Three Decades of Research Culminates in FDA Drug Approval
Indiana CTSI e-newsletter content from June 2018
By IU School of Medicine and CTSI Staff Reports:
Indiana CTSI-supported research led by two Indiana University School of Medicine faculty members has culminated in U.S. Food and Drug Administration approval of a new drug for patients with an uncommon, painful and deforming bone disease.
| IU School of Medicine researchers Michael Econs and Kenneth White (Photo courtesy of Indiana University) |
The drug, burosumab, treats X-linked hypophosphatemia, or XLH. It is being brought to market by Ultragenyx Pharmaceutical Inc. in collaboration with Kyowa Hakko Kirin Co., Ltd. and its European subsidiary, Kyowa Kirin International PLC, under the brand name Crysvita.
XLH is a phosphate-wasting disease that causes rickets and osteomalacia, or softening of the bones. It is typically inherited and affects more than 12,000 patients in the United States and one out of every 20,000 people worldwide. Patients are typically diagnosed as children and have bowed legs, short stature, and experience bone pain and dental abscesses.
The first ever human dose of burosumab was administered by an Indiana CTSI Clinical Research Center (CRC) nurse on January 27, 2009. CRC staff conducted study procedures for all clinic study visits associated with the Phase I – III studies (eight studies total), which included 46 patients (aged 1 year to 70 years) and 822 total study visits, and the CRC continues to support this important work with an upcoming Phase IV study.
Pawsitive results
CTSI-supported researchers at Purdue recently published a new study that shows how veterans with PTSD may benefit physiologically from using service dogs.
Growing talent
Central Indiana area high school students are exploring careers in science this summer by working in the labs of research mentors at CTSI partner institutions.
Meet Anna Roberts
Get to know the Indiana CTSI’s new Data Navigator, who serves as a resource for researchers looking to harness data expertise available through the Regenstrief Institute.
Bench to bedside
Early research supported by the CTSI has helped lead to the launch of a first-in-human clinical trial at IU School of Medicine that aims to treat an aggressive type of breast cancer.
Tune in
CTSI Director Anantha Shekhar will discuss the recent Indiana CTSI 3.0 grant renewal and what it means for the health of our state this weekend on Inside Indiana Business TV.
CTSI IN THE NEWS
- Do you want to be all in on good health? – Fort Wayne News-Sentinelcolumn featuring the Indiana CTSI’s new “All IN for Health” program.
- Hoosier health to get boost from $33M grant to Indiana institute – The Indianapolis Star story about the Indiana CTSI’s recent grant renewal from the NIH’s National Center for Advancing Translational Sciences.
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UPCOMING EVENTS
- Fourth Annual Diabetes Symposium
August 3, 2018 (Register by June 29) | Indianapolis - Big Ten–Ivy League 6th Annual Traumatic Brain Injury Summit
July 18-19, 2018 | Philadelphia, Pennsylvania - 2018 Indiana Health Advocacy Coalition Conference
July 26, 2018 | Indianapolis - Save the Date: Indiana CTSI Annual Meeting
September 14, 2018 | Indianapolis - Save the Date: Indiana CTSI Notre Dame Retreat
October 26, 2018 | South Bend
View all upcoming events.
