The U.S. Food and Drug Administration (FDA) has approved a new study that will test the efficacy of a stem cell technique used on children suffering from congenital heart disease.
The study is being hosted by Boston Children’s Hospital and Mesoblast Limited, a regenerative medicine company based in Australia. Their 24-patient trial will test the efficacy of Mesoblast’s proprietary allogenic mesenchymal precursor cells (MPCs) in a corrective heart surgery on children under the age of 5 that suffer from hypoplastic left heart syndrome (HLHS), according to an April 3 press release.
The new technique is designed to save more patients with HLHS because current treatment is not always effective. Right now physicians generally perform a single, ventricle palliation, which results in the patient using only the right ventricle to the support the heart’s entire circulation. However, the treatment is usually only a temporary fix because the right ventricle will eventually tire out, putting the patient at an increased risk for sudden cardiac arrest.
In the randomized, controlled trial, researchers will inject Mesoblast’s MPC-150-IM into the left ventricle during surgical recruitment procedures, which should improve ventricular mass and function and ultimately lead to a higher likelihood of biventricular conversion.
“The objective of combining Boston Children Hospital’s expertise in pioneering surgical approaches to treating hypoplastic heart syndrome with the regenerative potential of our lead cardiovascular product, MPC-150-IM, is to develop a highly innovative treatment for this complex congenital condition as well as other serious and life-threatening cardiac diseases in children,” said Kenneth Brow, the senior clinical development executive and head of cardiovascular diseases at Mesoblast, in a statement.