Sanford names gene pioneers $1 million Lorraine Cross winners

Sanford Health has named two women whose work reversed a form of blindness as the winners of its first $1 million Lorraine Cross award.

Drs. Jean Bennett and Katherine High were honored for their work with the RPE65 mutation that has reversed an inherited form of blindness.

Bennett and High pioneered the gene therapy, took it to clinical trials and then received FDA-approval for the treatment, the first FDA approval of a gene therapy for a genetic disease. High also co-founded Spark Therapeutics, a fully integrated, commercial gene therapy company working to accelerate the timeline for bringing new gene therapies to market. Bennett is a professor of ophthalmology at the University of Pennsylvania, and High is president and head of research and development at Spark Therapeutics.

“I would like to thank the Sanford group,” Bennett said. “This has been an incredible experience and you are setting a standard that is just amazing.”

Sanford Health created the prize, which it will award every other year, to be one of the top five most lucrative awards celebrating transformative breakthroughs in medical care.

“They’re all winners. But even more we are all winners. As the human race, people who will benefit from their treatments,” said Andy North, board chair of the Sanford International Board.

The candidates 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 for those who take action for their passions. Sanford Health uses it to symbolize innovation in health care.

“What you’re doing for us and what you’ve accomplished is a dream come true for this organization,” CEO Kelby Krabbenhoft told the finalists. “This company is moved forward by your efforts.”

Sanford is giving the award not just to honor their work but to inspire others, he said.

“We’re going to resolve sadness. We’re going to stop suffering, and we’re going to do this not trying to live forever, that’s not our pursuit but we do want to just live life without these sufferings.”

It was presented on a night with all the pomp and circumstance of a major awards ceremony.

A black-tie dinner preceded a program honoring the finalists.

Music was provided by vocalist Leslie Odom Jr.

CBS News was in town to cover it.

The finalists met with the Sanford International Board, which deliberated more than four hours and took three votes before naming the winner on an 8-1 vote. To symbolize the decision, blue smoke rose from the Sanford House.

The following is a piece published by Sanford that details those interviews.

James Wilson: “We only have one life.”

James Wilson is barely seated before he begins talking.

“I grew up the oldest of four kids in a suburb of Detroit,” he begins, and the board settles back to hear his roots. He went to Catholic school and loved to play sports -– and was competitive enough to know he wanted to break with tradition and go to the public high school, which was larger and offered more opportunity for sports.

He did, and you get the impression that Wilson usually is able to persuade people of things.

But in the next story, he shows he’s able to learn, too: “I was ready to accept a Division II scholarship, but my dad said, ‘Son, you’re too slow to make it to the pros, so you might want to rethink this one.’”

James Wilson

The room laughs, and so does Wilson.

Wilson says he listened to his dad and went on to a different college, realizing he could channel that competition into academics. He went on to study the molecular base of diseases, particularly in childhood diseases.

“It was an incredible time, but I realized the work I was doing wasn’t going to help them. If I was going to spend my life in science, I wanted it to be directed at therapy. I wanted it to be treatment.”

It was a turning point for Wilson, who left practice to begin his work in research, calling is last day at Mass General Hospital one of the saddest in his life.

“I was pretty good at science and probably a mediocre doctor,” he laughs.

For the rest of the hour, though, he talks about that phase: About this drive to solve problems, and the global responsibility he feels to make solutions accessible. He rattles off a list of phrases that begin with the obvious and end with the profound: Knowledge is power. Science self-corrects. The truth prevails, no matter what you think it may be.

And this: Science should be available to everyone.

You could follow these phrases to trace his career.

He goes back in time again -– to the turn of the century, when gene therapy was hot. “Everyone was riding the wave,” Wilson says. “There were many clinical trials, and none of them were working.”

Patients died, or developed cancer. The stock market tanked. Enthusiasm for the field evaporated.

“This was about a young man who had volunteered to participate in this. We had to figure out what happened,” Wilson says.

It was time to reinvent, and Wilson came back to what he knows: science. Science is the answer.

“That was the easy part,” he says. “Don’t point fingers. Take responsibility. Talk about resolve to getting it right –- I resolved to get it right.”

The board members who will decide the winner nod, begin asking questions. They want to know about collaboration. About roadblocks. About what keeps him up at night.

“How do you push forward but not make it so fast?” asks Robin Smith.

“It is getting ahead of itself,” he replies. “They call it Wilson’s warning. I wish they called it something else.”

He laughs, but there it is: The ever present comment on his past, on that story he tells as a warning and an inspiration to get it right. He doesn’t shy away from it, faces it head on.

“I say look, this is how I am,” Wilson says in his direct way. “You’re going to hear everything I worry about, not how I want it to be. Anything we’re doing now is not how we are going to be doing it in 10 years.”

The room looks ahead.

“With all your success, you’re still dreaming,” says Manny Ohonme, a member of the Sanford International Board.

Wilson is. He spends the rest of the discussion talking about the responsibility he feels to the world.

“One thing I enjoy is traveling to different parts of this planet and meeting families and patients. This is the physician in me,” Wilson says. “And they’re all the same. And we can cure those diseases in my lifetime. We just have to figure out how to do it. But it’s not going to be just me.”

Brian Kaspar: “It takes decades of work.”

Brian Kaspar talks about Falls Park, home to the Sioux Falls, South Dakota, namesake waterfalls. He drove through there the night before with his family to admire the holiday lights.

“It’s 100 percent decorated, and it’s just beautiful,” he says, before launching into a description of how he’s watched the lights go on in his work, using gene therapy to treat a deadly pediatric disease called spinal muscular atrophy, or SMA.

