2026 Biomedical Research Collaboration Award
Memphis, TN, October 20, 2026 -- The Hartwell Foundation officially announced a Biomedical Research Collaboration Award to
Pranam D. Chatterjee, Ph.D., 2022 Hartwell Investigator and Assistant Professor, Department of Bioengineering, University of Pennsylvania, and Roy Ben-Shalom, Ph.D., 2022 Hartwell Investigator and Assistant Professor, Department of Neurology and the MIND Institute, University of California, Davis, for their proposal "Rapid Screening and Generation of Therapies for Children with Neurodevelopmental Disabilities". The researchers will receive $770,000 in combined direct cost over two years.
Neurodevelopmental disabilities appear in about 1 in 6 children aged 3–17 years, affecting brain development and leading to challenges in learning, communication, social interaction, and motor skills. They are characterized by persistent deficits in cognitive, social, motor, or adaptive functioning, and severity can vary widely among individuals.
Neurons are the building blocks of the brain. They are specialized nerve cells that receive, process, and transmit information through electrical signals generated by the movement of certain charged ions (e.g., sodium, potassium, chloride, calcium, etc.) through specialized water-filled ion channels located in their cell membranes. Such ion channels are created by a specific type of pore-forming protein that allows rapid passive movement of the charged ions in and out of the nerve cell. Selective opening or closing of such membrane channels (gating), together with interconnected networks of neurons, form the functional circuitry of the brain.
During brain development, inherited mutations present in ion channel genes may produce a loss of function, gain of function, or altered regulation of ion channel function (channelopathy) that disrupts normal neuronal circuitry and contributes to neurodevelopmental conditions, including developmental delays, intellectual disabilities, autism, and epilepsy. With more than 400 human genes known to encode ion channels and each genetic mutation affecting the characteristics of the ion channel differently, mutations may be linked to hundreds of distinct channelopathies, with each mutation altering ion channel function in a unique way.
The unmet need for children with complex behavioral and cognitive conditions calls for a rapid and effective way to screen for medical treatments tailored to an individual’s genetic, molecular, and lifestyle profile, while minimizing side effects. To address this challenge, Chatterjee and Ben-Shalom propose to use generative machine learning and brain circuit models to identify precision therapies for children with neurodevelopmental disabilities caused by ion-channel disorders, using computational models to predict, prioritize, and identify effective therapeutic drug combinations.
Computational models are computer-based simulations that mathematically replicate real-world systems in order to analyze, predict, and optimize outcomes without physical experimentation. Using computational simulations that integrate the electrophysiological properties of individual neurons with known structure and connectivity architecture in the brain, Ben-Shalom has developed biophysically detailed brain circuit models that reflect neural activity and function. The models reveal how a particular change in a neuronal ion channel will alter the electrical activity of the neuron and the circuits it belongs, as well as how a particular intervention will enable a return of ion channel function to a normal or standard condition. In effect, the models connect a specific genetic mutation to neuronal function, defining how certain brain cortical circuits will behave in terms of observed clinical symptoms.
Addressing complimentary components of the same challenge, Chatterjee has developed generative machine learning methods to design therapeutic molecules and computational methods to simulate how proteins move and change shape. His approach will predict how gene mutations alter the shape and function of ion channels and guide the design of peptides (short chains of amino acids) that could help restore normal channel activity.
The Collaboration leverages how the Chatterjee laboratory will use existing channel structures and computer simulations to predict how individual gene mutations disrupt channel function and how precision therapies might correct it, while the Ben-Shalom laboratory will determine how those molecular changes alter the firing of neurons and the activity of cortical circuits, including testing the predictions in human neurons grown from stem cells that carry the patients’ own mutation. Together, their innovative approach links channel structure, channel function, neuronal excitability, circuit activity, and therapy, so that a prediction about a child’s mutation becomes a treatment option a physician can act upon.
"Together, the two groups will create a variant-to-therapy framework that connects molecular mechanism to treatment selection and therapeutic design," said Fred Dombrose, President of The Hartwell Foundation, adding that "each laboratory will address complementary components of the same challenge by contributing technologies that solve a critical part of the problem."
Fostering collaborations between investigators of complementary scientific strengths is one of the objectives of The Hartwell Foundation in its mission to fund innovative, early-stage applied biomedical research with the potential to benefit children.
For additional information see www.thehartwellfoundation.org
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