Rare Disease

A roundup of top healthcare news stories: January 27, 2017.

“Off-the-shelf” chimeric antigen receptor (CAR)-T cells, also known as universal donor cells, were used in 2 young infants with relapsed, refractory acute lymphoblastic leukemia resulted in molecular remission in 28 days in both infants.

Researchers from the Peter O’Donnell Jr. Brain Institute and Harold C. Simmons Comprehensive Cancer Center at the University of Texas Southwestern may have found the ultimate drivers of glioblastoma cell proliferation.

At the 58th annual meeting of the 58th American Society of Hematology, representatives from 2 national clinical trials, Beat AML and NCI-MATCH, detailed how they were incorporating genomic profiling to assign patients to different treatment arms.

A poster presented at the 58th Annual Meeting & Exposition of the American Society of Hematology compared the utilization patterns and associated costs for recently approved and older drugs in patients with relapsed/refractory multiple myeloma in the United States.

Patients with rare diseases often turn to the Internet for information on their illness, but an analysis of some of these websites found that their content often failed to meet important quality criteria and neglected key information categories.

The improved expansion of chimeric antigen receptor (CAR) T-cells indicates a deeper clinical response.

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

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.