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| June 1, 2010 |
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Stories |
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Clinical
Research |
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Association
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BMT Tandem Meetings |
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Calendar |
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Job &
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Monthly Journal |
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eNews
Archives |
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Calendar |
• June
8th Annual International Umbilical Cord Blood Transplantation Symposium
Cord Blood Forum
June 3-5
Hyatt Regency San Francisco at Embarcadero Center
San Francisco, California
American Society of Clinical Oncology (ASCO)
46th Annual Meeting
June 4-8
McCormick Place
Chicago, Illinois
European Hematology Association (EHA)
15th Congress
June 10-13
The Gran Via Conference Center
Barcelona, Spain
8th International Donor Registry Conference
World Marrow Donor Association (WMDA)
June 16-19
Trinity College
Dublin, Ireland
International Society for Stem Cell Research (ISSCR)
8th Annual Meeting
June 16-19
Moscone Center
San Francisco, California
Oncology Practice Reviews
Hilton Anatole
Dallas, TX
June 18-19, 2010
FOCIS 2010
Federation of Clinical Immunology Societies (FOCIS)
June 24-28
Boston Marriott Copley Place
Boston, Massachusetts
• July
UK National Stem Cell Network (UKNSCN)
Annual Science Meeting
July 12-14
University of Nottingham, East Midlands Conference Center
Nottingham, England, United Kingdom
Cryo 2010
Society for Cryobiology
47th Annual Meeting
July 17-20
Bristol Marriott Royal Hotel
Bristol, England, United Kingdom
• August
Canadian Society of Transplantation (CST)
Annual Scientific Conference
Aug. 13-14
Vancouver Congress Centre
Vancouver, British Columbia, Canada
• September
1st World Congress on Controversies in Hematology
Sept. 2-5
Rome, Italy
ISCT Europe Regional Meeting
International Society for Cellular Therapy (ISCT)
Sept. 11-14
Best Western Hotel Villa Carlotta
Belgirate, Italy
International Society for Experimental Hematology (ISEH)
39th Annual Scientific Meeting
Sept. 15-18
Melbourne Convention and Exhibition Center
Melbourne, Australia
American Society for Histocompatibility and Immunogenetics (ASHI)
36th Annual Meeting
Sept. 26-30
Westin Diplomat
Hollywood, Florida
• October
Fraunhofer Institute for Cell Therapy and Immunology
6th International Symposium on Neuroprotection and Neurorepair
October 1-4
Yachthafenresidenz Hohe Düne
Rostock-Warnemünde, Germany
European Society for Medical Oncology (ESMO)
35th Annual Meeting
Oct. 8-12
Milano Convention Centre
Milan, Italy
American Association of Blood Banks (AABB)
2010 Annual Meeting
Oct. 9-12
Baltimore Convention Center
Baltimore, Maryland
International Society of Hematology (ISH)
33rd World Congress
Oct. 10-13
ICC Jerusalem International Convention Center
Jerusalem, Israel
European School of Haematology
8th International Conference
Oct. 14-18
Cascais, Portugal
ISCT Asia-Pacific Regional Meeting
International Society for Cellular Therapy (ISCT)
Oct. 17-20
Phoenix Seagaia Resort
Miyazaki, Japan
Histiocytosis Association of America (HAA)
Oct. 18-20
Boston, Massachusetts
European Society of Gene & Cell Therapy (ESGCT)
18th Annual Congress
Oct. 22-25
Stella Polare Convention Centre
Milano, Italy
• December
American Society of Hematology (ASH)
52nd Annual Meeting
Dec. 4-7
Orange County Convention Center
Orlando, Florida
• 2011
BMT Tandem Meetings
Combined ASBMT and CIBMTR annual meetings
Feb. 17-21
Hawaii Convention Center
Honolulu, Hawaii
• 2012
BMT Tandem Meetings
Combined ASBMT and CIBMTR annual meetings
Feb. 1-5
Manchester Grand Hyatt Hotel
San Diego, California
• 2013
BMT Tandem Meetings
Combined ASBMT and CIBMTR annual meetings
Feb. 13-17
Salt Palace
Salt Lake City, Utah
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Top
Stories |
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European agency works on stem cell research guidance document
The European Medicines Agency held a workshop on May 10 as part of the public consultation process on the first dedicated regulatory guidance document on stem-cell research and development. Attendees discussed a draft reflection paper on stem cell-based medicinal products, and comments can be submitted until June 30. The reflection paper is expected to be finalized by the end of 2010. 
