Autoimmune summit highlights opportunities for innovative therapies

Brittan Kopittke calls herself living, breathing proof that autoimmune disease research and development matters.

“At 25 years old, I was knocking on death’s doorstep, and I stand here before you with a second chance at life,” said Kopittke, who shared her personal story to kick off “Shaping the Future of Autoimmune Therapies: Harnessing Artificial Intelligence Innovation,” a summit hosted by Eshelman Innovation on May 1 in Research Triangle Park.

Kopittke was diagnosed in January 2022 with an aggressive form of lupus that was resistant to standard treatments. Thanks to an experimental protocol of CAR T-cell therapy led by UNC School of Medicine Professor Dr. Saira Sheikh, one of the summit’s moderators, Kopittke is healthy and mostly symptom-free. Her story was a poignant reminder of the need for accelerated research into autoimmune diseases, such as lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes, which affect around 15 million Americans. The exact causes remain elusive. The science suggests that a complex interplay of genetic, environmental and immunological factors cause the disorders. Symptoms can vary widely, often making diagnosis and treatment challenging.

The Autoimmune Summit is part of Eshelman Innovation’s therapeutics curation strategy to accelerate the development of new therapies by bringing together leading researchers, clinicians and industry partners. Last spring’s 2024 Radiopharmaceuticals Summit resulted in two funded translational research projects. Eshelman Innovation will soon issue requests for proposals for autoimmune disorder research.

The promise of AI

The opening panel discussion focused on the potential for artificial intelligence (AI) and machine learning to accelerate drug development for novel autoimmunity therapy targets. One way to do so, said Marcel Frenkel, CEO of Ten63 Therapeutics, is by training AI to manage huge data sets. “AI can allow us to see things that would otherwise have been invisible, and that’s extremely exciting,” he said. But how do you train models when there’s no data? “What we’ve been focused on is using machine learning to accelerate quantum mechanical calculations, so we can simulate experiments at scale that would otherwise been impossible.”

Panelist Jenny Ting (in the top photo), genetics professor at the UNC School of Medicine, runs a traditional wet lab, where humans conduct experiments at the bench. “But discoveries are frequently made by looking at genomics and patient databases,” she said. “I don’t think a day goes by that we don’t look at AI-generated information.”

Ting is hopeful the technology can help overcome complexities stemming from the fact that autoimmune diseases are heterogeneous — meaning underlying causes and symptoms can vary significantly among individuals. “I think one of the great contributions of AI,” she said, “is understanding disease subsets” among individuals and groups.

Next, Alex Abuin, R&D Director for Eshelman Innovation’s Therapeutics Accelerator, led a fireside chat about emerging treatment paradigms. 

“We have addressed, through approved medicines, the low-hanging observational stuff in immunology — TNF [tumor necrosis factor] and interferon and cytokines that are present in lesions of patients with disease — and if you target those, you can alleviate symptoms,” said Michael Kalos, Senior Venture Partner and Head of Cell Therapy for the investment company Orange Grove Bio. “But that universe is kind of farmed out. What immunology needs, I think, is a better molecular understanding of disease ... so you’re not just treating the symptoms; you’re treating the cause of disease. I think that’s going to be the watershed.”

Fellow panelist UNC School of Medicine Professor Roland Tisch agreed. “The Holy Grail of a given immunotherapy is to be able to effectively suppress ongoing autoimmune response and reestablish some tolerance, so that an individual is not dependent on continued administration of an antibody or given drug,” he said. He’s hopeful that AI will help identify the key pathways that lead to such treatments.

Lessons from oncology

The final discussion, moderated by Dr. Sheikh, centered around improving patient outcomes. “I think that’s where the major gap is,” Sheikh said. “When we sit across from patients, we have no idea how to predict who’s going to respond to what therapy.” Add to that the fact that autoimmune diseases change and evolve over time and damage can come from the disease itself as well as from treatments that alleviate symptoms by suppressing the immune system. “There’s a lot of complexity and lots to unravel.”

“There are so many different things we don’t know about in the field of immunology. That’s why we need to learn from the oncology teams that have been so successful,” said Dr. Reginald Seeto, CEO and President of Scipher Medicine. “You can leapfrog, if you learn from others.”

“It hasn’t always been that way,” said Dr. Ned Sharpless, Professor of Cancer Policy and Innovation at the UNC School of Medicine, after raising his hand to comment from the audience. “When I started out in this field, we thought cardiology was how to do it; we did 8,000-patient trials for breast cancer. What really broke the case for oncology was the appreciation that we weren’t dealing with one kind of cancer but with heterogeneous cancers.”

Sharpless was one of many subject matter experts — researchers, clinicians, industry partners, patient advocates, policymakers, funders — in attendance.

“The goal of this therapeutics curation model is to engage stakeholders early to find out the needs and opportunities in a particular field,” said Dr. Sumitra Pati, Commercial Strategy Director for Eshelman Innovation’s Therapeutics Accelerator. “The science is hard enough. If we can be strategic about all the other pieces, including what the market wants, we’ll have a much better chance of success.”

In closing, Eshelman Innovation Executive Director John Bamforth addressed Kopittke: “You did a great job, Brittan, describing the challenge of working through your experience with lupus. But more importantly, you gave motivation to everyone in the room to continue the fight against this terrible set of diseases.”

The summit was co-sponsored by Alexandria Real Estate, Orange Grove Bio and the Triangle CERSI. Eshelman Innovation is part of the UNC Eshelman School of Pharmacy, the country’s number one pharmacy school.