Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: a phase 1/2 study

Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita

“…Two DC patients were treated with this combination in a different study, one with a matched unrelated donor who died and one with a matched related donor who is still alive. 18 Our data show encouraging OS of four of six patients (67%), including three of five patients (60%) from unrelated donor sources, a source that has been difficult in the past. 9,10,12,14,18,28 In addition, of particular note with our nonmyeloablative regimen has been the absence of pulmonary or liver complications during and immediately after HCT.…”

“…Various regimens have been examined, but there is not yet a commonly accepted standard. 9,12,14,[18][19][20][21][22][23][24][25][26][27][28][29][30] Table 3 summarizes the reported experience of HCT for patients with DC using reducedintensity conditioning, including specifics of donors, regimens and outcomes. Kaplan-Meier analysis revealed that overall cumulative survival was 65%, with related donor recipients having better survival compared with unrelated donor transplants (91 vs 30%, respectively, P ¼ 0.05).…”

Devouring the Hematopoietic Stem Cell: Setting the Table for Marrow Cell Transplantation

“…CAR T cells are genetically modified ex vivo to express a "chimeric antigen receptor" and are very promising cancer therapies that are still in development. 8 An advantage of the BiTE and CAR strategies is that they bypass the human leukocyte antigen (HLA) restriction for antigen recognition. Thus, neither the HLA-specific epitope presentation nor the recognition by a native T-cell receptor (TCR) is required.…”

“…7 Anti-CD45 monoclonal antibody administration along with immunosuppression was evaluated in one study of HCT for a variety of primary immunodeficiency diseases. 8 Although sufficient engraftment was seen, it was difficult to determine the exact role of the antibody in determining outcome. Finally, a potentially attractive approach that has been evaluated in a dog model © The American Society of Gene & Cell Therapy commentarỹ 20% chimerism.…”

Tearing RAGs apart

“…5 Similarly, hematopoietic progenitor cell depletion by administration of specific antibodies may be an important development. 6 Gene therapy strategies for ARTEMIS 7 and RAG 8,9 deficiencies have shown proof of concept in murine models and clinical trial protocols are in development. This may provide an option when compatible donors are unavailable but will still need to be coupled in ARTEMIS SCIDs with an appropriate conditioning regime.…”