(BPT) - This article was sponsored and developed by Neurocrine Biosciences, Inc.

Cerebral palsy (CP) is a neurodevelopmental disorder affecting movement and posture that begins in early childhood development. It is the most common cause of motor disability in children, affecting about one in 345 children in the United States.^1-2 It may happen as a result of lack of oxygen during or around birth, stroke, infection, a problem with metabolism or other problems that cause injury or affect the development of parts of the brain involved in movement control in the first few years of life.³ CP is a permanent condition, affecting a person for their entire life.

People living with CP typically have motor problems, which may include spasticity (abnormal muscle tightness), dyskinesia (uncontrolled movements) or ataxia (poor muscle control), and many people have a mix of these motor disorders.⁴ Dyskinesia due to CP (DCP) is one of the most disabling forms of CP and impacts approximately 10% to 20% of people living with CP.^5-6

According to the Cerebral Palsy Foundation (CPF), the combination of irregular and unpredictable movements (chorea) and twisting and repeating movements (dystonia) often disrupt activities and cause significant functional impairment, including the ability to maintain balance, walk or fine motor control.⁷ Heather Riordan, M.D., Neurologist and Movement Disorders Specialist, Director of the Phelps Center for Cerebral Palsy at the Kennedy Krieger Institute, discusses the symptoms of chorea and impact on patients in a video shared on the CPF website here.

About 30% of people with DCP are affected mostly by these involuntary and irregular movements (chorea), while dystonia is the predominant motor finding in the remaining 70%.⁸ However, it is common for both chorea and dystonia movements to be present together.⁶

For people living with DCP, these movements can occur in any region of the body, including the arms and legs, torso and face and may vary in severity. They are often triggered or made worse by stimulation or stress. Because dyskinesia can occur at rest and/or when actively using the body, the problems with movement are very burdensome in day-to-day living, with discomfort and pain affecting the quality of life for people and their caregivers.⁹

Jen Lyman, mom to a son with DCP, highlights how dyskinesia makes communicating more difficult. "The most difficult thing about dyskinesia is watching my son struggle to do things that he wants to do, but the extra movements get in the way…special things, such as using a touch screen to video chat with his grandmother, are nearly impossible despite his best efforts to use his hands and fingers. Something so simple, yet so special for him should be effortless."

DCP has a wide-ranging impact on the individual, including lifelong challenges with movement, a higher risk of other medical issues, a higher rate of mental health challenges and difficulties with communication.^10-12

"Those of us who have the privilege to serve patients with dyskinetic, hyperkinetic or mixed cerebral palsy see the functional impact of this very challenging type of tone every day," said Susan Biffl, M.D., Rehabilitation Medicine Specialist at Rady Children's Hospital-San Diego and Assistant Professor at UC San Diego School of Medicine. "Patients face challenges with self-care, independence, mobility, communication, fine motor skills that affect occupational and recreational activities, swallowing, social interactions and even sleep. As this tone is variable, it is much more challenging to treat than more consistent tone issues, such as spasticity."

Treatment Options

There are currently no Food and Drug Administration-approved treatments for DCP. Dystonia and chorea are often managed separately as they respond differently to various treatments, which can make treatment challenging.³ Most drugs being investigated for DCP have focused on dystonia, and more research is needed to determine potential treatments for chorea. New treatment options are needed to help manage these movement disorders and improve daily function and quality of life for those affected.

"After 20 years of caring for individuals with cerebral palsy, I continue to find those with dyskinetic cerebral palsy among the most challenging to treat, largely due to the limited effectiveness of available pharmacologic options," said Joyce Oleszek, M.D., Professor, Department of Physical Medicine and Rehabilitation, University of Colorado School of Medicine. "There is an urgent need for more robust evidence to support pharmacologic treatments for this condition, given its profound impact on function, comfort and quality of life."

Ongoing Research

Clinical studies are important in the development of treatment options, allowing researchers to evaluate the safety and effectiveness of new medicines. The data from clinical studies are used to determine if an investigational treatment can be approved for use to treat certain disorders.

There are ongoing clinical studies evaluating potential treatments for DCP, including one for chorea. Neurocrine Biosciences is conducting the KINECT^®-DCP clinical study, a Phase 3, randomized, double-blind placebo-controlled study investigating the study drug, valbenazine, for the treatment of DCP. The clinical study is currently enrolling children, adolescents and adults six to 70 years of age who have been diagnosed with DCP.

"The Cerebral Palsy Foundation is excited to partner with Neurocrine Biosciences on this study," said Rachel Byrne, Executive Director of the Cerebral Palsy Foundation. "We are proud to support strong research that can potentially bring meaningful change to the lives of those living with cerebral palsy."

If you or someone you know may be eligible for this research study, please visit the study website for additional information here: FindADCPStudy.com.

REFERENCES

1. Krigger KW. Cerebral palsy: a review. Am Fam Physician. 2006;73(1):91-100.
2. Michael-Asalu A, Taylor G, Campbell H, Lelea LL, Kirby RS. Cerebral palsy: diagnosis, epidemiology, genetics, and clinical update. Adv Pediatr2019;66:189-208. doi:10.1016/j.yapd.2019.04.002
3. Monbaliu E, Himmelmann K, Lin JP, et al. Clinical presentation and management of dyskinetic cerebral palsy. Lancet Neurol. 2017;16(9):741-749. doi:10.1016/S1474-4422(17)30252-1
4. Christine C, Dolk H, Platt MJ, Colver A, Prasauskiene A, Krägeloh-Mann I; SCPE Collaborative Group. Recommendations from the SCPE collaborative group for defining and classifying cerebral palsy. Dev Med Child Neurol Suppl. 2007;109:35-38. doi:10.1111/j.1469-8749.2007.tb12626.x
5. Lumsden DE, Crowe B, Basu A, et al. Pharmacological management of abnormal tone and movement in cerebral palsy. Arch Dis Child. 2019;104(8):775-780. doi:10.1136/archdischild-2018-316309
6. Monbaliu E, de Cock P, Ortibus E, Heyrman L, Klingels K, Feys H. Clinical patterns of dystonia and choreoathetosis in participants with dyskinetic cerebral palsy. Dev Med Child Neurol. 2016;58(2):138-144. doi:10.1111/dmcn.12846
7. Dyskinetic cerebral palsy. Cerebral Palsy Foundation. Published December 20, 2022. Accessed October 29, 2024. https://cpresource.org/understanding-cerebral-palsy/types-cerebral-palsy/dyskinetic-cerebral-palsy
8. Himmelmann K, Hagberg G, Wiklund LM, Eek MN, Uvebrant P. Dyskinetic cerebral palsy: a population-based study of children born between 1991 and 1998. Dev Med Child Neurol. 2007;49(4):246-251. doi:10.1111/j.1469-8749.2007.00246.x
9. Masson R, Pagliano E, Baranello G. Efficacy of oral pharmacological treatments in dyskinetic cerebral palsy: a systematic review. Dev Med Child Neurol. 2017; 59:1237-1248. doi:10.1111/dmcn.13532
10. What is cerebral palsy? Cerebral Palsy Foundation. Accessed November 13, 2024. https://cpresource.org/topic/what-cerebral-palsy
11. Adults & adolescence. Cerebral Palsy Foundation. Accessed November 13, 2024. https://cpresource.org/topic/adults
12. Communication. Cerebral Palsy Foundation. Accessed November 13, 2024. https://cpresource.org/topic/communication

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