Cancer Center Expands the Possibilities for Cord Blood Stem Cells

We are pleased to report another step forward for umbilical cord blood!  This month, the Fred Hutchinson Cancer Center in Seattle announced that they have been successful in treating leukemia with the infusion of umbilical cord blood that has been manipulated, in the lab, to increase the number of usable stem cells.  Results of the Center’s Phase I study have been published in the January 2010 issue of Nature Medicine.

The inherent issue with using cord blood in such infusion treatment for leukemia is the relatively low number of stem cells in a standard cord blood sample; thus, until now the greatest potential for cord blood has been limited to children or small adults.  With the ability to expand the number of stem cells in cord blood, however, comes the expanded possibility for treating disease in a greater portion of the population.

Results from the Hutch study in 10 leukemia patients show that the infusion of expanded cord blood stem cells is not only safe, but also effective in cutting the amount of time to engraftment by half, as compared to non-manipulated cord blood stem cells.  The bottom line: the quicker engraftment takes place, the better the patient’s chance of survival.   Seven of the study’s 10 patients are still alive today, and show no sign of the disease.

Cord blood continues to be a promising alternative to bone marrow transplant, as they are less prone to “graft vs. host disease” and other complications relating to the recipient body rejecting foreign cells, and don’t require a perfect donor match.  In an earlier blog entry, we reported on a similar process for expanding the cord blood stem cell population using carlecortemcel-l (aka StemEx), as published by The University of Texas M. D. Anderson Cancer Center in Houston.  While additional study is needed in order to fully realize the potential of these results, the continued news of these developments is both encouraging and exciting – both for us at NECBB and for families that may need cord blood in the future.

Banked Cord Blood Saves a Life

At NECBB, we believe cord blood has unlimited potential – this is proven time and again by the heartfelt stories that we receive from our clients. Just in time for the holidays, our founders, John and Cathy Rizza, received a letter from Leighsa M. Leighsa’s infant daughter Cameron was diagnosed with Fanconi’s Anemia, a rare blood disease that can be fatal: doctors did not expect her to live past her 10^th birthday.

The daughter of a scientist and researcher, Leighsa believed in the potential power of cord blood for treating many different disorders, including her daughter’s. She later gave birth to two boys and banked their cord blood with NECBB. One of the boys, Conlan, was a 6/6 tissue match for his sister. In 2007, Cameron’s disease had progressed to a point where a transplant was necessary, and doctors were able to use her brother’s cord blood for the procedure.

The transplant was a success and today, nearly 3 years later, Cameron is a happy – and healthy – eleven-year-old.

Stories like these remind us of why we are doing what we do at NECBB. While the science is exciting and brings the constant discovery of new ways to treat disease with cord blood, a story like Cameron’s – where a little life was touched and saved because of cord blood – inspires and motivates us to continue in our mission to make these treatments available to families when they need them.

Potential Use for Cord Blood in Treating Heart and Lung Disorders

Two new animal studies published in the current issue of Cell Transplantation show that umbilical cord-derived stem cells may have therapeutic applications in the heart and lungs.

In one of the studies, performed at Samsung Medical Center in Seoul, Korea, researchers found therapeutic benefit from transplanted cord blood mesenchymal stem cells (MSCs) in newborn rats with oxygen-deprived lung injuries. There is still a long way to go before human application, but if perfected, this type of treatment could be the first to address the currently untreatable hyperoxic neonatal lung disease, or bronchopulmonary dysplasia (BPD), in premature infants.

The second study, conducted in Germany, found that autologous (self-donated) transplant of umbilical cord mononuclear cells (UCMNCs) improved heart function in sheep with a common cyanotic congenital heart defect, deeming the procedure feasible and safe.

This research takes us yet another step forward, expanding the list of diseases that umbilical cord blood can potentially treat and further strengthening our belief in the importance of banking this cord blood – publicly or privately – for future use.

First Human Study Using Dental Stems Published

We are thrilled to report an exciting development in stem cell research: the first human study using dental stem cells was published November 12^th in the European Cells and Materials journal! This is great news for our National Dental Pulp Laboratory, which stores dental stem cells and has long believed in their potential for future medical use.