Guidelines released for stem cell use in MS patients
International guidelines have been released on the future of stem cell transplantation research for people with multiple sclerosis. The guidelines discuss different types of stem cells that might be used to treat MS, detail methods of delivering these stem cell therapies and highlight best practices in conducting clinical trials. 
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A Word from President John Barrett, MD
Mouse transplantation
For the well-bred laboratory mouse, treatment outcomes have never looked so bright in the field of stem cell transplantation (SCT). A huge diversity of transplant protocols is available to them in some of the top research institutes in the country, and (provided they are not allocated to a control group) the research subject can expect a complete cure of anything from GVHD to acute leukemia that may be induced during the trial. But how does all this industry reflect on the transplant prospects for the average mouse in the street, or for that matter human leukemia sufferers? Laboratory mice are different animals from their wild counterparts, let alone us. Mouse strains are inbred and humans are outbred. Mice have very different natural killer cells and mesenchymal stem cells, they have their own major histocompatibility complex molecules and antigens, they do not develop chronic GVHD and acute GVHD, and GVHD is hard to generate because of the improved hygiene of animal care. Despite these constraints, mouse transplant studies are popular. They are not only an easier option than going through the regulatory hoops required for clinical trials, but are also considered a purer form of science. Animal studies are more intellectually satisfying, command research funding and generate papers in high-quality journals because experimental conditions can be created (strain selection, knockouts etc) that are not available in man. Mouse transplant research is more likely to end up in the highest impact journals than human studies (who has not heard “But how can you be sure until you have proved it in the mouse?”). This state of affairs leads to a bias toward animal experimentation for its own sake at the expense of translational research in humans. Too many funded grants retain their endpoints within the murine world, meticulously proving self-fulfilling prophesies without any realistic forward look to human applications. While giving a nod to the idea that further development will follow the line of mouse experiments to large animal studies to human trials, the conclusions from animal experiments can be devoid of clinical relevance and become animal muddles rather than models. For example, approaches that test whether GVHD can be prevented while graft versus leukemia (GVL) is retained will fail to convince that the same is true in man if the “L” of the GVL refers to a leukemia cell line that bears little resemblance to a real disease.
Human studies
Fortunately today, clinically relevant SCT research is also flourishing. Human studies have been greatly facilitated by our increasing ability to characterize immune reactions, stem cell and tumor biology through molecular biology, cell culture and flow cytometry, and also by controlling transplant outcome through component cell therapy. When every donor-recipient pair represents a unique combination, the ability to study immune interactions at a molecular level is essential to interpreting outcome through cell biology. However, as we are all aware, human studies are slow to set up, slow to reach their endpoints and messy, and there are too many intervening factors that dilute the meaning unless the patient group is large enough. For a young investigator wanting to become established in research the prospect of, for example, designing some novel cell product, scaling it up to clinical grade and progressing through the regulatory hoops before setting up a clinical trial is daunting and time-consuming. Nevertheless, the most innovative studies can still find their place in high impact clinical journals, like our own BBMT.
Solutions
It is without question that mouse transplant studies have illuminated key principles in stem cell transplantation and critical components of the alloresponse. However, they serve us less well when it comes to the fine details of protocol design. Outbred animals, such as pigs, dogs and monkeys, offer more clinically relevant research, but the numbers of animals transplanted is limited by expense, and drug handling can be different. For example, the dog is not a good model for testing fludarabine, one of the most common components of reduced intensity preparative regimens, and when it comes to the higher primates (remember that chimpanzees share 98 percent of our genome) how comfortable do we feel about entering these trusting research subjects into potentially lethal procedures without their consent? There are other ways forward, like the SCID mouse and its ever more transplant-permissive variants that represent an informative middle ground where human cells can be put to work in a murine in vivo environment.
