Biotechnology cancer rare diseases

Study Reports Possible Novel Method for Stopping Untreatable Pediatric Brain Cancer

Researchers used an experimental molecular therapy in preclinical laboratory tests to effectively treat several types of deadly pediatric brain cancer and now propose advancing the treatment to clinical testing in children. Scientists at the Cincinnati Children’s Cancer and Blood Diseases Institute report in the journal Molecular Cancer Therapeutics testing the small molecule 6-thio-2’deoxyguanosine (6-thio-dG) in […]

Biotechnology rare diseases Uncategorized

Study of Mucus May Help Guide Sinusitis Treatment

By April, nearly one-third of the U.S. is already experiencing high pollen levels while the weather and temperatures continue to fluctuate, aggravating sinus symptoms. A patient’s mucus may predict the type of his or her chronic sinusitis, which could help doctors determine whether surgery or medical treatments can produce the best outcomes, according to a […]

Biotechnology cancer Immunotherapies rare diseases

Smart Bomb Virus Shows Promise as Brain Tumor Immunotherapy

A common cold virus engineered to attack the most common and deadly of brain tumors allowed 20 percent of patients with recurrent glioblastoma to live for three years or longer, researchers from The University of Texas MD Anderson Cancer Center report on a phase I clinical trial in the Journal of Clinical Oncology. The altered […]

Biotechnology cancer Gene Therapy rare diseases

IU Advances Fight Against Hepatitis B with ‘Virus-Cracking’ Molecules

Indiana University researchers have made an important step forward in the design of drugs that fight the hepatitis B virus, which can cause liver failure and liver cancer. It’s estimated that 2 billion people worldwide have had a hepatitis B virus infection in their lifetime, with about 250 million — including 2 million Americans — […]

Biotechnology Infectious Diseases rare diseases

Study Shows Evidence of Severe and Lingering Symptoms in Some after Treatment for Lyme Disease

In a study of 61 people treated for the bacteria that causes Lyme disease, Johns Hopkins researchers conclude that fatigue, pain, insomnia and depression do indeed persist over long periods of time for some people, despite largely normal physical exams and clinical laboratory testing. “Post-treatment Lyme disease syndrome (PTLDS) is a real disorder that causes […]

Biotechnology rare diseases Vaccines

New vaccine technology shows promise as a tool to combat the opioid crisis

Researchers with the U.S. Military HIV Research Program at the Walter Reed Army Institute of Research (WRAIR) report that an experimental heroin vaccine induced antibodies that prevented the drug from crossing the blood-brain barrier in mice and rats. The vaccine was co-developed at the National Institute on Drug Abuse (NIDA), part of the National Institutes […]

Biotechnology cancer Gene Therapy Prodrug rare diseases

Deadly Lung Cancers Are Driven by Multiple Genetic Changes

Blood-Based Cancer Tests Reveal Complex Genomic Landscape of Non-Small Cell Lung Cancers A new UC San Francisco–led study challenges the dogma in oncology that most cancers are caused by one dominant “driver” mutation that can be treated in isolation with a single targeted drug. Instead, the new research finds one of the world’s most deadly […]

Biotechnology Clinical Trials Gene Therapy rare diseases

Scientists make a major breakthrough to treat fibrotic diseases that cause organ failure

Researchers from Duke-NUS Medical School (Duke-NUS) and the National Heart Centre Singapore (NHCS) have discovered that a critical protein, known as interleukin 11 (IL11) is responsible for fibrosis and causes organ damage. While it is surprising that the importance of IL11 has been overlooked and misunderstood for so long, it has now been very clearly […]

Biotechnology Cell Therapy Clinical Trials Immunotherapies rare diseases Vaccines

Trained T-Cells to Target Toxic Viruses in Pediatric Patients New Cellular Therapy Approach for Children with Compromised Immune Systems

Michael Pulsipher, MD, of the Children’s Center for Cancer and Blood Diseases at Children’s Hospital Los Angeles, along with Michael Keller, MD from Children’s National Health System in Washington, DC, have been awarded $4.8 million by the California Institute for Regenerative Medicine (CIRM) to study the use of a new T-cell therapy to help fight […]

