Biotechnology Cell Therapy Infectious Diseases rare diseases

Cancer Drug and Antidepressants Provide Clues for Treating Fatal Brain-Eating Amoeba Infections

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 […]

Antibiotics Clinical Trials drug development rare diseases

Taking a Pill Can Effectively Treat Brutal Lung Disease

Researchers report in Nature Communications they figured out why air sacs in the lungs clog up with a thick substance called surfactant in a brutal disease called Pulmonary Alveolar Proteinosis (PAP), and they show taking cholesterol-busting pills called statins can effectively treat the disease. That’s good news for people with PAP because at present the […]

Biotechnology Pharmaceuticals rare diseases

Researchers Discover Llama-Derived Nanobody Can Be Used as Potential Therapy for Hard-to-Treat Diseases

Researchers from Case Western Reserve University School of Medicine have found a nanobody that holds promise to advance targeted therapies for a number of neurological diseases and cancer. In a recent study published in Nature Communications, Sahil Gulati, of the Department of Pharmacology at Case Western Reserve School of Medicine, and colleagues identified a nanobody derived […]

Biotechnology Clinical Trials Pharmaceuticals rare diseases

New Insight Into Huntington’s Disease May Open Door to Drug Development

McMaster University researchers have developed a new theory on Huntington’s disease which is being welcomed for showing promise to open new avenues of drug development for the condition. Huntington’s disease is caused by a mutation in the gene that makes the protein called huntingtin. A team of researchers led by McMaster has found there is […]

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 […]