When Sophie Lee was three years old, doctors noticed something odd in her blood work.
Aside from some sporadic leg discomfort, she seemed perfectly healthy. She was energetic, athletic and happy, so doctors decided to monitor her situation.
At 5 years old, she received a scary diagnosis.
Sophie had Myelodysplastic Syndrome, a rare blood disorder and precursor to an aggressive form of blood cancer called Acute Myeloid Leukemia (AML), which has the lowest survival rate of all types of leukemia.
Doctors told her parents, Anthony and Judy Lee, that Sophie would eventually develop AML if left untreated. She needed to flush out her own blood cells and replace them with healthy ones through a bone marrow transplant.
To get a transplant, Anthony and Judy needed to find a bone marrow donor match for their daughter. She didn’t match with either parent because she was adopted from Taiwan.
For months, they searched.
They checked public bone marrow donation registries in the United States, then Asia and Europe.
“Unfortunately after six months we never found a true good bone marrow match for her,” Anthony said.
But the Lees had another option: an umbilical cord blood donor.
Umbilical cord blood can work as an alternative for patients who can’t find bone marrow transplants. It is full of stem cells, which have some ability to turn into different types of cells, including blood cells. Because of this, cord blood can be transplanted into patients to treat certain types of cancers, metabolic diseases and blood disorders.
Without cord blood donations, the Lees would have been out of options. Donating cord blood is free of charge to parents and carries insignificant health risks, but health care providers say most mothers still choose to dispose of the cord blood as “medical waste.”
In Arizona, obstetrics health providers are required by law to educate expectant parents about the options to publicly donate or privately bank cord blood.
Scottsdale-based OB-GYN Kelly Helms said she tries to encourage her patients to donate, but many moms find the process tedious.
“You have to do a bunch of paperwork,” she said. “Some of them just don’t do it.”
Benefits of cord blood
As an alternative for transplants, cord blood stem cells offer some benefits over using bone marrow stem cells, according to Holly Miller, a doctor who performs between 40 and 50 stem cell transplants every year at the Phoenix Children’s hospital.
“What’s helpful for the umbilical cord blood transplants is that they tend to be more forgiving than bone marrow,” she said. “Patients undergoing cord blood transplant have a lower risk of immune complications to it because the cells tend to be more naive.”
They are naive, or immature, because the cells are so young and have not been exposed to outside elements, she said, and they carry a lower risk of graft-versus-host disease, in which the donor cells attack the recipient’s body.
This added flexibility means that doctors do not have to find a perfect 10 out of 10 genetic match for the recipient, an advantage when the recipient has a lower chance of finding a good match.
“Patients who are of Caucasian descent have the best chance of finding a match,” Miller said. “Unfortunately we do not have those same outcomes for patients who are of a minority, particularly, the hardest group that we have finding stem cells for are going to be our African American population.”
One drawback of cord blood is that the amount of cells in a given donated sample is small, so the samples are typically only big enough to use for transplants in smaller patients, such as children.
Limited public donation program
To make it easier to donate, Arizona has a state-funded public donation program that is operated in four locations: Dignity Health St. Joseph’s Hospital and Medical Center, Dignity Health Chandler Regional Medical Center, Valleywise Medical Center and Tucson Medical Center.
“If you’re not delivering at one of the participating hospitals, it is very difficult to be able to donate,” said Wendy Barrett, the cord blood program supervisor at Dignity Health St. Joseph’s Hospital and Medical Center and Chandler Regional Medical Center.
Those two facilities average about 800 donations of cord blood every year. Barrett would love to see more, but some mothers aren’t able to donate, and expanding the program would depend on additional funding from the Arizona Department of Health services, she said.
“There is a constant need for donations and we have some eligibility criteria that the mother has to meet,” she said. “It’s important that we keep that public system safe for the person who is going to receive the transplant.”
