Cerebral Palsy Research

1st FDA-APPROVED STEM CELL TRIAL IN PEDIATRIC CEREBRAL PALSY

Medical College of Georgia researchers are conducting the first FDA-approved clinical trial to determine whether an infusion of stem cells from umbilical cord blood can improve the quality of life for children with cerebral palsy.

The study will include 40 children age 2-12 whose parents have stored cord blood at the Cord Blood Registry in Tucson, Ariz.  Umbilical cord blood is rich in stem cells, which can divide and morph into different types of cells throughout the body, said Dr. James Carroll, professor and chief of pediatric neurology in MCG School of Medicine and principal investigator on the study.

Cerebral palsy, caused by a brain injury or lack of oxygen in the brain before birth or during the first few years of life, can impair movement, learning, hearing, vision and cognitive skills. Two to 3 children in 1,000 are affected by it, according to the Centers for Disease Control.  Animal studies indicate that infused stem cells help injured brain cells recover and replace brain cells that have died, Dr. Carroll said.  "Autologous stem cell transplantation, in which the transplant recipient is also the donor, is the safest form of stem cell transplantation because it carries virtually no threat of immune system rejection," he said.

While no controlled clinical trials have been conducted to date, previous studies have shown marked improvement in children with cerebral palsy about three months after an initial infusion of cord blood.  "Evidence up to this point has been purely anecdotal," Dr. Carroll said. "While a variety of cord blood stem cell therapies have been used successfully for more than 20 years, this study is breaking new ground in advancing therapies for brain injury -- a condition for which there is currently no cure."

Children will begin the study with a neurological exam by MCG pediatric neurologists Elizabeth Sekul and Nicole Brockway. Then, half of the study participants will receive an infusion of their own cord blood while the other half receive a placebo. Three months later, the children will be evaluated without physicians knowing which group received the stem cell infusion. Afterward, children who didn't get the cord blood initially will receive an infusion. Children will return three and six months later for evaluation.

Researchers will periodically assess the children's motor skills and neurological development.

"For the purposes of this study, we're not looking at stem cells as a possible cure; rather whether stem cells can help change the course of these types of brain injuries in children," Dr. Carroll said.  Study participants must have been unable to sit independently by 12 months or unable to walk by 18 months and must be seizure-free or have seizures that are adequately controlled.

To ensure consistency in cord blood stem cell processing, storage and release for infusion, the Cord Blood Registry is the only family stem cell bank participating in the study.

The trial is also receiving support from the Associazione Figli Inabili Banca d'Italia, a private organization in Italy that provides financial assistance to parents who can't pay for their children's medical treatments. 

CHILDREN WITH CEREBRAL PALSY COULD BE HELPED TO SPEAK MORE CLEARLY FOLLOWING RESEARCH BY A UNIVERSITY OF STRATHCLYDE ACADEMIC 

Dr. Anja Kuschmann will analyse the speech patterns of young people affected by the condition, in an effort to understand more about why they can have difficulties talking.  Cerebral palsy (CP) is usually caused by an injury to the brain before, during or after birth. Children with CP have difficulties in controlling muscles and movements as they grow and develop.

Discovering more about how the speech of children with CP is affected by muscular problems and brain damage could, Dr Kuschmann believes, lead to improvements in diagnosis and therapy.  She said: "Many children with CP have difficulties with speech melody, rhythm and stress. These difficulties, generally referred to as prosodic difficulties, can affect the intelligibility of a child's speech, and are therefore of great clinical importance.
"However, the development of prosody in children with CP is currently not well understood. As a result, it is not certain whether prosodic difficulties in CP are due to muscular problems during speaking or the inability to build and store the correct prosodic information in the brain.

"This research will investigate the prosodic abilities in children with CP to determine the underlying nature of their difficulties. It will contribute to the theoretical understanding of the causes of prosodic impairment in CP, and may help to improve diagnosis and therapy of prosodic difficulties in CP."  Dr Kuschmann's project will take three years to complete, and aims to help improve the lives of the one in 400 UK children affected by CP.

While there is no cure for CP, physiotherapy and other therapies can often help people with the condition become more independent. No two people will be affected by CP in the same way, and it is important to ensure treatments and therapies are tailored to children's individual needs.

Dr Kuschmann, of Strathclyde's School of Psychological Sciences and Health, said: "CP can affect speech because you need your muscles to control your tongue and lips to allow you to pronounce sounds properly. It influences the way you articulate single sounds -- and also, importantly, the melody of speech, allowing you to ask questions, make statements or convey irony.  This research, which will involve the analysis of the speech patterns of around 40 children aged between seven and 16, will hopefully inform the assessment and interventions for children with CP. By looking at how breathing might be improved, for example, some children with CP may be able to use their speech alongside assisted technology -- such as voice synthesisers -- to communicate.

"This research is extremely important for children with CP because struggling to communicate has a whole host of wider implications. It can affect children's educational progress, their ability to form friendships and, generally, to fully participate in the wider community."

UNIVERSITY OF DELAWARE: EFFECT OF BOTOX AND VIBRATION ON MUSCLE AND BONE DEVELOPMENT

Botox is commonly used to assess muscle spasticity in children with cerebral palsy (CP), but the effect of Botox on muscle and bone development and on physical activity is poorly studied. It is well-established that children with CP have weaker muscles and bones and participate in less physical activity.  One promising treatment for poor muscle and bone development is vibration. Low-level vibration has been shown to improve muscle and bone mass.  A few studies have included children with CP.  In this study we want to determine the effect of Botox and vibration treatment on muscle, bone and physical activity in children with spastic CP.

The total duration of the study is 6 months. The child will be tested at the baseline (0 month), 1 month, and 6 months. The testing at baseline (0 month) and 6 months is spread over 2 days and the testing at 1 month can be done on a single day. The schedule is very flexible and we try to work per the schedule of the family. Most ofour participants prefer being tested over a weekend. The tests are done at duPont Hospital for the Children at Wilmington, Delaware and the University of Delaware, Delaware.

Ages Eligible for Study: 2 Years to 12 Years
Genders Eligible for Study: Both

Inclusion Criteria:

  • Have spastic CP.
  • Between 2-12 years of age.
  • Recommended for Botox treatment by their physician as part of their clinical care. Those who accept Botox treatment and those who do not accept Botox treatment are both eligible for the study.
  • Have not had Botox treatment in the lower extremities within the last 1 year.
  • A score of 1-4 on the gross motor function scale (GMFC).
  • Do not have metal rods in both legs.

Contact Info: Harsh Singh: Phone number: 302.831.8137; Email - harsh@udel.edu

Although families from any part of the country are welcome to participate, participation would be most convenient for families living in Delaware, surrounding states like NJ, PA, NY, MD, or Washington DC.