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  • Clinical Implications of Basic Research

    For the past couple of decades, clinicians have watched the stem-cell field with a mixture of anticipation and skepticism. No group of patients has been more expectant than those with spinal cord injuries. Therapies for spinal cord injury have been promised almost since the dawning of the stem-cell…

    • May 17, 2012
    • Snyder E.Y. and Teng Y.D.
    • N Engl J Med 2012; 366:1940-1942

      For the past couple of decades, clinicians have watched the stem-cell field with a mixture of anticipation and skepticism. No group of patients has been more expectant than those with spinal cord injuries. Therapies for spinal cord injury have been ...

    • Clinical Implications of Basic Research

      Stem cell–based therapies have the potential to repair and even correct the defects related to human diseases. Although tantalizing niche applications have moved forward in the clinical setting, progress seems to be slow, and ethical challenges have yet to be definitively addressed. The goal of…

      • February 9, 2012
      • Sandhaus R.A.
      • N Engl J Med 2012; 366:567-569

        The holy grail of gene therapy is the treatment of disease caused by genetic mutations. A recent study in mice provides proof of principle that alpha1-antitrypsin deficiency can be corrected, if not cured, by the infusion of autologous induced pluripotent stem cells.

      • Perspective

        On December 1, 2011, in Flynn v. Holder, the U.S. Court of Appeals for the Ninth Circuit held that the ban on selling "bone marrow" that is part of the National Organ Transplant Act (NOTA) of 1984 does not encompass "peripheral blood stem cells" obtained through apheresis. This ruling means that…

        • January 26, 2012
        • Cohen I.G.
        • N Engl J Med 2012; 366:296-297
        • Free Full Text

        On December 1, 2011, in Flynn v. Holder, the U.S. Ninth Circuit Court of Appeals held that the ban on selling “bone marrow” that is part of the National Organ Transplantation Act of 1984 does not encompass “peripheral blood stem cells” obtained through apheresis.

      • Clinical Implications of Basic Research

        Hematopoietic stem-cell (HSC) transplantation remains the primary curative treatment for patients with a variety of hematologic cancers. Transplantation of either autologous or allogeneic stem cells requires the acquisition of sufficient numbers of HSCs to ensure rapid and consistent trilineage…

        • December 29, 2011
        • DiPersio J.F.
        • N Engl J Med 2011; 365:2536-2538

          Obtaining hematopoietic stem cells (HSCs) for transplantation depends on effective egress of HSCs from bone marrow into the peripheral circulation on induction. This process is impaired in a mouse model of diabetes and in patients undergoing autologous transplantation.

        • Editorial

          In this issue of the Journal, the findings of two case series suggest that in vivo treatment with interleukin-2 can suppress immune-mediated diseases. In one study, Koreth et al. found that low-dose interleukin-2 was associated with reversal of glucocorticoid-refractory chronic graft-versus-host…

          • December 1, 2011
          • Bluestone J.A.
          • N Engl J Med 2011; 365:2129-2131

            In this issue of the Journal, the findings of two case series suggest that in vivo treatment with interleukin-2 can suppress immune-mediated diseases. In one study, Koreth et al.1 found that low-dose interleukin-2 was associated with reversal of ...

          • Perspective

            Until recently, it seemed as if the question of future federal funding for embryonic stem-cell research had been settled. The Obama administration had lifted President George W. Bush's previous restrictions on funding, and the legality of that decision was upheld by federal courts. But the 2012…

            • November 17, 2011
            • Blendon R.J., Kim M.K., Benson J.M.
            • N Engl J Med 2011; 365:1853-1856
            • Free Full Text

            The 2012 presidential election campaign has created uncertainty about federal funding for human embryonic stem-cell research, with most Republican candidates suggesting that they would substantially reduce such funding. Where do U.S. voters stand on this issue? This Perspective summarizes polling results.

          • Editorial

            The therapeutic use of cells from healthy donors or patients is increasing. Decades ago, transfusion medicine and bone marrow transplantation provided the first successful cell therapeutics and established the foundations for cell delivery. Clinical investigation soon uncovered the double-edged…

            • November 3, 2011
            • Sadelain M.
            • N Engl J Med 2011; 365:1735-1737

              The therapeutic use of cells from healthy donors or patients is increasing. Decades ago, transfusion medicine and bone marrow transplantation provided the first successful cell therapeutics and established the foundations for cell delivery. Clinical ...

