Research Consortium Receives $1.6M to Develop Stem Cell Therapies for Skin Diseases

The Epidermolysis Bullosa iPS Cell Consortium has received $1.6 million in funding to develop stem cell-based therapies for people with inherited skin diseases such as epidermolysis bullosa (EB).

A grant from the National Institutes of Health (NIH) 21st Century Cures Act provided $800,000, which was quickly matched by a group of private foundations.

The grant follows prior awards given to the same team by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the NIH 21st Century Cures Act, and the U.S. Department of Defense. Since the initial NIAMS grant of $500,000 in 2017, the consortium has received more than $12 million.

“In drafting 21st Century Cures, our goal was simple: We wanted to find ways to safely speed up the development of new treatments and cures for some of the world’s most vexing medical challenges,” U.S. Rep. Diana DeGette, a main architect of expanded federal research funding, said in a press release.

The funding will help the consortium — a collaboration between the Gates Center for Regenerative Medicine at the University of Colorado, and both Stanford and Columbia universities — develop innovative treatments for EB and other skin diseases.

One of the novel treatments, developed outside of the consortium by the Gates Center team in collaboration with AVITA Medical, uses AVITA’s RECELL system technology to grow a suspension of “Spray-On Skin Cells” from a small sample of the patient’s own skin. The cells have the capacity to regenerate the outer layer of the skin, and can be prepared and applied in as little as 30 minutes.

The U.S. Food and Administration approved this delivery method in September 2018 for the treatment of severe thermal burns. In the case of EB patients, it may replace the use of genetically-corrected sheets of skin, which can be costly, carry transport challenges due to fragility, and take several weeks to produce.

“We very much look forward to collaborating with the Gates Center for Regenerative Medicine on this important research program,” said Mike Perry, CEO at AVITA and a professor at the Gates Center. “We are hopeful that the results of this collaboration will yield a significant advancement in the treatment of patients with EB.”

Future strategies include developing a safe and effective way of delivering regenerative cells to help repair scar tissue caused by EB in the esophagus and other internal parts of the body.

The consortium was co-founded in 2016 by the EB Research Partnership in New York and the California-based EB Medical Research Foundation (EMRF).

“We are extremely proud to have supported the EB iPS Cell Consortium since its inception,” said EMRF’s Andrea and Paul Joseph, whose son Brandon has EB. “The NIAMS’s ongoing funding is further validation of the incredible advancement the Consortium is making to develop a treatment for those living with EB.”

Alex Silver, chairman of the EB Research Partnership, said: “This Consortium was conceived to allow three leading EB research teams to come together to find a treatment or cure faster than any individual research approach we’ve seen. When time is the most precious resource of any child or adult living with EB, progressive collaboration like this is increasingly important.”