What it’s like to learn you’re going to live longer than you expected

Imagine if you knew, from your earliest awareness, that you were unlikely to live beyond your 20s. You would finish high school, but what about college? Would you allow yourself to fall in love? How much would you invest in a future that you might never see?

When Molly Pam was born in 1988, these impossible questions were the reality for people with cystic fibrosis, a genetic disease that leads to an early death from lung failure. But Ms. Pam is now 34. And thanks to a new drug that has revolutionised the treatment of this disease, she will likely live to celebrate her 40th and even 50th birthdays. Her life expectancy has shifted drastically within her lifetime.

It is a remarkable and complicated experience. And as science races forward with gene therapy and targeted cancer treatments that promise to turn terminal disease into chronic illness, it is also a lesson in what might be ahead.

Ms Pam was diagnosed with cystic fibrosis a few weeks before she turned 10. As a middle schooler, she went on a deep dive — discovering the memoir of a man with the disease who was amazed to live past 25. Was this what awaited her? Even though she followed her family legacy and went to college at Stanford, then moved to Manhattan, that question lingered.

As did the reality of her limited lung function. Early on after college, she realized that her body would not sustain full-time work. Still, she was able to build a life. She went to culinary school and built a career as a freelance chef. She fell in love and married.

Then, as she approached her 30th birthday, everything started to unravel. Her lung function plunged under multiple drug-resistant infections and eventual lung collapse. She stopped working and moved to California so that she and her husband could be near her family and her sister’s children. They would not have children of their own, that much was clear. The evaluation for a lung transplant began.

But as she prepared herself for the possibility of a transplant, Ms. Pam started on a new drug therapy that targets the underlying cause of her disease. And now, three years later, she is healthier than she had been in years. Her lung function has doubled. Possibility expands once again.

Since its approval in 2019, the drug therapy, Trikafta, has been heralded as a game changer for a majority of those with cystic fibrosis. The disease is caused by defects in a protein that affects the body’s cells and tissues, leading to mucus that accumulates in the lungs and other organs. Trikafta, a combination of three drugs, helps the faulty protein function more effectively. Without the thick mucus and infections and scarring that come with it, patients can ideally avoid the inevitable respiratory decline that characterizes life with C.F.

Some people have genetic mutations that do not allow them to benefit from Trikafta, which targets the most common C.F. mutation. Still, many of those born with the disease today can expect to live into their 60s — a prognosis that will most likely continue to improve during their lifetimes. They will grow up with an entirely different outlook, with a disease that for many will be chronic and manageable. But for those who are Ms. Pam’s age or older, many of whom have made decisions about career and family based on the expectation of a shortened life span, this staggering scientific advance has left them facing existential questions.

One woman in her 40s with cystic fibrosis withdrew her retirement money some years back to “live in the moment,” she said, because she assumed she would die before any traditional retirement. She is alive now, but she is adrift. There are others who never went to college or planned for a career because they and their parents believed that their health would make such a decision unnecessary. Internalizing the prognosis they had at birth, they never invested in adulthood, and yet here they are, adults, with years in front of them. Those who were sick enough before Trikafta to find themselves on the verge of transplant have been pulled back from the brink. But now what? And can they trust that this reprieve will last?

“Many patients are continuing to adjust to the impact of this transformative era on their lives,” said Dr. Manuela Cernadas, a pulmonologist who specializes in cystic fibrosis at Boston Children’s Hospital and Brigham and Women’s Hospital, where I work. Even as she celebrates the improving pulmonary function that so many of her patients have enjoyed since starting Trikafta, she thinks of the patients who died just before the drug was approved or who received lung transplants only to die nonetheless. They were so close, but there is so much randomness to this kind of luck, so many what-ifs. Dr. Cernadas is aware, too, that none of this is simple for patients who have lived their lives under the shadow of an early death.

This article originally appeared in The New York Times.