Category Archives: Stem Cell Human Trials

IMAGE:AIDS Research and Human Retroviruses, published monthly in print and online, presents papers, reviews, and case studies documenting the latest developments and research advances in the molecular biology of HIV... view more Credit: Mary Ann Liebert, Inc., publishers New Rochelle, NY, January 6, 2015--Timothy Ray Brown, long known only as the "Berlin Patient" had HIV for 12 years before he became the first person in the world to be cured of the infection following a stem cell transplant in 2007. He recalls his many years of illness, a series of difficult decisions, and his long road to recovery in the first-person account, "I Am the Berlin Patient: A Personal Reflection," published in AIDS Research and Human Retroviruses, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is part of a special issue on HIV Cure Research and is available free on the AIDS Research and Human Retroviruses website. Brown's Commentary describes the bold experiment of using a stem cell donor who was naturally resistant to HIV infection to treat the acute myeloid leukemia (AML) diagnosed 10 years after he became HIV-positive. The stem cell donor had a specific genetic mutation called CCR5 Delta 32 that can protect a … Continue reading →

In March, astrophysicists announced theyd detected the telltale signature of gravitational waves, ripples in space and time created almost immediately after the Big Bang. This would've confirmed cosmic inflation, the theory that the universe expanded extremely rapidly in the first moments of existence. But, follow-up analysis suggests the signal could have come from dust. More analysis is needed, but "the discovery of the century" is in doubt. BICEP2 Collaboration In type 1 diabetes, the immune system destroys pancreatic cells that make insulin, a hormone that prompts cells to absorb glucose from the blood. In the more common type 2 diabetes, these "beta cells" underperform. After decades of frustration, scientists finally succeeded this year in creating human beta cells from embryonic stem cells, and used them to ameliorate diabetes in mice. Human trials are still a few years off. Douglas Melton / Harvard Late in 2013, a two-year-old boy in a small Guinean village fell sick from Ebola. Less than a year later, thousands of west Africans were dead. The outbreak's lessons are ongoing: the irrationality of westerners fearing contagion, the dangers of relying on pharmaceutical companies to develop treatments for rare but potentially devastating diseases, the importance of local public … Continue reading →

Drugs that seem safe in animal studies are sometimes found to be harmful in humans. The health effects of many chemicals on the market in products such as cosmetics, food additives and industrial and household cleaners arent fully known. Theres a huge need to come up with better ways of testing compounds to see if theyre toxic, said James Thomson, the UW-Madison stem cell pioneer. Thomson, who first grew human embryonic stem cells in a lab in 1998 and co-discovered a way to reprogram mature cells to their embryonic state in 2007, is working with researchers across campus on a new quest. Theyre creating tissue chips clusters of interacting cells that mimic specific organs, such as a model of a developing brain. Using stem cells, miniature scaffolds and sophisticated computer programs, theyre crafting prototypes that could someday replace animal testing for drugs and serve as screening tools for environmental toxins. About 80 percent of experimental drugs fail in human clinical trials because they are unsafe or ineffective, with some 30 percent found to be toxic in people despite promising results in animal studies, according to the National Institutes of Health. Largely because of the expensive failures, it costs more than … Continue reading →

Keith Vanderlinde/NSF The BICEP2 telescope at the South Pole may have spied gravitational waves or dust. This year may be best remembered for how quickly scientific triumph morphed into disappointment, and even tragedy: breakthroughs in stem-cell research and cosmology were quickly discredited; commercial spaceflight faced major setbacks. Yet landing a probe on a comet, tracing humanitys origins and a concerted push to understand the brain provided reasons to celebrate. Asian nations soared into space this year. The Indian Space Research Organisation put a mission into orbit around Mars the first agency to do so on its first try. Japan launched the Hayabusa-2 probe, its second robotic voyage to bring back samples from an asteroid. And even as Chinas lunar rover Yutu (or Jade Rabbit) stopped gathering data on the Moons surface, mission controllers took the next step in the countrys lunar exploration programme by sending a test probe around the Moon and back to Earth. But for commercial spaceflight, it was a bad year. Virgin Galactics proposed tourism vehicle SpaceShipTwo disintegrated during a test flight in California and killed one of its pilots. That came just three days after a launch-pad explosion in Virginia destroyed an uncrewed private rocket intended … Continue reading →

PUBLIC RELEASE DATE: 4-Dec-2014 Contact: John Wallace wallacej@vcu.edu 804-628-1550 Virginia Commonwealth University @vcunews Is the human immune system similar to the weather, a seemingly random yet dynamical system that can be modeled based on past conditions to predict future states? Scientists at VCU Massey Cancer Center's award-winning Bone Marrow Transplant (BMT) Program believe it is, and they recently published several studies that support the possibility of using next-generation DNA sequencing and mathematical modeling to not only understand the variability observed in clinical outcomes of stem cell transplantation, but also to provide a theoretical framework to make transplantation a possibility for more patients who do not have a related donor. Despite efforts to match patients with genetically similar donors, it is still nearly impossible to predict whether a stem cell transplant recipient will develop potentially fatal graft-versus-host disease (GVHD), a condition where the donor's immune system attacks the recipient's body. Two studies recently published by the online journal Frontiers in Immunology explored data obtained from the whole exome sequencing of nine donor-recipient pairs (DRPs) and found that it could be possible to predict which patients are at greatest risk for developing GVHD and, therefore, in the future tailor immune suppression therapies … Continue reading →

