An infusion of cells from a child’s own umbilical cord blood appears to improve brain connectivity and motor function in children with spastic cerebral palsy.

The results of the randomized clinical trial were recently published by Stem Cells Translational Medicine.

The placebo-controlled, phase two trial included 63 children with varied types and severities of spastic cerebral palsy, a condition usually caused by brain damage before or at birth.

Children who received one intravenous dose of at least 25 million stem cells per kilogram of their body weight saw improvements in motor function a year later.

The improvements were greater than those typically observed for children of similar age and condition, and exceeded the gains made by children who received a lower dose of cells or a placebo.

“We are encouraged by the results of this study, which shows that appropriately dosed infusions of cord blood cells can help lessen symptoms in children with cerebral palsy,” said senior author Joanne Kurtzberg, M.D., director of Duke’s Pediatric Blood and Marrow Transplant Program and the Robertson Clinical and Translational Therapy Program.

“We still have a lot to learn about this therapy so that it can be optimized and accessible to more children with cerebral palsy,” said Kurtzberg, who is also director of the Carolinas Cord Blood Bank at Duke.

“Previous research has indicated it’s safe for children with cerebral palsy to receive an infusion of their own cord blood,” Kurtzberg said. “Now that we have identified a dosing threshold, we are planning additional studies testing the benefits of multiple doses of cells, as well as the use of donor cells for patients whose own cord blood was not banked.”

Kurtzberg has been a pioneer in testing the therapeutic potential of umbilical cord blood, which has been found to be a rich source of blood stem cells to treat cancers, neurological disorders and genetic diseases. Cord blood also contains other therapeutic cells that researchers believe could influence the formation of new neural connections in children with cerebral palsy.

In the cerebral palsy trial, Kurtzberg and colleagues tested doses from 10 million cells per kilogram of body weight up to 50 million cells per kilogram, based on the amount and quality of the cord blood each child had in storage. Among the tools used to evaluate the children’s progress were MRI to measure brain connectivity and the Gross Motor Function Measure (GMFM-66), a standardized analysis of a child’s ability to crawl, roll, kneel, and complete other movements based on age and development.

Children who have cerebral palsy are expected to gain motor function as they grow and develop and receive traditional therapies, including occupational and physical therapy, said Jessica Sun, M.D., a pediatric hematologist-oncologist at Duke and lead author of the paper. The GMFM-66 attempts to account for this expected growth based on age and the severity of the cerebral palsy, she said, and most participants improved when retested on the GMFM-66 a year after receiving an infusion, even those who received a placebo.

However, the improvements for children who received doses of at least 25 million cells per kilogram of body weight progressed beyond their expected increases when they were tested a year after infusion.

“For each child, the improvements are different and could be subtle, but sometimes even a seemingly small difference is significant,” Sun said. “For example, a child’s ability to turn their hand from facing down to facing up can change their ability to hold or grasp something, which can make a big difference in their everyday life.”

The study had some limitations, including the requirement that participants have cord blood in storage and be able to travel to Duke, both of which required financial means, the authors said. Being from well-resourced families, most participants were also receiving frequent physical and occupational therapy, Sun said, and those advantages could have influenced the results.

“We are hopeful that cord blood and cell therapy may have a role in treating children with cerebral palsy and brain injury and are encouraged to continue this promising research,” Sun said.

When he was born at Mount Sinai Hospital in Toronto, Jack was not breathing and was non-responsive. 

Doctors whisked him away from his mother and started efforts to resuscitate him. They managed to save his life but soon had to deliver some bad news to his parents, Stephen Pankratz and Kim Kucher. 

Their son had Hypoxic Ischemic Encephalopathy (HIE), brain damage caused by lack of oxygen and compounded by low blood flow to vital organs. Jack would likely suffer extensive cognitive and physical problems. 

But at only 12 days old Jack was re infused with his own stem cells – the youngest person ever to undergo stem cell therapy in Canada and the first person in the country to be treated for HIE with stem cells.

Doctors believe he may turn out to be the first of many. 

Two years later, Jack is thriving. He has cerebral palsy and faces many challenges, but his development has exceeded doctor’s expectations. His mother attributes his progress to the re-infusion.

Dr. Joanne Kurtzberg, a researcher at Duke University in Durham, N.C, has been leading clinical trials to determine whether cord blood can help to repair the brain. Research results published by Duke University indicate that early transfusion of cord stem cells is effective.

Jack’s parents discovered Kurtzberg’s findings while doing research on HIE in the days following their son’s birth. They brought them to the attention of his neonatologist, Dr Karen Pape. 

“She was open to hearing what we had to say and she read the research findings that we had given her,” says Pankratz. “She came to the same conclusion as us — that the potential benefits of a stem cell transplant outweighed the risks.

In scientific and medical circles, many stem cell experts are predicting the dawn of a new era in the treatment of HIE, autism and other brain disorders – with cord blood stem cells treatment playing a key role.

Umbilical cord blood could provide a lifesaving treatment for heart failure patients, a new study has found.

The report from the American Heart Association, published in September 2017, revealed that stem cells from cord blood boosted patients’ heart function when administered.

Experts are hopeful that the study could improve the lives of the 37 million people worldwide who live with heart failure, as current treatments involve invasive procedures and medications that take a toll on patients’ bodies.

The study involved 30 heart failure patients aged 18 to 75. ​While some were treated with umbilical cord blood, others got a placebo drug.

The heart muscles of those who were injected with umbilical cord cells saw ‘significant’ improvement during the year following the trial. This included an improved ability to pump blood and function at a higher level. 

The effects resulted in an improved quality of life for the patients who had received the cells, the study said.

Study author Dr Jorge Bartolucci said that the treatment could transform the way doctors think about heart failure treatments because current options for treating the fatal disease are complicated and ineffective.

‘Standard drug-based regimens can be suboptimal in controlling heart failure, and patients often have to progress to more invasive therapies such as mechanical ventricular assist devices and heart transplantation,’ Dr Bartolucci said.

Another researcher who worked on the study, Dr Fernando Figueroa, echoed the excitement over the study’s potential.

‘We are encouraged by our findings because they could pave the way to a non-invasive, promising new therapy for a group of patients who face grim odds,’ he said.

The study pointed out that, even though recent medical advances have improved these odds, half of the people who are diagnosed with heart failure die within five years of their diagnosis.

The American Heart Association journal, Circulation Research published the results of the study.