The Legacy of Angels Foundation Supports Krabbe Disease Research

The Legacy of Angels Foundation has awarded more than $900,000 to the Institute for Myelin and Glia Exploration at the Jacobs School of Medicine and Biomedical Sciences. The funding will support reserach at the institute that focuses on discovering effective treatments —and, ultimately, a cure—for Krabbe disease, a progressive and fatal neurologic disorder that usually causes death before age three.

The Legacy of Angels Foundation was established by Sue and Paul Rosenau, who lost their first grandchild, Makayla, to Krabbe disease in 2003, at age two. The circumstances that led to the establishment of the foundation are extraordinary and garnered national media attention at the time.

In 2008, Makayla’s parents had a second daughter. While driving to meet his new granddaughter for the first time, Paul stopped to buy a Power Ball ticket in his home state of Minnesota. Later that evening, he and Sue realized that they held the winning ticket and had won a jackpot totaling $180 million—exactly five years to the day after Makayla’s passing.

A few months later—spurred by their loss and touched by the remarkable timing of their winning—the Rosenaus founded The Legacy of Angels Foundation (TLOAF) to fund research into improved treatments for Krabbe disease, as well as for cystic fibrosis after having learned that their other daughter and son-in-law carry the gene for this disease. The foundation also promotes the expansion of newborn screening.

“We are extremely grateful for the support from The Legacy of Angels Foundation,” says M. Laura Feltri, MD, SUNY Distinguished Professor, professor of biochemistry and neurology, and acting director of the UB Institute for Myelin and Glia Exploration. “This two-component award provides crucial funding that supports the institute’s infrastructure and enables us to continue, unimpeded, our innovative research into how to better treat or even cure Krabbe disease.”

Paul Rosenau, TLOAF president, and Stacy Pike-Langenfeld, TLOAF executive director, collectively state: “We’re excited to see the progress that has occurred since the inception of TLOAF. Our foundation strives to bring researchers from across the world together to expedite the discovery of new therapeutic interventions for Krabbe disease. We’re thrilled to welcome the addition of Dr. Laura Feltri to our robust list of researchers.”

Traditionally, hematopoietic stem cell transplantation, also known as a bone marrow transplant, has improved the long-term survival and quality of life of patients with Krabbe disease, but it is not a cure. Recent studies conducted by Feltri and her group have led to a better understanding of possible ways to make bone marrow transplants and other experimental therapies such as gene therapy more effective.

The Legacy of Angels Foundation primarily focuses on providing support that strategically supplements gaps in research funding. Since 2008, it has helped to propel research on Krabbe’s disease and played a formidable role in the launch of two clinical trials offered to patients with infantile Krabbes. Recently, the foundation has leveraged this success to cultivate promising new collaborations with pharmaceutical companies.

—S. A. Unger