Not All Stem Cells are the Same

Transcription

1 Cord Blood Banking and Transplantation Jennifer Willert, M.D. Hematology/Oncology Blood and Marrow Transplant Rady Children s Hospital San Diego Clinical Professor UCSD Not All Stem Cells are the Same Embryonic Stem Cells Newborn Stem Cells Cord Blood Advantages High developmental potential High proliferative capacity Little risk of viral contamination Younger, excellent proliferation and differentiation abilities Immediately available Less stringent HLA requirements Lower risk of GvHD, infections Autologous transplants possible Limitations Limited supply Ethical issues No clinical data Biological constraints Delayed short-term engraftment One-time supply Adult Stem Cells Bone Marrow Peripheral Blood Ability to differentiate Rich concentration of stem cells Greatest amount of historical data Rapid engraftment Good historical data Less invasive than bone marrow Facilitates easier autologous transplants Complex harvest Minimal residual disease (MRD) Increased potential for GvHD Cord Blood Stem Cell Timeline Cord blood proposed as source of stem cells First family cord blood stem cell bank (for use by related individual) NEJM study demonstrated higher survival rates with related cord blood cell transplants than with unrelated More than 10,000 transplants have been performed to date First cord blood stem cell transplant from a related donor for Fanconi's anemia First successful transplantation from an unrelated donor Stem Cell Therapeutic and Research Act signed into law Adapted from: Emerging stem cell therapies. Supplement to OBG Management

2 Cord Blood Stem Cells Are Increasingly Being Used to Save Lives Cord Blood Stem Cell (CBSC) Transplants Cord Blood Banking Options: Private and Public Private Cord Blood Banking: Related Banks 98% of collected samples are banked Samples are reserved and available at any time for family use Cost is ~$1500 to $2000 upon collection plus $100 to $125 annual storage fee Is an option accessible to anyone in the United States Public Cord Blood Banking: Unrelated Donor Banks 48% 66% of all samples collected are banked Sample is available to any matched recipient; not all patients will find a match No cost for collection or storage; cost is ~$15,000 to $35,000 to the treating institution for sample retrieval Access denied to some due to limited number of public banks Both options help address the potential needs of patients Diseases That Could Currently Benefit From CBSC Transplants Blood Disorders Sickle Cell Anemia Aplastic Anemia Fanconi Anemia Beta Thalassemia Malignancies Leukemia Lymphoma Multiple Myeloma Metabolic Disorders Hurler Syndrome Niemann Pick Disease Severe Combined Immune Deficiency Many Emerging Therapies are Under Investigation Including Heart Disease, Diabetes, and Brain Injury 2

3 Blood and Marrow Transplant Approximately 500 CBSC transplants occur annually in the U.S. Many Patients Don t Find a Match Patients Needing Bone Marrow Transplants 33% Family Match 66% No Family Match Bone Marrow Transplant Registry 25% Find Suitable Match Up to 50% of All Patients Needing Stem Cell Transplants Are Unable to Find a Match Up to 75% Find No Suitable Match Source: 1) Perlow JH. Contemp Ob/Gyn Advantages of CBSC Over Bone Marrow Immediate availability Simpler collection process Elimination of surgical harvest Reduction in time to treatment Decrease in frequency of certain infections More tolerant of HLA mismatches Lower risk of GvHD Provide better long term immune function Source: 1) Emerging stem cell therapies. OBG Management. 2006;18(suppl):S1-S6. 3

4 Outcomes of CBSC Transplants Compared With Bone Marrow in Children Outcome Acute GvHD Chronic GvHD Time to Engraftment Early Transplant Related Mortality Relapse Survival Cord Blood Improved Improved Slightly Longer Equivalent Equivalent Equivalent Cord Blood Stem Cell Transplantation Provides Improved Outcomes for Patients Advantages of Related CBSC Genetically unique to child or sibling Increased chance of intra familial matching Known family history Decreased morbidity/mortality Readily available/accessible Source: 1) Gluckman E, et al. NEJM CBSC Transplants Are Highly Successful, Particularly in Related Stem Cell Transplants One Year Survival % 29% N=78 Provide best clinical outcomes Less GvHD Improved survival Related CBSC Transplants Unrelated CBSC Transplants Higher Clinical Success Rates With Related CBSC Transplants² 85% 72% 3% Engraftment Survival GvHD N=44 Genetically unique Known family history Immediately available/accessible Expected lower costs No sample procurement Less acute GvHD treatment Fewer chronic GvHD medication requirements Sources: 1. Gluckman E et al. N Engl J Med Wagner J et al. Lancet

