New results from clinical trials of immunotherapy and experimental targeted agents for patients with leukemia and lymphoma are being presented by Dana-Farber Cancer Institute researchers at the American Society of Hematology (ASH) Annual Meeting Dec. 1-4. Here are summaries of three presentations, including one that compared outcomes of CAR T-cell therapy in patients in clinical trials with outcomes in the “real world” of clinical practice: CAR T-cell treatment provides durable
Anti-malaria drugs known as chloroquines have been repurposed to treat cancer for decades, but until now no one knew exactly what the chloroquines were targeting when they attack a tumor. Now, researchers from the Abramson Cancer Center of the University of Pennsylvania say they have identified that target – an enzyme called PPT1 – opening up a new pathway for potential cancer treatments. The team also used CRISPR/Cas9 gene editing
In just a few years, CAR T-cell and other adoptive T-cell therapies have emerged as among the most promising forms of cancer immunotherapy. But even as these agents prove themselves against several forms of leukemia and lymphoma – and, potentially, certain solid tumors – basic questions remain about how they work. In a study published online today by the journal Immunity, scientists at Dana-Farber Cancer Institute, Harvard Medical School, Vanderbilt
The amoeba Naegleria fowleri is commonly found in warm swimming pools, lakes and rivers. On rare occasions, the amoeba can infect a healthy person and cause severe primary amebic meningoencephalitis, a “brain-eating” disease that is almost always fatal. Other than trial-and-error with general antifungal medications, there are no treatments for the infection. Researchers at Skaggs School of Pharmacy and Pharmaceutical Sciences at University of California San Diego have now identified
Controlling Immune System Fuel Puts the Brakes on Macrophage Damage When tissue is damaged, one of the body’s first inflammatory immune-system responders are macrophages, cells which are commonly thought of as “construction workers” that clear away damaged tissue debris and initiate repair. However, prolonged inflammation promotes the progression of many diseases, including obesity. Now, a common class of drugs used to treat diabetes has been found to exert a powerful
A new drug discovery system allows scientists to specifically target members of an important family of enzymes, called phosphatases, which were previously considered mostly "undruggable". Scientists from the Medical Research Council (MRC) Laboratory of Molecular Biology, in Cambridge, UK, demonstrated the capabilities of the new system by identifying a molecule that could successfully target a phosphatase to reduce the accumulation of Huntington's disease-associated proteins in the brains of mice. The
Pluristem Therapeutics Inc., a leading developer of placenta-based cell therapy products, announced positive top-line results from its multinational Phase II clinical study of PLX-PAD cells in the treatment of Intermittent Claudication (IC). PLX-PAD treatment reduced Incidence of revascularization and improved patients’ mobility. Study results also validate the design of Pluristem’s ongoing Pivotal Phase III study in CLI, a more severe stage of peripheral arterial disease (PAD) and confirm Pluristem’s proprietary
Researchers at UC Davis have confirmed that autologous hematopoietic stem cell transplant improves survival for people suffering from multiple myeloma, yet many potentially eligible patients never undergo the procedure. Using data from two extensive California databases, the team showed median overall survival for transplant patients was around 73 months, while controls who did not receive the procedure lived around 50 months. The study was published June 11 in the Journal
Changes in cellular struts called microtubules (MT) can affect the stiffness of diseased human heart muscle cells, and reversing these modifications can lessen the stiffness and improve the beating strength of these cells isolated from transplant patients with heart failure, found researchers from the Perelman School of Medicine at the University of Pennsylvania. This Nature Medicine new study is a continuation of research conducted two years ago on how MTs
Self-Condensation Process for Cells Generates Vascularized Organ Tissues for Transplant Researchers tissue-engineered human pancreatic islets in a laboratory that develop a circulatory system, secrete hormones like insulin and successfully treat sudden-onset type 1 diabetes in transplanted mice. In a study published by Cell Reports, the scientists use a new bioengineering process they developed called a self-condensation cell culture. The technology helps nudge medical science closer to one day growing human
