fiction writer who discovered geostationary satellites. He said, if a wisened expert in the field, which are either VCs or the consultants that VCs hired to advise them, tells you something is impossible, they are almost certainly wrong. And if a wisened expert in the field tells you something is possible, they are almost certainly right. So these VCs, they're risk averse, and it will always be difficult. What's needed is to be practical, to make a prototype, to show people that this is practical, to be persistent, to be ready to march through 99 no's for that one yes, and to finally communicate your idea in a stepwise sensible fashion. And entrepreneur nerds are doing this today, they did it 20 years ago, actually they did it 200 years ago.

Rebecca, I'm really inspired by the exponential growth in information technology. Ray Kurzweil, the author of 'Age of Spiritual Machines' and 'The Singularity is Near', he's on our board of directors. And he has actually had a very successful track record in terms of predicting new inventions that will come about as a result of the exponential growth in IT technology. So it seems to me that the natural extension of Ray's ideas is that we will get to a point where software is so sophisticated and so capable that it will do its best to persuade us that it's conscious. And at the beginning, everybody will say, 'No, you're not conscious, you're just a smart puppet.' Hackers and makers and software companies and DARPA contractors will work harder and harder to make this consciousness more and more affable, more feelable, more real. And there will come finally a tipping point when you can have a group of psychologists that would spend a year with a software mind, and after a year that group of psychologists say, 'This software mind is as human as any person, although it lacks a body.'

Sure, absolutely. There's the possibility of crazy software minds. In fact, about 1% of all human births result in a psychological condition where people just have a lack of affect and even an antisocial behavior type of disorder. But fire was used to burn people, and they used fire to say, 'If you're telling the truth, then your hand won't burn when you put your hand in.' That was just horrible, horrible things done in the name of fire. But our whole society is based on fire. So I believe that the answer to the negative face of technology is to emphasize the positive face of technology, build better and better positive technology, create safe harbors for positive technology, because ultimately it will take a good software mind to catch a bad software mind.

Well, one very practical way that we're doing that right now, Rebecca, is in a partnership between our company United Therapeutics and Craig Venter's company Synthetic Genomics and Human Longevity Inc. We're using the biological-to-digital and digital-to-biological expertise of Human Longevity Inc and Synthetic Genomics to decode the pig genome. The pig genome results in organs like hearts, lungs, kidneys, and livers that are the best size match for human organs of any animal in the animal kingdom, even better matched to us than chimpanzees, without the associated ethical issues. So we believe that our partnership with Human Longevity and Synthetic Genomics will allow us to make enough tweaks and changes in the pig genome using digital-to-biological and biological-back-to-digital techniques that we can create an unlimited supply of transplantable organs, starting with the lung and the heart. This means that when people reach the point of having end-stage heart disease or end-stage lung disease, instead of just calmly marching off to death, they can say, 'Hey, when my car needs a new engine, I replace it.' We have B-52 bombers that are still flying, built in the 50s, but so many parts have been replaced they're older than even the grandparents of some of the pilots. So we could replace our lungs and our hearts with an unlimited supply of transplantable organs, thanks to being able to walk back and forth from digital to biological.

Because still, people with pulmonary hypertension, and our daughter Genesis, they all take a handful of pills each day. Most people with pulmonary hypertension ultimately progress, their disease keeps progressing through the treatments, and the only absolute cure is a lung transplant. But as most people know, people end up rejecting the lung transplants. It's a similar situation with pulmonary fibrosis, cystic fibrosis, emphysema, even lung cancer. So what we believe we can do is by tweaking the pig genome using these digital and biological techniques, we can create a lung which is tolerable by the human body, so the patients will not have to take lifelong immunosuppressants, and replacing one's lungs will be a true cure for end-stage lung disease.

I believe the future for humans is taking the quality of life that we've built up among most of the people in the West and spreading that over more and more people. I'm a huge believer in Peter Diamandis's thesis of abundance. I think that positive thinking, hope, and belief in working hard in science and technology can overcome any problem. Yes, we have horrible problems: the Ebola pandemic in West Africa, the ISIS nihilism in the Middle East. However, if you look back in history, things were always worse. I mean, there were times when people just marched across all of the Near East and Europe and burned everything, burned people in their homes, and nobody could do anything about it, would do anything about it, would even know about it in distant parts of the world. We have now built a global network of mankind, which is our satellite and internet communications capabilities. We're creating a global empathy through meetings such as this Zeitgeist and journalists like you who can channel our stories to hundreds of millions of people. And we're building a global capability to get together and do something with organizations like the World Health Organization. The United States now sending thousands of soldiers into West Africa to try to contain this pandemic, many countries working together to contain ISIS. All of this is signs that we are, you know, legions away from utopia, but things are getting better all the time. And I believe the future will continue to get better and better.

That's an amazing question, but I would say... you really caught me. I caught me on that one. But I would say actually the story of the individual who is facing something that seems to be an absolute boundary, life or death, using their capabilities of communication, like the presentation yesterday with the young man dying from a sarcoma and using his ability to make beautiful music to marshal the talents of people throughout the world to come up with cures for those diseases. I think those are the stories that need to be told. That there is no limit to channel the great spirit of Audrey Hepburn: 'Nothing is impossible because the word itself says I'm possible.'