Cord blood is an accepted source of stem cells for any of the diseases where stem cell transplants are a standard therapy. This covers a long list of oncology and hematology diagnoses. Cord blood transplants have been in use since 1988, and by the end of 2009 there had been about 20,000 cord blood transplants world-wide (1). Yet, it was not until late 2011 that the US FDA finally licensed cord blood transplants for most of the diseases on the standard therapy list. While the FDA licensure does not change clinical practices at transplant centers, it does open up access to cord blood transplants for those patients whose health insurance providers had previously used the lack of licensure as a reason not to cover the transplants.
The definitive statistics on the rate of stem cell transplantation in the US were published in 2008 by Nietfeld et al. (2). The cumulative probability that a person will have some type of stem cell transplant by age 70, either from their own cells or from a donor, is approximately 1 in 200. This is not the probabiliity of needing a transplant, it is the probabilty of actually having one, assuming that there is no difficulty finding a matching donor. The study was based on transplant rates between 2001-2003 and relied on the national databases of diagnoses from SEER and transplantation from CIBMTR. The important caveat about this study, illustrated in the figure in the sidebar, is that rates of cancer increase with age, and thus the vast majority of transplant patients are older adults.
When parents contemplate cord blood banking, they consider the odds that their baby or an immediate family member will need a stem cell transplant. The cumulative probability that a child will have a stem cell transplant by age 10 is only 1 in 5,000 (.02%) for transplants from a donor and 1 in 10,000 (.01%) for transplants of the child's own cells. Medical societies have warned parents that the odds of their child needing stem cells from cord blood are very low, and if the only consideration is using the cord blood for a stem cell transplant, that is certainly true for the average person.
| Diagnosis | Transplant Type | Stage of Use | Odds by age 10 |
| blood disease or hereditary disorder |
stem cell transplant from donor | standard therapy | 1 in 5,000 |
| solid tumor | transplant of your own stem cells | standard therapy | 1 in 10,000 |
Emerging cell therapies is a catch-all phrase for any new applications of cord blood stem cells that are currently in clinical trials. Success stories from these trials have received a great deal of media attention. But not every patient is a success story, and until follow up studies against controls are completed, it is not clear what fraction of the patients benefited from the therapy and the measure of benefit they received.
Parents usually wish to bank cord blood for their family on the premise that many more uses for the cord blood are around the corner, and that is probably true, but it is not a certainty. For this reason, family cord blood banking is a form of "health insurance", where parents make an investment based on what is known now, as a hedge against future developments.There are a few pediatric disorders, summarized in Table 1 below, for which children are currently being treated with their own cord blood in clinical trials. What is most notable about these emerging therapies is that they all treat conditions that are not rare, conditions that a child is much more likely to experience than a stem cell transplant. The second important difference is that, whereas stem cell transplants require large cord blood collections, the emerging therapies can be successful with small collections.
The most well-known emerging therapy is to give a child his/her own cord blood stem cells for Cerebral Palsy. Since 2005, several hundred children world-wide have received this treatment for Cerebral Palsy and similar acquired neurological disorders (3). No one wants to imagine that their baby will be born with Cerebral Palsy, but the CDC estimates that 1 in 300 children in US elementary schools have Cerebral Palsy. The incidence of Cerebral Palsy among full term births is 0.2%, but among premature births the incidence is 10 times higher at 2% (4). For parents who are going through a pregnancy with a risk of premature birth, it is prudent to consider family cord blood banking in case the baby has this type of condition.
Lastly, there are currently (Feb. 2012) dozens of cell therapies in advanced (phase 3) clinical trials around the world that are using stem cells from bone marrow to treat medical conditions that afflict much of mankind: Stroke, heart disease, auto-immune disorders, neuro-degenerative disorders, and arthritis (ref: ClinicalTrials.gov). Just like cord blood can be substituted for bone marrow in stem cell transplants, any of the emerging therapies that are using stem cells from bone marrow could, in principle, use stem cells from cord blood or cord tissue. Table 2 below is a list of trials that are already using donor cord blood in cell thrapy for adult diseases.
| Diagnosis | Occurence in USA | Cell Therapy | Stage of Use | Clinical trial |
| Cerebral Palsy | 2 per 1000 full term births | child's own cord blood | clinical trials: phase 2 |
NCT01147653 NCT01072370 pending |
| Neonatal Oxygen Deprivation | 2 per 1000 full term births | child's own cord blood | clinical trials: phase 1 |
NCT00593242 NCT01506258 |
| Traumatic Brain Injury | 435,000 per year ages 0-14, #1 cause of death in kids | child's own cord blood | clinical trial: phase 1 |
NCT01251003 |
| Type 1 Diabetes | 1.7 per 1000 ages birth-19 | child's own cord blood | clinical trials: phase 1 & 2 |
NCT00989547 NCT00873925 |
| Hearing loss, sensorineural | 1 per 1000 births | child's own cord blood | clinical trial: phase 1 |
NCT01343394 |
| Hypoplastic Left Heart Syndrome | 0.2 per 1000 births | child's own cord blood | clinical trial: phase 1 |
NCT01445041 |
| Diagnosis | Occurence in USA | Cell Therapy | Stage of Use | Clinical trial |
| Cerebral Palsy | 2 per 1000 full term births | donor cord blood | clinical trial: phase 2 |
NCT01528436 |
| Type 1 Diabetes | 1.7 per 1000 ages birth-19 | donor cord blood | clinical trial: phase 2 |
NCT01350219 |
| cartilage repair | 10-25% adolescents have knee injuries | donor cord blood | Cartistem approved by Korean FDA | NCT01041001 |
| Critical Limb Ischemia | 2.5 per 1000 people, >80% of them diabetics | donor cord blood | clinical trial: phase 1 |
NCT01019681 |
References for body of page
References for tables of emerging therapies
