T1

Children

Supported by a CTSI Core Voucher Award, April Pyle, PhD, created functional skeletal muscle cells and used them in animal models of Duchene muscular dystrophy to restore dystrophin, the protein missing in the muscle-wasting disease that affects boys and young men.

Outcome. Her successful research was published in Nature Cell Biology [1].

T2

Children

Assisted by the CTSI’s study activation team in the CTRC, Noah Federman, MD, led UCLA’s participation in the multisite clinical trial of larotrectinib in children with bone and soft tissue sarcoma. 

Outcome. Results published in the NEJM led to FDA approval of the drug in children and adults with cancers that have an NTRK gene fusion, the second tissue-agnostic drug ever approved [2].

T3

Disparities and Older Adults

Work by KL2 scholar John Mafi, MD, demonstrated that a multipronged nurse-led, quality-improvement initiative at a Los Angeles safety-net hospital was associated with sustained reductions in low-value preoperative testing for cataract surgery compared with a control safety-net site. 

Outcome. This work led to county-wide implementation and to an R01 to incorporate behavioral economics approaches in partnership with UCLA Anderson School of Management faculty [3].

T3

Disparities

With funding from a CTSI Safety-Net Innovation Award, Dong Chang, MD of Harbor, developed an intervention that brought families and patients into the decision-making process to reduce non-beneficial ICU care. 

Outcome. Based on these results, Dr. Chang and his team received a foundation award to conduct a wider study involving additional hospitals across the county [4].

T4

Older Adults

As a KL2 Scholar, Joshua Pevnick, MD, of Cedars implemented a method to obtain accurate medication history for every high-risk patient upon admission. 

Outcome. His research contributed to passage of California Senate Bill 1254, which requires hospital pharmacy staff to obtain medication histories upon admission, enhancing patient safety [5, 6].