![[Lalitha Madhavan, MD, PhD, in her lab at the University of Arizona College of Medicine – Tucson]](https://deptmedicine.arizona.edu/sites/default/files/styles/medium_image/public/madhavan-slide.jpg?itok=l1je8vB- "Lalitha Madhavan, MD, PhD, in her lab at the University of Arizona College of Medicine – Tucson")
Parkinson’s disease (PD) 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 PD symptoms are available, no treatments exist to slow or prevent the progression of the disease. One major roadblock in developing such disease-modifying treatments has been an inadequate understanding of how PD develops.
That soon may change, as University of Arizona physician-turned-researcher Lalitha Madhavan, MD, PhD, is studying novel stem cell biology and its applications toward understanding and treating neurodegenerative disorders like Parkinson’s disease.
Dr. Madhavan, an assistant professor in the Department of Neurology in the UA College of Medicine – Tucson and head of the UA Stem Cells and Neurodegeneration Lab, always dreamed of being a physician. After completing medical school, for several years she was involved in clinical practice where she supported the care of patients with degenerative brain diseases like Parkinson’s and Alzheimer’s disease. After seeing the same patients deteriorate year after year, Dr. Madhavan realized that as a physician she had limited means to help her patients.
Driven by the desire to better understand the biological progression of neural degenerative diseases in order to develop truly curative therapies – instead of medications that only treat symptoms – Dr. Madhavan went back to school to get her PhD and pivoted from the clinic to the research bench.
Today, Dr. Madhavan’s lab is one of only a few in the United States 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 (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 stem cells into replicating the qualities of embryonic cells through genetic reprogramming provides incredible potential for innovation in regenerative medicine.
There long has been speculation about the ethics of using embryonic stem cells for scientific research, despite their potential for innovation in regenerative medicine. However, with the invention of iPSC the ethical argument becomes obsolete.
Dr. Madhavan recognized the implications of this discovery and applied for a grant to establish the technology for her 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 in her PD-related research. Recently, she also received funding from the Jim Himelic Foundation to extend this technology to studying another deadly neurodegenerative disease, amytrophic lateral sclerosis (ALS), or Lou Gehrig’s disease.
“We are the only lab in Arizona that is doing this type of work,” said Dr. Madhavan, also a member of the UA 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 PD and give rise to debilitating motor symptoms.
Dr. Madhavan’s research involves taking skin samples from patients with and without PD and generating relevant cells, such as dopamine neurons, to observe how cells from PD-affected and unaffected individuals differ. From these observations, Dr. Madhavan is gaining insight into the inner workings of the disease that she was unable 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,” Dr. Madhavan explained.
There are very specific advantages to using stem cells and iPSC technology, she says. “By modeling underlying cellular mechanisms, we are better able to understand the causes of the disease,” she said. Through working with this technology, she firmly believes significant advances in developing definitive cures for neurodegenerative diseases like PD will occur in the near future.
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 to develop personalized health solutions for PD.
Ultimately, Dr. Madhavan’s most ambitious goal is to develop a stem-cell based curative therapy via transplant that would replace, or support the survival of, the dopamine neurons and other relevant brain cells in patients suffering from PD. This treatment, particularly when applied early, would support recovery in PD patients by augmenting cellular life and preventing the progressive deterioration of brain cells by replacing or repairing them completely.
Dr. Madhavan is eager to share her knowledge of iPSC technology, believing it will spark the interest of 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,” Dr. Madhavan said. “It’s a friendly place that allows for ‘cross-pollination’ among different disciplines and researchers.”
Individuals interested in learning more or supporting Dr. Madhavan’s research should contact her at lmadhavan@email.arizona.edu or 520-626-2330.
About the University of Arizona BIO5 Institute
The BIO5 Institute at the University of Arizona mobilizes top researchers in agriculture, engineering, medicine, pharmacy and science to find creative solutions to humanity’s most pressing health and environmental challenges. Since 2001, this interdisciplinary approach has been an international model of how to conduct collaborative research, and has resulted in improved food crops, innovative diagnostics and devices and promising new therapies. Learn more at http://BIO5.org
About the University of Arizona Health Sciences
The University of Arizona Health Sciences is the statewide leader in biomedical research and health professions training. The UA Health Sciences includes the UA Colleges of Medicine (Phoenix and Tucson), Nursing, Pharmacy and Mel and Enid Zuckerman College of Public Health, with main campus locations in Tucson and the growing Phoenix Biomedical Campus in downtown Phoenix. From these vantage points, the UA Health Sciences reaches across the state of Arizona and the greater Southwest to provide cutting-edge health education, research, patient care and community outreach services. A major economic engine, the UA Health Sciences employs almost 5,000 people, has nearly 1,000 faculty members and garners more than $126 million in research grants and contracts annually. For more information: http://uahs.arizona.edu
Media Contact: Lisa Romero
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“UA physician-scientist tackling neurogenerative disorders” (KOLD-TV 13News Tucson) | Posted April 28, 2016