Meet the Sanford Lorraine Cross Award 2018 finalists

Sanford Health will reward one of four Lorraine Cross finalists $1 million for work in medicine, science and innovation.

The Sanford Lorraine Cross Award  will be given out in a special ceremony in Sioux Falls, South Dakota, on Tuesday, Dec. 4, 2018. The event will be livestreamed on Sanford Health’s social media channels on Twitter, Facebook and YouTube, along with regularly updated stories on Sanford Health News.

Candidates for the award were filtered through nominations from the public and a computer algorithm that sifted through medical publications in search of great discoveries. An interdisciplinary scientific advisory board identified top areas of innovation and the candidates were narrowed down to the top four.

The award is named after the Lorraine Cross, a symbol recognized around the world synonymous for those who take action for their passions.  In recent history, Sanford Health has used it to symbolize innovation in health care.

Here’s a look at the Lorraine Cross finalists, who have inspired others to develop gene therapy, reversed a rare form of blindness and enabled children to walk.

“These individuals are changing the world,” said David Shulkin, International Board member and Sanford Health chief of innovation. “At Sanford, we want to reward innovation and scientific breakthroughs. We’re committed to finding the next frontier in medicine and research, and recognizing others with the same goal.”

Jean Bennett and Katherine High: ‘Patients are the North Star’

Leaves falling. The reflection of sunlight on a pond. A mother’s gaze.

These visions slowly fade from focus and then go dark when someone has an inherited form of blindness.

Scientists Jean Bennett, M.D., Ph.D., and Katherine High, M.D., knew gene therapy had the potential to change that.

“The idea is to harness what we know about the defects, and then intervene,” said Bennett, a professor of ophthalmology at the University of Pennsylvania and a scientific co-founder of Spark Therapeutics, where High is the president and head of research and development.

They began by studying dogs who had the same gene mutation that causes blindness in humans — the RPE65 mutation. Bennett was searching for funding when High approached her, asking if she’d like to run a human clinical trial. Gene therapy was still in its infancy, though, with roadblocks from funding to regulation.

“But I was convinced we weren’t seeing any problems that couldn’t eventually be solved,” said High, who then co-founded Spark Therapeutics, a fully integrated, commercial gene therapy company working to accelerate the timeline for bringing new gene therapies to market. She knew she would have to help be part of the solution.

It worked, and they and their team received the first FDA approval of a gene therapy for a genetic disease. They credit the work of their team and their singular pursuit of a solution.

“Identify the best people, then entice them and motivate them to work on this problem so you can pull together and get to the goal,” High said.

Lighting someone’s future is still what drives their vision.

“Patients are the reason we do this work,” High said. “They are the North Star of what we do.”

James M. Wilson: ‘We’re not done’

The joy of the “a-ha” moment was short-lived for James Wilson.

He had been studying a rare childhood disease and had discovered the specific gene mutation that causes it. Better yet, he had discovered it while working with a young patient he had gotten to know.

“It was incredible,” Wilson said.

But when he went to tell the patient’s mother about it, she asked him simply: How will this help my son?

“And I was punched in the stomach,” Wilson said of that moment when the joy of the discovery couldn’t match the heartbreak of a mother.

Because the truth was, it wouldn’t help her son. Not yet. But it was part of what drove Wilson to develop a concept around how to treat the boy and others like him.

“Gene therapy treats the disease at its root,” Wilson said. “But we needed to develop approaches that just weren’t available.”

Wilson, M.D., Ph.D., is the director of the Gene Therapy Program, the Rose H. Weiss Professor and Director of the Orphan Disease Center, and a professor of Medicine and Pediatrics in the Perelman School of Medicine at the University of Pennsylvania.

He has spent decades trying to harness the power of a virus to deliver a therapeutic gene to treat disease. In 2008, Wilson and the University of Pennsylvania cofounded REGENXBIO Inc., a clinical-stage biotech company designing gene therapy products.

This is his life’s work.

“Until we’re done, we’re not done,” Wilson said.

Brian Kaspar: ‘We were holding our breath’

The answers were unacceptable to Brian Kaspar.

Spinal muscular atrophy, or SMA, is a motor neuron disease that affects infants. These children will never develop any motor milestones that normal children will, and they won’t make it to their second birthdays.

When parents would find out their child had SMA, the message was bleak.

“Take your baby home. Love them. Your baby is going to die.”

For Kaspar, Ph.D., there needed to be a better answer. His lab discovered a gene replacement therapy approach that seeks to change the course of SMA by addressing its genetic cause. There were improvements in early studies.

SMA is a devastating disease that robs babies of basic muscle functions, like breathing and swallowing, and in its most severe form (type 1), usually leads to death by age 2. An initial clinical trial using the AAV9 vector to treat SMA type 1 demonstrated a dramatic survival benefit and rapid improvement in motor milestones.

“An arm moving. A leg. We were holding our breath,” Kaspar said. “Is this going to last?”

He calls it a game-changer in the field and hopes it will be the start of something bigger, and to expand to treat other diseases.

Kaspar is the scientific founder and chief scientific officer of AveXis, a gene therapy company that was acquired by Novartis in 2018.

“Relish the potential to make a huge impact,” Kaspar said.

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