            • Original Article

              Although cellular therapies may be effective in cancer treatment, their potential for expansion, damage of normal organs,– and malignant transformation is a source of concern. In contrast, the toxic effects of small molecules usually diminish once the drugs are withdrawn. One approach to…

              • November 3, 2011
              • Di Stasi A., Tey S.-K., Dotti G., et al.
              • N Engl J Med 2011; 365:1673-1683
              • Free Full Text

              The introduction of a transgene into human donor T cells that can kill the cells when dimerization is induced by a pharmacologic agent has improved prospects for the exploitation of T-cell graft-versus-tumor effects while controlling graft-versus-host disease.

            • Original Article

              Hematopoietic stem-cell transplantation (HSCT) with the use of umbilical-cord blood is effective in patients for whom a sibling or matched, unrelated donor is not available.– Acute graft-versus-host disease (GVHD), a major cause of illness in patients undergoing HSCT, is less severe with cord…

              • September 1, 2011
              • Herrera A.F., Soriano G., Bellizzi A.M., et al.
              • N Engl J Med 2011; 365:815-824
              • Free Full Text

              The authors have identified an acute diarrheal syndrome that develops about 4 months after cord-blood hematopoietic stem-cell transplantation with the use of umbilical-cord blood. The disease is characterized by fever, watery diarrhea, and responsiveness to antibiotics.

            • Clinical Implications of Basic Research

              Stem cells hold promise in the treatment of many different types of disease. The generation of induced pluripotent stem cells (iPSCs) by reprogramming somatic cells has provided unprecedented opportunities for studying basic biology and modeling human diseases. In addition, the differentiation of…

              • July 28, 2011
              • Zhang K. and Ding S.
              • N Engl J Med 2011; 365:370-372

                A recent study suggests that the optic cup can form in vitro from clumps of cells made up of embryonic stem cells, which indicates that these cells may have a capacity for self-organizing.

              • Perspective

                The struggle over federal funding for research involving human embryonic stem cells (ESCs) continues — and may well be waged for years to come. On August 23, 2010, Chief Judge Royce Lamberth of the U.S. District Court of the District of Columbia granted the motion of plaintiffs James Sherley and…

                • June 2, 2011
                • Cohen I.G. and Adashi E.Y.
                • N Engl J Med 2011; 364:e48
                • Free Full Text

                The struggle over federal funding for research involving human embryonic stem cells (ESCs) continues — and may well be waged for years to come. On August 23, 2010, Chief Judge Royce Lamberth of the U.S. District Court of the District of Columbia granted ...

              • Clinical Implications of Basic Research

                In 2006, when Shinya Yamanaka found that introducing just four genes into normal mouse skin cells made those cells pluripotent — and thus able to generate all cell types of the body — the reaction was almost one of disbelief. But less than a year later, both he and James Thomson did the same…

                • June 2, 2011
                • Mummery C.
                • N Engl J Med 2011; 364:2160-2162

                  There are two sources of the human pluripotent stem cell — the differentiated somatic cell, such as a skin fibroblast, and the embryo. Recent studies suggest that an extra level of caution is warranted in using stem cells derived from somatic cells.

                • Perspective

                  The National Institutes of Health (NIH) and researchers using human embryonic stem cells (ESCs) have been operating under some uncertainty since the August 2010 ruling by federal districtcourt judge Royce Lamberth that the Dickey–Wicker Amendment (which Congress has attached to all appropriations…

                  • June 2, 2011
                  • Annas G.J.
                  • N Engl J Med 2011; 364:e47
                  • Free Full Text

                  The National Institutes of Health (NIH) and researchers using human embryonic stem cells (ESCs) have been operating under some uncertainty since the August 2010 ruling by federal districtcourt judge Royce Lamberth that the Dickey–Wicker Amendment (which ...

                • Original Article

                  The fundamental properties of stem cells are self-renewal, clonogenicity, and multipotentiality. Although tissue-specific stem cells have been identified,, they have not always been shown to exhibit stem-cell properties, particularly in the mammalian lung. Various cell populations with some of…

                  • May 12, 2011
                  • Kajstura J., Rota M., Hall S.R., et al.
                  • N Engl J Med 2011; 364:1795-1806
                  • Free Full Text

                  Cells in certain niches of the lower airways that express c-kit appear to be capable of self-renewal, clonogenicity, and differentiation into various lung-tissue components (including alveoli, muscle cells, and vessels) in vitro and may regenerate mouse lung tissue in vivo.

