A Moment with William J. Barson, MD
Back in the 70’s, Dr. Barson decided to complete his residency and fellowship training at what was then called Columbus Children’s Hospital because he knew he would receive great training and because he wanted to stay in Columbus. Training back then was quite different.
Dr. Barson shares, “We had no ED physicians, neonatologists, intensivists, or in fact many of the subspecialists that we are now so fortunate to have. You might think, how did things get done? Well, it was through the hard work of the residents, nurses, our handful of pediatric specialists, and the community physicians who graciously volunteered their time to attend on the wards.”
Drs. Milo Hilty and Dwight Powell, were Dr. Barson’s mentors during his infectious diseases fellowship training, and he describes them as “…awesome examples of professionalism and caring who taught me how to be a good clinician and teacher.”
As to what was most memorable about training at what is now Nationwide Children’s Hospital, Dr. Barson reflects on when, as a resident, he flew in the Ohio National Guard helicopter on patient transport missions and was responsible for the respirator settings while at the OSU NICU.
“As an intern, while in the OSU NICU under Dr. Rick McClead’s insight and ingenuity, we pieced together the first CPAP device to be used on a neonate in Columbus. I will always remember and remain indebted to the many fine mentors who trained me and the wonderful fellow residents, nurses, and ancillary health care providers with whom I worked side by side.”
But Dr. Barson’s most memorable and enduring experience was “…meeting my future wife, Darlene Terpenning, who was a neonatal surgical nurse here, when I rotated through her unit as an intern.”
After completion of his infectious diseases fellowship, Dr. Barson joined Columbus Children’s Hospital as an assistant professor dividing his time between the Ambulatory Pediatric Clinic at OSU and Infectious Diseases at Columbus Children’s. After a couple years, Dr. Barson became devoted full time to Infectious Diseases and in time became the Chief of the Section of Infectious Diseases and Director of the Infectious Diseases Fellowship. Dr. Barson says that because he pursued his career at the hospital where he trained, it has been wonderful to work with his former mentors and then, over the years, with the residents that he helped train.
When asked what the “today you” would say to the “resident/fellow you” Dr. Barson responded that there are three things to remember:
- Always treat the patient not their labs
- Always ask the question “why?”
- Be more flexible and become more adaptable to change
Promise of Gene Therapy Replacement for SMA
Spinal Muscular Atrophy (SMA), which can be diagnosed prenatally, is one of the leading genetic causes of death in infants. SMA is a progressive neuromuscular disease where patients lack the gene to make a protein called SMN that helps the motor neurons develop. Without this protein, the muscles do not get the innervation they need and thus weaken and become atrophic very early on in development. There are different forms of this disease based on how strong the patients are and what milestones they can reach. Up until now, the treatment for children with SMA Type 1, the most severe and lethal form, involved significant support including surgically placed feeding tubes, respiratory support with ventilators and frequent assessments from a multidisciplinary team of specialists to maintain survival.
Through a pre-clinical study in labs at both The Ohio State University and Nationwide Children’s Hospital, a novel treatment was developed using a virus that does not cause any significant human disease —adeno-associated virus. Effectively, unnecessary parts of this virus were removed and replaced with the gene for SMA. This virus is able to “infect” the patient’s cells, place the gene and replicating machinery where it needs to go and enable the cell to make the protein needed for development. At the end of the study period, all 15 infants enrolled in the study 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 were sitting for 30 seconds or longer. Two were able to crawl, pull to a stand and walk independently. Results of phase 1 of this first clinical trial to test the functional replacement of the mutated gene responsible for SMA1 were published in the New England Journal of Medicine in November 2017 and the study was recognized at the 2018 Top Ten Clinical Research Achievement Awards at the National Press Club in Washington, D.C., in April, 2018.
The gene therapy was developed in collaboration with AveXis, Inc. and builds on nearly three decades of foundational research led by teams at Nationwide Children’s and The Ohio State University College of Medicine exemplifying the strong basic science and clinical bonds between the two institutions. Next steps will include launching a phase 2 clinical trial.
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. PMID: 29091557.