A New Breed Of Organ Donors
Bloomberg Businessweek|August 02, 2021
Martine Rothblatt’s first multibillion-dollar medical business came from a desperate need to save her daughter’s life. Decades later, she’s racing to protect her again
By Adam Piore

I went right ahead and did research on my own and found out what he was saying was true,” Martine Rothblatt would later recall. “There were no medicines for it. Everybody did die.”

A doctor at Children’s National Medical Center in Washington, D.C., had just told Rothblatt and her wife, Bina, that the couple’s youngest daughter, 9-year-old Jenesis, had a rare medical condition that likely gave her three years to live. The arteries between Jenesis’s heart and lungs had narrowed, choking off oxygen and placing an unsustainable burden on her heart as it struggled to send blood through her thinning blood vessels, like trying to push water through a hose with a kink in it. The condition, known as pulmonary arterial hypertension, was progressive, and there were no approved treatments, short of a lung transplant— almost unheard-of in children.

Rothblatt set out to make one. On the cusp of 40 in the mid-1990s, she was a wealthy, pioneering aerospace attorney and communications entrepreneur. Her startups included the satellite navigation company GeoStar and the company that would later come to be known as SiriusXM Satellite Radio. On a personal level, Rothblatt was in the process of transforming into the person she’d always been meant to be. Within months, she would undergo sex reassignment surgery and come out to the world as Martine.

But Rothblatt spent the nights after Jenesis’s diagnosis in the hospital’s basement library, studying the molecular biology of her daughter’s disease in medical journals. She looked up unfamiliar terms and concepts in textbooks, tracked down obscure articles mentioned in cryptic footnotes, and grew convinced that technology could solve her daughter’s problem. “I just felt like I had no choice,” she would later say. “My only purpose in life now was not to help move to the stars with satellites and stuff like that. Instead, it was to save Jenesis.”

A pharmaceutical company in North Carolina had what Rothblatt considered a promising chemical compound sitting unused on the shelf. When the company’s lawyers refused to license it to an individual, Rothblatt started a biotech company, assembled a team of scientists, and persuaded the pharma company to take her money. Then, using her windfall from Sirius’s recent initial public offering, they developed the compound into a workable drug, took it all the way through clinical trials, and won approval from the U.S. Food and Drug Administration. The medication saved Jenesis and tens of thousands of other people.

Today, Rothblatt’s biotech company, United Therapeutics Corp., is worth about $8 billion. More than 100,000 people around the world rely on it to produce the lifesaving medicine, called Remodulin, and Rothblatt is the highest-paid female executive in the U.S. At age 36, Jenesis is working for the company, too.

And yet Rothblatt has always known the work was only half done. Remodulin is a treatment, not a cure, and it’s tough to be certain how long it will hold off the disease for each patient. At some unknown point, Jenesis’s lungs might still fail. The only permanent solution remains a transplant, and in a good year, not even 1% of the patients who need lung transplants receive them. In 2019 about 250,000 people died of end-stage lung failure, too far down the transplant list or otherwise unable to get one.

So once again, Rothblatt has vowed to solve the problem threatening her daughter’s life. This time, it’s the global organ shortage. “I did the math,” she says. “So I decided to change the math.”

Backed by United’s nine-figure annual research and development budget and about $2 billion in cash, Rothblatt and her team have been quietly working to create manufacturing techniques that don’t require human donors. Through acquisition and collaborations, what began several years ago at United’s headquarters in downtown Silver Spring, Md., as a small effort to solve the long-term organ shortage problem has grown into a network of small labs and research facilities scattered across the U.S. that are experimenting with possible solutions.

In Jacksonville, Fla., a team of United engineers at a Mayo Clinic facility is testing a method of lung perfusion, a technology that can help assess whether damaged donor lungs are still be usable for transplantation. In Manchester, N.H., and Research Triangle Park, N.C., United has hired teams of scientists to try to figure out how to seed decellularized animal organs with human stem cells, a first step toward 3D-printed organs using patients’ own cell samples.

Now Rothblatt’s company is getting ready to move forward with perhaps the most sci-fi of these efforts: growing genetically modified, human-compatible organs in pigs.

At a Rothblatt-designed facility at the University of Alabama at Birmingham, some of the people who cloned Dolly the Sheep have been helping to create a small army of swine with custom-edited genes. Each pig will carry a minimum of 10 genetic modifications that scientists say will make their organs, already comparable in size to those in the human body, acceptable to human hosts. Kidneys are the first goal, but they won’t be the last. Instead of using a pig valve in a heart transplant, which has been a common procedure for decades, why not the whole heart? Or the lungs that might someday save Jenesis’s life?

Rothblatt’s team is tackling a challenge that has frustrated some of the world’s top scientists for decades. The human immune system is notoriously fickle when it comes to accepting organs that come from another human, let alone a pig. “An organ must interact with a body along hundreds of biochemical links, whereas a drug needs to interact along only a few,” Rothblatt says. “The biochemistry is orders of magnitude more challenging.”

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