News from the field. A collection of industry, policy, government and other hot news from the field of hematology and stem cell.
Japan Stimulus to Boost Science
Japan's government approved a plan to spend $116 billion to jump-start the economy and set the stage for long-term growth. Sources in the Japanese press are hinting that research on renewable energy and on stem cells could land a significant chunk of the new cash.
U.S.-Based Gene Therapy Study Begins Treating Participants
Cooley's Anemia Foundation has learned that the first U.S.-based clinical trial in gene therapy for beta-thalassemia has begun treating trial participants. The Phase 1 study, the official title of which is "ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene,” is being conducted at Memorial Sloan-Kettering Cancer Center in Manhattan. The trial received FDA approval in the summer of 2012 and began recruiting study subjects soon thereafter.
Stem Cell Lawsuit Finally Over
The Supreme Court rejected a request to ban U.S.-funded research on human embryonic stem cells (hESCs). The decision brings to an end a long legal battle that has cast a shadow over hESC studies for over three years.
Genea Stem Cells (GSC): 25 New Disease Specific Pluripotent Stem Cell Lines Placed on the NIH Registry
Genea Stem Cells Pty Ltd (GSC), a supplier and developer of disease-specific human stem cells, announced that 25 of its disease specific embryonic pluripotent stem cell lines have been placed on the USA National Institutes of Health (NIH) human stem cell registry. These embryonic stem cell lines are now all available commercially for use in medical research.
First Gene Therapy Trial Launched for Fabry Disease
Researchers in Calgary, Alberta have launched the first gene therapy clinical trial in the world for Fabry disease that could ultimately lead to a permanent cure. Researchers will first remove a quantity of stem cells from a Fabry patient’s blood. Then a working copy of a new GLA gene will be inserted into the stem cells using a specially engineered virus. During the final phase of the trial, researchers hope to transplant these stem cells back into the donor patient and the new, working copy of the gene will make the missing enzyme.
One millionth blood stem cell transplant marks major medical milestone
The Worldwide Network for Blood and Marrow Transplantation reported that he collaborative work of medical scientists and physicians across the globe has resulted in a major medical milestone: the world’s 1 millionth blood stem cell transplant, a procedure that has become a proven and essential therapy for many patients battling blood cancers like leukemia and lymphoma, as well as other critical diseases.
Cell Therapeutics Begins Enrolment in Phase III PERSIST-1 Trial of Pacritinib for the Treatment of Myelofibrosis
Cell Therapeutics, Inc. (CTI) announced that the Company has initiated clinical trial sites and began enrolling patients in a Phase 3 clinical trial, known as PERSIST-1 or PAC325, for pacritinib, CTI's investigational JAK2 inhibitor, which is being evaluated for the treatment of patients with myelofibrosis
Seattle Genetics and Millennium Initiate Global Phase III Clinical Trial of ADCETRIS® (Brentuximab Vedotin) in Front-line CD30-Expressing Mature T-Cell Lymphoma (MTCL)
Seattle Genetics, Inc. and Millennium: The Takeda Oncology Company announced the initiation of a global phase III clinical trial evaluating ADCETRIS in combination with chemotherapy for the treatment of newly diagnosed CD30-positive MTCL patients, including patients with systemic anaplastic large cell lymphoma and other types of peripheral T-cell lymphomas.
Cellular Dynamics Announces the Production of Human iPSC Lines Under cGMP Conditions
Cellular Dynamics International, Inc., a leading commercial producer of human induced pluripotent stem cell (iPSC) lines and tissue cells for drug discovery and safety, announced that it is producing human iPSC master cell banks from five individual donors under current Good Manufacturing Practices (cGMPs). Manufacturing human iPSCs and ultimately human cells under cGMPs enables the possible use of iPSC-derived human cells within a clinical setting.
Gamida Cell’s StemEx® Achieves Primary Endpoint in Phase II/III Clinical Study
Gamida Cell announced today that its flagship product, StemEx, reached its primary endpoint of improving overall survival in a Phase II/III study which compared the use of StemEx as part of a transplantation regimen to historical controls in the treatment of patients with hematological malignancies such as leukemia and lymphoma.