Take It to the Bank- Saving Umbilical Cord Blood for Stem Cell Cures

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Take It to the Bank: Saving Umbilical Cord Blood for Stem Cell Cures Gerry Sotomayor, M.D. Northside Hospital Atlanta, GA Babies For Life Foundation  Babies for Life Foundation (BFL) is a private non-profit 501(c)(3) organization born from an idea of coordinating the generosity of parents who wish to donate their baby's umbilical cord blood with hospitals, medical providers and blood banks in order to increase the public registries of available stem cell units for research and transplantation. BFL and Cord Blood  Based in Atlanta, Georgia, Babies for Life Foundation (BFL) was incorporated in 2001 as a non-profit 501(c)(3) organization by Dr. Gerry Sotomayor, an OB/GYN, whose mission is dedicated to facilitate transplants and research for cures using umbilical cord blood and other post-natal tissues.  Umbilical cord blood contains millions of stem cells that have the potential to provide therapy for many diseases and is wasted in every delivery along with the placenta. BFL and Cord Blood Studies have shown cord blood stem cell therapies are superior to bone marrow transplants in treating many lifethreatening illnesses.  The current list of diseases in the world literature treated with ASC is over 70.  ASC are less differentiated, have more plasticity, have not been exposed to diseases or chemicals, are easier to obtain and match, overall less expensive.  I. Problem A great opportunity means a great challenge to meet the demand. 100,000,000 persons in the USA may benefit from stem cells but there is no formal policy in existence to assure their availability. Current policy is narrowly focused on a fraction of the potential benefits. II. Published Data Umbilical Cord Blood Success  In the early 1980s, scientists discovered that UCB contains high levels of Hematopoietic Progenitor Cells and that the number of the colony-forming units were similar in marrow collections, in which sustained hematopoietic engraftment was achieved. II. Published Data  1988- 1st Successful UCB Engraftment in child with Fanconi’s anemia- alive and well today. 1991- 1st UCB transplant performed on child with chronic myelogenous leukemia- alive and well today. RESULT: The stage is set for UCB to be used in any situation in which bone marrow is routinely utilized.   II. Published Data Cord Blood v. Bone Marrow Cord Blood Collection Bone Marrow Collection of Cord Blood is noninvasive, painless, and poses no risk to the donor. Greater of bone marrow is invasive and painful. Must be performed in a hospital surgical setting. to the maturity of the stem cells, it requires a greater HLA match to perform a transplant. GVHD occurs in 60% of all unrelated Bone Marrow transplants Bone Serious Due HLA compatibility due to decreased functionality of fetal lymphocytes. Graft v. Host Disease is reduced to 10% due to the absence of antibodies in the stem cells. Units are processed and ready for transplant. Significantly less expensive. Marrow is dependent on donor participation. II. Published Data Cost Structure Comparison  Lower Acquisition Costs Bone Marrow- $30,000 per case  UCB- $1,500 per case   Lower Treatment &  Average Case Costs  Follow-up Costs approx. $250,000 unrelated Bone Marrow  approx. $20,000-100,000 unrelated UCB II. Published Data Advantages of UCB over Bone Marrow □ Reduced Incidence Of Graft Versus Host Disease (GVHD) Why? Decrease functionality of fetal lymphocytes in children versus adults  Therefore…The same immunologic deficiency that makes neonates more susceptible to viral infections makes their lymphocytes less likely to cause GVHD. II. Published Data Advantages of UCB over Bone Marrow  Lessened Viral Contamination  40%-80% of U.S. population (geographical variances) have Cytomegalovirus (CMV)- a mild syndrome which nevertheless can be fatal to a depressed immune system.  CMV is typically not prevalent in infants. Two recent studies show CMV positive rate ranging from 0.1% to 0.3%. II. Published Data Advantages of UCB over Bone Marrow  Speed   Average 4 months- up to 6 months to identify marrow donor in NMDP UCB- approximately 3 weeks  HLA typing and viral testing completed before putting on registry  Size of Donor Pool - various ethnic backgrounds II. Published Data Cord Blood: a proven track record  Already, adult stem cells have successfully treated over seventy blood-related diseasessome previously considered fatal, including leukemia, Sickle Cell Anemia, Hodgkin’s lymphoma, Fanconi’s Anemia, and severe combined immunodeficiency diseases. For a list of all treatable diseases, please look at our website www.babiesforlife.org II. Published Data Cord Blood Collection Total Number of Units reported to BMDW, 2003. III. Current Trends in Research & Transplantation Early stage of research – basic science needs to be promoted more actively. Clinical trials – over 1,000 using ASC tissues.  33 years of original knowledge from bone marrow.  18 years of umbilical cord blood and other post-natal tissues.  Nasal mucosa, fat, and other tissues are currently being used. Heart attacks, strokes, diabetes, and other diseases (chronic illnesses) Sickle cell treatment and Thalassemia (first beneficiaries) IV. Umbilical Cord Blood Collections “This is the only way to guarantee universal access and unlimited supplies of stem cells for all of humanity.”     