Cord Blood Success Stories: 5 Inspiring Cases
Cord blood is saving lives and transforming treatments. From curing leukemia to helping children with cerebral palsy, cord blood stem cells are used in established transplant procedures for over 80 diseases, and research continues to explore their potential in new areas. Here's what you need to know:
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Leukemia: Cord blood saved a 3-year-old boy with a rare leukemia after traditional donor matches failed.
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Genetic Disorders: 90% of children with leukodystrophy survived 3+ years after cord blood treatment in one study.
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Cerebral Palsy: Children in a key clinical trial showed motor function improvement after receiving cord blood infusions.
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Sibling Donations: Families are using stored cord blood for established treatments like sickle cell disease.
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Emerging Uses: Conditions like autism and brain injuries are being studied in clinical trials using cord blood therapies.
With thousands of transplants performed yearly using hematopoietic stem cells (including those from cord blood), cord blood is proving to be a powerful medical resource. Read on to discover the life-changing potential of these treatments.
1. Leukemia Treatment Through Cord Blood
In 2023, three-year-old Tommy Bacon from Australia, diagnosed with juvenile myelomonocytic leukemia (JMML) - a rare and aggressive form of leukemia - underwent a life-saving stem cell transplant. The donor? His newborn sister Aria. This came after two unrelated donor matches fell through. JMML is typically fatal within a year if untreated, but Tommy reached remission in just four months, highlighting the impact of family cord blood banking for established treatments.
Recent studies back this success, showing cord blood transplants are effective for high-risk leukemia cases. For patients treated during their first complete remission (CR1), the data shows a 59% five-year overall survival rate and a 52% leukemia-free survival rate [2].
I would strongly recommend that if you're thinking about getting cord blood collected, do it! If it can change a life in such a huge way, why would you not? - Kylie, Tommy's Mum
Cord blood's less developed stem cells allow for more flexible HLA matching [3], making it a vital option when traditional donor matches aren't available. Dr. Filippo Milano, a researcher from Fred Hutch, highlights its importance:
Patients going into transplant with minimal residual disease, they have very dismal outcomes. [3]
Dr. Colleen Delaney from Fred Hutch's Cord Blood Program adds:
This brings home the point that cord blood shouldn't be called an alternative donor. The outcomes are the same as a conventional donor. [3]
2. Cord Blood Treatment for Genetic Disease
In 2020, researchers at UPMC Children's Hospital of Pittsburgh explored the use of cord blood to treat genetic disorders that are not cancer-related. Dr. Paul Szabolcs and his team treated 44 children with conditions like Krabbe disease, metachromatic leukodystrophy (MLD), and Hunter syndrome using established transplant protocols. Their method combined low-dose chemotherapy and immunosuppressants with intravenous cord blood infusions. Among 30 children with metabolic disorders who had shown neurological decline before the treatment, all reached normal enzyme levels within a year, and their decline stopped entirely [4].
Dr. Szabolcs highlighted the potential of this approach:
We designed an approach now proven to be efficacious for at least 20 diseases. And we believe it might be effective for many, many more. [4]
The study's findings were striking. Over 90% of leukodystrophy patients with symptoms survived at least three years after treatment, a significant improvement compared to the previous survival rate of 60% [4]. Additionally, this method showed potential advantages for diverse populations. Cord blood transplants allow for greater flexibility in donor matching, which is particularly beneficial for ethnic minorities. Dr. Szabolcs explained:
That's huge for ethnic minorities. The probability of a perfect match is very low, but with a cord blood graft, we have a chance to overcome this discrepancy over the course of a couple months and then taper immunosuppressants away. [4]
Cord blood's less mature stem cells help lower the risk of graft-versus-host disease (GVHD), a potential complication of transplants, making it safer for young patients. Cord blood is currently used in transplant medicine for over 80 diseases, reinforcing its growing role in addressing severe genetic conditions.
3. Cerebral Palsy Treatment with Cord Blood
A Phase II, double-blind trial conducted at Duke University studied the effects of autologous cord blood infusions on 63 children with cerebral palsy, with a median age of 2.1 years. The findings revealed that children receiving doses of at least 2×10⁷ cells/kg experienced improvements in their Gross Motor Function Measure-66 scores and brain connectivity [6]. It is important to note that this remains an area of active research, and cord blood infusion is not currently an FDA-approved standard treatment for cerebral palsy.
Dr. Jessica M. Sun from Duke highlighted that properly dosed cord blood infusions *can help improve* brain connectivity and motor function in children with cerebral palsy based on their trial data. She pointed out that even small changes - like better hand orientation - can have a meaningful impact on a child’s ability to perform everyday tasks [7][8].
Cord blood contains various stem and progenitor cells which researchers believe may aid in neural repair by reducing inflammation, supporting cell survival, and encouraging the formation of new blood vessels [8].
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.
Dr. Joanne Kurtzberg, director of Duke's Pediatric Blood and Marrow Transplant Program, underscored the potential of this treatment, while emphasizing the need for further research. The study's outcomes highlight the expanding possibilities being explored with cord blood therapies [8].
