Engles Advance Rare Disease Research

Engles Advance Rare Disease Research

The Engles family

This story appears in the Winter 2016 issue of the Life.Giving.Breakthroughs. donor newsletter.

Like most 4-year-olds, Lucy Jane Engles is full of life. That is why her parents, Gregg and Molly Engles, were shocked to discover that their youngest of four daughters has a rare genetic disease called Tuberous Sclerosis Complex (TSC). This condition causes benign tumors to grow in the brain and other vital organs, often resulting in seizures and a host of other serious medical issues.

“Our concerns about Lucy Jane’s disease were heightened when we learned how rare it is,” says Molly.  “With only two babies born with TSC on any given day in the United States, we wondered whether enough research was being done to find adequate answers. That concern catalyzed our decision to seek out extraordinary scientists who need funding to accelerate their work.”

Shortly after Lucy Jane’s diagnosis, the Engles, who live in Colorado, were introduced to TSC researcher Lisa Henske, MD, of Brigham and Women’s Hospital (BWH). Henske helped discover the mutations in TSC genes that cause a form of lymphangioleiomyomatosis (LAM), a rare, destructive disease that targets the lungs in women of childbearing age.

“In less than 15 years, the LAM research community has discovered the genetic cause of LAM and developed a treatment for a disease that previously could not be treated,” explains Henske. “This pace of discovery is unprecedented in lung disease.”

As a result, the outlook for patients like Lucy Jane has radically improved. But even with this progress, the Engles learned additional funding could build more momentum for Henske’s groundbreaking work. In honor of their daughter, they have given $5 million to establish the Lucy Jane Engles TSC/LAM Medical Research Program at BWH.

The funding will enable Henske’s team to bridge findings from their research and clinical trials with state-of-the-art, multidisciplinary clinical care for women with LAM.

“Thanks to the Engles’ groundbreaking gift, we are poised to continue on“We’re not interested in incremental advances,” Henske adds. “We are focused on major breakthroughs that will substantially improve outcomes for women with LAM.”

The strength of the research has helped the Engles overcome their initial fears, and has given them optimism for their daughter’s health and future well-being.

“Lucy Jane’s diagnosis has given greater meaning and urgency to our philanthropy,” says Gregg. “We hope that through our involvement with Lisa’s research and the TSC Alliance we will find additional promising research to invest in for the benefit of all those who suffer from TSC.” a fast track to new treatment options and, ultimately, a cure,” Henske says.