Anemia drug may prevent brain damage in high-risk newborns

One-third of newborns with hypoxic-ischemic encephalopathy treated with erythropoietin hours after birth showed no signs of brain damage five days later in a recent clinical trial.
By Stephen Feller  |  May 3, 2016 at 2:53 PM
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SAN FRANCISCO, May 3 (UPI) -- Brain damage caused by a condition at birth may be preventable, based on a recent study showing a nearly 30-year-old anemia drug helps the brain heal.

The drug erythropoietin, referred to as EPO, prevents damage and helps the brain heal after hypoxic-ischemic encephalopathy, researchers at the University of California San Francisco report in the study, published in the journal Pediatrics.

HIE is a dysfunction of the nervous system causing a drop in oxygen and blood flow to the brain and other organs, causing disabilities such as cerebral palsy or death in about 40 percent of newborns who have it, regardless of treatment.

EPO is a man-made version of erythropoietin, a hormone made in the kidney that tells stem cells in bone marrow to produce red blood cells. The drug was approved by the U.S. Food and Drug Administration in 1989 to treat anemia, but has been shown in studies to promote nervous tissue recovery and development after oxygen deprivation.

The standard treatment for HIE is hypothermia -- cooling the body to 33.5 degrees Celsius, or 92.3 degrees Fahrenheit, to speed healing -- but one-third of newborns in the new study given EPO as well had no injury to their brains after recovery, and just one in the group had a severe or moderate injury.

"We're hopeful that EPO not only reduces the extent of brain injury, but also allows the brain to be more effective at repairing itself during the recovery process," Dr. Yvonne Wu, a child neurologist and professor neurology and pediatrics at the University of California San Francisco, said in a press release. "While research with more participants is needed to determine whether EPO saves lives, we are heartened by the fact that among those infants that survived, the degree of brain injury was generally less severe than in the placebo group."

For the study, researchers treated 50 16.5-hour-old newborns with HIE, giving 26 hypothermia and placebo and 24 hypothermia and five injections of EPO.

At five days old, 33.3 percent of children in the EPO group had no brain injury detected on MRI, compared to 11.5 percent in the placebo group. In the placebo group, 11 children had moderate or severe brain injuries, compared to just one in the EPO group.

At around 12 months old, the researchers also found motor performance and developmental progress was better among children treated with EPO, compared to those only receiving hypothermia.

"It is clear that this therapy is safe as used in this study and there is a strong suggestion that the patients are doing better than would be expected long term," said Dr. Roberta Ballard, a neonatologist and professor of pediatrics at UCSF. "It would be very encouraging if we find that an inexpensive old drug, costing $60 per infusion, rather than a new drug that costs more than $2 billion to develop may prevent untold suffering. A larger trial will allow a definitive answer to whether there should be a change in clinical care for this devastating condition."

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