CAROLINE CAVANAUGH, NISHA LIPOWCAN, VALERIE KATULKA, WILL WATSON,
EMILY BRATLEE, SHAYNA SVARANOWIC, MAGGIE MCCALL

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https://www.youtube.com/watch?v=WGAcVLm9pBI

• Pre-Transplant
• Patients receive other forms of treatment
• Cord Blood is obtained
• Transplant
• Patient receives the cord blood intravenously
• Post-Transplant
• Stem cells travel to the patient’s bone marrow
• Stem cells produce new WBC, RBC and platelets

• In 1988, a pediatric patient with Fanconi’s Anemia was successfully treated with a transplant of hematopoietic stem cells (HSC) from umbilical cord blood (UCB)
• Fanconi’s Anemia: develops because of damage done to the bone marrow which is causing the bone marrow to slow or stop new blood cell production
• The Ende brothers are accredited with clinical observation, establishing the scientific basis of the observation and performing the first transplant
• Dr. Broxmeyer is accredited for his studies on the storage of the UCB
• Dr. Gluckman is accredited for her efforts that established the transplantation of HSC from UCB as an accepted therapy

• Treatment for over 80 diseases
• 1988 – 1 disease
• 2007 – 40 diseases
• 2010 – 80 diseases
• Not a cure but regenerates damaged tissues and restores lost function after injury or illness.
• Also used to regenerate healthy blood and immune systems after being damaged by diseases.
• Mostly used in children and small adults because not enough blood can be acquired from umbilical cord that is needed for the adult

Immune Disorders
Adenosine Deaminase Deficiency (SCID)
Bare Lymphocyte Syndrome (SCID)
Chediak-Higashi Syndrome (SCID)
Chronic Granulomatous Disease
Congenital Neutropenia
DiGeorge Syndrome
Evans Syndrome
Fucosidosis
Hemophagocytic Lymphohistiocytosis (HLH)
Hemophagocytosis Langerhans’ Cell Histiocytosis
(Histiocytosis X)
IKK Gamma Deficiency (NEMO Deficiency)
Immune Dysregulation, olyendocrinopathy, Enteropathy, X-linked (IPEX)
Syndrome
Kostmann Syndrome (SCID)
Myelokathexis
Omenn Syndrome (SCID)
Phosphorylase Deficiency (SCID)
Purine Nucleoside (SCID)
Reticular Dysgenesis (SCID)
Severe Combined Immunodeficiency Diseases (SCID)
Thymic Dysplasia
Wiskott-Aldrich Syndrome
X-linked Agammaglobulinemia
X-Linked Lymphoproliferative Disorder
X-Linked Hyper IgM Syndrome
Cancers
Acute Biphenotypic Leukemia
Acute Lymphocytic Leukemia (ALL)
Acute Myelogenous Leukemia (AML)
Acute Undifferentiated Leukemia
Adult T Cell Leukemia/Lymphoma
Chronic Active Epstein Barr
Chronic Lymphocytic Leukemia (CLL)
Chronic Myelogenous Leukemia (CML)
Ewing Sarcoma
Hodgkin’s Lymphoma
Juvenile Chronic Myelogenous Leukemia (JCML)
Juvenile Myelomonocytic Leukemia (JMML)
Myeloid/Natural Killer (NK) Cell PrecursorAcute
Leukemia
Non-Hodgkin’s Lymphoma
Prolymphocytic Leukemia
Plasma Cell Leukemia
Chronic Myelomonocytic Leukemia (CMML)
Leukocyte Adhesion Deficiency
Multiple Myeloma
Neuroblastoma
Rhabdomyosarcoma
Thymoma (Thymic Carcinoma)
Waldenstrom’s Macroglobulinemia
Wilms Tumor

Blood Disorders
Acute Myelofibrosis
Agnogenic Myeloid Metaplasia (Myelofibrosis)
Amyloidosis
Aplastic Anemia (Severe)
Beta Thalassemia Major
Blackfan-Diamond Anemia
Congenital Amegakaryocytic Thrombocytopenia (CAT)
Congenital Cytopenia
Congenital Dyserythropoietic Anemia
Dyskeratosis Congenita
Essential Thrombocythemia
Fanconi Anemia
Glanzmann’s Thrombasthenia
Myelodysplastic Syndrome
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Polycythemia Vera
Pure Red Cell Aplasia
Refractory Anemia with Excess Blasts (RAEB)
Refractory Anemia with Excess Blasts in Transition (RAEB-
T)
Refractory Anemia with Ringed Sideroblasts (RARS)
Shwachman-Diamond Syndrome
Sickle Cell Disease
Metabolic Disorders
Congenital Erythropoietic Porphyria (Gunther Disease)
Gaucher Disease
Hunter Syndrome (MPS-II)
Hurler Syndrome (MPS-IH)
Krabbe Disease
Lesch-Nyhan Syndrome
Mannosidosis
Maroteaux-Lamy Syndrome (MPS-VI)
Metachromatic Leukodystrophy
Mucolipidosis II (I-cell Disease)
Neuronal Ceroid Lipofuscinosis (Batten Disease)
Niemann-Pick Disease
Sandhoff Disease
Sanfilippo Syndrome (MPS-III)
Scheie Syndrome (MPS-IS)
Sly Syndrome (MPS-VII)
Tay Sachs
Wolman Disease
X-Linked Adrenoleukodystrophy