4 million births in the U.S. alone could satisfy sustained needs versus the proposed 400,000 frozen embryos (if used completely, then what?) 100,000 of HLA-typed umbilical cord blood and post natal tissues already exist and are ready for use 100,000 stem cells per mL in UCB Avg. collection is 100-150mL = 10-15 million stem cells in one collected transplantable unit. IV. Umbilical Cord Blood Collections Importance of Minority Donation  Minorities have more difficulty finding a match   With less representation in the population, the pool of potential donors is smaller. There is much greater variation in HLA-types among people with African ancestry than in any other group.  With cord blood a partial match is acceptable, thus increasing the donor possibilities. In fact, the Atlanta Journal-Constitution recently cited the treatment of Keone Penn, a sickle cell anemia patient from Snellville, using the “world’s first successful cord blood transplant from an unrelated donor.” V. Post Natal Tissues  All tissues developed during pregnancy (placenta, membranes, cord, amniotic fluid) are currently used as stem cell sources for research. The placental organ will be discarded always unless universal policy changes its usage. Other Proven Sources of Stem Cells              Olfactory mucosa Fat obtained during liposuction Muscle Bone Skin Blood Vessels Brain Dental Pulp Gastrointestinal tract Retina Liver Pancreas Cornea VI. Legislation     Law approved to create cord blood bank and collection system in the U.S. to supply the demands for currently approved treatment. HRSA regulates the cord blood industry, while NMDP standardizes and accredits the facilities. Laws banning fetal farming for organ retrieval. States currently promote either ESC or UCB, or both in some cases. VI. Legislation      Public Donation Banks – 20 plus established but now they are being accredited and regulated for the 1st time. SOPs are being developed (consents, sterile collection technique, packing, shipping, storage, classification, registration) Free donation and free transplant available from ONE bank to this day so far. Proven track record of engraftment, greater than the private sector. Private banks – growing numbers with venture capital backing - may be of use for those financially solvent only. VI. Legislation More to the solution…  Legislation requiring that expectant mothers be informed about the options of donating their cord blood and placentas for stem cell transplants, as well as for research will constitute a breakthrough in public policy and health care solutions. VII. Why This is Worth Pursuing   Cord blood is backed by a proven track record. In the US alone, cord blood is routinely discarded almost 11,000 times each day. So, despite the enormous potential found in cord blood, a medical treasure is routinely tossed as nothing more than medical waste. BFL is committed to increase the public benefits from cord blood stem cells through education, coordination of donations, and support of ethical research.  VIII. Conclusions    So how do we do it? We obtain the parent’s consent for donation of cord blood and placenta. The screening process identifies those who should store privately due to medical reasons versus those who could donate to the public cord blood bank. Those ineligible for either could still donate for research purposes. VIII. Conclusions     The doctor or midwife agrees to collect with a formal contract and adequate training. Our quality control person supervises the paperwork (NMDP) and the amount collected. All units require careful labeling including the mother’s blood samples. The units are carefully packaged and shipped via air carriers within 48 hours. VIII. Conclusions    The units are received by the blood bank and classified immediately. The data is entered into a computer system. The stem cells are now separated and stored in Liquid nitrogen while the cultures and disease screening tests are finished. HLA typing is done. Once the quarantine period is over, the information about the stem cells quickly goes to all the registries and becomes accessible to anyone in the world. VIII. Conclusions     Any doctor who has a patient that needs a stem cell transplant requests a specific match usually 3/6 and the unit is sent frozen. The patient then receives the transplant using the approved IRB protocol of choice. The period of engraftment begins and the patient is monitored for GVHD. If everything goes well that person can live a better life. VIII. Conclusions Our happy parents are always reminded to write in their baby’s book the following: “Your first act of charity was to give life to someone else.” Gerry Sotomayor, MD www.babiesforlife.org Questions How can we go about doing the same in our state? How much is involved, resources, money… Who are the reliable banks? Where can we send units for research? Do we need to legislate this thing? Are there any drawbacks in storing it privately? Can you do this in Cesareans? Thank you ! I would like to acknowledge all the persons involved in this activity and particularly Mr. Chip Maxwell and Sister Renee Mirkes for their kind invitation to share with you tonight. May God Bless the great state of Nebraska!

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