5 Brandyn 14 year old male Diagnosed with leukemia as a toddler Years of chemotherapy led to remission, until relapse and need for stem cell transplant Blood from younger siblings umbilical cord and placenta was banked at birth Stored at a private bank, alleviating the need to find a match from donor hospital or public registry Blood was rich in stem cells November 2, 1999 Brandon received his cord blood transplant and continues to do well. It was enough to do the transplant. It was the most wonderful news I ve ever heard. Brandyn s Mother Titus Diagnosed at 18 months with aplastic anemia He was unable to produce any red blood cells on his own Titus s mother became pregnant again; the hospital recommended that the family store the cord blood stem cells with Cord Blood Registry The baby s cord blood was collected at no cost, as part of CBR s Designated Transplant Program Stem cells were found to be a perfect match December 30, 2004,Titus received his cord blood transplant and is doing well As a mother, you ll do anything to help you son. I m really glad the hospital recommended Cord Blood Registry. Titus s Mother Joseph Jr. 6 year old male Diagnosed at birth with sickle cell anemia Joseph required frequent blood transfusions Joseph s mother became pregnant with a second child Amniocentesis revealed that the baby was a perfect match for Joseph Jr. Joseph s younger brother s umbilical cord blood was banked at birth free of charge as part of CBR s Designated Transplant Program The transplanted stem cells created new blood and a new immune system More than five years after the transplant, Joseph is healthy and vibrant It was about 20 days after the transplant when all of a sudden he started feeling better. Joseph s Father 5

6 Designated Transplant Program Established in 1996 to increase accessibility of stem cell transplantation to patients in need Eligibility criteria Affected family member is first or second degree relative of newborn Treating transplant physician confirms that affected family member is in need of a stem cell transplant All expenses of collection, processing, and storage are waived for DTP clients Through the DTP, CBR has stored cells for over 2000 families Empower Patients to Make an Informed Choice for the Future Who should receive information about cord blood banking? All expectant parents Family history of genetic disorders may pose particular need for education about cord blood banking Preparing for a Healthy Birth and Life CBSC banking discussed if multiple ethnicity exists (Initial visit) Maternal Serum Screen Ultrasound (15 20 weeks) Oral Glucose Challenge Test (28 weeks) BPP Non Stress Test (Post week 30) Weeks Initial hgc pregnancy test Prenatal Blood screening Rh incompatibility test & urinalysis CVS Nuchal Translucency Sono (Initial visit-14 weeks) Introduce CBSC banking options (20 weeks) Amniocentesis (Post week 16) PUBS (18 36 weeks) Cord Blood Collection (At delivery) Throughout Pregnancy Numerous Tests Are Conducted to Ensure the Present and Future Health of Mother and Baby 6

7 National and State Public Policy Continue to Drive Stem Cell Education and Awareness In addition to an Institute of Medicine (IOM) report and federal legislation centered on the creation of a national public bank, Cord Blood legislation has passed in 20 states. Odds of Use Odds Of Usage Over 70-year Span Based Solely On Current Indications 1 *: Autologous 1 in 400 chance of being a SC transplant recipient Autologous/Allogeneic 1 in 200 chance of being a SC transplant candidate Does Not Include Emerging, Potential Uses of Cord Blood Stem Cells to Treat Heart Disease, Diabetes, Parkinson s, Stroke, Muscular Dystrophy, and Spinal Cord Injury Source: 1) Pasquini MC, et al. Blood Factors Patients Need to Consider When Assessing a Cord Blood Bank Experience How many medical centers have utilized cells in transplant that were processed and stored with the cord blood bank? How recently? How many clients have samples stored at their facility? What is the longest time a unit has been stored and then used in a transplant? Cell recovery What percentage of cells do they recover from their processing? Publication of results Have they published data to support their processing and storage methods? Financial stability How long has the company been in operation and are they likely to be around in the future? Location of facility Are there risks associated with the location of the storage facility (tornadoes, hurricanes, airport closures, etc)? 7

8 Cord Blood Stem Cells Clinical studies over the past 20 years demonstrate the efficacy and safety of cord blood stem cells Cord blood stem cells can be easily collected and stored Cord blood stem cells are clinically useful in a variety of disease states and demonstrate tremendous future potential All expectant families should receive information about their options for collecting and storing their child s cord blood stem cells OBs play a vital role in ensuring families receive accurate information 8