He goes through his history, and his work at Ohio State University and Nationwide Children’s Hospital, before founding Avexis in 2015.

Sanford’s chief innovation offIcer, Dr. David Shulkin, greets Brian Kaspar.

“I’ve been in gene therapy for over two decades,” Kaspar says. He talks about Novartis, which acquired the company, and how that relationship developed.

“Novartis recognized that this is something that we had moved the needle on,” Kaspar says. “When they acquired Avexis, they said, ‘We like the miracles you’re doing each and every day. We consider you the speedboat. We’re the big steamship of Novartis, but when you need to come back and fill up with fuel, pull up.’”

He laughs, and so does the room, but everyone inside knows that funding matters, that getting therapies approved matters. That these are the steps that need to be taken before something can be treated or cured.

And for Kaspar, it always comes back to that.

Children born with SMA don’t live to see their second birthdays.

“We’ve worked diligently throughout my career to change these diseases,” he says, and then he credits Wilson, a fellow award finalist, as a pioneer in the field.

Kaspar shows slides of babies who can’t hold their heads up.

“These may be hard to watch,” he tells the room. He describes SMA and says it doesn’t have any ethnic boundaries. “This happens around the entire world. I’ve heard the same stories in China. In Argentina.”

It’s the most common genetic cause of infant death. He calls it a childhood version of Lou Gehrig’s disease, so they’ll understand.

It’s a death sentence.

He’s used a gene replacement therapy, starting in mice. Then pigs. Eventually, children.

It’s a one-time administration into the blood, and the human clinical trials at first made people uncomfortable. “We would receive phone calls saying, ‘Don’t do this,’” Kaspar says. “This is the largest amount of gene therapy in a human. This is scary.”

The procedure takes about an hour. The child sleeps, or rests in a parent’s arms.

These children are still alive. Some are turning age 5. They’re sitting up on their own. He shows more videos.

“These kids are eating normally,” Kaspar marvels. “One of these has achieved a milestone I haven’t: They’re bilingual.”

It’s clear he loves what he does and knows why he does it. And he spends as much time crediting his team as he does the science itself.

“We’ve broken the barrier of giving the world’s largest amount of gene therapy safely,” he says. “But how do you scale this?”

It’s the same driver: Make it accessible.

For Kaspar, that means making a test for SMA part of the standard battery of tests in a newborn screening. It’s a blood draw.

“I’m blessed to have two healthy children and a healthy wife,” Kaspar tells the room. “But you see how fragile that is. And you see that we are not superhumans.”

Jean Bennett and Katherine High: “We were lucky we were naïve.”

Jean Bennett and Katherine High say they faced dozens of challenges on the way to discovering the first and only approved gene therapy for inherited disease in the United States -– and creating a path for genetic treatments for blindness and other devastating diseases.

They begin with a slide show.

And the facts.

“Let me tell you about this disorder,” says Bennett, her long hair falling out of her barettes as she gestures, as she pulls the room into a conversation, not a presentation. High sits next to her, lets Bennett talk about how people with this inherited form of blindness slowly lose their sight.

Finalists Katherine High and Jean Bennett

They show a video of a boy holding his parents’ hands as he walks down the hallway, unable to see enough to navigate the space. Then he’s 9 years old, and he’s riding a bike. Now, he’s 18 and he picked up his date for prom. In a car he drove himself.

This is the result of their work.

To deliver a correct copy of a gene for some of the 3,000 people in the United States with this form of blindness.

They started in animals, and when they were ready to move to humans, they had a list of obstacles to overcome.

There was no path for pediatric drug development. They needed to figure out how to document clinical meaningfulness –- the test of reading an eye chart wouldn’t work. These patients couldn’t even see the eye chart. They had to come up with an obstacle course for patients to navigate –- to measure changes in functional vision.

They did. Then had to come up with dozens of configurations so people couldn’t memorize it.

It was just one setback, creating the test, in what High calls a “bewildering array of problems.”

That included the rarity of the disease, the lack of natural histories of it, and what would be the controls in a trial. And then, of course, the funding.

“We were getting great results, but we realized we wouldn’t be able to assemble everything we needed to move this across the finish line,” High says. “So in 2013 we started Spark Therapeutics.”

Bennett is a scientific founder but has no financial stake in the company. This was to protect conflicts of interest for her.

The conversation switches as High explains the company and the why: Bringing this treatment to a commercial level, who could help, how to solve issues of access and reimbursement.

They figured it out, just like they had been doing together for years, and in 2017 Luxturna was approved.

“This is how we felt,” High laughs as she shows a slide of fireworks.

The board asks them about their early work. Bennett talks about working with gene therapy. They talk about their families. High discusses working in internal medicine and hematology. They found each other at the right time.

High wanted to focus on gene therapy but was told it couldn’t just be in hematology. She liked Bennett’s work.

They pause.

“No two people can do this alone,” Bennett says.

High reflects on how to move faster.

“If I didn’t believe the clinical guidelines would shorten, I couldn’t come to work every day,” she says. “I believe that every time somebody does a trial, more problems are solved and the timelines are shortened.”

They know this was their path, and it will be for others, too.

“We chose a good target, and we executed,” High says. “We solved the problems in our path. For young people, if you want to get across the finish line, you own all the problems. Not just the ones that will lead to a good journal article, but the ones that are boring and need to be done. No problem is too small.”

Gene therapy pioneers named finalists for $1 million Sanford Lorraine Cross Award

Sanford names gene pioneers $1 million Lorraine Cross winners

Sanford Health has named two women whose work reversed a form of blindness as the winners of its first $1 million Lorraine Cross award.

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