Page 18 - Gates-AnnualReport-2019

There is no current treatment or cure for EB, only attention to its symptoms. Research has focused on both external applications of materials that could restore skin cells, or injections of modified cells that might eventually override the patient’s damaged cells.
The Gates Center has grants to work in both areas and more. In its second 21st Century Cures Act award, the Center said “the funds will advance innovative Consortium research and expansion of treatment in areas including a potential “Spray- On SkinTM” application of regenerative cells, and a promising treatment of internal diseased tissue with systemically targeted-delivery cells.”
Spray-on delivery of new cells “has the potential to revolutionize treatment” for kids with EB, Roop said.
Still, even if proven successful, transforming significant portions of skin will be time-consuming. That’s why researchers also are zeroing in on internal delivery of modified cells that would travel through the blood stream to damaged cells and replace them.
“We’re going for the moon shot, we’re going for the complete cure,” Roop said. Moreover, knowledge gained in EB research has already and will continue to inform other medical disciplines – new methods of skin growth and targeted cell delivery could help millions.
Preparing for meetings with EB parents, Roop sometimes has to temper those words in his own head. “You realize you have to be very very cautious, and very realistic. The FDA is very cautious. And it always takes longer than the parents want,” Roop said. “We don’t know exactly what the timeline is going to be to get FDA approval. I’m an eternal optimist by nature, and I’m sure it shows when I start talking, and parents get excited.”
Human clinical trials of promising EB treatments are still some time away. The approval process may seem even slower than on other new drugs, as therapies that involve altering cells may need longer observation times to ensure there are no unwanted cell mutations.
At the Attars’ meeting with Gates researchers and their primary EB clinician, Dr. Bruckner, Chris Attar mentioned that at their last research update, they had looked into a
Music therapy sessions for Brady in the living room often turn into a family jam session with brother Leo and more.
Brady shows off his improved walking skills with caregiver Kristina Byrne, RN, in between clinic exams.
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