was World Cord Blood Day (WCBD), a day organized and hosted by Save the Cord Foundation to promote awareness and education on the topic of cord blood. The day’s presentations centered around the research and incredible life-saving potential of cord blood stem cells. Several great speakers virtually tuned in to provide the general public with exciting new research advancements and inspiring stories from cord blood transplant families. Don’t fret if you missed the events of the day-- we’re here to provide you with the highlights from the keynote researchers!
Cord Blood Stem Cell Expansion
One common topic focused on cord blood expansion. Researchers have been working on expanding cells from cord blood or other blood sources for three decades. Today, we know of at least three technologies that do expand stem cells in the cord blood. The most advanced and farthest along in the commercialization process is technology discovered by the company, Gamida Cell. They have been able to isolate the CD133 cells and grow them in nicotinamide and growth factors for three weeks to expand the cell count 400-fold. As this clinical research progresses, it will prove to have a revolutionary ripple effect in the medical community and widen the treatment possibilities for patients.
Cryo-Cell’s medical director, Dr. Joanne Kurtzberg, and her team at Duke Medical Center have been a part of the Gamida trials from its initial launch. Phase I and Phase II have been completed in children with sickle cell anemia using the Gamida product. Duke Medical is also involved in clinical trials for an expansion technology developed by Magenta Therapeutics; this product is being used in trials for children with leukemia and children with storage diseases. Dr. Kurtzberg ‘s afternoon presentation gave a very thorough overview of ongoing and forthcoming clinical trials. She was recently present at Cryo-Cell’s past conference to deliver more thrilling advancements concerning these trials and to share her work at Duke with a cord blood-derived cellular therapy; stay tuned
for more details on this.
30+ Years of Cord Blood Transplant Success
Dr. Kurtzberg kicked off the WCBD presentations with a short overview of the last 30+ years of successful cord blood transplants. In 1988, she joined forces with Dr. Eliane Gluckman on the first cord blood transplant patient. Matthew Farrow was diagnosed with Fanconi anemia at five years old and was able to use the cord blood of his younger sister. He engrafted after 19 days, and, today, is a father and a strong cord blood advocate. From this initial transplant, sprung over 40,000 more transplantations worldwide utilizing umbilical cord blood. Dr. Kurtzberg reviewed some of the advantages of cord blood transplantation over bone marrow, which included the fact that cord blood units are more readily available and show more success against relapse. She stated, “Cord blood has huge potential to turn into therapeutic products.” Because of this, she predicts, “Cord blood banks, particularly public banks, will need to increase cord blood units. Likely, the need will grow exponentially.”
Access to Cord Blood Transplant Care
Professor of Medicine and Section Chief of Hematology at the University of Virginia Cancer Center, Dr. Karen Ballen, also reiterated this idea and spoke about increasing access to cord blood transplant care. She revealed recent case studies in her work with patients with acute myeloid leukemia, acute lymphoid leukemia, myelodysplasia, myeloproliferative neoplasms, aplastic anemia, and lymphoma. She explained the typical timeline of admission for a cord blood transplant recipient using allogeneic cord blood with a scenario of discharge ranging from day 15-30 with a return to work and regular routine around months 9-12 post-transplant. It was revealed that the odds of finding a matched sibling bone marrow donor, which is a doctor’s first consideration, is roughly 30% in the United States.
Therefore, doctors search for alternative graft sources which include umbilical cord blood, haploidentical (half-matched), or 7/8 HLA matched unrelated donors. Dr. Ballen expressed the advantages of using cord blood specimens in transplants and spoke about ex. vivo expansion and homing techniques. These techniques have reportedly allowed almost half of all the pediatric patients in her program to receive a cord blood transplant. However, she also shared there is a trend of older patients receiving allogeneic transplants in the U.S, and noted a dramatic increase in patients over 60 years of age. She addressed the difficulty of some patients to access a transplant center location-wise, and her team continues to work to make cord blood transplantation as much of an out-patient procedure as possible by offering patients telehealth options, remote monitoring strategies, and performing HLA typing at the time of leukemia diagnosis.
Ex Vivo Cord Blood Cell Expansion
Dr. Elizabeth Shpall of Texas MD Anderson Cancer Center highlighted some of their team’s work with ex. vivo cord blood expansion. Her early work revealed that when isolated and used in vitro in combination with cytokines, mesenchymal stem cells (MSCs), markedly increased the expansion of cord blood hematopoietic progenitors. Furthermore, they discovered ex vivo expansion coupled with fucosylation (adding fucose sugar units to cells), led to enhanced homing and engraftment. So far, five patients at MD Anderson have been treated with this method. She aspires to achieve a method able to deliver expansion within just seven days. She reports that many other expansion technologies have come close to this goal, naming NiCord as one with an 11 day expansion period. This product was found to pose less of a risk of bacterial infections and led to fewer days in the hospital during the first 100 days for the patient post-transplantation.
Likewise, Cohen and Sauvageaux’s Phase I and Phase II clinical trials revealed a seven-day expansion period with a technology that can almost double the expansion time of cord blood MSCs vs. bone marrow MSCs. She stated, “The MSC is a magical cell that has huge potential if expanded in the right way.” When asked if she predicts cord blood expansion becoming a standard of practice, she replied, “If the FDA approved it and if it would be covered by insurance, I think it could. It could become a home-run in terms of making it safer and cheaper.”
