# Assessment of Chemotherapy-Induced Peripheral Neuropathy Susceptibility Using Patient-derived iPSC Technology

> **NIH NIH R01** · MAYO CLINIC ROCHESTER · 2020 · $363,713

## Abstract

PROJECT SUMMARY/ABSTRACT
Chemotherapy-induced peripheral neuropathy (CIPN) is a serious side effect that causes morbidity
and limits the dose of chemotherapy allowed to treat cancers. Of those receiving neurotoxic
chemotherapy, approximately 30-40% of patients develop CIPN, yet the risk factors for developing
this are poorly understood. The goal of this project is to test whether susceptibility to CIPN can be
predicted in vitro by employing our novel CIPN-in-a-dish neurotoxicology assay that uses iPSC-
derived sensory neuron from patient samples. In the first Specific Aim, sensory neurons (iSN) will
be derived from patients with Charcot-Marie-Tooth disease (hereditary peripheral neuropathy).
First, the CIPN-in-a-dish assay will be used to compare susceptibility between iSN from CMT
patients and healthy controls. Subsequently CMT samples will have their deleterious gene mutation
corrected using gene-editing technology (CRISPR/Cas) and CIPN susceptibility will be compared
between the pathologic iSN and gene-corrected iSN. In the second Specific Aim, iSN will be derived
from a cohort of patients with breast cancer that have received standard adjuvant paclitaxel
chemotherapy. Again using the CIPN-in-a-dish assay, iSN from patients that have clearly
developed CIPN from paclitaxel will be compared in a blinded fashion with patients that clearly
have not. These studies will serve two important functions: 1) they are a “proof-of-principle” study
that determines whether this approach using patient samples can be used to predict CIPN in
individual patients, 2) a hypothesis-generating study wherein patient samples will allow for
directed studies of mechanisms of CIPN susceptibility. The potential future application of this
technology will be to use a patient's own neurons to determine their susceptibility to the neurotoxic
effects of specific chemotherapy, thus allowing for personalized precision medicine for the patient
with cancer.

## Key facts

- **NIH application ID:** 9991777
- **Project number:** 5R01CA211887-04
- **Recipient organization:** MAYO CLINIC ROCHESTER
- **Principal Investigator:** Nathan P Staff
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $363,713
- **Award type:** 5
- **Project period:** 2017-09-18 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9991777

## Citation

> US National Institutes of Health, RePORTER application 9991777, Assessment of Chemotherapy-Induced Peripheral Neuropathy Susceptibility Using Patient-derived iPSC Technology (5R01CA211887-04). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/9991777. Licensed CC0.

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