Dr. Joseph Lillegard: What if We Could Actually Cure Diseases?

Dr. Lillegard started his presentation with a slide showing the short and powerful mission statement of his team at Minnesota Children’s Hospital, “Keep the tiny humans alive.” He then explained why he has dedicated his career to finding cures for the tiniest of humans, “While all healthcare professionals do important work and add value to people’s lives when you talk about fetal interventions, you’re talking about contributing eighty years of life and productivity.” Dr. Lillegard’s work is fueled by the belief that early interventions put the least burden on the healthcare system and provide the most positive value to patients’ lives. In this talk, he explained that his team works to discover the earliest and smallest forms of disease intervention because intervening early makes the most significant and longest-lasting contributions to the lives of individuals and society as a whole.

Dr. Lillegard’s track record, and the stats of fetal surgery in general, demonstrate the value of conducting surgical interventions as early as possible. Conducting surgery on fetuses with spina bifida led to those babies having a higher quality of life than the babies with spina bifida who were only treated post-natal. The benefits include walking sooner and performing at higher cognitive levels as measured by the Bayley Scales of Infant Development. The advantages of early interventions extend beyond the quality of life for the babies and their families. Dr. Lillegard explained, “When you look at a kid with spina bifida that received a fetal repair and one that received a post-natal repair, the fetal repair put roughly ten percent of the cost on the health care system as the post-natal repair.” 

As the audience members were either in silent awe or wiping away heartfelt tears, Dr. Lillegard described the details of conducting fetal surgery on twin fetuses. He released the emotional buildup in the room with a joke explaining how nervous he gets during the procedure, “The uterus sits on mom’s aorta, and is moving at 80 beats per minute, and the babies’ heartrates are about 150 beats per minute, and my heartrate because of those two things is about 300 per minute." His research around twin to twin transfusion syndrome, and his ability to keep a steady hand under pressure, has led to increasing the national survival rate of fetuses with twin to twin transfusion syndrome by over fitty percent in the last ten years. 

Unfortunately, not everyone has access to facilities like Minnesota Children’s and surgeons like Dr. Lillegard. There are fewer than ten advanced fetal surgery centers in North America. The sad reality that many can’t access fetal surgery centers combined with the slim likelihood of that reality changing anytime soon has led Dr. Lillegard and his team to shift their focus to researching technology that is even smaller than fetal surgery and that could have an even bigger impact on people’s lives.

Dr. Lillegard believes gene correction therapy has the potential to be the great equalizer in healthcare. His teams’ focus on gene correction therapy stems from the same passion that fueled their work in fetal surgery. It’s better for patients and the healthcare system to intervene with diseases earlier rather than later and to cure diseases rather than manage diseases. Dr. Lillegard stated that the latest research regarding gene correction therapy demonstrates it is in position to be the early interventive cure that patients and society are searching for.

“Only 18-20% of people that need an organ transplant get one—the rest die waiting.”

To illustrate the inefficacy of the current approach to treating diseases he cited some stats related to organ transplants, “Only 18-20% of people that need an organ transplant get one. The rest of the people die while waiting on the transplant list.” The tragedy of that situation is amplified when you consider that most patients on the waiting list for organ transplants only have a problem with one gene within their problematic organ. The rest of the organ works properly. Dr. Lillegard used a construction metaphor to highlight the inefficiency intrinsic to the current approach of dealing with liver diseases, “Right now we replace the whole house, just if the house needs a new doorknob.” Sticking with the metaphor, Dr. Lillegard’s team now works on developing ways to leave a fully functional house intact, while replacing the faulty doorknobs.

Dr. Lillegard’s team achieved their goal by developing a way to extract a diseased gene from a person, remove the disease from the extracted gene, grow healthy cells of the patient’s own genes in a bio-simulator, and then reinsert the healthy genes back into the patient. The academic publication Science Translational Medicine explained the significance of this work, “Having demonstrated in large animals the use of materials that are safe for use in people, the technology is now poised to move into patients, to regenerate their own livers and spare them the long wait times on the liver transplant list.”

“In the future we are not going to be treating diseases. We are going to be curing them.”

This research leads Dr. Lillegard to the optimistic claim that, “In the future, we are not going to be treating diseases. We are going to be curing them.” Unlike the fetal surgeries Dr. Lillegard conducts, the gene correction technology his team has developed will not require extensive equipment and rare surgical specialties. Instead of having to find one of the top surgeons in the world, access to cures could be widely accessible. Dr. Lillegard explained, “You only need a vial and a syringe to administer this. You’re talking about being able to do this in rural America and third world countries, you’re talking about unburdening the healthcare system.” You can watch the full talk above to learn more about the great work Dr. Lillegard and his team are conducting.

At Worrell, we are committed to designing the bright future Dr. Lillegard describes, where diseases will be cured, and the healthcare system will be able to provide better patient outcomes for everyone. Please reach out if you would like to learn more about the healthcare innovations we are working on, the experts and institutions we collaborate with, and how we might be able to help your organization solve the trickiest problems in healthcare.