In 1990, 6-year-old Jenesis Rothblatt's lips turned blue. The little girl gasped for breath, could not climb stairs and was soon in the intensive care unit of Children's National Hospital in Washington, D.C.Um... yes. That happens sometimes.
The disaster began a transformation for Jeni's father--and a change in the way drugs for rare diseases are developed. Martin Rothblatt, a lawyer and businessman, turned his childhood passion for outer space into founding a series of companies that made him millions of dollars. First came GeoStar, a GPS-based navigation system, in 1986, then an early satellite radio company called Worldspace and finally Sirius Satellite Radio ( SIRI - news - people ) in 1990. In 1994 he underwent gender reassignment surgery and changed his name to Martine. Now a she, 55, Martine remains married to Jeni's mom.
When Jeni became sick, Martine felt useless. "I was an expert in satellites, and I didn't know anything about medicine," she says. Jeni had pulmonary arterial hypertension (PAH), a rare and fatal disease that is caused when the artery between the heart and lungs is damaged. With 200,000 cases worldwide, PAH was too uncommon to win drug-development money. Martine Rothblatt sold her telecom stock and started a $3 million foundation to fund research, but three years went by without any progress.As you do when your child's life is at stake.
Everybody else in biotech had a Ph.D. or an M.D., so Martine decided to get one, too. The late John Vane, a 1982 Nobel laureate in medicine who was advising United Therapeutics, hooked Rothblatt up with bioethicists at the University of London. At the time there was a raging controversy over efforts by Novartis to create genetically modified pigs whose lungs and other organs could be transplanted into people. Some virologists worried that retroviruses in the pigs could be transmitted to people via the transplants. This infuriated Rothblatt, because those lungs could save the lives of PAH patients who had run out of options. She wrote her thesis on how the research could be ethically conducted and earned her Ph.D. in bioethics in 2001. Pig-to-human organ transplant research remains banned.*SIGH* She tried. And yes, after transition, you have so much more ability to deal with things. Need a PhD? Go get one. Even when under the stress of knowing your child is dying, and depending on you.
It's no easier after transition, that's for sure. It's just that your capabilities expand.
At first telling investors that she was going to charge a high price for a treatment for people like her daughter gave Rothblatt "a sour taste" in her mouth. But the availability of treatments changed the nature of PAH. There were 75 specialists in PAH in the U.S. when Jeni got sick. Now 10,000 doctors treat it. Pfizer, Actelion and Gilead Sciences ( GILD - news - people ) all market pills for PAH. The disease is still invariably fatal, but patients are living much longer, in some cases decades after diagnosis.Look, our peculiar neurology gives us unusual talents - some of us at least. Just as we have unusual difficulties. To those that have been given much, much is expected, and it would only be ... right? balanced? correct? just? appropriate? all of the above? to put whatever talents we have to good use for the species. A Duty of Care, if you will. "Radically extending human life span" is something that should be doable. And worth doing.
In 2004 Rothblatt and her wife, Bina, cofounded a nonprofit, the Terasem Movement, dedicated to promoting the idea that science can radically extend the human life span. She says that medicine will allow human beings to "transcend commonly accepted limits" of human life and calls futurist Raymond Kurzweil, who promotes similar ideas, "the smartest guy on the planet." Kurzweil also serves on United Therapeutics' board of directors.
Jeni Rothblatt, now 26, doesn't disclose what medicines she takes, but she is doing fine. She works at United Therapeutics, setting up town halls in the online virtual world of Second Life, where Martine can interact with all 410 of her employees, whom she calls Unitherians.
Hmmm... I must contact my co-author. Wonder if our work could be useful to them...
She's a gal after my own heart. Just a bit brighter than me though.