Special Needs Resources The Promise of Cord Blood for Kids with Special Needs « Previous Next » Megan Krueger • March 27, 2012 1 Comment Tweet Photos by Kristen Hines In April 2011, Allison Thurman, then 2 years old, pulled herself up on her small metal walker. As big sister Audrey, 4, cheered her on and her parents Erica and Mike watched in anticipation, Allison took her first independent steps down the long, hardwood hallway of their St. Clair Shores home. "It was really very exciting," Mike says. Allison's first steps were monumental considering her circumstances. Allison was diagnosed with cerebral palsy, a brain abnormality that affects the nervous system and motor skills, just three months prior to taking steps with her walker. The Thurmans credit Allison's rapid and significant progress that spring to an infusion of her umbilical cord blood cells, which they had banked after her birth. They did not know that the measures they took as an "insurance policy" for their child would someday help their daughter – and contribute to groundbreaking research. Last year, Allison was accepted to participate in the first FDA-regulated clinical trial studying the use of cord blood cells to treat cerebral palsy – something one of Allison's many doctors encouraged the family to look into. It was merely two weeks after her first injection that Allison took those steps with her walker. Before the injections, she was unable to pull herself up to it without additional support. For the Thurmans and many other parents of children with special needs, cord blood cells have been viewed as a potential miracle, one that could change their children's lives for the better and perhaps even eradicate many of the challenges of their disability. And yet the full potential of cord blood cells has yet to be fully explored. According to the Cord Blood Registry (CBR), the world's largest private stem cell bank, only about 5 percent of expectant parents donate or bank the cells. Because of that low rate, studies are delayed or incomplete, hindering further scientific exploration and development of their potential – and an avenue of hope has been lost to scores of children with special needs. How cord blood cells work Cord blood cells are stem cells extracted from a newborn's umbilical cord just after the cord is cut. These cells are "the body's building blocks for organ tissue, blood and the immune system," according to CBR. "Cord blood is rich in hematopoietic stem cells (HSC) – the type of stem cell that gives rise to blood and the immune system," explains Kate Brown, content scientist at CBR. In particular, cord blood stem cells have been found to have "regenerative potential," meaning they're able to "find injured cells and tissue in the body and initiate a healing process," CBR's website says. Once the blood is taken from the cord, it is picked up from the hospital by a bank and processed to deplete red blood cells and plasma, leaving just stem cells. Then, it is frozen and stored, Brown explains. Brown says cord blood cells "have been used for over 20 years in applications in traditional transplant medicine." In these cases, she says, cord blood cells have been used to treat cancers, diseases or blood and immune disorders that are also treated with bone marrow transplants. Today, Brown says researchers are exploring "a whole new area" of cord blood cell use. "The emerging investigation is into regenerative medicine – which is looking at cerebral palsy, traumatic brain injury and hearing loss," she says. "These are conditions where there's no cure at this point." Clinical trials in regenerative medicine are focused on the use of a child's own cord blood cells to treat these non-life threatening conditions because it is viewed as "the best and safest option for their treatment," Brown says. "Donor stem cells, even from a close relative such as a sibling, are not a perfect genetic match to the patient," she adds, "and so the cells will be rejected unless the patient undergoes a pre-treatment conditioning regimen to… suppress their immune system." With a patient's own cells, however, "There is no risk that the cells will be rejected by the patient's immune system." Parents have a few options when it comes to cord blood, though. They can bank the cells at a private bank for family use, she says, or they have the option of donating to a public bank, which would designate the cells for public use in transplant medicine. The third option – the one chosen by most new parents – is to throw cord blood away. Brown says she hopes most OB-GYNs are explaining the value of cord blood that is being discarded as "medical waste," and that families are "at least educated on it and know their options." However, it somewhat depends on the state, she says, adding that some states have legislation requiring education on the practice. The law in Michigan encourages health care facilities to educate the public on the value of cord blood and to educate expecting mothers on their options for banking and donating, Brown says. In Michigan, public health code specifies that health care professionals, facilities and agencies distribute "information to a pregnant woman before her third trimester of pregnancy." It's definitely on the radar. And, for one local family, those stars aligned. Allison's story When Mike and Erica Thurman's OB-GYN presented them with a pamphlet about banking cord blood cells, Erica was about 15 weeks pregnant with their first daughter, Audrey. She read through the informational materials and signed up to receive