Approximately one-third of the children born with cerebral palsy (CP) have both legs affected yet can walk either independently or with a walker. The most common CP movement disorder in these children is spasticity hence many receive botulinum toxin A (BTA) injections to temporarily relieve the effects of spasticity. Some smaller percentage of these children go on to get a significant neurosurgery called selective dorsal rhizotomy (SDR) in an attempt to permanently remove that spasticity as it interferes with their functional abilities. While both of these treatment paradigms have been rigorously tested to determine their benefit, the most common treatment protocols have never been compared. The Cerebral Palsy Research Network (CPRN) applied to the National Institute of Neurological Disorders and Stroke (NINDS), one of the National Institutes of Health (NIH), for a large grant this past February to compare these treatment paradigms. Functional Improvement Related to Spasticity Treatments (FIRST) has been in the planning stages for several years. FIRST, if funded, would be conducted with children being seen at 25 clinical centers across North America. FIRST seeks to compare how well children walk after receiving serial BTA injections to those that go on to receive an SDR.
Several CPRN investigators worked on the development of FIRST which was submitted by three co-principal investigators including Kristie Bjornson, PT, PhD from Seattle Children’s Hospital, Paul Gross from the University of Utah and Jeffrey Leonard, MD from Nationwide Children’s Hospital. FIRST plans to enroll 1624 subjects across the 25 sites. The study design is observational in nature (patients are not assigned to interventions). The observational design is important because it enables the testing of effectiveness of interventions in the real world clinical practice. The particular funding opportunity at NINDS to which this grant was submitted was specifically created to accommodate observational studies for conditions like CP.
The review process at NIH is thorough but also lengthy. The study team does not expect a funding decision before the Fall of 2020. But the process of assembling the study team, gathering preliminary data from the network and developing an application of this scale was a success in of itself. It demonstrated the deep commitment of the CPRN site investigators to improve outcomes for people with CP.