[Good Brain 2026] KAIST Professor Min Lee: "For New GLP-1 Brain Disorder Drugs, Patient Identification Is the Top Priority"

Different Pathogenic Mechanisms Despite the Same Diagnosis
"Patient-Derived Cells and AI Are Key to Identifying Responsive Groups"

"The key question is not whether glucagon-like peptide-1 (GLP-1) based drugs are effective for diseases such as Parkinson's, but rather which specific patients they are effective for. The core of strengthening K-bio competitiveness lies not in the number of candidate substances, but in developing the ability to identify responsive patients."

On September 3, Min Lee, a professor in the Department of Brain and Cognitive Sciences at the Korea Advanced Institute of Science and Technology (KAIST), made these recommendations at the '2026 Good Brain Conference' hosted by The Asia Business Daily at the Westin Chosun Hotel Seoul in Jung-gu, Seoul, under the theme 'Brain Revolution: The Future of Humanity Changed by GLP-1.' She pointed out that, since causes of disease differ greatly among individuals, conducting studies by averaging patients under a single disease label can often reduce drug efficacy.

Professor Lee is regarded as one of the pioneering scholars who initiated the discussion of using GLP-1 drugs for brain disorder treatments. During her graduate studies at the University of Cambridge in the United Kingdom, she generated induced pluripotent stem cells (iPSCs) from the somatic cells of Parkinson's patients, differentiated them into neurons, and investigated the disease's pathogenesis. In 2017, she published Phase 2 clinical trial results for the candidate substance exenatide in the international journal The Lancet. The administration of this drug to Parkinson's patients produced improvements in motor symptoms, drawing attention from the academic community. However, the Phase 3 clinical trial results announced last year failed to demonstrate disease control effects in the entire group of moderate Parkinson's patients.

Min Lee, a professor in the Department of Brain and Cognitive Sciences at KAIST, is giving a lecture at the '2026 Good Brain Conference' held on the 3rd at the Westin Chosun Hotel Seoul in Jung-gu, Seoul, under the theme 'Brain Revolution: The Future of Humanity Changed by GLP-1.' Photo by Kang Jin-hyung

Professor Lee reflected that one limitation in the clinical trial design was grouping together patients with different disease mechanisms and administering the same drug to all. She explained, "Although clinical symptoms are similar, the pathogenic mechanisms leading to Parkinson's disease differ by patient—for some, it's cell death due to mitochondrial dysfunction; for others, the accumulation of aggregated proteins in lysosomes; and for yet others, issues with insulin signaling or inflammation are primary."

She continued, "Research conducted in 2023 and 2024 proposed four subtypes, but the actual number could be seven or even well into the double digits." She added, "It is clear that these pathological subtypes differ, but since we don't yet have methods to distinguish them, we are currently conducting clinical trials without differentiating patients who receive the same diagnosis." This means that, even if a certain drug works for some patients, if patients with different pathological traits are grouped together in analysis, the treatment effect may be diluted overall.

As a solution, she proposed a subtype classification method combining patient-derived iPSC neurons and artificial intelligence (AI). By differentiating patient cells into neurons and comparing their morphological and functional characteristics using AI, it is possible to distinguish the main causal mechanisms even among patients diagnosed with the same Parkinson’s disease. This approach allows researchers to determine in preclinical stages which specific patients will benefit from particular drugs.

Professor Lee also believes that for current research to lead to new drug development, it is necessary to standardize not only drug manufacturing batches, purity, formulations, and storage conditions, but also cell lines, differentiation batches, testing institutions, and analysis criteria. She emphasized the need for organizations equipped with capital and systematic capabilities, such as major pharmaceutical companies, to play a more proactive role in these efforts.

Building on these ideas, Professor Lee proposed the creation of a K-bio alliance for the development of next-generation GLP-1 therapeutics. In this model, hospitals would contribute clinical, imaging, and long-term follow-up data; universities would provide patient-derived iPSCs, organoids, and AI capabilities; companies would offer manufacturing, quality control, and clinical development expertise; and regulatory agencies would be responsible for regulatory science related to the assessment of biomarkers and companion diagnostics. She stressed, "These four entities must be interconnected from the beginning, centering on common patient subtypes and shared biomarkers."

This content was produced with the assistance of AI translation services.