the collection kit to take with her to the hospital. "It was the 'just in case' for insurance," Erica says. "We're just lucky that (Erica's) OB talked to us about all of the options," Mike says. So, when she was pregnant with Allison, "There wasn't really any question if we were going to do it," Erica says. "We said, 'We did it with one, we can't not do it for the other.'" In March 2009, Allison was born at 32 weeks – almost two months premature – and spent about two weeks in the neonatal intensive care unit. It wasn't until Allison was around 7 or 8 months old that the Thurmans started realizing she wasn't reaching her milestones, Erica says. "We talked to our pediatrician(s), and they were indicating that we had to allow some time for prematurity," Erica says. Allison eventually began physical therapy around age 1, and the family began taking her to a neurologist, as well as a physical medicine and rehabilitation doctor. In December 2010, she was clinically diagnosed with cerebral palsy. And, after an MRI, they were given the definite diagnosis in January 2011, the Thurmans say. "About half of the kids who have cerebral palsy were born premature," says professor Seth Warschausky, who works with the Cerebral Palsy Program at the University of Michigan in Ann Arbor. Cerebral palsy can affect multiple things, including mobility, hearing, vision, attention span and thinking in general, he explains. According to the U.S. Centers for Disease Control and Prevention, cerebral palsy is the "most common motor disability in childhood." It was after the clinical diagnosis in late 2010 that Erica mentioned to one of Allison's doctors that they had banked her cord blood cells at birth. That doctor encouraged the family to research clinical trials involving cord blood cells and cerebral palsy – which led them to Dr. James Carroll's clinical trial at Georgia Health Sciences University. The cerebral palsy trial About two years ago, Carroll, chief of pediatric neurology at GHSU, launched the first FDA-regulated cerebral palsy clinical trial. It's evaluating the use of a child's cord blood cells to ease cerebral palsy symptoms, making it one of a small number of studies looking into regenerative medicine. "The hope is it would promote healing in the injured part of the brain," Carroll says. "We want to see if it does make a difference." The study, Carroll explains, requires the participant to make four visits over the course of a year. Of the four visits, there are two infusions as part of the "blinded" study – meaning one infusion is a placebo and the other is of the participant's stem cells. It's unknown at which visit a participant will receive their stem cells. The other two visits are evaluations, Carroll says. "A comparison is being made at the three-month period. Then, half of children would have received cord blood cells," he explains. "The third and fourth visits are check-ups to see how children are progressing or reacting to treatment." The study requires participants to have cerebral palsy and access to their own cord blood cells. Because of the requirements, the study has thus far been limited and slow going, Carroll says. "We're aiming for 40 (participants), but they've been very slow in being accessed – because most children with cerebral palsy don't have their umbilical cord blood," he says. "So we've had about 10 (participants)." Since its launch, a second, similar trial at Duke University has kicked off. Like the Georgia study, Duke's study started about two years ago and is still in need of more participants. Seeing results Allison's first visit to Georgia was in April 2011, the Thurmans say – a trip made possible by CBR's nonprofit, the Newborn Possibilities Fund. The Thurmans banked Allison's blood with CBR, which qualified them for the study. During that initial visit, Allison received her first infusion. The infusion itself took about five minutes, Erica recalls, but the hospital required the Thurmans to stay for 15 hours to ensure Allison's vitals were clear. "I was thinking, 'How are we going to keep her entertained for that length of time?'" Mike says. "She was hooked up to a lot of monitors, so that's asking a lot of a 2-year-old to be hooked up for that amount of time." As medical staff checked on Allison every half hour, the family kept her occupied with bubbles, toys, snacks and her favorite movie: Toy Story. "Allison handled it much better than we did," Mike recalls. "She was an angel the whole day." Afterward, the family returned to St. Clair Shores. And, about two weeks after the infusion, they were astounded by what they saw. "We believe that she got the stem cells after her first visit," Erica says. "Within days of us returning from Georgia, we saw a dramatic increase in her vocabulary – and, within the month, she graduated from speech therapy." The Thurmans say that the October prior to that April infusion, Allison tested around the 12-month mark in speech. By mid-May 2011, the speech pathologist reported Allison had tested above her age group, Erica says. Mike and Erica also noticed improvements in Allison's mobility after the April infusion. "Allison was walking with a walker during therapy and needed to be supported by her hips before we left," Erica says, "and within two weeks after the first visit, she started independently pulling herself up in her walker – and walking by herself in the walker." Why are so few parents banking? Multiple studies are working to prove the benefits of saving a child's cord blood cells. And, for studies in regenerative medicine, it