Kwang-Soo Kim, PhD

Dr. Kim’s Molecular Neurobiology Laboratory, founded in 1998, studies the biology of brain cells that rely on the chemical dopamine to communicate. Major brain disorders such as PD, ADHD, and schizophrenia are related to abnormalities affecting these cells.

Dr. Kim and his group work to identify defective components from these cells—like the orphan nuclear receptor Nurr1, critical for dopamine neurons and inflammation—to understand and reverse the damage. This transcription factor appears to be key to the loss of A9 subtype dopamine neurons of the substantia nigra in PD.

Nurr1 represents a potential drug target for associated human disorders, so the lab is exploring promising drug candidates targeting Nurr1 that could slow PD progression in a mechanism-based and neuroprotective manner. Although Nurr1 is a ligand-independent nuclear receptor, Dr. Kim hypothesizes it may have endogenous ligand(s) and is pursuing identification and characterization of potential endogenous ligand(s).

In addition, Dr. Kim’s lab pioneered the development of safe, patient-specific stem cells using a new technique—a promising breakthrough for treating and studying human diseases. Cell replacement therapies require clinically safe stem cells to generate many (possibly all) cell types. Most iPSCs have been derived using viral vectors and are not ideal for studying or treating human diseases. The lab pioneered the generation of safe human iPSCs via direct delivery of reprogramming proteins.

More recently, Dr. Kim’s lab identified novel mechanisms underlying the reprogramming process through metabolic reprogramming during human-induced pluripotency, enabling more efficient and safer methods to generate biomedically and clinically ideal iPSCs. The lab continues to refine in vitro differentiation of iPSCs into functional midbrain dopaminergic (mDA) neurons and optimize critical transplantation parameters, providing tools for disease modeling and personalized cell therapy.

These innovations culminated in a landmark achievement: the first treatment of a PD patient in 2017 using mDA progenitors derived from the patient’s own fibroblasts and iPSCs.

In 2023, the lab’s Investigational New Drug (IND) application for personalized cell therapy targeting PD received approval from the U.S. Food and Drug Administration (FDA). The team is now partnering with researchers at Massachusetts General Hospital (MGH) to initiate a first-in-human clinical trial—marking a pivotal step toward realizing personalized regenerative therapies for neurodegenerative disease.

Building on transcriptional mechanisms in midbrain dopamine neurons, Dr. Kim’s lab also develops novel, neuroprotective therapeutics for PD and related disorders. Ongoing research explores how inflammation pathways contribute to neurodegeneration and other conditions such as autism.

The lab identified Nurr1 as a key transcriptional repressor of neurotoxic cytokine production, and small molecules enhancing Nurr1 function have been shown to suppress neuroinflammation—highlighting potential treatments for inflammation-related diseases both in and outside the brain.