# Accelerating Functional Maturation of Human Neurons for High-Throughput Screening

> **NIH NIH R44** · BRAINXELL, INC. · 2020 · $609,162

## Abstract

Project Summary/Abstract
 Neurological and psychiatric disorders, including drug abuse and addiction, exert a devastating
personal and economic toll on patients, families, caregivers, and society. This is in part due to our failure in
developing effective medications, which in turn is due to the use of drug discovery platforms that are often not
relevant to target diseases. The recent development of induced pluripotent stem cells (iPSCs) from humans
makes it possible to screen and validate candidate compounds on human neurons, including those from
patients, thus potentially increasing the success rate and speeding up the pace of CNS drug development.
 BrainXell, Inc. has been a pioneer in developing human patient neural cell-based platforms for CNS
drug discovery. We are able to produce large quantities of highly enriched, functionally specialized neurons of
consistent quality from human iPSCs through our platform technology of directed neural differentiation and
expansion of committed progenitors. However, iPSC-derived neurons are immature, comparable to those at
the fetal stage, which makes it difficult for presentation of disease phenotypes and for high-throughput
screening (HTS) for drug leads intended for those whose brains are fully mature. In our Phase I project, we
identified several molecules that accelerate the expression of synaptophysin (SYP) expression specifically in
cortical glutamatergic (Glut) neurons or spinal motor neurons (MN), and formulated Glut and MN maturation
supplements that yield mature neurons expressing synaptic markers within a week.
 In this Phase II project, we propose to develop maturation supplements for the other two neuron
subtypes, cortical GABAergic neurons and midbrain dopaminergic neurons. The overarching goal of this
proposal is to accelerate neuronal maturation while maintaining a highly pure and homogenous population of
neurons, which would allow the practical use of mature human neurons in HTS for neuron activity and function.
Formulation of an effective cocktail for rapidly generating human mature neurons and determining the optimal
conditions for its application will remove the major roadblock in establishing human patient neuron-based HTS
for CNS drug discovery.

## Key facts

- **NIH application ID:** 10017190
- **Project number:** 5R44DA042659-03
- **Recipient organization:** BRAINXELL, INC.
- **Principal Investigator:** Zhong-wei Du
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $609,162
- **Award type:** 5
- **Project period:** 2016-09-15 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10017190

## Citation

> US National Institutes of Health, RePORTER application 10017190, Accelerating Functional Maturation of Human Neurons for High-Throughput Screening (5R44DA042659-03). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10017190. Licensed CC0.

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