• Unlike other medications that focus to control the behavior, targets the observed molecular mechanisms.
• Abnormal neurotransmitter regulation, mitochondrial dysfunction, bloodbrain barrier disruptions
• Study
• Combined transplantation of human cord blood mononuclear cells
(CBMNCs) and umbilical cord derived mesenchymal stem cells (UCMSCs)
• Improved behavior and function
• Safe to use

• Umbilical cord blood shown to lessen the impact of injury and facilitate neural cell repair resulting in improved function for patients with cerebral palsy
• Study
• Peripheral mononuclear blood stem cells used
• Resulted in functional improvements
• Safe to use

• CB stem cells are capable of giving rise to hematopoietic, epithelial, endothelial and neural tissues
• Study
• Cord blood-derived unrestricted somatic stem cells (USSC) used in patients with acute ischemic myocardium
• Mesenchymal stem cells
• Functional improvements observed but not full recovery

• Help in stroke recovery and brain repair by angiogenesis and neurogenesis with restoration to cortical tissue
• Study
• CD34+ of umbilical cord blood
• Successful outcomes in animal testing

• Allogenic mesenchymal stem cells from donated human umbilical cords are administered into the affected joint(s) (intra-articular injection) and intravenously
• Vet-Stem tested and uses stem cells on horses with various joint deformities to accelerate healing
• Stem cells can protect cartilage from further destruction and facilitate regeneration
• Typical treatment protocol at Stem Cell Institute is about 5 days
(Riordan, 2012)

• Other treatments have long-term adverse effects and do not address the issue of damage that has already occurred
• Mesenchymal stem cells produce anti-inflammatory agents, which act locally and do not suppress the immune response of the patient’s whole body
• Induce the production of T regulatory cells, a type of immune cell whose function is to protect the body against immunological selfattack
• Typical treatment protocol is 4 days
(Riordan, 2012)

• Typical treatment protocol calls for 16 intravenous and intrathecal injections over the course of 4 weeks and uses a combination of umbilical cord derived stem cells and bone-marrow derived stem cells
• Intrathecal injections enable the stem cells to bypass the blood-brain barrier and migrate to the injury site
• Study
• Treatment was effective in 13 out of 22 patients (81.25% effective in incomplete
SCI, not effective in any patients with complete SCI)
• Patients reported significantly improved tactile sensation, motion and activities of daily living
• Real-life Success Story: Jamie Richie
(Riordan, 2012)

• Proven to treat hematopoietic diseases
• One umbilical cord generally contains enough stem cells to treat a child or small adult
• Previously, treating with two different donors has been effective which is reassuring in showing how adaptable cord blood cells are
(Apurva, 2009)
• Expensive
• Chance that patient is exposed to a rare genetic disorder of the immune system or blood
• It may take a decade or two before there is a definite cure to deadly diseases
• The average-sized or larger adults were not thought to be able to benefit from this type of treatment

• Respect for human dignity and integrity
• Autonomy
• Justice and Solidarity
• Beneficence
• Non-maleficence
• Proportionality
(Petrini, 2013)

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• Sufficient donations from different ethic groups
• Public storage when possible
(Petrini, 2013)

• Public or Private cord banking
• Public: Even discarded blood can benefit
• Private: The use for self is rare (0.005%)
(Petrini, 2013)

Fig. 3. Monte Carlo simulation; 7,500 trials performed. Points that lie below the dotted line represent trials in which private cord blood banking was cost-effective at a threshold of
$100,000.Kaimal. Cost-
Effectiveness of Cord
Blood Banking. Obstet
Gynecol 2009.

• Informed Consent
• Autonomy: right to self-determination
• USA: Laws aimed at educating parents about CB
• Process of obtaining informed consent should begin BEFORE labor and delivery
(Petrini, 2013)

• New model of business that sells only “the hope on future use.”
• High use of Propaganda or Persuasion by private blood bank company’s on potential clients.

