United Therapeutics CEO Martine Rothblatt talks about pig cloning, lung transplants and electric … [+] helicopters at the Forbes Women’s Summit on Tuesday.
From cofounding Sirius Satellite Radio to launching a biotech company to find a cure for her daughter's illness, Martine Rothblatt has had so much career success that any one of her accomplishments would be a crowning achievement for another entrepreneur. "I always try to convert a moonshot into an earthshot," Rothblatt told hundreds at the Forbes Women's Summit on Tuesday.
As the CEO of United Therapeutics, which now sells five FDA-approved pills for pulmonary arterial hypertension, Rothblatt is constantly innovating. Her company has been experimenting with pig cloning and genetic modification to create organ transplants the body doesn't reject. She's also now figured out how to save the some 80% of donated lungs that end up unusable. On the side, Rothblatt is also working on the first electric helicopter and, as a transhumanist, Rothblatt experiments with robots. Her Terasem Movement Foundation helps anyone "upload" their brains to computers to one day prepare for "mind clones" to live in the technological world.
Forbes sat down with Rothblatt to hear about her effort to save lungs awaiting transplants, how her company became the largest pig cloner in the world, the moonshot she still wants to work on and advice for launching a new company.
FORBES: Given all that you’re doing, and the way that your career has evolved, given the audience that we have today, what would you say, right now, is your passion and what you're most focused on?
MARTINE ROTHBLATT: My next project that gets me most excited is creating an unlimited supply of transplantable organs. In my business at United Therapeutics, I've come across too many patients who unfortunately have passed away while waiting for an organ transplant. To give you a horrible but, on the other hand, perhaps a helpful image of how bad the organ transplant situation is, the number of people that die waiting for an organ transplant is equal to the number of people that would be killed in a jumbo jet that crashed every single day, 365 days a year. It's a ridiculous total. My feeling is that if we can keep buildings going essentially forever, by continuing to repair and place their parts, if we can keep cars and planes going essentially forever—today there are B-52 bombers being flown by the grandchildren of the people that built those planes originally, by having replacement parts—we should also, in this day and age of genetic engineering and regenerative medicine, be able to make replaceable organs, something that could keep going longer and longer.
FORBES: There's a huge number of donated lungs that doctors can't use for transplants. You figured out a way, at least on a small scale right now, to basically take them out of the garbage, so to speak, and make them workable. Tell me what you're doing to save those lungs.
ROTHBLATT: I always try to convert a moonshot into an earthshot. The moonshot is to have an unlimited supply of transplantable organs. The first thing I ask myself is: How can I make that an earthshot? I knew that more than half of Americans are kind enough to donate their organs upon unexpected death. I wondered, why are there only 2,000 lung transplants done each year when hundreds of thousands of Americans died each year? I found out that the vast majority of organs that people donate are not usable at the time of their death. Even among the just 1% of organs that are usable at the time of the person's death because they've died in a hospital and they died without cancer or infectious disease, those sorts of things, that of that 1%, about 80% of the lungs were thrown in the trash and were deemed unusable for transplantation. I think, oh, my God, this is a horrible waste. This is a disrespect to the patients who wanted to donate their organs and to the families that wanted their loved ones to continue. I investigated the reason why. It turns out that the lung is a very fragile organ. It's got a very thin membrane. Upon the body dying, fluids begin to invade the lung and fill up the lung with mucus and with liquid. By the time a transplant surgeon gets ready to take that lung, most of the time it's filled with mucus and fluids, and it's no longer usable within somebody else who would, of course, be on their last legs needing a lung transplant. I thought, why don’t you take these lungs out of the dying body and put them in a kind of an artificial body where they have none of the toxic chemicals associated with the processes of death? I said we'd build a real-time network that we could show transplant surgeons throughout the country in real time exactly how good shape those lungs were by putting cameras in the end of bronchoscopes so we could do what the surgeon wanted to show down different branches of the lungs. Do real-time X-rays. Lo and behold, with the benefit of a very good team of scientists and engineers, we now do this every day, 24 hours a day, 365 days a year. We’ve already saved hundreds of patients' lives with this grabbing procedure called ex vivo lung perfusion. That's the earthshot that's kind of helping me, step by step, get to the moonshot.
FORBES: And tell us what you're doing with pigs now.
ROTHBLATT: Aside from the shortage of lungs, there's a horrible shortage of kidneys and of hearts. In fact, the largest number of people on the organ wait list are for kidneys, and the largest cause of untimely deaths are heart attacks and strokes. We have worked together with the team that decoded the human genome. We asked them to decode the pig genome and to decode it at such a high level of resolution that we would be able to figure out which parts of the pig genome gave rise to hyperacute rejection when a pig organ was placed into a human. We have identified those genes. Surprisingly it's only about ten genes that cause the pig's organs to be viciously rejected when placed into a human body. We have now modified those ten genes. We’ve replaced those ten genes with humanized forms of the genes or just knocked them out of the pig genome completely. We are in the phase of animal testing of these what we call xenografts. Xeno means like from a pig or from another animal. Xenografts to create an unlimited supply of transplantable kidneys, hearts, livers and lungs for everybody. I believe very confidently that during the 2020s—which like, whoa, are in 24 months, right?—during the 2020s, there will be thousands of people whose lives have been saved with xenotransplantation.
FORBES: The thing to me that is so amazing is that you actually got into the healthcare field kind of happenstance. Your daughter Jenesis was 8 years old when you got a really horrible diagnosis and you were told that she might not live. You took that as a challenge and threw yourself into healthcare and figured out a way to save her and thousands of others. Can you tell us a little bit about that story?