In the study, patients had wisdom teeth that were impacted, which caused bone loss (resorption) at the site of impaction. Because the bone defect would not repair on its own after the wisdom teeth were removed, the researchers used a mixture of dental pulp stem cells harvested from the patient’s non-impacted, upper wisdom teeth and placed them onto a “scaffold” made of collagen sponge. This mixture was then used to fill in the injured areas that were left when the impacted teeth were removed from the lower jaw. (The upper jaw area served as a control, or comparison, area—no dental stem cells were used there).

Three months after treatment, bone had completely regenerated at the injury site and the periodontal tissue had been restored. Optimal bone regeneration was seen in the seven patients who returned for the one year follow up. The investigators concluded that this clinical study demonstrates that dental stem cells and a collagen sponge scaffold can completely restore bone defects in the human jaw and indicates that these cells have the potential to repair and/or regenerate tissues and organs

Previously, jaw defects had been repaired using dental stem cells in an animal model only—never in humans. In fact, no dental stem cell therapies have ever been shown in humans. As you can guess, this bone grafting study is very exciting for all of us who believe in the future promise of dental stem cell therapies—whether a dental stem cell banking facility like our own NDPL, or individuals who want to preserve their own or their children’s pulp in order to have a source of stem cells that they might be able to put to use for future medical needs.

Good Reasons to Bank Umbilical Cord Blood- Publicly or Privately

There are plenty of good reasons to bank cord blood. Not only is collecting cord blood simple, painless, and harmless to the mother and baby, but cord blood stem cells are currently being used to fight a wide range of diseases—over 80 of them! And, every day, research is being conducted worldwide to find more and better ways to use the stem cells from cord blood.

The question faced by expectant parents who are considering cord blood banking, then, is whether to donate the blood to a public cord blood bank (in which case the blood would most likely go to an unrelated donor) or to privately bank the blood for possible later use by the child or family. Some organizations have spoken out against private banking, arguing that the benefits to the child (autologous use) are limited and that donation to public banks should be encouraged instead.

We at the New England Cord Blood Bank (NECBB) think that families should have the option to publicly bank their child’s cord blood if they would like to do so for altruistic reasons, but we also believe that parents should have the option of saving their child’s own cord blood in case a life-threatening condition should arise for their child or other family members in the future.

Should a life-threatening condition that requires a stem cell transplant arise in your child or family, private banking provides immediate access to a source of stem cells with a higher probability of being an exact or acceptable match to the child or a family member as compared to stem cells from a non-related donor. For example, the probability that the stem cells will be an exact match is 1/1 for the child from whom the stem cells were collected, 1/2 for the parents of the child and 1/4 for the child’s siblings.

In addition, as research progresses and more uses for cord blood stem cells are found, privately banking this blood may become even more relevant.

Fortunately, we are not alone in this thinking. In a recent journal article by Hollands and McCauley in the journal Stem Cell Reviews and Reports, the authors state that the scientific evidence shows that private banking is a reasonable and rational choice, and that many of the critics of private cord blood banking have overlooked the potential benefit to the siblings or parents of the child from whom cord blood has been collected: cord blood transplants from related donors are superior to transplants from unrelated donors in terms of clinical outcome. In addition, the immediate availability of privately stored cord blood is an important benefit, as the sooner treatment is available, the lower the risk of death or advanced illness.

Growing the Potential for Umbilical Cord Stem Cells

An exciting development is expanding the possibilities for umbilical cord stem cells, according to an article published in the November 2009 issue of Expert Opinion on Biological Therapy. The article discusses the development of carlecortemcel-l (brand name StemEx), an umbilical cord blood stem cell graft created by encouraging proliferation of the stem cells – without allowing them to differentiate.

What does this mean, and why is it important?

While there is a great amount of evidence on the healing potential for stem cells extracted from umbilical cord blood, it has been argued that the limited number of stem cells available from a single banked unit is not enough to treat patients other than children or very small adults. By creating a method for multiplying these stem cells in their vital pre-differentiated state, researchers can potentially overcome this challenge and widen the applicability of many stem cell treatments.