In the end, if we are to avoid the divergence of transplantation research into two different non-communicating universes, we need to continue to use animal models where they are best applied in defining the big picture, while developing and promoting human biology, using the increasingly powerful tools at our disposal. We should consider whether a particular line of research requires us to follow the classical path of mouse to large animals to man, or whether it is possible to advance our understanding and ultimately treatment success through studies of human biological mechanisms that underpin major transplant outcomes. To help clinical researchers with these issues, the ASBMT offers an annual Clinical Research Training Course where young physician scientists spend five days with leaders in the BMT field writing and refining their clinical transplant-related protocols. Maybe a change of emphasis towards human research is not as radical as it seems; after all what happened to that icon of medical animal experimentation in the guinea pig in stem cell transplant research?
-- John
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Legislation and Regulation |
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MDS Update
As you may recall, last fall a number of leading blood and marrow organizations including the ASBMT and National Marrow Donor Program (NMDP) submitted a formal request to the Centers for Medicare & Medicaid Services (CMS) to make a make a formal decision to cover allogeneic transplant for myelodysplastic syndrome. On May 6, CMS posted its proposed decision on MDS coverage. A copy of the decision can be found at: https://www.cms.gov/mcd/viewdraftdecisionmemo.asp?id=238
In the decision, CMS does not agree that coverage for allogeneic transplant of MDS should be allowed but does believe that enough information has been submitted to provide for coverage under the Coverage with Evidence Development process. This allows for coverage for individuals who receive a transplant provided that the transplant is done under a clinical study designed to meet CMS requirements as described in the proposed decision. While this is not the best outcome, it is a good result nonetheless as Medicare-eligible individuals will have access to transplant, provided we can organize a clinical study. 
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Clinical Research |
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Research may lead to treatment for hearing loss
Working in mice, scientists have created hair cells from the ear canal that are damaged during hearing loss. Researchers at Stanford University used both embryonic stem cells and induced pluripotent stem cells to recreate the hair cells, which are covered with bristles called “stereocilia.” 
Hox genes regulate types of stem cells produced
Scientists have discovered a function that regulates how stem cells produce different types of cells in different parts of the nervous system. According to a report in PLoS Biology, the Hox genes that work to give each piece of the body its unique regional identity also control the stem cells produced. For example, in the head region, specific nerve cells are produced by the Hox genes turn them into another type of cells.

Endometrial stem cells may help treat Parkinson’s disease
When injected into mice whose brains had damage resembling Parkinson’s disease, endometrial stem cells transformed into brain cells. Scientists at Yale University School of Medicine said endometrial stem cells generate less of an immune system rejection response than other stem cell types, and they are readily available.

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Association
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Scholars selected for 2010 research training course
Twelve young clinicians and investigators have been selected to participate in the fourth annual ASBMT Transplant Clinical Research Training Course, to be held in July in Park City, Utah.

New cord blood licensure website
A website has been created to support the volunteer working groups formed at the Cord Blood Licensure Workshop on March 10. The contents of the site are the result of collaborative efforts to facilitate the process of obtaining a Biologics License Application. 
BBMT online access
ASBMT members and individual subscribers receive online access to the full text of articles with their print subscription at http://www.bbmt.org. All you need to claim access is your ASBMT member number. Authors and reviewers can access the online submission system via a separate login at http://ees.elsevier.com/ybbmt. For more details, please visit http://www.bbmt.org/content/additionalinformation.
Reimbursement tips and tactics
Tips and tactics for transplant procedure reimbursement are being posted on the ASBMT Web site. 
Volunteer opportunities
Health Volunteers Overseas is an organization committed to providing teaching and training to health professionals in developing countries. For more information, contact Health Volunteers Overseas directly at http://www.hvousa.org.
‘Be the Match’ continues its recruitment
The National Marrow Donor Program’s “Be the Match” registry continues its efforts to recruit donors. 
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