When considering designing stem-cell-based treatments for retinal diseases, Dennis Clegg, Ph. M., is one of the go-to researchers. He not just heads his own lab at the University of California at Santa Barbara; heâs the recipient of a CIRM grant for a treatment of age-related macular degeneration, and can turn complicated technology into a compelling narrative, as this TED Talk demonstrates.
Itâs thus no real surprise that Dr. Clegg is a part of an all-star research team developing a âretinal patchâ project funded simply by FFB. He and I recently talked about his work as well because the great promise of stem-cell analysis.
Please describe the âretinal patchâ task and your role in it.
The idea is to generate a spot that would consist of a scaffold plus two different retinal cell sorts that are sometimes missing in people along with retinal diseasesâphotoreceptors and retinal color epithelium (RPE) cells. Both will be derived from induced pluripotent stem (iPS) cells, where you can take a skin biopsy and convert it into what ever kind of cell you need.
My team was the first to show you could create functional RPE cells from iPS cells. Now, weâre teaming plan Dr. David Gammâs group, which can make the photoreceptors. Then weâll attempt to combine them and basically restore the outer retina.
And youâre supervising another project, targeting ADVANCED MICRO DEVICES,
The FFB-funded project is really building on four many years of another project using human wanting stem cells to make RPE for your treatment of age-related macular degeneration. Thatâs a $20 million project financed by the California Institute for Regenerative Medicine, our state-funded stem-cell-research plan. That project involves five colleges, and our role, again, would be to make the RPE cells.
Weâre focusing on the dry form of AMD, that there is no treatment. Weâve been successful inside a rat model, and weâre today carrying out crucial studies the FOOD AND DRUG ADMINISTRATION needs to see for an application to get a Phase I clinical trial. Weâre hoping to launch that at the end of 2014, beginning of 2015.
What inspires you to do this work,
Thereâs incredible potential in stem-cell analysis to treat blinding eye diseases. The eye is a really good place to create cellular therapies, as compared to other tissue. Refined surgical techniques have already been created, and the eye is easily accessible. Thanks to sophisticated imaging technology, additionally, there are many ways to measure visual awareness to see if a treatment is operating.
Thatâs compared to treating, state, a spinal cord injury, where you provide millions of cells into a spinal cord, after which itâs hard to know the results. The eye is a really good place to start.
It also seems as if stem-cell studies advancing relatively quickly.
It is. The first human wanting stem cells were isolated within 1998, but these iPS cells had been first described in humans within 2007, so thatâs just a few years back. And already weâre pushing towards clinical trials. In fact, within Japan, theyâre going forward with a demo for the wet version of ADVANCED MICRO DEVICES. Thatâs the speed of light with regards to scientific research, when you move therefore quickly from the basic level, breakthrough in a laboratory, to application inside a clinical trial.
In developing retinal-disease treatments, what role does FFB play,
The Foundationâs function is crucial. It supports plenty of whatâs called translational, or pre-clinical, researchâwhat goes on before projects get to clinical trials. And to go from your basic discovery of making a cellular in a lab to assembling all of the data you need to present to the FOOD AND DRUG ADMINISTRATION for a clinical trial is an region where FFB provides support plus basically moves the needle ahead in very significant ways.
What do you foresee happening with possible treatments in the next decade,
Itâs a very exciting time. Not just our group but a number of other groupings are going forward with clinical studies using RPE cells for macular dystrophies. If you think about it, stem-cell research is similar to the space program within the 1960sâitâs the very early days, very fresh. Weâre making these constructs plus trying them out, and we donât understand which ones are going to be successful.
Different scaffolds are being tried, different cell outlines, different types of applications. I think itâs a terrific way to go at it. Itâs never going to happen overnight, but when we appearance back 10 years from today, weâre going to