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Vikram Khurana leads development of rapid ‘In a Dish’ Parkinson’s model
The study introduced a method to rapidly produce Parkinson’s disease in a petri dish, offering unprecedented opportunities for personalized diagnosis and treatment.
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Vikram Khurana/ Representative Image / Image- The Harvard Gazette
A groundbreaking model developed by Dr. Vikram Khurana and his team at Harvard-affiliated Brigham and Women’s Hospital (BWH) promises to revolutionize the treatment of Parkinson’s disease (PD).
The study, published in Neuron, introduced a method to rapidly produce Parkinson’s disease in a petri dish, offering unprecedented opportunities for personalized diagnosis and treatment.
Khurana, chief of the movement disorders division at BWH, spearheaded the research that addresses the limitations of existing “Parkinson’s in a dish” models, which transform stem cells into brain cells but lack the speed and specificity needed to study patient-specific cellular pathologies. His team’s technique achieves this transformation within weeks instead of months and replicates the diverse protein misfolding pathologies associated with PD.
“We sought to assess how quickly we could make human brain cells in the lab that give us a window into key processes occurring in the brains of patients with Parkinson’s disease and related disorders like multiple system atrophy and Lewy body dementia,” said Khurana.
“And, unlike previous models, we wanted to do this in a short enough time frame for these models to be useful for high-throughput genetic and drug screens and easy enough for many labs to use across academia and industry,” he added.
Parkinson’s disease is a progressive neurodegenerative disorder characterized by symptoms such as tremors, muscle stiffness, and speech impairment. The disease, like Alzheimer’s, is marked by the buildup of misfolded proteins in neurons, leading to impaired cell function. Current therapies for PD manage symptoms but fail to address the underlying protein misfolding.
The innovative model uses PiggyBac vectors to introduce transcription factors into stem cells, rapidly converting them into brain cells. These cells are then exposed to aggregation-prone proteins like alpha-synuclein, which play a key role in the formation of protein clusters in PD.
“This technology will pave the way for rapidly developing ‘personalized stem cell models’ from individual patients. These models are already being used to efficiently test new diagnostic and treatment strategies ‘in a dish’ before jumping into clinical trials so we target the right drug to the right patient,” said Dr. Isabel Lam, co-first author of the study.
The research also acknowledges the current limitations of the model, such as its production of immature neurons. Future work aims to replicate the model using mature neurons to better understand the effects of aging on protein aggregates.
Dr. Khurana, who is also a co-founder of CNS-focused companies DaCapo Brainscience and Yumanity Therapeutics, noted that the work represents a significant step forward in the quest to create effective, personalized treatments for Parkinson’s and related disorders.
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