The Scleroderma Foundation has awarded eight research grants for projects seeking to advance personalized medicine, lab-grown skin, new therapeutic targets, and more, in the field of scleroderma research.
“The proposals from these scientists received the highest ranking from the Peer Review committee for quality of scientific design and the prospect of advancing the body of knowledge on scleroderma,” the foundation stated on its website.
Each grant supports the associated project for two to three years.
Stephanie Stanford, PhD, an investigator at the University of California at San Diego, won a three-year grant to develop a method for analyzing the gene activity changes that occur in small areas of a patient’s skin.
This method, called laser capture microdissection-RNA sequencing, uses a laser to cut out small portions of skin. The samples are analyzed to understand disease-promoting signals in blood vessels, signals that are thought to induce the production of excessive collagen — a hallmark of scleroderma — in nearby cells.
Jonathan Garlick, PhD, from Tufts University School of Dental Medicine, won a two-year grant, with which he aims to create “lab-grown skin” using cells from people with scleroderma. Garlick’s team will evaluate how similar this artificial skin is to patient skin. In contrast to existing approaches that use single skin layers on lab dishes, this project intends to grow skin with four different cell types in the hopes that the lab-grown skin can help scientists to study scleroderma and test potential therapies.
Ido Amit, PhD, from the Weizmann Institute of Science in Israel, won a two-year grant to use single-cell sequencing to explore the mechanisms leading to scleroderma. By analyzing individual cells, Amit hopes to identify rare populations in patients’ skin and blood, which might help find biomarkers for early disease detection and identification of new therapeutic targets. The project will study cells at a much greater resolution than possible before, according to Amit.
Another emerging single-cell technology may enable Mengqi Huang, PhD, of the University of Pittsburgh, to investigate small numbers of patient-derived endothelial cells — those lining blood vessels — without significantly altering their genetic and epigenetic features. Epigenetics refers to changes in how genes work that do not alter the underlying DNA sequence.
More “traditional” laboratory techniques typically require larger quantities of these cells, which, in the case of scleroderma, had to be collected from difficult-to-access patient tissues. In addition, these methods were prone to inducing changes to cells.
Huang intends to use her three-year grant to apply more precise technologies to the collection, growth, and analysis of endothelial cells from scleroderma patients. Her research may help develop therapies that target those cells specifically.
Likewise, the other four grantees will use their funds to explore other aspects of scleroderma biology, in pursuit of safe and effective treatments for this group of disorders.
Among them is Kimberly Showalter, MD, from the Hospital for Special Surgery, who received a three-year grant to study if fibroblasts — a key cell type in scleroderma that produces collagen — can help predict clinical improvement in response to treatment. Ultimately, this may help offer treatment to people more likely to benefit, and spare from toxic effects those unlikely to improve.
Fibroblasts will also be the focus of Harry Karmouty-Quintana, PhD, at University of Texas Health Science Center at Houston. His team will study if two genes, SIX1 and EYA2, drive the excessive activity of fibroblasts responsible for lung scarring. This could help develop new medicines to stop lung scarring in scleroderma, according to the researchers.