In the spirit of retrospection, as we have entered a new decade, we wanted to pay homage to the advancements this past decade has seen in regards to cord blood and cord tissue research. The past ten years have yielded a tremendous amount of research in what is possible in the way of regenerative treatments in several conditions that were otherwise thought of as untreatable through the use of umbilical cord blood and cord tissue stem cells. And as of today, there have now been over 40,000 transplantations worldwide for bone marrow reconstitution.
A simple search in clinicaltrials.gov reveals that from 01/01/2009-12/31/2019, there were 676 studies
listed using the term “cord blood” with the status, “recruiting,” “active/not recruiting,” “completed,” or “enrolling by invitation.” To further narrow it down, there were 560 studies listed under the term “umbilical cord blood.” Likewise, there were 227 studies
listed using the term “cord tissue” with the same status criteria as listed above.
Of these studies, research findings were revealed relating to umbilical stem cells’ role in the treatment of autism, cerebral palsy, multiple sclerosis, ischemic stroke, hypoxic-ischemic encephalopathy, spinal cord injury, and many other conditions. The past three decades have seen an explosion of breakthroughs and revelations regarding the life-saving potential of cord blood and cord tissue stem cells. Let’s briefly focus on some of the pivotal milestones that the years 2009-2019 have given us in the field of umbilical cord stem cell therapies.
By this year, cord blood had become the second most common source
of stem cells for cell therapy, second to bone marrow.
Dr. Dong Ku Kim and a group of colleagues published research findings
comparing the hematopoietic activities of CD34+
cells derived from human bone marrow and umbilical cord blood (UCB) and demonstrated that UCB is a better source of immature hematopoietic cells and that CD34+
cells encourage hematopoietic cell repopulation. Several other studies have been published since then exploring the advantages of cord blood stem cells
; in particular, mesenchymal stem cells and hematopoietic stem progenitor cells over cells found in blood marrow and other sources. In turn, there has been a recent outpouring of interest and research regarding mesenchymal stem cells derived from cord blood in the therapeutic setting as a means of regenerative therapy
in many degenerative conditions.
2009 also marked a significant year for Cryo-Cell International. A new advanced technology for processing cord blood known as Prepa-Cyte CB
was developed to yield the maximum recovery of healthy stem cells while removing up to 99% of red blood cell contaminants. Cryo-Cell became the first major cord blood bank to embrace this technology by offering this type of processing to our clients.
Dr. Colleen Delaney and colleagues developed an innovative approach to produce cord blood stem cells in the lab using a manufactured protein to expand the number of cells before a transplant occurred as part of a decade-long effort at the Fred Hutchinson Cancer Research Center.
Dr. Joanne Kurtzberg’s research group at Duke University Medical Center published a pilot study report
documenting 184 patients who were the first to be treated with their own cord blood as therapy for acquired neurological disorders including cerebral palsy, hypoxic-ischemic encephalopathy, birth asphyxia, stroke, etc.
Hemacord, which would later be named HPC Cord Blood, became the first stem cell product to be FDA-approved. Hemacord is intended for patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment. These conditions include patients with hematologic malignancies, primary immunodeficiency diseases, bone marrow failure, and beta-thalassemia.
During this time, Cryo-Cell International initiated a premium service that cryo-preserves cord tissue stem cells in addition to cord blood. This would prove significant for families, as cord tissue MSCs would become a topic of attention for their regenerative properties and capability to differentiate into other types of cells.
Dr. Garbuzova-Davis and colleagues published a paper about their studies
involving multiple intravenous transfusions of human umbilical cord blood stem cells (UCB) in mice models and their successful outcomes in the treatment of amyotrophic lateral sclerosis (ALS). It was found that these cells were responsible for protecting motor neurons from inflammatory damage. Subsequent studies
would later reveal that these cells also played a key role in motor performance improvement and astrogliosis in the spinal cord.
Dr. Elizabeth Shpall, along with colleagues at the MD Anderson Cancer Center, found that they could expand cord blood stem cells 30-fold by manipulating them in a cultured environment that mimicked bone marrow. The clinical trial
reported that the 31 patients diagnosed with blood cancers who were treated with the expanded cord blood stem cells showed better engraftment results and immune system recovery within two weeks of treatment.
The first human case study is performed using autologous (child’s own) cord blood mononuclear cells in treating a patient with cerebral palsy specifically caused by hypoxia-induced brain damage after a cardiac arrest. The results
of this procedure demonstrated that cord blood stem cells contributed to the child’s improved motor control, eyesight recovery, spastic paresis, speech, and overall functional neuroregeneration.
This year saw a number of clinical trials investigating the potential of nonmanipulated cord blood stem cells to treat Type 1 diabetes. A study
confirmed that the autologous infusion of cord blood in patients with Type 1 diabetes proved to be safe and feasible both given alone and also when coupled with daily vitamin D dosage.
Other notable case studies took place this year involving Dr. John Wagner’s cord blood transplant on a child diagnosed with leukemia and HIV, as well as Dr. Timothy Nelson’s trial involving treating children with serious heart defects with their own cord blood stem cells in combination with surgery.
In June of this year, Duke University Medicine was awarded $15 million to support an innovative research program to explore the use of umbilical cord blood cells to treat autism, stroke, cerebral palsy, and related brain disorders. This would launch several clinical trials in various phases of development- some completed and which show promising results, and some still ongoing and progressing to other phases.
Dr. Joanne Kurtzberg and colleagues at Duke Medical Center initiated a Phase I trial
to determine the safety and feasibility of a single infusion of autologous cord blood in children with autism spectrum disorder. The results of this completed study would be published in 2017, demonstrating that not only was it safe, but cord blood infusion led to significant behavioral and nonverbal IQ improvement. There have been numerous other studies both in the U.S. and around the world that have stemmed from these findings in the diagnosis of autism.
Cryo-Cell International also receives FACT accreditation and becomes the first privately owned U.S. cord blood bank to voluntarily adhere to the most stringent quality standards in the cord blood industry.
of the safety of allogeneic umbilical cord blood infusions in children with cerebral palsy is published by Dr. Kurtzberg and the medical research team at Duke. Their observations disclosed that children who received higher cord blood cell doses showed superior gains in motor function and whole- brain connectivity during a one-year follow-up.
During this time, it was reported that clinical trials with perinatal mesenchymal stem cells (MSCs) comprised more than half of the annual total of clinical trials to date. In 2015, they reached 70% of the annual trials, as reported in the Regenerative Medicine
journal. The MSCs from cord tissue were and are being used in clinical trials in the treatment of conditions such as Alzheimer’s, Parkinson’s, liver failure, osteoarthritis, multiple sclerosis, stroke, cartilage repair, and various other conditions
A clinical trial
with 45 patients diagnosed with hepatitis-B-induced liver disease was published. The results of this trial demonstrated that mesenchymal stem cells derived from cord blood were shown to improve hepatic function and survival.
A Phase II clinical trial
began in June to study patients with mild to moderate dementia due to Alzheimer’s with the use of human mesenchymal stem cells, the type of cell which is found in cord tissue. The study completion date is scheduled for June 2020.
This year also brought forth more attention regarding cord tissue MSCs. One study
revealed that umbilical cord tissue MSCs significantly improved survival in mice suffering from acute lung injury and also dramatically reduced pulmonary inflammation.
At this time, researchers gathered and analyzed 15 studies
published between 1995 and 2016 involving 764 people with various forms of multiple sclerosis who underwent transplantations using stem cells like those in cord blood, giving us our most comprehensive look at how these stem cells can be used to treat the condition.
Umbilical cord stem cells are also studied to treat inflammatory bowel disease
, corneal disease, renal disease
, and collagen‐induced arthritis.
In addition, this year uncovered encouraging results from Duke’s preliminary, Phase 1 study on the safety of treating children with autism with an intravenous infusion of their own umbilical cord blood. The study disclosed improvements in behavior observed in the first 6 months post‐infusion and sustained at 12 months. Moreover, a higher baseline nonverbal IQ was associated with a greater degree of improvement in participants.
Expanded access was given for cord blood therapies for the treatment of neurological disorders, including autism and cerebral palsy, which allowed parents of impacted children to register for treatment at Duke Medical Center.
In September, the umbilical cord blood transplant and newborn stem cell banking communities celebrated the 30th
anniversary of the first hematopoietic stem cell transplant using cord blood in treating a child with Fanconi’s anemia. Since then, there have been more than 40,000 transplants using cord blood.
It was also an important year for Cryo-Cell International as we expanded our role to include public cord blood banking services through our partnership with Duke University. We also announced the appointment of Dr. Joanne Kurtzberg, as our medical director. Dr. Kurtzberg has been a revolutionary contributor in the cord blood field by performing the first unrelated cord blood stem cell transplant in 1993. Currently, she also serves as lead investigator in numerous clinical trials involving cord blood in regenerative medicine as well as in her work regarding ex vivo cell expansion.
In the scope of cell expansion, Nohla Therapeutics
sponsored a clinical trial looking at donor umbilical cord blood transplant with and without ex-vivo expanded cord blood progenitor cells in treating patients with Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, or myelodysplastic syndromes. Other companies such as Gamida Cell and Fate Therapeutics are also focusing on cord blood expansion technologies to deliver “off-the-shelf” cord blood products for treatment.
A mentionable clinical trial includes a study for the safety of autologous umbilical cord blood therapy for acquired sensorineural hearing loss in children, with results showing safety in this method of treatment. Although improved structural, behavioral, and functional outcomes were witnessed, the need for a larger Phase II trial is recognized.
A concise overview published in 2019 looked at the application of mesenchymal stem cell therapy for the treatment of osteoarthritis of the knee over other methods of treatment such as anti-inflammatory drugs and surgery. The publication stated that umbilical cord MSCs can serve as allogeneic stem cell drugs, which can replace damaged tissue in the microenvironment of the implanted site, creating a new approach to treatment.
A relevant Phase I/II study of umbilical cord blood and MSCs in children with cerebral palsy is intended to complete in 2021 and will look at motor function 12 months post-treatment in 90 participants who were given a single dose of allogeneic cord blood or multiple doses of cord tissue-derived MSCs.
According to CordBlood News, their most popular tweet of 2019 was a study published in Stem Cells Translational Medicine on allogeneic human umbilical cord mesenchymal stem cells for the treatment of autism spectrum disorder in children looking at the safety profile and effect on cytokine levels.
Several pivotal research highlights and findings were announced during the 2019 World Cord Blood Day. These were involved with the discussion of new stem cell expansion technologies, 30+ years of cord blood transplant success, ways to deliver more access to cord blood transplant care patients, and Dr. Wise Young’s work with using cord blood to treat spinal cord injury. His phase III trial is set to take place in 2020.
The cord blood and newborn stem cells industry have seen dynamic advancements in research and innovative developments in the applications of cord blood and cord tissue. This past decade, alone, has proven to be a springboard into what the future holds in regards to new treatments and the importance of cord blood’s role as an alternative source to bone marrow. Cryo-Cell International has also seen a great deal of evolving to ensure that we provide more than 500,000 parents from 87 countries with the highest quality of cryopreservation service possible. As of 2020, Cryo-Cell is the first private cord blood bank to claim specimen longevity of 23+ years with optimal viability.
As we journey into the frontier of umbilical stem cell research, we hope to continue our efforts to spread awareness about the importance surrounding the once-in-a-lifetime opportunity to save these precious cells. Cord blood and cord tissue stem cells are internationally recognized for their potential to treat a host of diseases, and the list of diseases continues to grow through the work of dedicated industry leaders. We cannot predict the magnitude of research that will prove beneficial for countless patients and their families.
What will this next decade in umbilical stem cell research reveal? We are optimistic about future outcomes and eagerly look forward to sharing with you!