Results of a Phase I study of carlecortemcel-l conducted at The University of Texas M. D. Anderson Cancer Center, as reported in the article, suggest that the infusion of these expanded umbilical cord stem cells is safe – and may be beneficial for adults and adolescents with leukemia and lymphoma. A follow-up Phase II/III safety and efficacy study called ExCell is currently underway, enrolling adolescents and adults with high-risk hematologic malignancies at clinical sites in the U.S., Italy, Spain, Hungary and Israel. Gamida Cell and Teva Pharmaceuticals, co-developers of StemEx, anticipate the product officially reaching the bedside by 2011.

Reprogramming Cord Blood Stem Cells

Induced pluripotent stem cells (iPSCs) have been in the news quite a bit of late, specifically with a report over the summer of two Chinese studies showing that stem cells created from adult mouse skin cells had the ability to regenerate in much the same way as embryonic stem cells, resulting in both cases in the birth of baby mice.

It’s looking like cord blood cells can be reprogrammed in much the same way as the adult stem cells in those studies – but with fewer factors and in less time, according to Juan-Carlos Izpisúa Belmonte, Ph.D., a professor in the Salk Institute’s Gene Expression Laboratory, who led his own study published in the October issue of the journal Cell Stem Cell.

This news takes some wind from the sails of one argument against using one’s own banked cord blood stem cells to treat disease: that the stem cells may contain the same genetic mutation, or be predisposed to the mutation, that caused the disease in the first place. By reprogramming the cord blood stem cells, however, scientists can potentially remove the possibility of this ever happening.

Still Potential for Prochymal?

Today’s news that Prochymal, Osirus Therapeutics’ mesenchymal stem cell (MSC) therapy, failed in late stage clinical trials in acute graft-versus-host disease (GvHD) is disappointing. GvHD is a serious complication that occurs in some individuals who have received bone marrow from a donor. If the trials had been successful, Prochymal would likely have been the first commercial stem cell product approved by the FDA.

However, there are still reasons for hope. First, in one of the GvHD trials, patients with GvHD affecting their liver or gastrointestinal tract who did not respond to steroid treatment appeared to benefit from Prochymal, although the results are still preliminary. In addition, Prochymal is still currently being tested in human trials for several other indications, including type 1 diabetes, heart attack, and chronic obstructive pulmonary disease (COPD). The same type of stem cells utilized in Prochymal – MSCs – can also be found in dental pulp, which is currently extracted and stored privately for families by our affiliate, the National Dental Pulp Laboratory. We and many others will continue to follow the news and hope for the best as this potential stem cell treatment is tested in these areas.

Enhancing technology, emphasizing our promise to families

We are pleased to announce that we have officially started processing cord blood using our new automated blood processing system, the AutoXpress System (AXP). This state-of-the-art system maximizes the number of valuable stem cells extracted from the cord blood – the AXP can harvest greater than 97% of the mononuclear cell population, which is rich in stem cells, without the use of additives.

AXP also minimizes the risk of contamination and produces data that will help us create the highest quality blood samples.

In addition to the AXP processing, we will continue to manually process samples that are too small for the automated system.

We believe that by heightening our technology, we can provide an added feeling of confidence and security to the families that choose to trust us with their most precious gift.

The Today Show showcases NDPL and a family’s hope for dental pulp

NBC Today aired a segment today featuring our affiliate National Dental Pulp Laboratory (NDPL) and a family that banked their daughter’s dental pulp with us. Dental pulp is found in baby (and other) teeth and contains the potential for treating many diseases – including type 1 diabetes.

Mesenchymal stem cells (MSCs), the stem cells found in dental pulp, can differentiate into other cell types, such as cardiac and muscle cells. Animal testing has shown great progress in regenerating teeth with dental pulp cells, and human trials are not far behind.

The family assured Today Show viewers that their decision to bank their daughter’s dental pulp was an informed one, and they feel that it is an investment in their daughter’s well-being. We take pride in the fact that we provide families with detailed information on dental pulp banking, and counsel them personally before they make a decision.