Importantly, some of the work supported by the grant will address the fundamental question of the underlying mechanisms of the proposed therapy.
"We want to learn how these cells do what they do," says Friedlander. "By understanding the molecular events initiated by these cells, we hope we might improve upon the individual cells themselves."
One intriguing possibility that will be explored under the grant is that stem cells could be engineered to express additional therapeutic molecules to further enhance neuroprotection or to interfere with the proliferation of blood vessels. If successful, this approach could have broad treatment implications not only for eye disease, but also for some types of tumors. "We know that these particular stem cells like to target certain connective tissue cells of the central nervous system (CNS) called glia," adds Friedlander. "It turns out that most neovascular eye diseases have an associated gliosis (proliferation of glial cells) and we can exploit this affinity by targeting the injected cells to these sites. Glioblastoma multiforme, a highly malignant brain tumor, is another CNS disease to which we can target these cells. In collaboration with neurosurgeon Faith Barnett, we have shown that it may be possible to use these cells to deliver potent anti-angiogenic molecules to the tumor and, thus, kill it."
A major strength of the new grant is bringing together expertise from a variety of fields represented in different Scripps Research labs to address the use of stem cell therapy for treating vision loss. The project combines expertise from the Friedlander lab in retinal vascular and degenerative biology, stem cells, and disease; from the Torbett and Crisa labs on differentiation and transcriptional regulation of myeloid progenitor cells; from the Ruf lab in vascular endothelial protease and prote
Contact: Keith McKeown
Scripps Research Institute