Biotechnology Cell Therapy Clinical Trials rare diseases Vaccines

FDA Announces First Approval of Targeted Therapy Based on Basket Study

Precision medicine clinical trial leads to approval of first treatment for Erdheim-Chester disease The US Food and Drug Administration (FDA) has announced that it has approved the drug vemurafenib for the treatment of patients with BRAF V600-mutant Erdheim-Chester disease (ECD). This is the first approval of a targeted therapy based on a basket study and […]

Biotechnology cancer Cell Therapy Clinical Trials rare diseases

Breast cancer researchers track changes in normal mammary duct cells leading to disease

Breast cancer researchers have mapped early genetic alterations in normal-looking cells at various distances from primary tumours to show how changes along the lining of mammary ducts can lead to disease. The findings of the multidisciplinary team of surgeons, pathologists and scientists led by principal investigator Dr. Susan Done are published online today in Nature […]

rare diseases

FDA awards 15 grants for clinical trials to stimulate product development for rare diseases

The U.S. Food and Drug Administration today announced that it has awarded 15 new clinical trial research grants totaling more than $22 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry across the country. “Given […]

Biotechnology Immunotherapies Infectious Diseases rare diseases

Good-Guy Bacteria May Help Cancer Immunotherapies Do Their Job

Individuals with certain types of bacteria in their gut may be more likely to respond well to cancer immunotherapy, researchers at the Harold C. Simmons Comprehensive Cancer Center found in a study of patients with metastatic melanoma. The incidence of melanoma has been increasing over the past 40 years. Immunotherapies have dramatically improved the outlook […]

Biotechnology cancer rare diseases

Drug combination may improve impact of immunotherapy in head and neck cancer

Checkpoint inhibitor-based immunotherapy has been shown to be very effective in recurrent and metastatic head and neck cancer but only in a minority of patients. University of California San Diego School of Medicine researchers may have found a way to double down on immunotherapy’s effectiveness. In a paper published in the journal JCI Insights on […]

Biotechnology Gene Therapy Pharmaceuticals rare diseases

MDI Biological Laboratory study finds immune system is critical to regeneration

The answer to regenerative medicine’s most compelling question — why some organisms can regenerate major body parts such as hearts and limbs while others, such as humans, cannot — may lie with the body’s innate immune system, according to a new study of heart regeneration in the axolotl, or Mexican salamander, an organism that takes […]

Biotechnology Cell Therapy Clinical Trials rare diseases stem cells

CRI Scientists Discover Vitamin C Regulates Stem Cell Function and Suppresses Leukemia Development

Not much is known about stem cell metabolism, but a new study from the Children’s Medical Center Research Institute at UT Southwestern (CRI) has found that stem cells take up unusually high levels of vitamin C, which then regulates their function and suppresses the development of leukemia. “We have known for a while that people with […]

Biotechnology cancer Prodrug rare diseases

Combination of Traditional Chemotherapy, New Drug Kills Rare Cancer Cells in Mice

An experimental drug combined with the traditional chemotherapy drug cisplatin, when used in mice, destroyed a rare form of salivary gland tumor and prevented a recurrence within 300 days, a University of Michigan study found. Called adenoid cystic carcinoma, or ACC, this rare cancer affects 3,000-4,000 people annually, and typically arises in the salivary glands. […]

Biotechnology cancer Cancer Discovery rare diseases

Prostate Cancer Cells Become ‘Shapeshifters’ to Spread to Distant Organs

Johns Hopkins Kimmel Cancer Center scientists report they have discovered a biochemical process that gives prostate cancer cells the almost unnatural ability to change their shape, squeeze into other organs and take root in other parts of the body. The scientists say their cell culture and mouse studies of the process, which involves a cancer-related […]

Biotechnology cancer Clinical Trials rare diseases

New Therapeutic Approach for Difficult-to-Treat Subtype of Ovarian Cancer Identified

Scientists from The Wistar Institute demonstrate how a mutation in ovarian clear cell carcinoma can be exploited to design a targeted treatment. A potential new therapeutic strategy for a difficult-to-treat form of ovarian cancer has been discovered by Wistar scientists. The findings were published online in Nature Cell Biology. Ovarian clear cell carcinoma accounts for approximately […]