rare diseases

FDA approves first treatment specifically for patients with rare and life-threatening type of immune disease

The U.S. Food and Drug Administration today approved Gamifant (emapalumab-lzsg) for the treatment of pediatric (newborn and above) and adult patients with primary hemophagocytic lymphohistiocytosis (HLH) who have refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. This FDA approval is the first for a drug specifically for HLH. "Primary HLH is a rare and life-threatening condition typically affecting children and this approval fills an unmet medical need
Biotechnology cancer Cell Therapy Immunotherapies rare diseases

Study Uncovers Key Parts of Mechanism for Activating T Cells to Fight Cancer and Other Diseases

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
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 School of Pharmacy and Pharmaceutical Sciences at University of California San Diego have now identified
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How Malaria Parasites Take Over Human Red Blood Cells, According to Newly Published Research

Researchers at Iowa State University have gained new insight into how the parasitic protozoa that cause malaria hijack human red blood cells, a development with the potential to lead to new ways to treat the disease. The parasites that cause malaria symptoms in humans enter the red blood cells of a host and quickly rearrange things to their liking by inserting their own proteins into the red blood cell. But
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 current standard treatment is something called a whole lung lavage. Essentially, it involves flushing patient
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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 from a llama that targets signaling of G protein-coupled receptors (GPCRs), a large family of
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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 a unique type of signalling coming from damaged DNA, that signals huntingtin activity in DNA
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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 brain cancer stem cells derived from tumor cells donated by patients. Researchers also tested the
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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 recently published Vanderbilt study. Justin Turner, MD, PhD, associate professor of Otolaryngology-Head and Neck Surgery,
Biotechnology rare diseases

Unexpected Finding May Deter Disabling Diabetic Eye Disease

Diabetic retinopathy is considered one of the most disabling complications of diabetes and the leading cause of new cases of vision loss among adults. A new Michigan State University study is the first to find that a particular type of lipid, or fat, thought to only exist in the skin, now lives in your eye and might play a major role in deterring the eye disease. “Our study presents an
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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 adenovirus, called Delta-24-RGD or DNX-2401, was injected one time directly into the tumors of 25
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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 -- living with chronic infection. Although a vaccine exists, there is no cure. The study, published Jan.
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 severe symptoms in the absence of clinically detectable infection,” says John N. Aucott, M.D., associate
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 of Health, which funded the preclinical research. "By eliciting antibodies that bind with heroin in
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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 forms of lung cancer is driven by changes in multiple different genes, which appear to
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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 demonstrated by this work. A protein known as transforming growth factor beta 12 ("TGFB1") has
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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 active viral infections in children with severe immune deficiencies. In what will be the largest
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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 the first-ever drug approved for ECD, a rare blood disorder. This landmark approval came as
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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 Communications. Dr. Done, a pathologist affiliated with The Campbell Family Institute for Breast Cancer Research
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 the often small number of patients facing certain rare diseases, there can be limited resources