Our goal is to speed the development of stem cell treatments for the many blinding diseases that affect the eye and specifically the retina, such as age-related macular degeneration (AMD). Cell transplantation has been attempted for 2 decades without real success. This scientific project will help fill gaps in our knowledge in two areas. 1) It will help us answer how the stem cells get to where they where they need to be to help the eye. 2) How do we make the cells stay (for a long time) and work where need to be. We feel the immune systems reaction to the cells will play a big role in answering these two question. Plus we will do our research in such a way we will meet the standards mandated by the Food and Drug Administration for use in human treatments. This will help us move forward quickly to the treatment of real vision loss in patients. Lastly we will compare stem cells from different sources to find out which source will be the best for eye and retina disease.
To answer the question how do the cells get to where they need to be, we have found a way to study cell movement in the eye. This new model allows us to study cells as the move to the retina. Using this model, we can figure out how our delivery of the cells to the eye affects where they ultimately end up in the retina. Also we will see how different types of stem cells may travel more appropriately than others, which will help us chose the best ones for patients. Plus we feel that the immune system whose job it is to keep bad germs away from us, also will influence how these cells travel.
We also want to find what keeps the cells where they need to be. We think the stem cell type may make a difference and again we think the immune system will play a role in their long-term survival. Our studies will be aimed at understanding these things that keep the cells from staying where they need to be. We will take steps to figure out the specific proteins or molecules that are involved. If we can figure this out we can take action to change those factors, and provide a way for long-term survival of the cells in the retina.
Blindness is tragedy, which more and more Americans suffer with. We believe that stem cell transplantation to the eye and retina could prevent and in some cases reverse blindness. Filling these gaps in our knowledge regarding stem cell movement and how the immune system affect both movement and survival will allow us to advance towards our goal of treating blind patients in the near future.
Statement of Benefit to California:
Vision loss to an individual can be catastrophic. Blindness can be overwhelming, robbing one of independence, hindering communication, and worsening other health care problems. Vision loss can also prevent gainful employment, as only 1 out of 3 visually impaired people of employment age are in the workforce. In the United States, blindness is one of the public’s biggest health fears only behind cancer and AIDS. Vision loss increases with age as over 20% of those over 65 of age report vision loss. In California, 16% (1.7 million) over 45 years of age have vision loss, and as the Baby Boomers generation continues to age, these numbers are expected to greatly increase. In fact, the Eye Diseases Prevalence Research Group estimates that by the year 2020 the percent of Americans blind will increase by 70%.
Problems with the retina encompass the great majority of the reasons for blindness and vision loss in the American population. For those 60 years and over, age-related macular degeneration is the leading cause of vision loss. For people between the age of 25 and 74, the leading cause of blindness is damage to the retina caused by diabetes (diabetic retinopathy). Even for infants, the leading cause of blindness is retinopathy of prematurity.
We believe our proposed studies will be of significant value to Californians. Our goal is to speed the development of stem cell therapies for the many blinding diseases of the retina. First, funding this project will help establish California as a leader in retinal stem cell therapies. These advances will allow Californian patients with many blinding disorders to benefit by directly participating in the clinical trials. In addition, answers found in these studies of the eye will apply to other stem cell treatments. Moreover, as retinal problems are common in Californians, the number of Californians that could directly benefit is great. Our proposed projects are focused on the application of human stem cells towards human retinal disease, specifically addressing how stem cells get to where they need to be in the eye what keeps them from staying there. In addition, we will study how the immune system affects the transplantation of these cells and how cell changes (differentiation) makes them more or less successful in migration and survival. Understanding these immune responses will be critical for the success of most future stem cell treatments.
Finally, many physicians and even moreso retinal specialists do not stay in academics and research because the financial and personal benefits of private practice are great and research funding continues to decline. These realities mean that there is not an abundance of physicians that commit their careers towards improving eye care in fields such as retinal stem cell therapies. This type of funding of a physician-scientist in the study of retinal disease will help reverse this trend here in California.