A Genetic Condition

PUBLISHED DECEMBER 2024

Steven Leeder saw it coming. Three years before Children’s Mercy Kansas City opened its new research tower to explore new frontiers in pediatric pharmacology, the future director of that site assessed where genomic and precision medicine were heading.

“As an industry, we need to get away from this idea that giving everybody the same dose works. Instead, we need to give the right exposure and target of drug that will work for a particular individual, not what works on the ‘average’ population,” he said. “At the end of the day, individualized patient dosing means better and quicker treatment. Right now, physicians prescribe a medication and wait for a period of time to determine if the medication works or not. What’s going to be a game-changer is knowing how a person is going to respond to a medication before that person ever takes the drug.”

The institute where he works opened its doors in 2021, immediately thrusting the Kansas City region into the forefront of pediatric research. But Children’s is not exploring that space on its own. The largest research institutions in the metropolitan area have stepped up their studies into emerging fields of medical treatment. Their work is fraught with both promise and peril: The potential to bring life-saving and life-extending treatments to patients, at the risk of raising costs as economies of scale are lost to personalization of care.

Regional research efforts are exploring new avenues in treatment along multiple pathways. One is the contemporary view that treatments designed for broad patient populations too often miss the mark. Another is that some diseases are too rare for drug manufacturers to explore production but affect an estimated 10 percent of the population—33.8 million people—when considered as a whole.   

So the research potential is almost unfathomable. And those nearly infinite possibilities have been powerful incentives for large health systems and small starts-up alike to draw on increasingly powerful research tools like artificial intelligence, large-scale data analytics and machine learning. 

Consider cancer research. Within the realm of patient population differences, not every cancer case is the same. Even with a specific cancer type, no two cases are precisely alike, given the variances from one patient to another that can be genetic, related to weight, age, or ethnicity, compounded by co-morbidities or lifestyle factors—daunting numbers of factors that can determine whether a treatment regimen succeeds or fails.

Here’s a thought experiment. Consider one illness. In this case, liver cancer. In the U.S., some 40,000 cases are diagnosed each year. Some will hit otherwise healthy people; some will befall patients with a co-morbidity. Some will have multiple chronic illnesses. Consider that population across half a dozen ethnic groups, then sort them by age into decadal cohorts—six groups for patients between the ages of 20 and 70. Toss in other factors like environmental conditions, work history, drug or alcohol abuse and smoking. 

Already, you’re closing in on a thousand potential differences in the way a particular liver cancer presents at diagnosis. You can’t prescribe the same drug, dosage or duration for all of them. 

However, you can develop processes that, based on an individual’s genetic profile, suggest treatment variations specific to that particular patient. 

That’s what inspires the work at the Kansas Institute for Precision Medicine, founded in 2019 by The University of Kansas Health System. The system says a $11.4 million grant from a National Institutes of Health division funds training for physicians and scientists in precision medicine and supporting their research.

“It’s been a passion of mine for my entire career, trying to understand how you move things from discovery into change in clinical practice,” says Andrew Godwin, founding director of the KIPM. “The KIPM helps us promote the next generation of scientists, many of whom are physician-scientists, who can then move forward with new discoveries and change the way we treat patients.”

The breadth of that research, Godwin says, is substantial, “including asthma, juvenile arthritis, schizophrenia and sleep disturbances, Alzheimer’s disease, cystic fibrosis, thrombocytopenia, implementation of genomic medicine, mathematical models of biomarker selection, and the study of biomarkers and therapies for multiple types of cancer.”

Genomics is a fertile field of research, considering that the human body has between 20,000 and 25,000 genes. That’s a lot of potential for something to go haywire. 

And the NIH classifies more than 7,000 diseases as “rare,” with the vast majority—an estimated 95 percent—having no known treatments at this point. Laws of supply and demand drive the lack of pharmaceutical development to address those conditions: It would simply be prohibitively expensive to develop a drug that can treat only a comparative handful of cases. 

At Saint Luke’s Center for Precision Oncology, researchers have sequenced 20,000 cancer genes to help identify any given patient’s exact genomic mutation and prescribe the best possible treatment. Sifting through oceans of patient data using AI, they can discern accurate diagnoses and produce personalized treatment plans. The technology is better at predicting treatment outcomes, leading to better decision-making by providers, often with fewer side effects for patients.