                • Editorial

                  During the fifth gestational week, the human lung begins to develop from epithelial buds of a primordial endodermal tube that is destined to give rise not only to the lung but also to most abdominal organs. Using tools to trace individual cells, biologists have determined that virtually all of the…

                  • May 12, 2011
                  • Chapman H.A.
                  • N Engl J Med 2011; 364:1867-1868

                    Urine-sediment microscopy is useful for diagnosing and managing kidney and genitourinary disease, but photodocumentation is often unavailable. This letter describes a point-of-care method involving a microscope eyepiece and inexpensive digital or cell-phone cameras.

                  • Clinical Implications of Basic Research

                    Once researchers recognized that adult stem cells can generate multiple cell types and contribute to tissue homeostasis, it became conceivable to exploit this potential to treat genetic or acquired disorders characterized by tissue degeneration or organ dysfunction. The concept of regenerative…

                    • March 24, 2011
                    • Parolini O.
                    • N Engl J Med 2011; 364:1174-1175

                      Engraftment of transplanted hematopoietic stem cells in the developing fetus is achieved with variable rates of success. A recent study suggests that immune surveillance by maternal T cells is an obstacle to engraftment.

                    • Review Article

                      Multiple myeloma is a neoplastic plasma-cell disorder that is characterized by clonal proliferation of malignant plasma cells in the bone marrow microenvironment, monoclonal protein in the blood or urine, and associated organ dysfunction. It accounts for approximately 1% of neoplastic diseases and…

                      • March 17, 2011
                      • Palumbo A. and Anderson K.
                      • N Engl J Med 2011; 364:1046-1060
                      • CME

                      Autologous stem-cell transplantation and agents such as thalidomide, lenalidomide, and bortezomib have changed the management of myeloma and extended overall survival. This review discusses both the biologic features and the management of multiple myeloma.

                    • Editorial

                      In the early 1970s, patients with high-risk hematologic cancers had a small chance of long-term survival. This chance began to increase with allogeneic blood or bone marrow transplantation, which combines high-dose chemotherapy, total-body irradiation, and graft-versus-leukemia effects. In…

                      • November 25, 2010
                      • Kersey J.H.
                      • N Engl J Med 2010; 363:2158-2159

                        In the early 1970s, patients with high-risk hematologic cancers had a small chance of long-term survival. This chance began to increase with allogeneic blood or bone marrow transplantation, which combines high-dose chemotherapy, total-body irradiation, ...

                      • Original Article

                        The Wiskott–Aldrich syndrome (WAS) is a complex primary immunodeficiency disorder that is characterized by recurrent infections, thrombocytopenia, eczema, and autoimmunity, and caused by mutations in WAS. Its gene product, WAS protein (WASP), is a key regulator of actin polymerization in…

                        • November 11, 2010
                        • Boztug K., Schmidt M., Schwarzer A., et al.
                        • N Engl J Med 2010; 363:1918-1927
                        • Free Full Text

                        The authors report the long-term (up to 3 years) correction of the Wiskott–Aldrich syndrome in two patients through retroviral infection of CD34+ hematopoietic cells after busulfan-induced transient myelosuppression.

                      • Perspective

                        Embryo research was born political. Expressions of shock and surprise at the August 23 ruling of federal district court judge Royce Lamberth enjoining federal funding of stem-cell research — which was based largely on his reading of an amendment to an appropriations bill — are thus not terribly…

                        • October 28, 2010
                        • Annas G.J.
                        • N Engl J Med 2010; 363:1687-1689
                        • Free Full Text

                        Embryo research was born political. Expressions of shock and surprise at the August 23 ruling of federal district court judge Royce Lamberth enjoining federal funding of stem-cell research — which was based largely on his reading of an amendment to an ...

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                      Medical Meetings Pediatrics Conferences and Meetings

                      2012 Certifying Examinations of the American Board of Pediatrics

                      The general pediatrics examination will be held in various cities, Oct. 16-18. Registration for first-time applicants is ongoing through May 3. Registration for re-registrants is ongoing through May 24. The following subspecialty examinations will be held in various cities: "Hospice and Palliative Medicine" (Oct. 4); "Pediatric Transplant Hepatology" (Oct. 11); "Pediatric Cardiology" (Nov. 7); "Pediatric Pulmonology" (Nov. 8); "Medical Toxicology" (Nov. 12); and "Pediatric Critical Care Medicine" (Nov. 14). Registration for first-time applicants is ongoing through April 30. Registration for re-registrants is ongoing through June 15.

                      Contact the American Board of Pediatrics, 111 Silver Cedar Court, Chapel Hill, NC 27514-1513; or call (919) 929-0461; or fax (919) 918-7114 or (919) 929-9255; or see http://www.abp.org .

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