Researchers in China have discovered that an enzyme called UGT8 drives the progression of basal-like breast cancer, an aggressive form of the disease that is largely untreatable. But the study, which will be published May 4 in the Journal of Experimental Medicine, reveals that the widely used osteoporosis drug zoledronic acid inhibits UGT8 and prevents the spread of basal-like breast cancer in mice, suggesting that this drug could also be
The U.S. Food and Drug Administration (FDA) has expanded approval for a personalized cellular therapy developed at the University of Pennsylvania’s Abramson Cancer Center, this time for the treatment of adult patients with relapsed or refractory large B-Cell lymphoma after two or more lines of systemic therapy. Today’s approval includes treatment of diffuse large B-cell lymphoma (DLBCL) – the most common form of non-Hodgkin’s lymphoma – as well as high
Columbia biomedical engineers invent innovative technique to help injured hearts regenerate, through therapeutic application of extracellular vesicles secreted by cardiomyocytes derived from human pluripotent stem cells Heart disease is a major global health problem--myocardial infarction annually affects more than one million people in the U.S. alone, and there is still no effective treatment. The adult human heart cannot regenerate itself after injury, and the death of cardiac muscle cells, known
A drug given to early stage lung cancer patients before they undergo surgery showed major tumor responses in the removed tumor and an increase in anti-tumor T-cells that remained after the tumor was removed, which resulted in fewer relapse cases in the patients. The research teams at the Johns Hopkins Bloomberg~Kimmel Institute for Cancer Immunotherapy, the Johns Hopkins Sidney Kimmel Cancer Center and the Memorial Sloan-Kettering Cancer Center wanted to
Inhibition of the oncogenic kinase AKT, a key protein governing the cell cycle, was found to arrest cancer cell proliferation and triggered their programmed death by apoptosis. The study, published today in Oncogene, represents significant progress in the clinical translation of previous basic scientific discoveries. “Understanding the molecular features that govern cancer cell behavior is the basis for the design of the so-called ‘targeted therapies’ which constitute modern precision medicine,”
HDAC inhibitors are effective in treating ovarian tumors with mutations in the ARID1A gene. Wistar researchers have found rationale for repurposing a class of antitumor compounds called HDAC inhibitors, already approved by the FDA for the treatment of diseases such as leukemia, as a new therapeutic option for ovarian cancer with mutations in the ARID1A gene. Study results were published online in Cell Reports. Ovarian cancer is the most lethal
A study conducted at The Wistar Institute in collaboration with The University of Texas Southwestern Medical Center has demonstrated the efficacy of targeting aberrantly active telomerase to treat therapy-resistant melanoma. The research was published in the journal Clinical Cancer Research. The introduction of targeted therapies and immune checkpoint blockade therapies has revolutionized the therapeutic options for patients with advanced melanoma. However, the long-term therapeutic benefit of these new approaches is
Creating enough nanovesicles to inexpensively serve as a drug delivery system may be as simple as putting the cells through a sieve, according to an international team of researchers who used mouse autologous -- their own -- immune cells to create large amounts of fillable nanovesicles to deliver drugs to tumors in mice. Nanovesicles are tiny sacs released by cells that carry chemical messages between cells. These nanovesicles are natural
A prospective new method of treating patients with multiple sclerosis has been proposed by researchers of the Mainz University Medical Center working in cooperation with researchers of the University of Montreal. In model trials and experiments employing human endothelial cells, they discovered that the EGFL7 protein hinders the migration of immune cells into the central nervous system by stabilizing the blood-brain barrier. These findings have recently been published in Nature
Frontiers in Medicine has published key findings from a study of PLX-R18, a cell-based therapy using human placenta, demonstrated the cells’ efficacy in improving human hematopoietic engraftment. The article titled, “Posttransplant Intramuscular Injection of PLX-R18 Mesenchymal-Like Adherent Stromal Cells Improves Human Hematopoietic Engraftment in A Murine Transplant Model” was published in the peer-reviewed journal’s February 2018 issue. In the published study, mice were injected intramuscularly (IM) with PLX-R18 following human
