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
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 brain cancer stem cells derived from tumor cells donated by patients. Researchers also tested the
Cancer Discovery

Study Suggests Way to Attack Deadly, Untreatable Nerve Tumors

Photo caption: This microscopic image uses immunostaining to highlight the presence of TAZ/YAP (shown in green) in human malignant peripheral nerve sheath tumors that grew from Schwann cells. The job of Schwann cells is to form the protective nerve sheath. The cell nuclei are shown in blue. The image is from a study by scientists at Cincinnati Children’s published by the journal Cancer Cell. Genomic profiling of mostly untreatable and
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 for patients with metastatic melanoma in the past half-dozen years, but still only about half
Biotechnology Infectious Diseases rare diseases

Childhood obesity major link to hip diseases

New research from the University of Liverpool, published in the Archives of Disease in Childhood journal, shows a strong link between childhood obesity and hip diseases in childhood. Significant hip deformities affect around 1 in 500 children. Slipped Capital Femoral Epiphysis (SCFE) is the most common hip disease of adolescence. The condition always requires surgery, can cause significant pain, and often leads to a hip replacement in adolescence or early
Biotechnology Gene Therapy

One gene closer to regenerative therapy for muscular disorders

A detour on the road to regenerative medicine for people with muscular disorders is figuring out how to coax muscle stem cells to fuse together and form functioning skeletal muscle tissues. A study published June 1 by Nature Communications reports scientists identify a new gene essential to this process, shedding new light on possible new therapeutic strategies. Led by researchers at the Cincinnati Children's Hospital Medical Center Heart Institute, the
Biotechnology

Engineering Researcher at MSU Helps Design Artificial Lung Device

Children with chronic lung diseases often must wait months or even years for a transplant, while large, immobile hospital equipment that could help them breathe easier actually may worsen their condition by overtaxing already damaged lungs. Additionally, the required bed confinement can bring about a decline in these young patients’ overall physical and mental states. At Mississippi State, Greg Burgreen is part of a team at the university’s Center for
cancer rare diseases

Scientists Find Possible Achilles Heel of Treatment Resistant Cancers

Scientists identify two signaling proteins in cancer cells that make them resistant to chemotherapy, and show that blocking the proteins along with chemotherapy eliminate human leukemia in mouse models. Reporting results March 20 in Nature Medicine, researchers at Cincinnati Children’s Hospital Medical Center suggest that blocking the signaling proteins c-Fos and Dusp1 as part of combination therapy might cure several types of kinase-driven, treatment-resistant leukemia and solid tumor cancers. These
Biotechnology

Predicting Autism: Researchers Find Autism Biomarkers in Infancy

By using magnetic resonance imaging (MRI) to study the brains of infants who have older siblings with autism, scientists were able to correctly identify 80 percent of the babies who would be subsequently diagnosed with autism at 2 years of age. Researchers from the University of Washington were part of a North American effort led by the University of North Carolina to use MRI to measure the brains of "low-risk"
cancer Cancer Discovery

Protein Network Linked To Cancer Is Critical To Male Fertility

Researchers studying reproductive science identified a network of proteins often linked to cancer as also important to male fertility and the birth of healthy offspring, according to a study in the Oct. 18 online issue of Cell Reports. The study by Satoshi Namewaka, PhD, and colleagues at Cincinnati Children’s Hospital Medical Center focuses on the precise epigenetic regulation of the sex chromosomes, which is important to germline cells that make
Biotechnology Cardiology

In Child Heart Patients, a Novel Approach Improves Symptoms of Hazardous Lymph Blockage

Pediatric researchers have devised an innovative, safe and minimally invasive procedure that helps relieve rare but potentially life-threatening airway blockages occurring in children who had surgery for congenital heart defects. The physician-researchers developed new imaging tools and used minimally invasive catheterization techniques to treat plastic bronchitis, a condition in which abnormal circulation causes lymphatic fluid to dry into solid casts that clog a child’s airways. The authors reported their retrospective
Biotechnology Genomes rare diseases

Drug Candidate Halts Crippling Excess Bone Growth in Animal Model of a Rare Bone Disease

New research in laboratory animals suggests that the drug palovarotene may prevent multiple skeletal problems caused by a rare but extremely disabling genetic bone disease, and may even be a candidate for use in newborn babies with the condition. Scientists at The Children’s Hospital of Philadelphia, who previously repurposed the drug to prevent excess bone formation in animal models of fibrodysplasia ossificans progressiva (FOP), have extended that research in animals