Martine Rothblatt wants to transform the way we transplant organs and she doesn’t take no for an answer. When her daughter came down with a rare, life-threatening illness several decades ago, Rothblatt started researching the illness, and founded a company to develop a treatment for it.
In the mid-1990s, Rothblatt’s daughter Jenesis developed pulmonary arterial hypertension, a rare condition in which the arteries in the lungs and the heart become constricted, making it hard for blood to flow through them. Doctors gave her a grim prognosis: Without a lung transplant, she had roughly three-to-five years to live.
Rothblatt found that unacceptable.
Although she had no formal medical background (she was trained as a lawyer), Rothblatt decided to take matters into her own hands.
She started by founding a nonprofit foundation geared at finding cures for pulmonary arterial hypertension, called the PPH Cure Foundation. Then she created a pharmaceutical company called United Therapeutics Inc., which designs drugs and machinery to treat the condition. (According to UT’s website, they’re also researching treatments for cancer and viral illnesses.)
Rothblatt wasn’t starting entirely from scratch, of course. In fact, she’s had a history of creating and leading highly successful companies. Before entering the biotech industry, Rothblatt created the satellite radio service Sirius XM and the satellite navigation company GeoStar. Last year, she was the second highest paid female CEO in the United States, earning a total of $US31.6 million. As a transgender woman, she has also been a strong advocate for transgender rights.
Overcoming the odds
When Jenesis received her diagnosis, a medicine for treating pulmonary hypertension did exist, which worked by dilating the collapsed arteries in the lungs, but it had to be delivered by intravenous or subcutaneous injections.
The only “cure” for her disease was grim: she needed a new lung. And only slightly more than half of all patients who get a lung transplant survive more than five years.
Rothblatt’s company, United Therapeutics, first developed a drug called Remodulin, delivered by injection or a pump. Because many patients experienced intense pain at the injection site, her company collaborated with the University of Rochester to create a patch to treat the skin pain. Jenesis, now 31, has been taking the drug and wearing the patch ever since.
United Therapeutics later developed a pill version of the drug, called Orenitram (Martine Ro. spelled backward), which is now FDA-approved.
But it’s not a cure, and Jenesis still needs a new lung.
The organ shortage
Sadly, new lungs are hard to come by. About 21 people die in the US every day because there aren’t enough organs for transplant, according to the US Department of Health and Human Services. And the problem isn’t just that there aren’t enough donors — more than 80% of lungs are discarded because they aren’t in usable condition.
So her company is developing machines that can fix up and preserve some of the discarded lungs, so they can be used for transplants.
In partnership with the Silver Spring-based company PERFUSIX, Rothblatt’s company developed ex-vivo lung perfusion (EVLP) machines, a fancy term that stands for a machine that sits outside the body and supplies the lungs with a consistent supply of fresh blood.
These machines act as an artificial body, free of the toxic molecules released by a dying body or the drugs used to preserve it.
Once harvested for transplant, organs remain viable for a limited amount of time, so long as they’re kept chilled. This depends on the organ, but lungs typically last about four-to-six hours outside the body. But the EVLP machine effectively “resets the clock” on transplantation, Rothblatt told Business Insider. The device is now approved by the Canadian equivalent of the FDA and is undergoing clinical trials in the US.
While Rothblatt understands that her daughter’s struggle to survive is very real, she also belongs to a school of thought called transhumanism, the belief that all humans will eventually transcend the limitations of our current biological form.
She encourages people to think of it this way: Old aeroplanes and antique cars still function on replacement parts, “so why can’t we have an unlimited supply of replacement organs for the body?” she said at a conference this week hosted by the Defence Advanced Research Projects Agency (DARPA) in New York.
In 2004, she launched the transhumanist Terasem Movement Foundation, which supports the idea that it will eventually be possible to upload our minds to a computer or an artificial body in order to achieve digital immortality.
But even today, technology has enabled humans to overcome biological limitations — such as Rothblatt’s daughter’s illness — that would have seemed impossible a few decades ago.
“To me, we are already transhuman,” Rothblatt said.
Beyond human organs
Once her company has mastered the revival of human organs, Rothblatt plans to find ways of transplanting animal organs, tissues or cells into humans, a practice known as xenotransplantation. (These organs are often structurally and functionally similar to human ones, but there is concern about the risk of infections from the donor or rejection by the recipient’s immune system.)
The first animal-to-human organ transplants were performed in the 1960s, but many of the patients didn’t survive long because their immune systems rejected the organs. Pigs have been proposed as a better source of organs, and pig valves are already widely used in humans.
But Rothblatt doesn’t plan to stop there. Once we can use animal organs, she aims to find ways to remove the animal cells from the organ (a process known as decellularization) and replace them with the human patient’s own cells. Decellurized tissues and organs have been used in a wide array of applications in both animals and humans, from skin burns to bone grafts.
Ultimately, Rothblatt plans to create 3D-printed organs from scratch that can be seeded with the patient’s cells. This process would entail printing the “scaffold” of an organ out of materials compatible with the body, isolating cells from the patient and converting them into the desired tissue type (bone, muscle, etc), and growing these cells on the artificial scaffold.
In theory, you could then transplant the new printed organ into the patient’s body.
While ambitious, these goals aren’t outside the realm of scientific feasibility. While the idea of creating whole organs from scratch remains science fiction at this point, 3D-printed tissues are already being used in medicine, and patients’ cells are being used to grow replacement tissue.
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