Rare Disease

Among the 9 new orphan drugs approved by the FDA in 2016 were 3 treatments for rare diseases that, so far, had no approved treatments: Duchenne muscular dystrophy, spinal muscular atrophy, and severe hepatic veno-occlusive disease.

This study measured the value of survival gains attributable to the introduction of 3 novel therapies for myelodysplastic syndromes.

At the 58th American Society of Hematology Annual Meeting & Exposition, Jan Joseph Melenhorst, PhD, presented results of a study evaluating biomarkers of response to anti-CD19 CAR T-cell treatment in patients diagnosed with chronic lymphocytic leukemia (CLL).

In high-risk patients with chronic lymphocytic leukemia (CLL), CD19 chimeric antigen receptor (CAR)-T cells of defined composition can be administered with an acceptable early toxicity.

A new initiative has been launched at the University of North Carolina to create and share tools to fight rare diseases.

Researchers from the Memorial Sloan Kettering Cancer Center have developed a novel method using modified donor T cells to make stem cell transplants both safer and more effective for treating blood cancers

A new single-patient case study, published in the New England Journal of Medicine, has raised the possibility of using CAR-T cells in the treatment of glioblastoma, an aggressive form of brain tumor.

The first patient has been treated with ruxolitinib (Jakafi), by Incyte Corporation, for steroid-refractory acute graft-versus-host disease, as part of the REACH-1 phase 2 trial. The trial is evaluating ruxolitinib in combination with corticosteroids.

Caring at home for children with special healthcare needs brings high costs to families. A study by researchers at the University of Southern California, Boston Children’s Hospital, and RAND Corp., placed the uncompensated medical cost at nearly $36 billion annually.

A new clinical study to reduce the unwanted side effects of radiation in patients with human papillomavirus (HPV)—positive oropharynx cancers has found that reducing the dose of radiation can maintain the cure rates but avoid some of the late treatment-associated toxicities.

The Lancet study shows addition of bortezomib significantly improves survival in treatment-naïve patients with multiple myeloma.

Using angiogenesis inhibitors in patients with glioblastoma that is highly vascularized could yield much improved outcomes, the analysis found.

A report card published by the National Organization for Rare Disorders issued grades to each state for 7 categories of rare disease policies.

Researchers at the Moffitt Cancer Center have developed a genomics model called genomic-adjusted radiation dose that can guide the radiation dose to match a tumor’s radiosensitivity.

Craig Sager, a sideline reporter for CNN and Turner Sports, has died a little over 2 and a half years after being diagnosed with acute myeloid leukemia (AML).

In high-risk patients with chronic lymphocytic leukemia (CLL), CD19 chimeric antigen receptor (CAR) T-cells of defined composition can be administered with an acceptable early toxicity.

Representatives from the Beat acute myeloid leukemia (AML) and National Cancer Institute-Molecular Analysis for Therapy Choice (NCI-MATCH), which incorporate genomic profiling to assign patients to different treatment arms, provided an insight on trial design and a progress report.

Older patients diagnosed with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) continue to present a favorable response to single-agent ibrutinib, at a follow-up of 29 months, in phase 3 of the RESONATE-2 trial.

Early phase 1 results show that including the cyclin-dependent kinase 4/6 inhibitor palbociclib in the treatment of patients with mantle cell lymphoma (MCL) can help overcome resistance to ibrutinib.

Chimeric antigen receptor (CAR) T-cells have been dramatically effective in treating B-cell cancers, according to David L. Porter, MD, of the University of Pennsylvania Health System. He also identified the use of CAR T-cells for treating solid tumors as a research area that will see more development in the coming years.

Interim data analysis of a phase 1 trial of chimeric antigen receptor-T cells (CAR-T) targeting the B-cell maturation antigen in heavily pretreated patients with multiple myeloma has identified an objective anti-tumor response, with limited toxicity.

Statin use was associated with a reduction in all-cause and multiple myeloma-specific mortality.

A study published in the Annals of Oncology has analyzed data on young patients with acute lymphoblastic leukemia (ALL) to evaluate risk factors for febrile neutropenia and infection-related complications.

Within 4 months of the last reported incident, Juno Therapeutics has again halted the phase 2 “Rocket” trial of JCAR015 in patients with relapsed or refractory B cell acute lymphoblastic leukemia (ALL).

A study published by researchers from the Fred Hutchinson Cancer Research Center in Seattle has found that admission to the intensive care unit reduced survival and increased the cost of care among patients undergoing treatment for acute myeloid leukemia.

At the Annual Meeting of the American College of Rheumatology in Washington, DC, several experts presented their progress in harnessing technology to help teenagers and young adults manage their juvenile arthritis.

David Hafler, MD, chairman of the Department of Neurology at the Yale School of Medicine, spoke of exciting times in the field of genome-wide association study-specifically speaking to neurology, genetics, the environment, and the autoimmune response.