“If you look at just 2024, with the new oncology drug approvals by the FDA, about half of them were for targeted therapies and precision therapeutics. That number grows each year,” says Marc Roth, a hematology oncologist at Saint Luke’s. “It’s not just the drug development, but also the ways we identify who will benefit from these drugs. With sequencing platforms or testing on patients with advanced cancer, as those become more sensitive and are better able to detect specific molecular aberrations, locations and numbers of genes they detect, we’re able to find more patients with these targetable aberrations and find new ones.”

A Saint Luke’s colleague, clinical pharmacist Blake Buzard, noted that the region is awash in clinical trials for various gene-based therapies. “Not only Saint Luke’s but a lot of centers in the area have certain trials open right now. One thing we do as far as our precision trials is screen for other trials in the area. It’s hard to put a number on it at any one time, but definitely, the number of trials is in the high two-digits, if not over 100, across solid tumors and hematologic malignancies.”

Beyond that, he notes, “oncologists, not just in large academic centers, but also in community practices, are ordering more and more of these tests, so we’re finding more patients.”

The research effort at Children’s Mercy has shown that a robust research effort can attract outside funding at scale. After $32.4 million from grants and contracts last year, the hospital announced nearly $30 million in research grants for 2024, ranging from $1,000 microgrants up to nearly $12 million for multi-year research projects. 

The largest was a grant from the Centers for Disease Control and Prevention to researchers specializing in infectious diseases ($11.7 million); another exploration into additional health risks facing children with chronic kidney disease secured a $5.8 million award from the National Institute of Diabetes and Digestive and Kidney Diseases and NIH. 

Children’s also developed the Genomic Answers for Kids program, which it says may be the world’s most advanced system for rare disease diagnosis. “The team has surpassed 1,000 rare diagnoses through their groundbreaking work—far out-pacing other rare disease research programs—and continues to conduct cutting-edge science that has led to a number of ‘firsts’ in the field of genomics research, hospital officials say.

One of the most high-profile expansions of regional medical research has been at The University of Kansas Cancer Center, which won a prized designation from the National Cancer Institutes in 2012 as an NCI care center and followed that up with Comprehensive Care Center recognition a decade later. 

Under the direction of Roy Jensen, research operations at KUCC have yielded benefits that go far beyond improved patient care, generating an estimated $2.5 billion in economic impact for the broader region since the center began working toward NCI designation in 2007. 

And HCA Midwest Health, as part of Nashville-based HCA Healthcare, is working on its own molecular profile studies through the national Sarah Cannon Cancer Center. Physicians within that network identify and order the most appropriate molecular tests from commercial laboratories for more than 400 genes present in solid tumors or blood cancers. 

All of this helps make the Kansas City region attractive to talented health-care professionals and researchers; Roth, for example, was working in an emerging health-care  center of excellence when he came here from Nashville a couple of years ago, albeit with family considerations driving that decision. 

“I was surprised there was so much going on in Kanas City,” Roth says. “I have worked at various bigger academic medical centers, but what I’m seeing is that more and more community practices and hybrid practices are developing centers for precision oncology, which I would put up against any of the bigger, high-powered academic centers. The work they do is impactful for patients.”

As the growth in that field continues across the region, it poses the question: Where will Kansas City find the additional research talent and supporting work force to keep the momentum going? Attempting to address that need is BioNexusKC, whose mission includes serving as a clearinghouse for information on life-sciences developments, capital formation and workforce developments. 

“Our region has been investing in the life sciences ecosystem for decades, but in recent years, we have been intentionally focused on building out the support for the development of biologic vaccines and therapeutics,” said president and CEO Dennis Ridenour. “Biologics enable precision medicine, and as the industry pivots toward biologics, their pipelines increasingly feature these therapies, reflecting a broader trend toward personalized and targeted medicine that aligns with advancements in technology and patient needs.”

The economic impact as the life-sciences sector grow here will be massive, says Melissa Roberts Chapman, president of KC BioHub.

“We project the creation of more than 10,000 good jobs over the next 10 years,” she says. “Just as important, the growth of this industry will on-shore critical biomanufacturing activities, contributing to our country’s national security.”