It Takes Many Villages

Loren Eng didn’t set out to run a non-profit.

That changed when her daughter, Arya, was diagnosed with spinal muscular atrophy (SMA), a severe, progressive neuromuscular disease, when she was about two years old. Arya was a beautiful baby and developing perfectly, but soon Loren recognized there was something wrong: Arya wasn’t walking properly, and soon, she was losing function. The diagnosis was heartbreaking, as Loren learned that SMA was leading genetic killer of young children and there were no treatments available at that time.

The more she researched, the more she became determined to do something to help her child. The space had many challenges, including a lack of awareness and funding. No one, it seemed, had heard of the disease. That said, there were positives, too: SMA was a druggable target and the genetics were unique. Loren saw an opportunity: she could bring a new perspective to the space through her investment banking and business development background. She decided to start the SMA Foundation with express purpose of trying to develop drugs to treat SMA, and to do anything to be a catalyst to achieve that goal.

She approached this as any other business problem – the Foundation made a list of potential partners, created an investment banking-style roadshow, and went out on the road to pitch them. With Loren at the helm, the Foundation wanted to show they could think like their partners and speak their language, showcasing the science and that it was possible and profitable. Deeply understanding the patient experience was critical, too, because how could you make a drug without knowing the patient, how they live and what challenges them, and how to measure the success?

When they met PTC, her first impressions of the company were the extremely high value placed on the patient. Loren saw that patient interaction in the drug development process was integrated early, frequently and deeply. She saw their passion, and because it was a small biotech, the Foundation would be able to work directly with the decision makers. The SMA Foundation and PTC began to work together to develop a treatment to target the underlying cause of SMA.

It was a perfect match that became a collaboration between PTC and the SMA Foundation that eventually expanded to include and Roche, who was also committed to ensuring patient voices were part of the process. Loren knew that starting a project with so many entities might be challenging – and sometimes, it was tough – but the collaboration was a success, and the diversity of thought was part of that success.

As the saying goes, it takes a village to raise a child. But in Loren’s words, “it takes hundreds of villages and cities and towns working together to develop a drug.” The Foundation had that cohesiveness of many villages working together to improve the lives of children living with SMA. After many years of collaboration, research, development, and testing, the first oral treatment for SMA was approved. There was an amazing sense of joy and pride of their accomplishment, and to know they had that kind of transformative impact on SMA was momentous and fulfilling.

Loren feels that it’s important to know suffering to make you a more empathic person, that to be able to overcome hardship gives you confidence that you can overcome anything. For her daughter Arya and other SMA patients, that resolve and grit is part of the patient experience. But she believes that if you channel those hardships with the right support, kindness, and love from people around you, it can propel you to do great things and pay it forward. For Loren, what PTC has done enables Arya to pay it forward.