Upcoming Cord Blood Clinical Trials
Dr. Filippo Milano of the Fred Hutchinson Cancer Research Center also echoed the importance of cord blood stem cells by proclaiming, “Cord blood has much potential. We have not unleashed all of it. The potential is out there; it’s just for us to embrace.” Furthermore, “There are aspects that are keeping cord blood from being used. We need to spread the positive data. These days, it’s becoming more difficult to find HLA-matched donors. We need to spread the use of cord blood. Transplant is just a minimal part of what cord blood could do.” In addition to several ongoing and upcoming projects, his work at the center includes two recent clinical trials, one of which involves using cord blood in HIV patients; and is in the process of gaining approval.
Dr. Wise Young, a professor at Rutgers University and founding director of the W.M. Keck Center for Collaborative Neuroscience, spoke with great optimism regarding his work with cord blood to treat spinal cord injury. Like so many others, he also sees a “tide of pessimism” in his field. This is due to a misconception of what is possible due to a lack of understanding or education about the subject of cord blood. His early work has shown that animal models showed very positive results. More than 100 laboratories have demonstrated that spontaneous regeneration in the spinal cord has occurred in animal models.
He recalls, “A lot of scientists believe that regeneration is not possible. It takes a long time, less than a millimeter a day, similar to the growth of your hair.” He goes on to state, “In the 1970s, this idea of regeneration was heretical.” Medical scientists would demonstrate spinal injury using a wrong model, which essentially cut the spinal cord in half. He explained the error in this by elaborating, “It’s like meeting the Grand Canyon and expecting to jump over it. These axons don’t jump--they crawl.” A group of doctors and scientists came together to create a more accurate model to represent what often happens in spinal cord injuries. We now know that axons cannot regenerate through gaps, but they can cross over an injury site through the bridging of cells.
With this finding, Dr. Young and colleagues began animal trials testing the safety and dosage of cord blood stem cells in animal models. Studies showed that rats, mice, and dogs with umbilical cord blood mononuclear cells started walking. This led to a Phase I clinical trial of 8 patients in Hong Kong and a Phase II trial involving 20 patients. It was observed that higher cord blood stem cell dosage equated to better patient walking outcomes. In fact, 75% of patients were walking after the one-year follow-up, and 65% of patients recovered bowel and bladder function. What’s even more surprising was that, lithium, a drug used for manic depression, was responsible for producing more axonal growth, which formed a bridge over the injury site.
In 2016, Young applied to perform a study in the U.S., in which the FDA required more animal studies to be completed before granting permission. After subsequent proven success, the FDA approved a Phase III clinical trial to take place in 2020. It will be the first clinical trial directed for patients of complete spinal cord injury. There will be three groups of patients: 1 group will receive 6/6/6 walking training—meaning, walking 6 hours/day, 6 times a week, for a 6 month duration; 1 group will receive cord blood stem cells in addition to 6/6/6 training; 1 group will receive cord blood stem cells, with lithium, and 6/6/6/ training. After the trial, those in the control group, who did not receive cells, will have the opportunity to receive cell treatment. Those participating in this trial will be 18-64 years of age. However, there are also plans for a clinical trial involving children ages 8-17, and also one which will include patients 65-80 years of age.
Even more promising, Dr. Young reported, “There are almost just as many animal trials showing a benefit of umbilical cord blood in treating stroke.” Additionally, he states that trials are showing cord blood may help in treating macular degeneration (a leading cause of vision loss). He concluded by emphasizing the need for more expecting parents to donate their children’s cord blood. As of now, there are 900,000 allogenic umbilical cord blood units in the public registry—which is simply not enough. He postulates, “We’ll have a therapy that will work, but not a business model to support the need.” He continued by saying, “We need to make collection a matter of course to ensure that umbilical cord blood is not thrown away…we are throwing away something that is very valuable; it’s sad.” “We either throw them away or save them and save lives. I think the choice is straightforward and simple.”
Cord Blood Education is Key
A common plea among all speakers was for the public to understand the vital potential cord blood stem cells hold; that they should not be discarded. There are several advantages to banking privately, but regardless of whether stored publicly or privately, families should make an informed choice to store. The topic of cord blood stem cell expansion was at the forefront of most of the research presentations and provided us a glimpse into what the future holds. From the prestigious and renown pioneers in the industry to the cord blood recipients, such as the Fryer family and Dr. Alexes Harris, who spoke of their gratitude for cord blood stem cells, the message of importance was relayed throughout.
Many highlighted the fact that these cells, which are commonly thrown away and considered as medical waste, actually hold a power that is yet to be fully discovered. Currently, there are a combined 200 clinical trials taking place pertaining to cord blood improving engraftment, and being used in a regenerative therapy setting. Treatment of conditions such as autism, cerebral palsy, stroke, spinal cord injury, diabetes, etc. is being explored through the use of cord blood and cord tissue in clinical trials. As this year’s World Cord Blood Day passes, let us take the time to educate ourselves on the topic and celebrate the many who have been impacted through the work and efforts of cord blood research.