Efforts to understand and treat degenerative eye diseases utilizing stem cell technology just got a boost. The California Institute for Regenerative Medicine (CIRM) has awarded Buck Institute faculty Deepak Lamba, MBBS, PhD, $1.5 million to create a 3-D model of the retina, the delicate, multilayered, light-sensitive membrane that lines the inner eyeball. Using a combination of stem cells and bioengineering approaches, the model will enable novel studies of retinitis pigmentosa, an inherited degenerative disease which leaves most of its sufferers legally blind by age 40.
Lamba, who was a practicing physician in India before going into research, will use scaffolds to grow eye cells from induced pluripotent stem cells (iPSCs), which are reprogramed from adult tissues. The iPSCs will be generated from both patients with the disease and from those who are disease-free. “Even though we have identified a number of gene mutations which cause retinitis pigmentosa, we do not know how those mutations ultimately lead to the loss of photoreceptor cells in the retina,” Lamba said. “Directly comparing normal and diseased retinal cells will allow us to identify the processes that ultimately lead to the cell death.” Lamba hopes to extend the work to identify new drugs which would help halt or at least slow the degenerative process.
CIRM awarded more than $38 million to a total of 28 projects as part of its Basic Biology awards program, focusing on basic research, to gain a better understanding of the different kinds of stem cells and how to work with them. “This kind of basic research is essential to helping us answer some essential questions about stem cells,” says Pat Olson, PhD, Executive Director of Scientific Studies at CIRM. “The knowledge we gain from these studies will ultimately inform other work and advance our understanding of the fundamental mechanism of stem cell biology, and move us ever closer to knowing how best to use stem cells to help patients."
Lamba, who got a Master’s Degree in Bioengineering before doing his doctoral thesis and post-doctoral work on generating and transplanting retinal cells derived from human embryonic stem cells and iPSCs, says the techniques and models developed from the three-year grant will aid research on other degenerative eye diseases. Officials from CIRM acknowledged that possibility in this grant review statement: “the proposed scaffold design is novel and represents a potentially innovative breakthrough for retinal modeling."
Lamba’s research is also focused on macular degeneration, a disease of the retina which impacts central vision. An estimated 11 million people in the United States have some form of macular degeneration, making it the leading cause of vision loss in Americans 60 years of age and older. The disease impacts the ability to read, drive and do daily activities of life. Retinitis pigmentosa affects 1 in 4,000 people and runs in families. The disease is usually diagnosed in adolescents and young adults; most patients are legally blind by middle age. “I want to thank CIRM for their support,” said Lamba. “Stem cell technology holds much promise for those suffering from degenerative eye diseases. I am committed to taking full advantage of this grant to advance efforts to find effective treatments for the diseases.”
About the Buck Institute for Research on Aging
The Buck Institute is the U.S.’s first and foremost independent research organization devoted to Geroscience – focused on the connection between normal aging and chronic disease. Based in Novato, California, The Buck is dedicated to extending “healthspan”, the healthy years of human life and does so utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and those focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related conditions such as Alzheimer’s and Parkinson’s diseases, cancer, cardiovascular disease, macular degeneration, osteoporosis, diabetes and stroke. In their collaborative research, they are supported by the most recent developments in genomics, proteomics, stem cell technology, and bioinformatics. For more information: www.thebuck.org.