Nationwide Children’s Researcher Wins Distinguished Clinical Research Achievement Award

April 24, 2018

(COLUMBUS, Ohio) – The Clinical Research (CR) Forum, a non-profit membership association of top clinical research experts and thought leaders from the nation’s leading academic health centers, presented the Distinguished Clinical Research Achievement Award to two outstanding studies at the Top Ten Clinical Research Achievement Awards on April 18th at the National Press Club in Washington, D.C.

Nationwide Children’s Hospital is honored to announce that the first clinical trial to test the functional replacement of the mutated gene responsible for SMA1 led by Jerry Mendell, MD, was recognized as a recipient of the Distinguished Clinical Research Achievement Award. This award comes with a cash prize of $5,000. 

The CR Forum presents its annual Top Ten Clinical Research Achievement Awards to highlight outstanding research advances that involve both innovation and impact on human diseases. A complete list of the 2018 Top Ten Award Winners can be found at www.clinicalresearchforum.org.

The Distinguished Clinical Research Achievement Award is presented to the top two studies that show creativity, innovation, or a novel approach that demonstrates an immediate impact on the health and well-being of patients. Results of the phase 1 clinical trial of AVXS-101, published in the New England Journal of Medicine in November 2017, was one of this year’s honorees.

SMA1 is a progressive, childhood, neuromuscular disease caused by a mutation in a single gene. Children with SMA1 fail to meet motor milestones and typically die or require permanent mechanical ventilation by two years of age. According to the natural history of the disease, patients require nutritional and respiratory support by 12 months of age, and are unable to swallow or speak effectively.

At the end of the study period, all 15 infants had a favorable safety outcome. Of the 12 patients treated with the highest dose, 92 percent achieved head control, 75 percent rolled over and 92 percent sat with assistance. Seventy-five percent of the infants are now sitting for 30 seconds or longer. Two can crawl, pull to a stand and walk independently.

This study built on nearly three decades of foundational research led by teams at Nationwide Children’s and Ohio State’s Wexner Medical Center and exemplifies the strong basic science and clinical bonds between the two institutions. Dr. Mendell and his team at Nationwide Children’s led the clinical trail in collaboration with Arthur Burghes, PhD, of Ohio State, who created a ground-breaking SMA mouse model that remains the standard by which all therapies, including AVXS-101, are initially tested.

Brian Kaspar, PhD, an investigator of the study and Chief Scientific Officer for AveXis, during his appointment at Nationwide Children’s, discovered that the AAV9 vector was capable of crossing the blood brain barrier when injected into the vascular system to deliver genes directly to motor neurons. The Ohio State and Nationwide Children’s laboratories then collaborated to show that scAAV9-SMN, when delivered to SMA mice shortly after birth, completely prevented their neuromuscular disorder. They successfully prove that reversing a protein deficiency through gene therapy is effective in improving and stabilizing SMA in a large animal model.

AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXS-101, which is the company’s initial proprietary gene therapy candidate.

Since it was founded in 1996, the Clinical Research Forum has enabled the sharing of best practices in clinical research, informed meaningful policy dialogues and increasingly played a national advocacy role in support of clinical research. For more information, visit www.clinicalresearchforum.org.

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About Nationwide Children's Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report’s 2017-18 list of “America’s Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric healthcare systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children’s has a staff of nearly 13,000 providing state-of-the-art pediatric care during more than 1.4 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s physicians train the next generation of pediatricians and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at NationwideChildrens.org.