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Posted On: 08/30/2017 3:33:13 PM
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Dr. Urano's department gets a $46M NIH grant. Here's the part on him. Recall last week Senator Roy Blunt of Missouri and NIH visited his lab. Shout out to Missouri coming up big lately in our investment world. Chemia corp too.
Another example of the breadth of ICTS’ success is the recent development of a translational pipeline for the treatment of Wolfram syndrome.
The syndrome is a rare cause of diabetes and visual impairment in children that also affects many other organs, including the brain. With ICTS support, Washington University researchers led by Fumihiko Urano, MD, PhD, created a clinical and translational research program to study the disease, which led to fundamental laboratory studies that uncovered the molecular genetic defect, and ultimately to the identification of a therapeutic target that is now being tested in patients [dantrolene]. According to Urano, the Samuel E. Schechter Professor of Medicine, national resources offered through the NCATS Rare Disease Program combined with patient organizations and local ICTS support have directly supported the first clinical trial of a repurposed drug for the treatment of Wolfram syndrome.
A new grant led by the Urano team and funded by NIH/NCATS will combine elements of collaborative preclinical science with the national drug development team at NCATS, along with later clinical trial staging. The novel bench testing approach paired with therapeutic strategies will deliver personalized medicine for patients with Wolfram syndrome and other forms of juvenile diabetes.
Recall Dr. Urano's May 26 blog. MANF gene therapy is in play.
https://medicine.wustl.edu/news/46-million-su...ign=buffer
Another example of the breadth of ICTS’ success is the recent development of a translational pipeline for the treatment of Wolfram syndrome.
The syndrome is a rare cause of diabetes and visual impairment in children that also affects many other organs, including the brain. With ICTS support, Washington University researchers led by Fumihiko Urano, MD, PhD, created a clinical and translational research program to study the disease, which led to fundamental laboratory studies that uncovered the molecular genetic defect, and ultimately to the identification of a therapeutic target that is now being tested in patients [dantrolene]. According to Urano, the Samuel E. Schechter Professor of Medicine, national resources offered through the NCATS Rare Disease Program combined with patient organizations and local ICTS support have directly supported the first clinical trial of a repurposed drug for the treatment of Wolfram syndrome.
A new grant led by the Urano team and funded by NIH/NCATS will combine elements of collaborative preclinical science with the national drug development team at NCATS, along with later clinical trial staging. The novel bench testing approach paired with therapeutic strategies will deliver personalized medicine for patients with Wolfram syndrome and other forms of juvenile diabetes.
Recall Dr. Urano's May 26 blog. MANF gene therapy is in play.
https://medicine.wustl.edu/news/46-million-su...ign=buffer
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