By Suzanne Hodsden, contributing writer Oxford BioTherapeutics (OBT) and the Menarini Group are prepared to begin human trials for OBT357 (MEN1112), indicated for the treatment of acute myeloid leukemia (AML). This project is the first of five projected collaborations on cancer-fighting antibody drug conjugates (ADCs) to proceed to phase 1 human trials. ADCs are a biologic/chemical combination that attaches cancer-fighting drugs to antibodies, leading to a more targeted and less toxic approach to cancer treatment. In 2012, OBT and Menarini partnered to share expertise in developing, testing and marketing ADCs. According to the terms of the $1 billion agreement, OBT provides the proprietary cancer target, antibody and delivery technologies, and Menarini provides for the manufacture and clinical development of each newly developed ADC. Menarini would be responsible for regulatory approval measures in its territories: Europe, Asia, and Latin America, while OBT would seek approval in North America and Japan. This ADC indicated for AML treatment is their first collaboration, launched in 2013. Andrew Slade, CEO of Menarini commented then, The first result of our researchers has arrived after a year of intense work, in the form of a monoclonal antibody which shows remarkable ability to target cells and induce their … Continue reading →

PUBLIC RELEASE DATE: 3-Dec-2014 Contact: Glenna Picton picton@bcm.edu 713-798-4710 Baylor College of Medicine @bcmhouston HOUSTON - (Dec. 3, 2014) - A novel mechanism - similar to how normal tissue stem cells respond to wounding - might explain why bladder cancer stem cells actively contribute to chemo-resistance after multiple cycles of chemotherapy drug treatment. Targeting this "wound response" of cancer stem cells can potentially provide a novel approach for therapeutic invention, said researchers from the National Cancer Institute-designated Dan L. Duncan Cancer Center at Baylor College of Medicine. The results of their study appear online in the journal Nature today. "Treatment for advanced bladder cancer is limited to surgery and chemotherapy. There are no targeted treatments available," said Dr. Keith Syson Chan, an assistant professor of molecular and cellular biology and of urology and the corresponding author on the report. "The chemotherapy response is far from ideal so the clinical goal is to advance research into this area and uncover a much more targeted approach." Together with co-lead authors Antonina Kurtova, a graduate student in the Translational Biology and Molecular Medicine Program at Baylor, and Dr. Jing Xiao, research assistant in urology at Baylor, Chan and his team sought out to … Continue reading →

THOUSAND OAKS, Calif. and SOUTH SAN FRANCISCO, Calif., Dec. 3, 2014 /PRNewswire/ -- Amgen (NASDAQ: AMGN) today announced that the U.S. Food and Drug Administration (FDA) has granted approval of BLINCYTO (blinatumomab) for the treatment of patients with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). This indication is approved under accelerated approval. Continued approval for this indication may be contingent upon verification of clinical benefit in subsequent trials. With this approval, BLINCYTO becomes the first FDA-approved bispecific CD19-directed CD3 T-cell engager (BiTE) antibody construct product, and the first single-agent immunotherapy to be approved for the treatment of patients with Ph- relapsed or refractory B-cell precursor ALL, a rare and rapidly progressing cancer of the blood and bone marrow.1-3 "The FDA's breakthrough therapy designation and accelerated approval of BLINCYTO underscores the critical need for new treatment options for patients with relapsed or refractory B-cell precursor ALL, who are often young adults," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "BLINCYTO is the first clinical and regulatory validation of the BiTE platform, a new and innovative approach that helps the body's own immune system fight cancer." The BLINCYTO approval is based … Continue reading →

SEATTLE, Dec. 1, 2014 /PRNewswire/ -- Juno Therapeutics today announced the appointment of Jeffrey Bluestone, PhD, as scientific advisor. He will advise the company on T cell therapies, including potential applications in autoimmune disease and organ transplantation. "We are delighted to have the opportunity to work with Dr. Bluestone, one of the world's outstanding T cell biologists, who has made major contributions in basic immunology and in the translation of those discoveries to clinical trials," said Juno CEO Hans Bishop. Bluestone's scientific work includes building a broad understanding of human immune responses at the molecular level, with significant contributions to stem cell research and immunology. He has published more than 400 peer-reviewed publications in journals including Nature, Nature Immunology, the Journal of Immunology, and Diabetes. "I am excited to be working with Juno's outstanding scientific founders and team to translate discoveries in immune tolerance into medical applications," said Bluestone. Bluestone is the A.W. and Mary Margaret Clausen Distinguished Professor and Director of the Hormone Research Institute at the University of California San Francisco, where he guides the institute's autoimmunity research and clinical initiatives in immune tolerance and T cell therapies. He has held appointments at the University of Chicago and … Continue reading →

PUBLIC RELEASE DATE: 26-Nov-2014 Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center @sumedicine Induced pluripotent stem cells made from patients with a form of blistering skin disease can be genetically corrected and used to grow back healthy skin cells in laboratory dishes, researchers at the Stanford University School of Medicine have found. They've termed the new technique "therapeutic reprogramming." The skin cells formed normal human skin when grafted onto the backs of laboratory mice, they said. The findings represent a major advance in the battle against the disease, epidermolysis bullosa, in which the top layer of skin, called the epidermis, sloughs off with the slightest friction, leaving open wounds that are difficult to heal. Severely stricken children who survive into their late teens or early 20s often die from invasive squamous cell carcinoma, a skin cancer that can arise during repeated cycles of skin wounding and healing. "Epidermolysis bullosa is a truly horrible, debilitating skin disease in which the top layer of skin is not properly anchored to the underlying layers," said Anthony Oro, MD, PhD, professor of dermatology. "When they are born, the trauma of birth rips away their skin, and they continue to suffer severe skin wounds … Continue reading →