The California Institute of Regenerative Medicine has awarded US$4.96 million to research into neural stem cell-based therapies for perinatal hypoxic-ischemic brain injury, such as cerebral palsy, caused by oxygen deprivation at birth.
Cerebral palsy caused by perinatal hypoxic-ischemic brain injury (HII) could be a thing of the past, thanks to new funding awarded to help fund studies into neural stem cell-based therapies. Evan Snyder, director of the Center for Stem Cells & Regenerative Medicine at Sanford Burnham Prebys (CA, USA), was awarded US$4.96 million from the California Institute of Regenerative Medicine (CA, USA) to fund preclinical study into the effect of neural stem cells on the disorder.
HII can lead to permanent neurological disorders such as cerebral palsy or epilepsy. However, Snyder has been studying human neural stem cells since 1998 and believes they may hold promise for treating a range of neurological disorders, such as Parkinson’s disease and amyotrophic lateral sclerosis (ALS).
“These cells are exceptionally well characterized,” explained Snyder. “We know that they are drawn to injured regions like a magnet, we know they make a cocktail of healing factors and we know they soak up toxins in damaged areas. These characteristics make them very promising therapies, particularly for the developing pediatric brain.”
Beyond therapeutic hypothermia
The CIRM funding will enable preclinical, animal studies to assess when, where and in what dose to administer the neural stem cells to salvage undamaged, native cells and restore some function. This phase of study is expected to take 2 and a half years, and Snyder hopes at this point he will be ready to present the data to FDA, with a phase Ib/IIa trial to investigate the protective qualities of the neural stem cells coming after that.
“In the past 25 years that I have worked with these critically ill newborns, the only treatment advance for HII has been therapeutic hypothermia–or whole-body cooling–to prevent brain damage,” commented Snyder. “Today, we cross an important threshold. We are one step closer to a future where we may be able to protect some newborns from an unanticipated birth injury–and help parents give their children the best possible start in the world.”
What causes perinatal hypoxic-ischemic brain injury?
HII occurs when a baby’s brain doesn’t receive enough oxygen or blood and is a leading cause of mortality in the USA, where 0.2-0.4% of babies experience it. It can occur before, during and after a baby is born, but possible intrapartum complications that could lead to HII are low maternal blood pressure, pressure or damage to the placenta or uterus, and very long late stages of labor.
What are the treatments for perinatal hypoxic-ischemic brain injury?
The treatment for HII depends on its cause; therapeutic hypothermia, or targeted temperature management, has been recommended for newborn infants with moderate or severe HII. Therapeutic hypothermia involves cooling either the head or whole body of the infant and aims to improve brain function and/or survival by reducing the brain’s demand for oxygen and reducing molecules such as neurotransmitters or free radicals, which could damage the brain further.
Could stem cells treat perinatal hypoxic-ischemic brain injury?
Stem cell-based therapies to treat HII are currently still in the investigational stage; however, they are currently being investigated for the treatment of adult neurological disorders such as stroke, where they have been shown to be safe and, in some cases, effective