L. Andrew Koman, M.D. was recently awarded the Orthopaedic Research and Education Foundation (OREF) Clinical Research Award the 2019 Annual Meeting of the American Academy of Orthopaedic Surgeons (AAOS). Dr. Koman, along with co-authors Beth Paterson Smith, Ph.D., and Thomas L. Smith, Ph.D., studied the impact of botulinum toxins on the musculoskeletal system over the past 30 years, defining its pharmacologic properties and documenting its positive value in patients with cerebral palsy.
In 1988, Dr. Koman and Dr. Beth Smith were the first to inject botulinum A toxin—a naturally-occurring poisonous substance—to stabilize muscle imbalance in children with cerebral palsy. They found that botulinum toxins improved function for cerebral palsy patients.
“For severe patients, it facilitates their care and makes it easier for their caregivers to help them,” said Dr. Koman, professor and chair of the Department of Orthopaedic Surgery at Wake Forest School of Medicine, Winston-Salem, North Carolina.
“For patients with upper body functional impairment and mobility challenges, it was easier to get their clothes on and off, to raise their arms, and to get them in and out of wheelchairs. For less severe patients, it was easier for them to walk and it decreased pain. Using botulinum toxin delayed surgery, a significant amount of time, decreased hospitalizations, improved the quality of life for patients, and it is cost effective.”
Dr. Koman and his colleagues built a multidisciplinary lab that conducted over 110 pre-clinical and clinical trials and established a database with more than 25,000 patient years of data. This information allowed researchers to look at factors as the cost impact of botulinum use and caregiver stress associated with the level of severity of cerebral palsy.
“If used appropriately with the right dilution and the right number of units, it is a very safe drug,” said Dr. Koman. “In the randomized, controlled trials, the patients who received placebo had more pain than the patients who had toxins. We found that by increasing the volume, we can decrease the amount of toxin, which makes it safer and less expensive, but we get the same efficacy.”
Asked for details on the challenges involved in defining the pharmacologic properties of botulinum A toxin, Dr. Koman told OTW, “In order to define the neuromuscular junction [NMJ] subtype properties required the development of a model and research protocols we had to do anatomic studies to determine the NMJ distribution. The most important clinical pharmacologic properties were the time activity curves or the length of time the drug worked and its expected decreased effect for weakness—this required a model and protocols. Also crucial was the effect of dilution which required a model and protocols.”
This doctor made me a patient advocate for others.