4. Family Cord Blood Donation Success
Family cord blood donations have proven to be lifesaving, especially for established treatments when other donor options fall short. They’ve been effective in treating a variety of medical conditions, highlighting the importance of saving newborn stem cells for potential family use.
For instance, sibling cord blood donations have shown promise. Take Charlotte, who struggled with basic tasks like holding a water bottle or speaking due to cerebral palsy. After receiving cord blood cells from her younger sister Emma in an investigational setting, her progress was evident according to her family.
Charlotte was quite static in her development and progress up until the infusion and then we saw this noticeable difference, shares Laura West, Charlotte's mother [9].
Statistics back up the potential of sibling donations: there’s a 25% chance of a perfect HLA match for cord blood stem cells between full siblings, and a 50% chance of a partial match, which is often sufficient for cord blood transplants. Since 1988, over 40,000 cord blood transplants have been carried out globally [13].
Another inspiring case is that of ten-year-old Eli Cola-Achille. In 2021, Eli received cord blood and bone marrow stem cells from his younger brother Gus at the Dana-Farber Cancer Institute. The treatment led to a significant reduction in his sickle cell disease symptoms, allowing him to return to school and resume everyday activities [13].
Our study found infusing matched sibling cord blood cells into children with cerebral palsy is a relatively safe procedure; however, it should only be conducted in tertiary hospitals with facilities to treat the infrequent adverse reactions [9].
These examples highlight how preserving cord blood can offer critical medical options for FDA-approved conditions and provide access to ongoing clinical trials.
5. New Medical Uses for Cord Blood Under Investigation
Advances in regenerative medicine research have opened up new possibilities for using cord blood to treat conditions once considered untreatable, although these applications are still largely **experimental and require more research**.
Individual stories reflect growing research interest. Reflecting this trend, reports from some *private banks* indicate that a significant portion (nearly half or more) of their *client sample releases* are used for investigational regenerative medicine therapies. While future projections suggest regenerative medicine *could potentially* benefit a large portion of the population someday, this depends heavily on continued research and therapeutic breakthroughs.
Ongoing research is pushing the boundaries even further. Hundreds of clinical trials worldwide are currently exploring cord blood and cord tissue therapies for conditions like brain injuries, stroke, hearing loss, heart attack recovery, type 1 diabetes, and even corneal repair.
To support access to both established therapies and potential future breakthroughs identified through research, services such as those provided by Americord Registry offer options to preserve cord blood.
Conclusion
Cord blood therapies discussed in these five cases highlight the progress made in medicine. Since the first cord blood transplant in 1988, hematopoietic stem cell transplants (using sources like cord blood) have become standard care for dozens of conditions, with thousands performed globally each year[1], showcasing advancements in medical science.
Cord blood is a great option that really should be considered in line with the other donor sources. It shouldn't be considered a second or third or fourth choice for patients. That's especially true for pediatric patients where we're trying to provide them with quality of life for decades to come as well as curing their disease.[5]
For me, the number one benefit of cord blood transplantation is the marked decrease in chronic graft-versus-host disease that cord blood patients have after their transplant, presuming everything else is going well. That is probably the number one, two and three reason why we are such big fans of cord blood.[5]
The growing market reflects this confidence. Valued at $1.3 billion in 2020, the cord blood market is expected to hit $4.5 billion by 2030[10], driven by established uses for nearly 80 diseases[10] and ongoing research into new applications. Cord blood transplants also require less strict HLA matching compared to bone marrow transplants[1], making them accessible to more patients, and have a lower risk of transmitting certain infections. By 2021, over 700,000 units were stored in public banks and 5 million in private facilities worldwide[10].
From established treatments for leukemia to investigational therapies for cerebral palsy and autism, these cases showcase cord blood's vital role and future potential. While it's important to distinguish between standard treatments and experimental procedures, the clinical and economic advancements underline cord blood's growing importance in medicine, offering hope to countless families. With ongoing research, cord blood is set to play an even larger role in medicine.
References
https://www.dukehealth.org/blog/value-of-saving-umbilical-cord-bloodhttps://pmc.ncbi.nlm.nih.gov/articles/PMC5569913/https://www.fredhutch.org/en/news/releases/2016/09/Umbilical-cord-blood-transplant-is-associated-with-high-survival-rate-among-high-risk-leukemia-patients.htmlhttps://www.chp.edu/media/news/071420-ucb-for-congenital-diseaseshttps://network.nmdp.org/partners/transplant-centers/cord-blood-transplant/cord-blood-benefits-and-use-considerationshttps://pubmed.ncbi.nlm.nih.gov/29080265/https://medicalxpress.com/news/2017-11-cord-blood-motor-function-cerebral.htmlhttps://corporate.dukehealth.org/news/umbilical-cord-blood-improves-motor-skills-some-children-cerebral-palsyhttps://cerebralpalsy.org.au/news-stories/landmark-study-finds-sibling-umbilical-cord-blood-can-be-safely-used-as-a-treatment-for-cerebral-palsy/https://www.dovepress.com/advancements-in-umbilical-cord-biobanking-a-comprehensive-review-of-cu-peer-reviewed-fulltext-article-SCCAA
