Hospital study targets Surfaxin use for premature babies

March 11, 2005
Pennsylvania hospital selected for drug study to boost long-term survival of premature babies

It was an uncomplicated pregnancy until the sixth month when June Frye of Hazelton, Pa., started into pre-term labor. At 28 weeks, the risks were high and the local hospital was ill-equipped to handle such high-risk cases, so June was transported to Lehigh Valley Hospital in Allentown, Pa., with its maternal fetal program and Level III Neonatal Intensive Care Unit (NICU).

After unsuccessful attempts to halt labor, June gave birth on Oct. 14, 2004, to a boy weighing 2 lb 9 oz. The tiny preemie, named Peyton, seemed to do well initially but a few days later needed heart surgery. He was intubated and put on a ventilator for the surgery and then again a few days later when fluid started collecting in his lungs. As a result, Peyton developed bronchopulmonary dysplasia (BPD), a chronic lung disease associated with premature infants and the use of ventilators and supplemental oxygen.

In January, Lehigh Valley Hospital and Health Network (LVHHN) was picked as the first site in a 25-site multinational clinical study of Surfaxin, a drug to help premature babies survive without developing BPD. The Phase 2 study will explore whether or not Surfaxin, developed by Discovery Laboratories, Inc., Doylestown, Pa., can prevent or reduce the incidence of BPD in these high risk babies.

Surfaxin is a precision-engineered, peptide-containing substance designed to closely mimic the function of natural human lung surfactant. In just completed Phase 3 clinical trials, Surfaxin (lucinactant) therapy increased survival of premature babies with Respiratory Distress Syndrome (RDS), and incidentally, seemed to prevent BPD in these babies as well. This new study will seek to verify Surfaxin's role in preventing BPD.

Premature infants, born 6 or more weeks early and weighing less than 4.5 pounds, typically have underdeveloped lungs that lack a lipid/protein substance or surfactant essential to oxygen absorption and proper airflow through the respiratory system. Without the natural lung surfactant, many of these infants are subject to life-threatening RDS at birth and require mechanical ventilation and oxygen to survive. While ventilators help them breathe, prolonged use of the ventilators and oxygen can lead to bronchopulmonary dysplasia characterized by inflammation and scarring in the lungs and chronic health problems.

BPD is costly. According to National Heart, Lung and Blood Institute, between 5,000 and 10,000 babies in the U.S. develop BPD each year. The overall cost of treating infants with BPD is estimated to be approximately $2.4 billion and treatment of a single patient can cost up to $250,000.

"At this point we have no specific therapy for BPD, so this clinical trial is an exciting opportunity for Lehigh Valley Hospital to improve the lives of these babies and their families," said Christopher Morabito, M.D., chief, division of neonatology and the study's principal investigator. "BPD can have a long term impact on children who may suffer with breathing and neurological problems throughout their lives."

No one knows that better than Heather Maeding, a nurse practitioner in a neonatal intensive care unit and mother of a BPD-child. At 3 1/2 years old, Luke Maeding should have outgrown the BPD, but instead he has been hospitalized on average twice a year and, most recently, for lung abscesses that required lung surgery. Born at 26 weeks and weighing barely 2 pounds, Luke has had significant respiratory challenges throughout his short life. He is on supplemental oxygen and has a gastronomy tube (g-tube) to address his feeding problems and to improve his nutrition.

Over a year ago, Luke was put on prednisolone to help control the inflammation in his lungs. While steroid therapy improved his respiration and kept him out of the hospital for a while, it compromised his immune system leading to the abscesses and he stopped growing - both common side effects of prolonged steroid use.

"The impact on the family can be huge," notes LVHHN pediatric pulmonologist, Robert Miller, M.D. "Once the family gets through the short-term acute phase when they don't know if the baby will live, there are a host of ongoing issues they may have to face. In addition to breathing, there could be feeding, speech, and cardiac problems as well as poor physical growth, susceptibility to respiratory and other illnesses, emotional development and neurological issues."

Luke and his family experience the bulk of these challenges as well as others. "The child needs round-the-clock surveillance and can't go to day care because of their weak immune systems," explains Maeding. "Finances are another consideration. We have good health insurance and, because Luke was in the foster care system, there were funds available to help with his medical treatments. A lot of families don't have that and the burden can be enormous. Having a chronically ill child challenges the best of relationships," she adds, "and many families split up under the strain."

"Parents can be more hopeful because we have the technologies to help these very tiny infants survive but there are quality of life issues...for the children and for the families," Miller said. "If the clinical trial shows that Surfaxin is successful in preventing BPD, that would be a major step forward to improve the lives of these highly vulnerable children."

LVHHN is a regional referral center for a large portion of eastern Pennsylvania and includes a the state's busiest Level I Trauma Center, Regional Burn Center, Pediatric and Neonatal Intensive Care Units, Primary Stroke Center, Regional Heart Center and Cancer Center.