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For first time, drug developed based on zebrafish studies passes Phase I clinical trial
Date:10/21/2013

Boston, Mass., October 18, 2013 Zebrafish research achieved a significant milestone when the first drug developed through studies utilizing the tiny animal and then put into clinical trials passed a Phase 1 trial aimed at establishing its safety. The drug, discovered in the laboratory of Leonard Zon, MD, at Boston Children's Hospital, has already advanced to Phase II studies designed to determine its efficacy.

Results of the safety trial were reported recently in the journal Blood. At only six years after Zon's laboratory reported the discovery of the chemical from which the drug is derived, the Phase 1 data underscore the potential of zebrafish research to accelerate the journey from bench to bedside.

"The zebrafish is a very good system for evaluating potential drugs," Zon said. "When you discover a new treatment option and can see it go into patients, it's quite a remarkable feeling."

The drug, which is being developed by Fate Therapeutics under the name ProHema, is the result of the Zon laboratory's search for a way to improve the success of hematopoietic stem cell (HSC) transplants using umbilical cord blood. ProHema is a chemical derivative of prostaglandin E2 (PGE2) that, according to preclinical and clinical data, might improve engraftment of transplanted umbilical cord blood cells by helping donated cells home in on the bone marrow.

Although umbilical cords are an effective transplant source in patients for whom a suitable donor cannot be found, a single umbilical cord rarely contains enough HSCs for a transplant for an adult patient. The current method is to use two cord blood units per transplant, raising the risk that the immune cells that arise from the two cords may start to attack each other. In addition, umbilical cords are expensive and in limited supply.

This problem has led Zon, a co-author of the Blood study, and other researchers to search for molecules that could help expand co
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Contact: Irene Sege
irene.sege@childrens.harvard.edu
617-919-7379
Boston Children's Hospital
Source:Eurekalert  

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For first time, drug developed based on zebrafish studies passes Phase I clinical trial
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