Meet the finalists for Sanford’s $1 million prize

Nov. 29, 2018

Some of the most accomplished researchers in the medical world will be in Sioux Falls next week, and one will leave with the first $1 million prize awarded by Sanford Health.

The Lorraine Cross award is designed to recognize those who are driving innovation in health care.

Sanford created the prize, which it will award every other year, to be one of the top five most lucrative honors celebrating transformative breakthroughs in medical care and the scientists who dedicate their lives to the work of health and healing.

Contenders were identified using a computer algorithm that sifted through medical research globally in search of great discoveries. An interdisciplinary scientific advisory board identified the finalists and the Sanford International Board will select a winner, who will be named the night of Dec. 4.

Here’s a closer look at their transformative work.

 Dr. Jean Bennett and Dr. Katherine High

The work of Drs. Jean Bennett and Katherine High is literally helping reverse blindness.

Bennett has been drawn to the field since the early 1980s, but at that point the tools hadn’t been developed yet to pioneer gene therapy.

She and her husband, who is a retina surgeon, spent 15 years developing the tools, surgical techniques and methods for evaluating safety and efficacy of retinal gene therapy.

Then, in the late 1990s, mutations in the RPE65 gene were identified in both blind children and blind puppies.

“It was a natural step to put everything together to try to treat this condition in the puppies, particularly since three RPE65-mutant puppies had just been born in our animal facility and were available for study,” Bennett said.

“We knew within weeks after the injections, which took place in July 2000, that the intervention had reversed blindness in the puppies. We then spent several years optimizing the reagent, including dose, volume, age of intervention and promoter, and then trying to obtain funding and approvals for carrying out a human clinical trial.”

The first clinical trial occurred in 2007, followed by two more, and the reagent – Luxturna – finally was granted FDA approval one year ago.

The first patient was injected with it in March, and at least 10 U.S. centers now are delivering it. Luxturna was granted approved for use in the European Union just last week.

Patients receive a one-time injection under general anesthesia, and the gene stays active in targeted cells, Bennett said.

“The exact results depend upon how long the disease has progressed because cells in the retina die off over time due to the disease,” she said.

“The more cells that remain viable, the more function that can be restored. However, the vast majority of patients become able to see well in environments that were completely invisible to them previously; everything is brighter and they have greatly expanded side vision. Many people have improved visual acuity — fine visual resolution — and can now recognize faces and facial expressions, read books, appreciate colors, see what the teacher is writing on the board, play sports, walk and ride bicycles independently instead of using a blind cane and work at office jobs.”

Seeing their work come to fruition in treatments is an achievement of a lifetime, High added.

“There were many times along the way when we despaired of reaching the finish line, but every day we got up and tried to solve that day’s problems and found ourselves a little further along the path,” she said.

“It has been a lifelong dream for us to see gene therapy become a real treatment option for patients born with serious genetic conditions. We believe that Luxturna has helped to make that dream a reality.”

The team thinks the same approach can be used to treat other forms of inherited blindness.

While theirs was the first, there now are more than 40 different retinal gene therapy clinical trials targeting more than one dozen inherited forms of blindness.

“There are many other disease processes for which gene therapy approaches are also being tested, including blood coagulation disorders, muscular dystrophy, metabolic diseases, neurodegenerative disorders, deafness, etc.,” she said.

High and Bennett also co-founded Spark Therapeutics in 2013 to further their gene therapy research.

“Spark has built a fully integrated, commercial gene therapy company that strives to challenge the inevitability of genetic disease,” High said, adding the company “has several investigational gene therapies near or in clinical trials, including investigational gene therapies for hemophilia A and Pompe disease.”

Brian Kaspar

Brian Kaspar is an innovator in the field of gene replacement therapy — taking a cloned, intact gene and delivering it to patients who have diseases stemming from a missing or mutated gene.

He and his team have used the therapy on diseases such as spinal muscular atrophy. Affecting babies from birth, SMA robs young children of the ability to walk, talk, crawl and eat. Most patients won’t live to see their second birthdays.

“To see SMA not only affect children but to see its toll on the parents, the grandparents, the aunts and the uncles,” Kaspar said. “These diseases spread throughout the family emotionally. And I think that’s something that keeps us grounded … to understand that we’re working for a true purpose.”

That purpose is eliminating SMA. Since 2004, Kaspar has been working on gene replacement therapy that can halt the disease, with clinical trials showing children walking, talking and living a full life at 5 years old.

“We saw some of the kids standing … most of the kids sitting unassisted, doing things that the textbooks would say they shouldn’t be able to do,” he said. “This is unheard of in the disease called SMA type 1.”

After seeing those clinical results, Kaspar faced a new challenge: getting this life-changing drug to market. To overcome the financial and regulatory hurdles of drug distribution, Kaspar thought outside the box, partnering with investors to create AveXis, a company aimed at getting therapies through the finish line to make a global impact. It was acquired by Novartis this year.

“We took a different approach,” he said. “We were looking at the finish line, and that is distributing this globally and treating every single patient. That is our end goal, and until we get there, we will not rest.”

Today, Kaspar’s SMA therapy has achieved special designation in the European Union and is currently undergoing FDA approval in the U.S. Additional research shows similar gene therapies are yielding extraordinary results with other motor neuron diseases such as Rett syndrome. These breakthroughs are giving new hope to families around the globe.

“Don’t just do the simple experiment,” Kaspar said. “Don’t do the thing that seems most obvious. Relish the potential to make a huge impact.”

Dr. -James Wilson

Dr. James Wilson’s work has focused on developing the science of gene therapy necessary to support successful clinical trials.

In 2008, Wilson and the University of Pennsylvania co-founded REGENXBIO Inc., a clinical stage biotech company designing gene therapy products.

“We discovered a family of viruses endogenous to primates that when made vectors have become best in class in the commercial development of gene therapy,” he explained.

“There currently are 85 different disease-based programs in 28 companies that use vectors that fall under our patents. Approximately one-third of these are in clinical trials.”

His work helped define the scientific and ethical standards for advancing gene therapies through FDA-approved clinical trials.

“Gene therapy represents a platform where common elements of the product are deployed across a broad array of diseases,” he said. “It is very helpful if the experiences of each sponsor are shared with others. For example, this may allow us to identify safety concerns relevant across multiple programs.”

Reputation builder

The Lorraine Cross prize also is serving to elevate Sanford’s stature in the broader research community.

“I knew that Sanford Health was an outstanding health network in the central USA, but honestly I knew very little about the extent to which this organization carries out and supports research for rare diseases,” Bennett said.

“I have learned a lot over the past month about this amazing and powerful entity and am frankly overwhelmed by what I have seen and read. The scientists, board members and staff are absolutely amazing people as well. It is great to see such wonderful work going on in our country.”

High agreed.

“What impresses me about this award is the emphasis on bringing an innovative concept all the way through from first idea to actual clinical practice,” she said.

“As anyone who does this type of work knows, the number of hurdles and obstacles along the way, especially for a completely new class of drugs like gene therapy, can be truly daunting. And success comes from finding solutions for all the problems, not just those that are flashiest or most interesting scientifically.”

Wilson knew of Sanford’s work before the prize and said he’s particularly impressed with the organization’s focus on rare diseases, which is his specialty.

“I am very familiar with Sanford’s commitment to excellence in clinical care, research and education,” he said. “It appears to me that the organization has the resources and resolve to make a positive impact on patients’ lives throughout the world.”

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Meet the finalists for Sanford’s $1 million prize

Some of the most accomplished researchers in the medical world will be in Sioux Falls next week, and one will leave with the first $1 million prize awarded by Sanford Health.

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