For many children battling cancer, a blood or marrow transplant is their best hope for a cure. Yet transplants often pose big risks: a weakened immune system and greater risk of infections. These can lead to serious complications and even death.
Today researchers at the University of Minnesota, a leader in childhood cancer research, are rewriting this story, helping doctors speed recovery so children can come home sooner and healthier.
Mikaela Gerick started ninth grade like many students—easing into a new school and meeting new friends. But unlike her classmates, Mikaela spent the next three years battling cancer. Her treatment journey took her to a clinical trial that led to extraordinary results.
In September 2012 Mikaela came home from school pale and lethargic, with an unusually high temperature. Doctors confirmed that Mikaela had acute myeloid leukemia. Her first treatment was rough. She was admitted to the intensive care unit where her heart stopped, and she suffered multiple organ failures.
Then, nearly two years after her initial diagnosis, treatment and months of remission, Mikaela came home from camp with a rash on her face and legs. She had relapsed.
A blood or marrow transplant was the only solution. Mikaela’s family chose the University of Minnesota Masonic Children’s Hospital for and the promising cord blood expansion research of John Wagner, MD, co-chief medical advisor of Children’s Cancer Research Fund and director of the Pediatric Blood and Marrow Transplant Program.
Doctors have recognized the challenges patients face since the first umbilical cord blood transplant for leukemia was performed 25 years ago. A critical post-transplant milestone is the day of engraftment—when a patient’s new cells start to grow. Unfortunately, some patients never engraft, leaving them prone to infection, bleeding and even a need for a second transplant.
Improving engraftment is one of the most important goals of Dr. Wagner’s research. He hypothesized that increasing the number of hematopoietic stem cells in a cord blood transplant could speed up engraftment. And he was right. To further examine the idea, Dr. Wagner looked to a new molecule called StemRegenin 1 (SR1).
With the support of Novartis, a pharmaceutical company, Dr. Wagner’s team prepared for a clinical trial on 30 childhood and adult cancer patients. After chemotherapy and radiation, all patients were transplanted with an unprecedented number of SR1-expanded hematopoietic stem cells—up to 5 billion instead of the 200,000 typically found in umbilical cord blood.
At 16 years old, Mikaela became the 19th patient in the first in-human clinical trial for Dr. Wagner’s cord blood expansion protocol.
She engrafted on day seven—nearly 20 days sooner than she would have without the cord blood expansion protocol. And, she had far less post-transplant infection risks compared to children who did not participate in the trial.
In fact, the 30 clinical trial patients engrafted in an average of eight days—the fastest recovery ever documented. A typical engraftment takes an average of 26 days.
“If I had her cancer when I was her age, I would be dead, because the mortality rates were extremely high then,” says Mikaela’s mom. “If it weren’t for this research, Mikaela wouldn’t be with us, either. For the odds to shift that dramatically in a 30-year period is a big deal.”
Despite the positive results from the SR1 clinical trial, Dr. Wagner and team are not done.
Patients can still get infections after engraftment while their immune systems recover. The process takes six months or more, potentially leading to viral infections or relapse because immune cells are not functioning.
Using grants from Children’s Cancer Research Fund, Heather Stefanski, MD, PhD, Bruce Blazar, MD and Dr. Wagner can now focus on speeding recovery of the immune system after transplants.
In particular, they’re looking at ways to bridge the gap between the beginning of a patient’s recovery period (when the immune system declines) and the end of the recovery period (when the immune system is reinvigorated).
“Cells called thymic progenitors may help build a temporary healing bridge until the patient’s natural immune responses are ready to take over,” says Dr. Wagner.
Time is not on the side of children fighting cancer, so funding researchers is critical.
Even when a research project shows promise, the road ahead can be lengthy. Each step could take months or even years to secure funding, complete the research and get approval to make sure it’s safe for patients. And federal funding is becoming more competitive—fewer dollars are granted to childhood cancer researchers and not all stages of research receive funding.
We need your help. More funding is required to move the cord blood expansion research to a Phase 3 clinical trial—the final phase before it can become a treatment option that leads to faster recovery and better chances of survival for all patients. “Faster recovery also means fewer transfusions, shorter hospitalization and lower costs of the transplant procedure,” says Dr. Wagner. “This could revolutionize the way we do transplants.”
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Read more about blood and marrow transplants and the SR1 clinical trial in our 2015 Annual Report.