ROTHBLATT: There is nothing worse than being told that your daughter is going to die. I can't really say that I even intellectualized it to the point of, like, this is a challenge. It's just, like, what can I do to save my daughter? I did at first try to find other doctors researching this area and to donate money to those doctors. It was not producing any results. There were no medicines approved by the FDA. I just said, it's either I put my whole previous satellite communications life aside and cure Jenesis, I will find a way, or she was going to die because all of the previous people with this illness had died. They died within three to five years, very, very quickly. Again, with the good blessings and the good efforts of more people than I could ever count, we were able to create a village of people committed to developing new treatments—first treatments—for pulmonary arterial hypertension. Within five years, we got the first FDA-approved medicine for pulmonary hypertension. We were able to get Jenesis, our daughter, on the medicine. Thousands of other kids, it's mostly girls, like 70% female have this condition. When I started, only 2,000 people in the U.S. had pulmonary hypertension because everybody who got it died. There was never a time when the numbers increased. They got it, they died. Today, there are 40,000 people in the United States with pulmonary hypertension, the vast majority of them living normal lives. We've made a medicine that can keep people alive indefinitely.
FORBES: Let's shift gears to gender. Martine went through sexual reassignment surgery more than two decades ago. You may be the only person in this room who has the perspective of having been a male in the boardroom and a female in the boardroom. I'm curious, what can you tell us to help us understand?
ROTHBLATT: Fortunately, there were a few motion pictures that gave me clues of how that would work. It's been a fascinating journey looking back. It's true that while I've always been a woman, I had my womanhood deeply closeted inside male trappings for more than half my life. When there was disrespectful instances of, I want to say, locker-room talk or whatever, I wasn't one [to speak out] when I was closeted as a male, because you can't go up and say, hey, that's wrong. Then, as I came out of the closet, transitioned openly into my authentic self, things that I had seen from a woman's perspective I was able to articulate. For example, I remember, just a month ago, I was in a meeting with a bunch of xenotransplantation scientists and engineers working on the xeno-kidney. There was a reference to the work of an absolutely incredible scientist in our midst today, Luhan Yang. She is the chief science officer at a company called eGenesis. She's the first person in history to have made 26 simultaneous genetic edits in any animal. In her case it was a pig. These 26 simultaneous genetic edits got rid of all of the viruses in a pig that could conceivably infect humans if you put the organs of the pig in a human. Previous people had knocked out one gene, 2 genes, 3 genes. 26, everyone said it was impossible, but Luhan Yang did it through a brilliant scientific breakthrough. This guy who was a transplant surgeon, he's giving a PowerPoint to the group of other scientists. He said, "Of course, we're not doing anything about the viruses that girl genetically removed." It hit me like a slap in the face. I'm that girl. We're all that girl. I'm going to leave his name out of it, but I said, "You know, I'm sorry, but what you just said is wrong. You can say 'that scientist,' you can say 'Doctor Yang,' but it’s not 'that girl.'" Of course, you could hear a pin drop because it was kind of like a #MeToo-ish moment. He said, of course, "I’m sorry. I didn't mean anything by that." I’m just trying to use this as an anecdote that you do see things differently from a woman's perspective and a man's perspective. I feel honored and blessed to have a chance for the second half of my life to speak up for myself.
FORBES: Have you made any mistakes over your career?
ROTHBLATT: Yes. The biggest mistake is to not do anything because you're afraid of making a mistake. I actually think mistakes are good because mistakes show that you're trying to do something. The person that makes no mistakes is making the biggest mistake because they're just standing still and doing nothing. I say at our company all the time that it's not a problem. People say, "I’m sorry, I made a mistake." No, there's nothing to be sorry about that you made a mistake. It's good that you made a mistake. The important thing is to see what can we do about this going forward. How do we change our behavior going forward from the point where you made the mistake? For example, those of you who are MBAs, probably quite a few, it's fundamental to the entire concept of net present value. What you spent in the past is completely irrelevant. Don't count it, don't think about it. All that matters in net present value are expenses and revenues going forward. The same kind of thing with mistakes. A mistake is a past thing. Okay? Don't worry about the past. Just ask yourself: How can I do better going forward? That's like a general thing.
FORBES: What advice would you give to us?
ROTHBLATT: As the CEO of a publicly traded company—and we went public in 2001 at the equivalent of like $3 a share; now we’re over $100 a share—I still say that the really big mistake I made was going public. I have learned over the years that all of the benefits that our company gets from being public, I now know ways to accrue all of those benefits to our stakeholders, to our employees, to everybody as a private company without all the bureaucracy and the legal frustration that's associated with being public. If you're out there listening, stay private.
FORBES: You constantly have new moonshots. Can you tell us about a moonshot that you haven't shared with others?
ROTHBLATT: I think this is another earthshot. We have figured out a way—there's a broad spectrum of stem cells, there’s a particular type of stem cell that’s not a pure stem cell. It's called an endothelial stem cell, which means that it's a stem cell kind of on its way to the becoming the cell that lines your blood vessels. Of course, you've got gazillions of them. We figured out a way to take some blood, spin it down so we get these kind of proto-stem cells out of their blood and then make a genetic modification on those stem cells to correct something that, in this case, is causing pulmonary hypertension, and then send those cells back to the patient and reinject them. Those cells, with one beat of the heart—every time your heart beats, a bunch of blood comes to your lungs. With two or three heartbeats, all of your blood is going through your lungs. These tiny cells, they get trapped in parts of the lung, the pulmonary arterials. They get pressed. When they get pressed, they express the gene product. We found a way to turn your very own cells into your very own little medicine factories. That's now in phase three trials. I believe it will be a complete cure for the illness that afflicts my daughter and 40,000 other people.
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