is necessary for participants to have access to their own cells. However, many studies like the one Allison participated in are delayed due to a low number of children having access to their cord blood cells. The CBR reports that "95 percent of the time, this source of potentially life-saving stem cells is thrown away as medical waste." Three out of every four expectant mothers "consider themselves 'minimally informed' about cord blood banking," CBR adds. "It is difficult to know exactly why the number of families that choose to bank is not greater," CBR's Brown says. "One possibility is that many families are simply unaware of the value of the stem cells in cord blood." CBR, she adds, is working to educate more people on the benefits of cord blood cells so that fewer cells are discarded after birth. The bank, Brown says, recently launched a website offering an interactive outline of options for cord blood banking. It also works with the medical community to provide resources and information, she says. Current clinical trials could also help spread the word on cord blood cells, Brown adds. "Hopefully what will come out (of trials) is a wider knowledge to the public about the potential these cells have," Brown says, "so that everybody does have access to their own." Cost may play a role, as well. "The cost of banking can vary between banks, with costs usually in the $1,500-$2,000 range," Brown says. Health insurance, she notes, does not cover the cost of banking cord blood, but CBR has partnered with some insurance providers to "offer special savings" on banking. After the initial start-up cost, banks charge an annual storage fee. That cost can vary, depending on the bank, but is about $125. Some providers also may help cover doctors' fees for collecting, Brown adds. CBR offers payment plans and a gift registry, so friends and family can contribute to the costs. The future for Allison Over the past year, the Thurmans have begun to notice a difference in Allison's muscle tone. Her once-rigid and stiff muscles have loosened up, Erica says. Now, they're working with her physical therapist, Donna Tavalieri of Pediatric Potentials in St. Clair Shores, to strengthen the weakened muscles. Tavalieri has been working with Allison since she was a year old. She says Allison was "moving in a really good direction" before the infusion, but "during and after infusion, she seems to move smoother. "We're trying to capitalize on that as best we can," she says. Today, Tavalieri is still working with Allison on her walker – and has been for about a year, she says. "It's a slow and steady process, but she has really continued (improving)," Tavalieri adds. Erica says Allison "still has issues with sitting up, but she's better." The Thurmans took Allison for her fourth and final observation visit to Georgia on March 5 – a moment Erica calls "bittersweet," when thinking back on the past year. Allison, who turned 3 at the end of March, is on a packed daily schedule with school and therapy sessions, Erica says, which keeps the whole family busy. She receives physical therapy and occupational therapy every week – in and out of school, which she now attends Monday through Friday. "Her teachers at school have remarked that she's very independent," dad Mike says, "and she likes to do things on her own and try different things. Even though she's got the physical disability, she seems to be participating in a lot of things." In the meantime, Allison – who her dad says has "a great sense of humor" and "sparkling personality" – is a bubbly kid who enjoys snuggling and cooking in the kitchen. The family bought her a special stool for Christmas that helps to boost her up to the counter, Mike says. Allison and Audrey both enjoy playing, reading books and swimming together, Erica says. "(Allison's), in my opinion, very normal and very smart for her age," Mike says. "Other than the fact that she's not walking independently right now, you wouldn't know that there's really anything wrong with her." Cells and special needs Erica and Mike both say they want to see Allison "be happy and reach her maximum potential, whatever that may be" – in addition to seeing her walk independently and "live life to the fullest." "One thing we try to stay away from is to say we want her to be 'normal,' because to us she is normal," Erica says. "You really can't say that, just because some people may look at her and say she's not normal. Because to us she is." But without completed clinical trials, experts say it's difficult to say what the future of cord blood cells will be in regenerative medicine. "We're all very excited about regenerative medicine," Brown says. "We're excited for the sort of things we're seeing. Just the fact that we're seeing these trials, that says a lot as to where this could go." Erica says there's only a small sample of Allison's cells left that they most likely won't be able to use for anything. But that's OK with her. "It was a decision that we had to make," she says. "We had to ask ourselves, 'Is this something we want to use?'" she says. "It's a catch-22, and it was a decision – but it wasn't a hard decision, just because you have to take that chance. Even though it's a gamble, that's why we had them." The Thurmans would "definitely encourage other parents" to save their child's cord blood cells. "You just never know. We didn't know there (were) any problems with Allison. And we did it with our first daughter," Erica says. "We think it's a very special gift you can give your child as an insurance policy."