Researchers have long hoped to discover effective new treatments for difficult-to-treat heart conditions – and they may be getting closer. Stem cell treatments for congenital heart conditions have in recent years been proven safe, and are advancing, while drawing increased interest from the medical community. These innovative therapies are bringing the promise of advancements in regenerative medicine closer for those that choose to bank stem cells.
Hypoplastic Left Heart Syndrome
Hypoplastic left heart syndrome (HLHS) is a congenital heart condition in which the left side of a baby’s heart cannot effectively pump blood to the body, leaving the right side of the heart to perform all of the vital work. The condition can sometimes be detected by doctors during an ultrasound as early as the second trimester, but is commonly detected and diagnosed shortly after birth.
The condition is serious: without surgery or heart transplant, it is usually fatal within the first few weeks after birth, and even with successful surgery, potentially dangerous complications are common afterwards and specialized, lifelong medical care is a necessity.
There are two clinical trials of note seeking to improve the results of these surgeries, potentially providing patients with improved long-term health outcomes. The goal is not to replace surgery, but rather to help patients receive better results.
The first trial involves the use of cord blood stem cells during the Norwood procedure, a surgery typically performed within the first two weeks of a baby’s life that allows the right side of the heart to pump blood more effectively to the lungs and rest of the body. The primary measure of this trial is still safety, but researchers are monitoring the potential for change in heart function and structure and even broader measurements of the newborn’s health, like body weight.
The other trial is investigating the safety and efficacy of using cord blood stem cells during the Glenn surgical procedure, typically performed 3 to 6 months after birth. The procedure is usually performed after a successful Norwood procedure, redirecting blood flow with the assistance of an existing large vein that reduces the workload of the right ventricle. The hope of this trial is to strengthen the heart muscles that pump blood from the right side of the heart to the rest of the body, a supplemental treatment to improve function of the heart.
HLHS and Transposition of the Great Arteries
Transposition of the great arteries (TGA) is a congenital condition in which the two main arteries leaving the heart are reversed, resulting in oxygen-deficient blood traveling to most of the body, while blood high in oxygen is sent back to the lungs, instead of to the rest of the body. The deprivation of oxygen can be dangerous, with potential brain damage a risk, and the heart and lungs are also at risk of damage, so surgery is usually performed to re-route the arteries within the first week.
An upcoming Israeli clinical trial intends to supplement treatment of both HLHS and TGA with cord blood stem cells after surgery. As safety is confirmed, the researchers will also assess any health benefits. The hypothesis is not just a reduction of chronic issues of heart health, but also of reduced brain injury that can be assessed with an MRI. The researchers hope to establish long-term benefits by continuing to assess neurological development and motor function for a year after treatment.
What do these trials mean for families?
The full potential of these treatments is not yet known, but could very well improve the long-term health outcomes of babies born with these congenital heart conditions.
It’s important to remember how early these surgeries take place in a baby’s life. If these clinical trials demonstrate favorable results, the decision to bank a newborn’s cord blood would become incredibly valuable, as finding a matching source of stem cells in such a short period of time would not just be costly, but also incredibly difficult.
For parents who know their baby will be born with one of these conditions, the decision to bank cord blood may become obvious. For other parents, these developments may serve as one more reason to bank their newborn’s cord blood. And cord blood from one sibling has a 50% chance to match another, and is already the preferred source of stem cells for a number of serious medical conditions, opening up the possibility of further treatment options.
With hundreds of advanced clinical trials taking place around the world, new FDA-approved stem cell treatments for a wide range of medical conditions are becoming reality, and the future of regenerative medicine has never been brighter.