UA Researcher Uses Adult Stem-Cell Technology
April is Parkinson’s Awareness Month, and innovative research by Dr. Lalitha Madhavan is working toward a cure for the neurological and progressive disorder that affects an estimated 7 million to 10 million people worldwide.

By Lisa Romero, BIO5 Institute
April 14, 2016


Dr. Lalitha Madhavan: "I really enjoyed my job as a physician, but I knew that if I really wanted to make a difference and change things, I needed to pair it with research."
Dr. Lalitha Madhavan: "I really enjoyed my job as a physician, but I knew that if I really wanted to make a difference and change things, I needed to pair it with research."

Parkinson’s disease is a common age-related neurological disorder that affects about 1.5 million people in the United States alone. Although drugs and surgical approaches that alleviate its symptoms are available, no treatments can slow or prevent the progression of the disease.

One major roadblock in developing such disease-modifying treatments has been an inadequate understanding of how Parkinson's develops. That soon may change, as a University of Arizona physician-turned-researcher, Dr. Lalitha Madhavan, is studying novel stem-cell biology and its applications toward understanding and treating neurodegenerative disorders such as Parkinson's. 

Madhavan, assistant professor in the Department of Neurology in the UA College of Medicine – Tucson and head of the Stem Cells and Neurodegeneration Lab at the UA, always dreamed of being a physician. After completing medical school, she was involved in clinical practice for several years, supporting the care of patients with degenerative brain diseases such as Parkinson’s and Alzheimer's. After seeing the same patients return year after year continually deteriorating, Madhavan realized that she was limited in helping her patients. 

Driven by the desire to better understand the biological progression of neural degenerative diseases in order to develop curative therapies instead of medications that treat only symptoms, Madhavan went back to school to get her Ph.D. and pivoted from the clinic to the research bench.

Today, her lab is one of only a few in the U.S. using a novel methodology that takes easily obtainable adult cells, such as skin cells (fibroblasts), and reprograms them back to an embryonic-like state from which they can be differentiated into any cell type in the human body.

This method is known as induced pluripotent stem cell, or iPSC, technology and was introduced by scientists John B. Gurdon and Shinya Yamanka, who won the 2012 Nobel Prize in Medicine for their findings. Their discovery of how to manipulate adult cells into replicating the qualities of embryonic cells through genetic reprogramming provides incredible potential for innovation in regenerative medicine.

There has long been speculation about the ethics of using embryonic stem cells for scientific research, despite their potential for innovation in regenerative medicine. However, through the invention of induced pluripotent stem cells — adult stem cells that replicate the qualities of embryonic cells through genetic reprogramming — the ethical argument becomes obsolete.

Pilot Grant for Technology

Madhavan recognized the implications of this discovery, and she applied for a grant in order to establish the technology for her specific work in neural degeneration at the UA. She received a pilot grant from the Arizona Center for the Biology of Complex Diseases and implemented the technology into her Parkinson's-related research. More recently, she also has received funding from the Jim Himelic Foundation to extend this technology in studying another neurodegenerative disease, Amytrophic Lateral Sclerosis, or ALS.

"We are the only lab in Arizona that is doing this type of work," said Madhavan, also a member of UA’s BIO5 Institute. "It is a very unique, cutting-edge technology because it allows scientists to obtain embryonic cells without actually using embryos."

In the Madhavan lab, iPS cells represent a virtually unlimited source for generating disease-relevant neuronal cells such as dopamine neurons, which degenerate in patients suffering from Parkinson's and give rise to debilitating motor symptoms.

Madhavan’s current research involves taking skin samples from patients with and without Parkinson's, and generating relevant cells such as dopamine neurons, in order to observe how cells from Parkinson's-affected and -unaffected individuals differ.

From these observations, Madhavan is gaining insight into the inner workings of the disease that she never was able to obtain while practicing medicine.

"I really enjoyed my job as a physician, but I knew that if I really wanted to make a difference and change things, I needed to pair it with research," she said.

Madhavan said there are specific advantages to using stem cells and iPSC technology.

"By modeling underlying cellular mechanisms, we are better able to understand the causes of the disease," she said.

Through working with this technology, she has come to believe that there will be significant advances in developing definitive cures for neurodegenerative diseases in the near future.

A System for Testing Drugs

In particular, the initial human fibroblasts and iPS-derived cells will provide a powerful system to test promising drugs, as well as generate specific cell types that can be implanted into afflicted patients in order to develop personalized health solutions for Parkinson's.

Ultimately, Madhavan’s hope is to be able to develop a stem-cell-based curative therapy done via transplant that would replace or support the survival of the dopamine neurons and other relevant brain cells in patients suffering from Parkinson’s. This treatment, particularly when applied early, would support recovery in Parkinson's-affected individuals by augmenting cellular life and preventing the progressive deterioration of brain cells by replacing or repairing them completely.

Madhavan is eager to share her knowledge of induced pluripotent stem-cell technology, hoping that it will spark interest in other scientists working in translational regenerative medicine.

"The best thing about the University of Arizona is that there is a lot of absolutely high-impact science and it’s easy to collaborate and communicate with people," Madhavan said. "It’s a friendly place that allows for cross-pollination between different disciplines and researchers."

Extra info

Those interested in learning more or supporting Dr. Lalitha Madhavan’s research should contact her at or 520-626-2330.




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Lisa Romero

BIO5 Institute