Navigation Links
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 cord blood stem cells or improve the efficiency of cord blood transplants and eliminate the need for cells from a second cord.

Zon's laboratory discovered PGE2's properties after screening 2,500 chemicals for their effects on blood stem cell production in zebrafish, a popular and cost-effective research model for stem cell, genetic and developmental research. Not only are zebrafish genes surprisingly similar to human genes, but they can be inexpensively housed at high densities and female zebrafish lay 300 eggs per week, making them a promising vehicle for quickly and cheaply discovering new drugs.

Zon and his colleagues reported their initial PGE2 findings in Nature in 2007.

"We think PGE2 acts as a kind of priming mechanism," Zon said. "It gets the cell set so that it will function better once it is introduced into the recipient's body." Subsequent preclinical studies showed that PGE2 can trigger a four-fold increase in efficiency of stem cell engraftment, compared to untreated controls, by helping stem cells home more effectively to the bone marrow.

The Phase I trial of ProHema, the drug derived from PGE2, was launched in 2009 at Dana-Farber Cancer Institute (DFCI) and Massachusetts General Hospital under the direction of DFCI's Corey Cutler, MD, MPH. It showed that treatment of donated umbilical cord blood stem cells with the drug before transplant was safe. In addition, treated cells could engraft and rebuild a patient's blood system more quickly than untreated ones.

"These are very promising results," Cutler said. "They suggest that by generating more effective stem cells, we might be able to lower the dose of stem cells needed for a successful transplant. And because this approach takes substantially less time than techniques that increase the number of stem cells prior to transplant, it can easily be performed by most stem cell-processing facilities."

Because the Food and Drug Administration has already approved PGE2 for other uses, researchers were able to move quickly into clinical trials. The Phase II study is underway at seven institutions nationwide.


'/>"/>

Contact: Irene Sege
irene.sege@childrens.harvard.edu
617-919-7379
Boston Children's Hospital
Source:Eurekalert  

Related medicine news :

1. First targeted nanomedicine to enter human clinical studies
2. First contact: Early intervention key in diagnosis and treatment of serious mental illness
3. Donor Kidney Re-Used in Second Patient After Failing in First
4. Many Who First Misuse Prescription Pills Get Them From Friends, Family: Report
5. First-of-its-kind Menopause Map helps women navigate treatment
6. First recipients of AcademyHealths Presidential Scholarship announced
7. Kessler Foundation researchers present at first International Congress on Cognition in MS
8. Whos the Dad? First-Trimester Blood Test May Tell
9. AGA releases first independently developed ABIM-approved Practice Improvement Module in GI
10. Dr. Yael Mosse will receive first Nachman Award in Pediatric Oncology at national conference
11. First study investigating possible link between sunscreen ingredient and endometriosis
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
For first time, drug developed based on zebrafish studies passes Phase I clinical trial
(Date:2/7/2016)... Michigan (PRWEB) , ... February 07, 2016 , ... ... organ prolapse with the latest techniques and the most minimally invasive approaches. , ... organ prolapse, particularly after menopause. Other risk factors include surgery to the pelvic ...
(Date:2/7/2016)... Birmingham, Lake Orion, Clarkston, Metamora, Michigan (PRWEB) , ... February 07, ... ... women on Friday in recognition of National Wear Red Day. National Wear Red ... heart disease and stroke in women. Heart disease and stroke cause 1 in 3 ...
(Date:2/7/2016)... ... February 07, 2016 , ... HealthSmart Holdings, Inc. ... organizations with the tools and information to lower the costs, and increase the ... cost of providing employee healthcare benefits by as much as 22%:, + ...
(Date:2/7/2016)... ... February 07, 2016 , ... ... Plastics, has added Kybella® to his medical and surgical expertise. Technically known as ... FDA injectable medication used as a non-surgical alternative for reduction of fat below ...
(Date:2/6/2016)... ... February 06, 2016 , ... ... EXPECTED AT AORN SURGICAL CONFERENCE & EXPO , WHAT:     , This conference ... estimated 5000 perioperative nurses in attendance to study the latest evidence-based recommendations ...
Breaking Medicine News(10 mins):
(Date:2/5/2016)... CHAPEL HILL, N.C. , Feb. 5, 2016 /PRNewswire/ ... sector has been slower than other industries to embrace ... that come with utilization. On the medical side, organizations ... to improve everything from clinical trials to adherence. ... recent research from benchmarking firm Best Practices, LLC, Big ...
(Date:2/5/2016)... , Feb. 5, 2016 Amgen (NASDAQ: ... Annual Global Healthcare Conference at 9:15 a.m. ET on ... . David W. Meline , executive vice president ... conference. Live audio of the presentation can be accessed from ... Investors. A replay of the webcast will also be available ...
(Date:2/5/2016)...  Redwood Scientific Technologies, Inc. announced today the development ... women balance their hormones. This product will be featuring ... Jason Cardiff , President and CEO. "I am proud ... millions of women across the country and around the ... Our research and development team is confident that through ...
Breaking Medicine Technology: