What are stem cells?

Single cells that can

Single-cell

Differentiate into any one of 220 different specialised cells in the body or

What-are-stem-cells-manequin

…replicate themselves

Multiple-cells

Stem cells are often called MASTER CELLS. They form the foundation of your entire body and act as building blocks for the blood, immune system, tissue and organs. The can REPLICATE or REGENERATE themselves and have the ability to DIFFERENTIATE into any kind of specialised cell in the body.

What are blood stem cell transplants?

New York, 1956

American doctor E. Donnall Thomas did the first successful bone marrow transplant to cure leukemia.

bone

Blood stem cell transplantation, using stem blood cells from sources such as bone marrow, has been performed for MORE THAN 50 YEARS, with more than 1 MILLION BLOOD STEM CELL TRANSPLANTS across the world playing an important role in the treatment of bone marrow failures, blood cancers, blood disorders, metabolic diseases, immune deficiencies and autoimmune diseases.

Why may a bone marrow transplant (also called a blood stem cell transplant) be needed?

To treat and/or cure certain types of blood related disease, for example to:

  • replace diseased bone marrow with healthy bone marrow to treat/cure patients with e.g. blood cancer
  • replace non-functioning bone marrow with healthy functioning bone marrow in patients with e.g. acquired bone marrow failure.
  • regenerate a healthy immune system in patients with e.g. immune deficiencies, autoimmune disease
  • replace bone marrow with genetically healthy functioning bone marrow in patients with inherited blood related diseases.

Blood stem cells (tissue stem cells) are responsible to maintain the blood and the immune system and can defferentiate into red blood cells, white blood cells and platelets.

Red blood cells deliver oxygen to all organs and tissue and also remove carbon dioxide from your body, transporting it to the lungs for you to exhale.

Platelets are tiny cells that have the important job to stop bleeding.

White blood cells are the cells of the immune system that are involved in protecting the body against both infectious disease and foreign invaders

Blood stem cells used in transplants can be collected from:

bone-marrow
circulatig-blood
cord-blood

To date:1 000 000+blood stem cell transplants have been done

with more than 1/3 usingcord blood stem cells

human

What are your chances of finding a blood stem cell donor in SA?

70%

graph

of patients in need of a blood stem cell transplant won’t have a sibling donor

Depending on a person’s tissue type, the chance of finding a bone marrow donor may be less than

1 in a 100 000

helix

Bone marrow transplants require a perfect tissue match in most cases

Only 75 000

healthy donors are registered on the South African Bone Marrow Registry

Why should we store umbilical cord stem cells?

There is no public umbilical cord blood bank in

South Africa

1 in 217
The chances of needing a stem cell transplant in a lifetime of 70 years
>35 000
cord blood transplants performed in almost 30 years
>80 diseases
treated
>5 million
cord blood units stored in private banks

When facing illness of a child or loved one, families want hope and doctors want options. Banked umbilical cord blood stem cells provides both.Patients who require a haematopoietic stem cell transplant will need to obtain cells from one of three sources: bone marrow, circulating blood or umbilical cord blood. Finding a stem cell donor who is a match (and able to donate stem cells) is often difficult and, in many instances, no suitable donor can be found either within a family or through a National or International Bone Marrow Registry.Storing your baby’s umbilical cord blood at birth provides a source of stem cells, an option for the Transplant Team, should a stem cell transplant be required either for your child or, depending on the degree of matching, for a sibling.

How is it collected?

Collecting stem cells from umbilical cord blood is QUICK, PAINLESS and NON-INVASIVE – posing no medical risk to mother or baby.

After birth, the umbilical cord is clamped and cut before the baby is taken away for care. The blood is collected from the umbilical cord. A 20-25cm segment of the umbilical cord is collected in a quick, painless and non-invasive procedure.

Who can use banked umbilical cord blood?

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THE GOAL OF A BONE MARROW TRANSPLANT / BLOOD STEM CELL TRANSPLANT IS TO TREAT AND/OR CURE CERTAIN TYPES OF BLOOD RELATED DISEASES.

Banked umbilical cord blood can potentially provide easier access to a stem cell transplant.

Studies have shown that cord blood transplants can be performed in cases where the donor and the recipient are only partially matched, depending, for example, on the number of stem cells in the collection. In contrast, bone marrow transplants require a perfect match in most cases

Your baby’s cord blood stem cells will always be:

100%/PERFECT MATCH

AT LEAST A HALF MATCH

Autologous:

using your own stem cells

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own-stem-cells

Syngeneic:

using stem cells from an identical twin

arrow
identical-twin

Haplo-identical:

using stem cells from biological parents

bilogical-parents

25% CHANCE OF BEING A PERFECT MATCH FOR

1:100 000 CHANCE OF BEING A PERFECT MATCH

Allogeneic:

using your sibling’s stem cells

siblings

Allogeneic:

using an unrelated donor’s stem cells (recruited through the South African Bone Marrow Registry)

donor-stem-cells

The good news is that with umbilical cord blood stem cells, you don’t always need a perfect match

This increases your chances of finding a matching donor.

What is the difference between umbilical cord blood and cord tissue stem cells?

CORD BLOOD

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How is it collected

After birth the umbilical cord is clamped and cut before the baby is taken away for care. The blood is collected from the umbilical cord in a quick, painless and non-invasive procedure.
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What type of stem cells it contains

Blood stem cells/ Haematopoietic stem cells (HSCs) in umbilical cord blood are blood-forming stem cells that can differentiate into red blood cells, white blood cells and platelets.
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What is it used for

Blood-forming stem cells are used to treat bone marrow failure, blood cancer and other blood disorders.
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First used

Cord blood stem cells were 1st successfully transplanted in 1988, and now it is used to treat over 80 blood-related diseases.
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Evolving medicine

In addition to the current treatments, FDA-regulated clinical trails are underway to study ways in which cord blood stem cells can be used as treatment for:

  • cerebral palsy
  • traumatic brain injury
  • acquired hearing loss
  • juvenile diabetes

CORD TISSUE

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How is it collected

A 20-25cm segment of the umbilical cord is collected after birth.
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What type of stem cells it contains

The stem cells found in the umbilical cord tissue (Wharton’s jelly) is called mesenchymal stem cells (MSC). The umbilical cord vein and arteries contain endothelial stem cells and the umbilical cord lining contains both mesenchymal stem cells and epithelial stem cells.
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What is it used for

MSCs are able to differentiate into cartilage cells, muscle cells, bone cells, nerve cells, etc. Future treatments may include Alzheimer’s, liver and heart failure, bone regeneration, HIV, Type 1 diabetes, and more.
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First used

Clinical trials using MSCs started in 1995, with more than 200 trials currently underway.
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Evolving medicine

Cord tissue MSC are being evaluated in laboratory studies and clinical trials to treat:

  • heart disease
  • spinal cord injury
  • cartilage injury
  • liver disease

What have stem cells done so far?

More than35 000cord blood transplants have been performed to date

Stem cell research

microscope

UMBILICAL CORD BLOOD RESEARCH

Clinical trials where children use their own (autologous) cord blood stem cells

  • Cerebral palsy
  • Autism
  • Neonatal oxygen deprivation
  • Acquired hearing loss
  • Encephalopathy (neonatal)
  • Hypoplastic left heart syndrome
  • Stroke (pre/peri-natal)
  • Traumatic brain injury
  • Type 1 Diabetes

Clinical trials where children use donor (allogeneic) cord blood stem cells

  • Alzheimer’s disease
  • Bronchopulmonary dysplasia
  • Cartilage repair
  • Cerebral palsy
  • Critical limb ischemia
  • Global development delay
  • Graft versus host diseases
  • HIV
  • Fertility
  • Intraventricular haemorrhage
  • Liver cisshosis
  • Neurodegenerative disorders
  • Stroke
  • Sweat gland regeneration
  • Type 1 Diabetes

CLINICAL TRIALS USING MSCs FOR TREATMENT OF:

NEUROLOGICAL DISORDER

  • Autism (Phase 2)
  • Cerebral palsy (Phase 2)
  • Hearing loss (acquired sensori-neural) (Phase 2)
  • Hypoxic Ischemic Encephalopathy (HIE) (Phase 1)
  • Spinal cord injury (Phase 2)

CARDIOVASCULAR

  • Critical Limb Ischemia (Phase 3)
  • Ischemic stroke (Phase 2 placenta) (Phase 3)
  • Myocardial Infarction (Phase 3)
  • Cardiomyopathy (Phase 3)

NEUROLOGICAL DISORDER

  • Crohn’s Disease (Phase 3)
  • Diabetes, Type 1 (Phase 2)
  • Graft-versus-Host Disease (GvHD) (Phase 3)
  • Kidney plus stem cell transplant (Phase 2)
  • Systemic Lupus (SLE) (Phase 2)
  • Multiple Sclerosis (Phase 1)
  • Rheumatoid Arthritis (Phase 2)
  • Scleroderma (Phase 2)

Clinical trials: Autism & cerebral palsy

Cerebral palsy

Joanne Kurztberg: “Children who received a high dose of cord blood cells had statistically significant changes in their motor function, more than 30% above where they would have been predicted to be without cord blood therapy. These children made new connections in their brain and that’s why their function was better.”

1 in 326 children are affected

Autism spectrum disorder

Joanne Kurztberg: “Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech. Many children were able to attend to play and have meaningful communication in a way that they weren’t before. Some children had less repetitive behaviours than they did when they came onto the study.”

1 in 68 children are affected

1 in 217
The chances of needing a stem cell transplant in a lifetime of 70 years
>35 000
cord blood transplants performed in almost 30 years
>80 diseases
treated
>5 million
cord blood units stored in private banks

Umbilical cord blood – not just a bag of stem cells

Cord blood therapies have shown highly promising results to treat diseases for which there are currently no available therapies. Results using autologous (own) cells in babies and children with HIE, cerebral palsy and autism spectrum disorder are safe and results are promising. The safety of partially or fully matched sibling cord blood infusions has also been demonstrated preliminarily

Featured advanced cell therapy trial: Expanded access cord blood therapy for autism and cerebral palsy

The Duke University Medical Center has received permission from the FDA to offer cord blood therapy for conditions like autism spectrum disorder and cerebral palsy under an expanded access clinical trial. This protocol establishes an umbrella clinical trial, registered on 31 October 2017. This trial enables children who have these neurological disorders to receive therapy with their own cord blood or cord blood from a sibling, regardless of whether they qualify for a targeted clinical trial.

The registration of this clinical trial opens the door for many children who are af icted with an acquired neurological disorder to travel to Duke University for cord blood therapy, provided they have a suf ciently matching cord blood unit in a family bank. Sibling therapy only requires a partial match, not a perfect match.

Michael Chez (Sutter Neurological Institute) speaks about the recent progress in clinical trials using cord blood to potentially treat autism. He discusses his own research in this area and gives an overview of other research currently underway at Duke by Dr Joanne Kurtzberg.

Umbilical cord blood approved stem cell therapies

blood-bag

First used
Cord blood stem cells were first successfully transplanted in 1988, and now it is used to treat over 80 blood-related diseases.

BLOOD CANCERS

  • Acute Lymphoblastic Leukaemia (ALL)
  • Acute Myeloid Leukaemia (AML)
  • Chronic Myeloid Leukaemia (CML)
  • Myelodysplastic Syndrome (MDS)
  • Multiple Myeloma
  • Hodgkin’s Lymphoma
  • Non-Hodgkin’s Lymphoma
  • Bone marrow failure syndromes
  • Severe Aplastic Anaemia, Unspecified
  • Fanconi Anaemia

BLOOD DISORDERS

  • Sickle Cell Disease
  • Thalassemia

IMMUNODEFICIENCIES

  • Severe Combined Immune
  • Deficiency (SCID)
  • Wiskott-Aldrich Syndrome

INHERITED METABOLIC DISORDERS

  • Hurler Disease (MPS type IH)
  • Osteopetrosis
  • Adrenoleukodystrophy
  • Krabbe Disease

AUTOIMMUNE DISEASES

  • Systemic Lupus (SLE)

Seven-year-old Gina Rugari is a living example of how cord blood stem cell research is helping children born with Krabbe, a genetic disorder that almost always results in death before age two. Gina’s life was saved through a cord blood stem cell treatment.

Erik Praskins speaks at the Arizona Cord Blood Conference 2017 about the importance of cord blood donors. His son, Dylan, was diagnosed with leukaemia as a baby. He received a cord blood transplant just prior to turning 6 months old. That transplant saved his life. He is now a young boy, attending school and enjoying all of the activities a young child his age loves.

Deathly sick with Hodgkin’s Lymphoma and facing the 5th recurrence of the disease, Diane Paradise pushed doctors to help her. Educated about her options and her own body, she opted to participate in a clinical trial with cord blood instead of pursuing the usual bone marrow transplant and extended chemotherapy. This was a choice she does not regret. Diane is living proof that #WeCanICan: Beat Cancer with Cord Blood.

Improving the collection to ensure the best possible outcome

Speak to your doctor or midwife about the importance of collecting enough cord blood.

COLLECTING CORD BLOOD IS A SAFE, EASY AND PAINLESS PROCEDURE THAT CAN SUCCESSFULLY BE COMBINED WITH DELAYED CORD CLAMPING.

The placenta and umbilical cord hold enough blood to allow delaying the cord clamping by 1 – 3 minutes (the recommended time given by the World Health Organization) and still have enough for a successful cord blood collection. Chances of a successful collection will naturally increase by combing in+ex utero collection.

The most important consideration is collecting enough cord blood because in general, there is a correlation between the volume of cord blood collected and the number of stem cells in a collection.

Combining in- and ex-utero collection is the best-practice solution to collect enough cord blood. Combining these methods is particularly useful when the in-utero volume is low, for example due to delayed cord clamping.

Greater volume of blood = more stem cells.
For a collection to be potentially successful, a minimum collection of 80ml of cord blood is recommended.
Info-Card-9-Stage2-Labour

Stage 2 Labour
Cord blood collection takes place after birth and only once the umbilical cord has been clamped and cut. It is a painless procedure that poses no risk to mother or baby.

Info-Card-9-In-Utero-Collection

In-utero collection
With in-utero collection, the cord blood is collected immediately after the umbilical cord has been cut and before the expulsion of the placenta. This extraction mode requires about 10 minutes and is faster because uterine contractions allows cord blood to flow more easily.

Info-Card-9-Ex-Utero-Collection

Ex-utero collection
Ex-utero collection takes place after the expulsion of the placenta and takes about 25 minutes to complete.

Other benefits of combining these collection methods include:

  • There is a significantly higher total nucleated cell counts (TNC) in the in- & ex-utero collection vs. inutero collection.
  • There is no statistically significant difference in the microbial contamination rate in in-utero vs. in- & exutero collection, however it is lower in the ex-utero collection compared to either the in-utero or ex utero collections.
  • In- and-ex utero collection of umbilical cord blood for banking is safe and results in significantly higher TNCs than either technique alone.

Sample Releases

Within the Cryo-Save Group internationally, there have been 18 stem cell release to date – to centres such as Duke University North Carolina, USA, Kliniek Pediatrische Hematologie en Oncologie Frankfurt, Germany and Kinderspital Zurich, Switzerland.

We also had one South African release, which was for diagnostic genetic testing for acute lymphocytic leukaemia.

This confirms that Cryo-Save South Africa laboratory aligns with international standards give you an indication of the standards of our cord
blood products.

14/18

have been for autologous (own stem cells) & sibling transplants

4/18

have been for clinical trails for cerebral palsy

TRANSPLANTATION CENTRES

List of transplantation centres where Cryo-Save samples have been accepted for transplantation

  • Duke University North Carolina, USA
  • Kliniek Pediatrische Hematologie en Onclogie Frankfurt, Germany
  • Pirogov Sofia, Bulgaria
  • Centro De Transfusiones de la, Comunidad de Madrid, Spain
  • Kinderspital Zurich, Switzerland
  • Hospital Infantil Nino Jesus Madrid, Spain
  • Hospital Universitario Madrid, Spain
  • John’s Hopkins Hospital, Maryland, USA
RELEASE DATE DISEASE SOURCE
January 2004 Aplastic Anaemia Autologous
January 2007 Congenital Immunodeficiency Allogeneic (sibling)
October 2007 Subarachnoidal Haemorrhage Autologous
March 2009 Medulloblastoma Autologous
April 2009 Acute Lymphoblastic Leukaemia Allogeneic (sibling)
May 2009 Cerebral Palsy Autologous
December 2010 Cerebral Palsy Autologous
May 2011 Cerebral Palsy Autologous
August 2011 Cerebral Palsy Autologous
March 2013 Diagnostic Purpose
April 2013 Blackfan-Diamond Anaemia Allogeneic (sibling)
July 2013 Cerebral Palsy Autologous
July 2013 Cerebral Palsy Autologous
December 2013 Cerebral Palsy Autologous
October 2014 Beta Thalassemia Major Allogeneic (sibling)
January 2015 Diagnostic Purpose
March 2016 Beta Thalassaemia Major Allogeneic (sibling)
April 2017 Beta Thalassaemia Major Allogeneic (sibling)

Reference:

National Cord Blood Program, http://www.nationalcordbloodprogram.org/patients/ncbp_diseases.pdf
https://parentsguidecordblood.org/en/diseases#standard