Breaking the ‘stalemate’ in the most common soft tissue sarcoma in children: First randomized clinical trial to show positive results in rhabdomyosarcoma since 1974

A phase 2 clinical trial has found that combining a molecular targeted drug called temsirolimus with chemotherapy shows promise in the treatment of rhabdomyosarcoma, the most common soft tissue sarcoma in childhood. The Children’s Oncology Group trial was led by Leo Mascarenhas, MD, MS, Deputy Director of the Children’s Center…

Promising approach to reducing plaque in arteries

In a new Yale-led study, investigators have revealed previously unknown factors that contribute to the hardening of arteries and plaque growth, which cause heart disease. Their insight is the basis for a promising therapeutic approach to halt and potentially reverse plaque buildup and the progression of disease, the researchers said….

Finnish people’s unique genetic makeup offers clues to disease: Study of Finns, with their similar DNA, gives new hints to cardiovascular, metabolic disease risk

A new study harnessed the unique genetic history of the people of Finland to identify variations in DNA that might predispose certain individuals to disease, whether or not they are Finnish themselves. The study was conducted by researchers at Washington University School of Medicine in St. Louis, in collaboration with…

Mutation discovery leads to precise treatment for child with severe lymphatic disorder: A repurposed drug reshapes anatomy, brings dramatic clinical improvement

Faced with a preteen boy in pain and struggling to breathe from a severe, deteriorating rare condition, researchers at Children’s Hospital of Philadelphia identified the responsible gene mutation and harnessed that knowledge to develop a novel treatment that dramatically improved the problem. The patient had been born with a complex…

Lymphoma trial finds combination targeted therapy effective prior to chemotherapy: MD Anderson Phase II study suggests patients with lymphoma subtype may be able to avoid chemotherapy in the future

Results of a Phase II clinical trial conducted at The University of Texas MD Anderson Cancer Center revealed that combination targeted therapy, consisting of rituximab, lenalidomide and ibrutinib (RLI), had an 84.6 percent overall response rate (ORR) and 38.5 percent complete response rate (CRR) when given prior to any chemotherapy…

Clinical trial improves treatment of genetic rickets

A new study shows a drug developed in conjunction with investigators at Indiana University School of Medicine to alleviate symptoms of a rare musculoskeletal condition is significantly more effective than conventional therapies. The findings are published in Lancet. X-linked hypophosphatemia, or XLH, is a phosphate-wasting disease that causes rickets and…

Multiple myeloma: DNA rearrangement may predict poor outcomes

A certain type of DNA marker predicts poor outcomes in multiple myeloma, researchers at Winship Cancer Institute, Emory University have found. The marker is a kind of rearrangement of chromosomes that is rarely tested for, but may indicate resistance to immunomodulatory drugs such as lenalidomide, which have become standard therapies…