• Claim 1: Your child will most likely need their stored blood, the one company claims 1 in every 50 children.
• Claim 2: The child can use their own cord blood to treat leukemia.
• Claim 3: Stem cells in cord blood can be used as a cure-all for adults in treating many diseases.
• Claim 4: Finding a stem cell donor is often impossible.
• The Society of Obstetricians and Gynecologists and The American Academy of Pediatrics does not recommend cord blood banking.
(Marketplace: Busted: Cord Blood Banks)

“DUTY TO FEEDBACK” CHANGES IN LAW FOR THE
COMMERCIAL SECTOR
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Allow donors to have the right to know what exactly their donations will be used for and how they are being used.
Allow donors to obtain profits from products or tests developed from their samples.
• A donor should have the right to know if the researcher has come across a potential risk for their health within the study.

• Tracey and Victor’s son Anthony diagnosed with Osteopetrosis at 4 months old
• Osteopetrosis, "stone bone", also known as marble bone disease and Albers-
Schönberg disease, is an extremely rare inherited disorder whereby the bones harden, becoming denser
• Chose to save and store Anthony’s cord blood at birth
• Why were they unable to use the stem cell’s from the cord blood

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"The materials provided by the bank said this was Anthony's life insurance and could save him if he needed it. They never mentioned that the cells could be diseased.”

• Mixed race woman Diagnosed with Acute Myelogenous Leukemia as an adult and was selected to participate in a study done by the SCCA working with cord blood transplants in Adults
• The nature of cord cells is unique (cells haven’t been educated against foreign invaders like bacteria and viruses)
• Naïve immune system of cord blood means that close HLA tissue-type
DNA matching is much less important for cord blood than it is for bone marrow transplants

• It takes more time to restore a patient’s blood counts after cord blood transplantation than with bone marrow or adult blood cells.
This makes cord blood transplant patients more vulnerable to infections early.
• Doctor’s used a public cord blood bank with 2 units of primary cord blood and one unit of “expanded” cord blood (to overcome the delay in WBC production/decrease the risk of Jessie acquiring an infection soon after transplantation)
• Expanded cord blood cells are frozen and made readily available for patients

ABC News. (2010, May 6). Questions about cord blood banking. Retrieved from https://www.youtube.com/watch?v=BnzRzKsvlDo&index=44&list=PLturoXTpuYxDZkWOka_1SJ1icT-fxZqfP
Aplastic anemia. (n.d.). Retrieved March 10, 2015, from http://www.mayoclinic.org/ diseases- conditions/aplastic-
20019296
Apurva. (2009, January 11). Advantages and Disadvantages of Cord Blood Treatment. Retrieved from http://www.disabledworld.com/news/research/stemcells/cordblood-treatment.php
anemia/basics/causes/con-
Brown, N., & Kraft, A. (2007). Blood Ties: Banking the Stem Cell Promise. Technology Analysis & Strategic Management, 313-
March 14, 2015, from Taylor & Francis Online
Cord Blood Transplants. (n.d.). Retrieved March 10, 2015.
327. Retrieved
Crompton, K. E., Elwood, N., Kirkland, M., Clark, P., Novak, I., & Reddihough, D. (2014). Feasibility of trialling cord blood stem cerebral palsy in Australia. Journal Of Paediatrics & Child Health, 50(7), 540-544. doi:10.1111/jpc.12618 cell treatments for
Ghodsizad, A., Ungerer, M. N., Bordel, V., Kallenbach, K., Kögler, G., Bruckner, B., & ... Ruhparwar, A. (2011). Transplanted human cord blood-derived unrestricted somatic stem cells preserve high-energy reserves at the site of acute
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Glasser, L. (n.d.). The Ende brothers and the arcane history of the first umbilical cord blood hematopoietic stem cell transplant.
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Gluckman, E., Broxmeyer, H., Auerbach, A., Friedman, H., Douglas, G., Devergie, A., Boyse, E. (1989). Hematopoietic
Fanconi's Anemia by Means of Umbilical-Cord Blood from an HLA-Identical Sibling.
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Kiatpongsan, S. (2008). Business on Hope: A Case Study on Private Cord Blood Stem Cell Banking. Chot Mai Het Thang Phaet,
91(4), 577-580. Retrieved March 1, 2015, from PubMed.gov.
Kaimal, A., Smith, C., Laros, R., Caughey, A., & Cheng, Y. (2009). Cost-effectiveness Of Private Umbilical Cord Blood Banking.
Obstetrics & Gynecology, 114(4), 848-855. Retrieved March 12, 2015, from http://journals.lww.com/greenjournal/Fulltext/2009/10000/Cost_effectiveness_ofPrivate_Umbilical_C ord_Blood.21.aspx?WT.mc_id=EmxALLx20100222xxFRIEND#P27
Marketplace: Busted: Cord Blood Banks. Retrieved from https://www.youtube.com/watch?v=f0qr8Xpoa2Q
Moninger, J. (2014) The cord blood controversy.
Peterson, D. A. (2004). Umbilical cord blood cells and brain stroke injury: bringing in fresh blood to address an old problem. Journal
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