The criteria include the mother being free of any major health issues and not having travelled to any disease-risk areas, such as Mexico, where there is a greater risk of mothers contracting the Zika virus.
If a mother meets all the criteria, she can still run into issues on the day of delivery. The public cord blood donation bank is not open on Sundays or on major holidays, so if a mother delivers on one of those days, her donation can’t be collected in time to be frozen at ClinImmune Labs, an FDA-approved blood banking facility in Colorado that the state works with.
There, Brian Freed, the lab’s executive director, said samples are analyzed to see whether they’re large enough to be used in a transplant. Since babies are small and cord blood is limited, only 10% of donations contain enough cells for transplants, he said.
“Getting enough for transplant can be kind of tricky, but we can’t predict that,” Barrett said.
Every year, Freed said the lab transplants 120 cord blood samples, and banks about 150 cord blood samples out of the 1,500 or so donations that they receive.
“We’re basically replacing the ones that come out of the bank with new, bigger units,” he said.
Donating for research
If transplant isn’t an option, parents can decide whether they would like their samples to be donated to research.
Much of this research focuses on the potential for cord blood stem cells to be used in regenerative medicine, according to David Harris, a University of Arizona umbilical cord blood stem cell researcher.
In the past two decades, Harris said he has been involved with studies to treat kids with conditions like cerebral palsy, strokes, traumatic brain injuries and diabetes.
“There’s two where we’ve seen really good success. One is orthopedic injuries … we’ve seen that work very well in terms of relieving that pain — sometimes it actually helps to regrow the tissue,” he said. “The other one we have very good success with is stroke…There’s like an 80-85% cure rate for kids with strokes.”
While other hospitals may offer the option to donate cord blood, the benefit of donating through the state program is that it ensures the cord blood will be used for FDA-approved purposes.
“We won’t deal with anybody who’s not legitimate,” Freed said.
Because of the hype around stem cells and their potential for regenerative medicine, there has been an explosion of stem cell clinics throughout the country. Those clinics inject patients with stem cells, some of which they claim come from cord blood. Many of these procedures are not FDA approved and it’s unclear where doctors get cord blood stem cells, but Harris thinks some hospital donations may end up going to stem cell clinics.
“I don’t think the moms would be happy if you’re selling it to some of these clinics,” he said.
Freed said ClinImmune Labs gets a lot of wonky requests from people who haven’t gone through the FDA but who want some of their banked cord blood samples.
“We have been contacted by groups that want to use it for injecting the patients knees and stuff,” he said.
To check the legitimacy of a customer’s request before releasing a sample, a ClinImmune Labs representative sets up a phone call to discuss the planned use of the cord blood and requests a letter with a description of the research project.
The lures of regenerative medicine potential also drives some parents to pay for private banking of their children’s cord blood instead of donating the cord blood, in the hopes that their children will use it in the future.
“I think most people think about umbilical cords nowadays and think about how they can use it for themselves, and not how they can use it as a treatment for others,” Judy Lee said.
Without a donated cord blood transplant, Sophie’s prognosis would have been poor, she said. Because of the life-saving potential of the blood, Judy said she encourages people to donate cord blood if they can — because you never know who might need it.
“The question is, what would we have done?” she asked.
Fortunately, she and her family didn’t need to face the scary prospect of not having a transplant donor.
Sophie is now 10 years old. At four years post-treatment, she remains cancer-free and is a competitive child who plays ice hockey, shoots basketball with her father in the backyard, and tries to beat her mother at Wii tennis. She is active and healthy, all thanks to a cord blood transplant.
PRIVATE BANKS: Cord blood banks sell parents on promising stem cell research, but with no guarantees
Amanda Morris covers all things bioscience, which includes health care, technology, new research and the environment. Send her tips, story ideas, or dog memes at [email protected] and follow her on Twitter @amandamomorris for the latest bioscience updates.
Independent coverage of bioscience in Arizona is supported by a grant from the Flinn Foundation.
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