Cellular pharmacology involves the study of drug action on complex cellular functions. Scientists at the Indiana Biosciences Research Institute (IBRI) use this technology to investigate disease processes and evaluate the potential of therapeutic molecules.
Our cellular pharmacologists conduct in vitro (outside of a living organism) studies on cells to:
- Understand the biochemical and physiological actions of drugs, which act on cellular signaling pathways.
- Investigate how drug molecules affect cellular responses (in vitro).
- Gather quantifiable information on compound efficacy, potency and side effect liabilities.
- Help prioritize molecules for in vivo studies.
Cellular pharmacology projects at the IBRI
The development of new disease-fighting drugs requires a multidisciplinary team effort. The IBRI teams work collaboratively to advance drug discoveries on a growing portfolio of programs.
Accelerating therapies for Alzheimer’s disease
IBRI cellular pharmacologists are key players in an Alzheimer’s disease research initiative called Target Enablement to Accelerate Therapy Development for Alzheimer’s Disease (TREAT-AD). Our work is part of the Indiana University (IU) School of Medicine-Purdue University TREAT-AD Drug Discovery Center.
IBRI scientists are developing therapies to test hypotheses for regulating microglia (immune cells in the brain) function that contribute to neuroinflammation in Alzheimer’s disease. Through this collaboration, IBRI scientists are expanding on biology and clinical research to create potential new therapeutic molecules and share findings with the broader Alzheimer’s disease research community.
Seeking treatments for neurofibromatosis type 2 (NF2)
In collaboration with Riley Children’s Health at IU Health, IBRI cellular pharmacologists are exploring ways to target a gene mutation that causes NF2.
This rare pediatric disease causes benign (noncancerous) tumors to grow along the auditory nerve that carries information between the brain and the inner ear, leading to hearing loss, tinnitus and dizziness. This disease does not have any highly effective treatments or a cure, and surgical removal of the tumor often leads to serious consequences.
The cellular pharmacology team at the IBRI is using in vitro cellular bioassays to test the efficacy of potential drug molecules on multiple cell signaling pathways that cause tumors to form in children with NF2. We are studying drugs individually and in combination with the hope of finding regimens with improved efficacy and safety.
Using human iPSCs to find answers and create personalized medicine
Successful cellular pharmacology drug discovery efforts depend on appropriate human cell models. For example, in Alzheimer’s disease, human microglia play a critical role in the disease proinflammatory response. However, it is difficult to obtain sufficient primary cells from human subjects to carry out in vitro studies.
To address this, IBRI is employing human induced pluripotent stem cell (iPSC)-derived cell lines as an in vitro neuroinflammation model of human disease. These cells are generated and validated in the IBRI iPSC Lab and then the effects of potential drug candidates on multiple endpoints related to cellular signaling and functions are evaluated.
Because iPSCs are derived from individual patients, measured responses are reflective of their unique genetic background and disease. Studies using these cell lines can lay the foundation for personalized therapeutics.