CLINICAL TRIAL. A new drug based on research at the University of Gothenburg seems to be able to prevent both stroke and severe lung disease in children born extremely prematurely. In a smaller first study in which the drug was administered to premature children, the risk of these complications was cut in half.
“This is the first time that a medical intervention was found to have such a great effect on the most serious complications of extremely premature birth. If the results continue to be this favorable, we will be able to offer a new preventive treatment to improve the care of premature children,” said Ann Hellström, ophthalmologist and professor at Sahlgrenska Academy, University of Gothenburg.
Growth protein prevents complications
The study is a Phase II clinical trial, which means that the potential drug is first tested in premature infants. In the study, 121 extremely premature children (born between gestational weeks 23 and 27) were randomly assigned to receive either treatment with growth protein IGF-1/GFBP-3, or to receive the treatment usually given to premature children. The growth protein was administered in droplets until the children reached 30 weeks of age.
“There are a number of vital substances that the child normally has access to in the womb, but that are drastically reduced in connection with premature birth. One of these factors is growth protein IGF-1. The new treatment is to restore IGF-1 to the levels normally found in utero, which seems to be able to prevent more serious complications that can affect premature children,” said Ann Hellström.
Lung diseases and stroke decreased
The children who were administered drops of growth protein had significantly lower levels of both severe chronic lung disease and intraventricular hemorrhage, both of which are common and serious complications in children born extremely prematurely. Lung diseases decreased by just over fifty percent, but in the children who had the desired range of the protein in the blood, lung diseases decreased by nearly 90 percent. The occurrence of severe intraventricular hemorrhage decreased by 44 percent in all children receiving treatment, and by over 60 percent in the children who had the desired level of protein in the blood.
“Our main goal in developing drug candidates has been to prevent retinopathy of prematurity, an eye disease common among children born extremely prematurely. The study was unable to show that the drug can protect the eyes of children, but the other clinical effects are highly relevant to the care of premature children,” said Ann Hellström.
International cooperation
The planning of the next phase of the clinical study is in progress, and is expected to start early next year.
“We have studied this factor for over twenty years, so it will take many years before the research can lead to a drug that can be used in the care of premature children,” said Ann Hellström.
The candidate drugs have been researched in an international research project, involving researchers from Lund University Hospital, Karolinska University Hospital, Harvard Medical School, the University of Cambridge, the University of Amsterdam, Istituto Giannina Gaslini in Italy, the pharmaceutical company Premacure AB, the biotechnology company Mediagnost and the research organization Smerud. The project is funded through a research grant of SEK50 million from the EU and was taken over by the pharmaceutical company Shire in 2013.
Text. Elin Lindström Claessen
Photo: Mattostock
