Rare Disease

The new immunotherapy of chimeric antigen receptor (CAR)-T cells has demonstrated the ability to increase clinical remission in multiple myeloma patients by targeting the B-cell maturation protein that participates in disease progression.

Enasidenib (Idhifa), developed by Celgene and Agios, has been granted FDA approval in tandem with a companion diagnostic test to detect IDH-2 mutations.

The FDA has expanded the indication of Bristol-Myers Squibb’s Yervoy (ipilimumab) to include the treatment of pediatric patients 12 years of age and older with unresectable or metastatic melanoma.

Venetoclax (Venclexta), in combination with low dose cytarabine, has been granted a breakthrough therapy designation for use in elderly, treatment-naive patients with acute myeloid leukemia (AML) who cannot handle intensive chemotherapy.

Patient-reported outcomes (PROs) can demonstrate the effects of a disease or treatment. By using patient questionnaires, the results can be analyzed to predict clinical outcomes in medical research for diseases, including multiple sclerosis (MS).

Researchers have identified a cellular signaling pathway that when activated by mutations may prevent immune cells from infiltrating bladder tumors, thus making the cancer resistant to immune checkpoint inhibitors.

In a late-breaking abstract at the 2017 American Society of Clinical Oncology Annual Meeting, researchers presented an interim analysis of a randomized phase 2 trial of second- or third-line nivolumab, with or without ipilimumab, in patients with second- or third-line malignant pleural mesothelioma.

Report notes that 74% of drugs that are currently in development are potentially first-in-class medicines, and 822 drugs in development are for rare diseases.

Managing heavily pretreated, often less fit, patients with relapsed/refractory multiple myeloma is a challenge in routine practice, as illustrated by the fact progression-free survival remains short, although daratumumab-based combination therapies are proven effective, according to results presented during a poster session at the 2017 American Society of Clinical Oncology Annual Meeting.

A phase 1 study, presented at the 2017 Annual Meeting of the American Society of Clinical Oncology, found that using daratumumab, an antibody that binds and inhibits the CD38 receptor, can improve patient response to treatment.

Updated trial results at the 2017 Annual Meeting of the American Society of Clinical Oncology showed the combinations of daratumumab with either lenalidomide and dexamethasone or bortezomib and dexamethasone prolonged progression-free survival for patients with relapsed refractory multiple myeloma.

A new study examines the impact that a pharmaceutical’s designation as an orphan drug can have on its manufacturer’s stock prices, and finds that the largest returns were for oncology drugs.

Blinatumomab is now fully approved as a treatment for relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), following FDA assessment of new trial data on survival outcomes and its use in certain clinical subgroups.

Severe hemophilia often results in a significant economic and psychological burden on patients, caregivers, and the healthcare system as a whole, according to recent research from Europe.

An analysis of whole-genome sequencing (WGS) in healthy adults in the primary care setting found that 1 in 5 had a previously unrecognized variant associated with a rare disease, according to a study published in Annals of Internal Medicine.

Novartis’ chimeric antigen receptor T-cell (CAR-T) therapy for treating pediatric leukemia is on the cusp of being the first FDA-approved gene therapy, which will lead to new developments and utilizations of CAR-T therapy for treating other advanced blood cancers.

A study published by BloodAdvances, a Journal of the American Society of Hematology, demonstrated the potential of the 2-pronged approach in treating non-Hodgkin lymphoma, multiple myeloma, and acute myeloid leukemia.

Sarepta Therapeutics has announced a new collaboration with Genethon for gene therapy research in efforts to develop new Duchenne muscular dystrophy (DMD) treatments.

Whipple’s disease (WD) is a rare disease that predominantly affects middle-aged white men, and its diagnosis is often delayed because it is misdiagnosed as rheumatoid arthritis. A new study in Orphanet Journal of Rare Diseases aimed to describe clinical symptoms of WD and appropriate treatment.

Among a group of patients with chronic lymphocytic leukemia (CLL) who were previously unresponsive to rituximab, a new antibody could help enable the immune system to attack the cancer cells, according to a study in PLOS One.

The study, published in Lancet Oncology, has found that nivolumab reduced the rate of clinically meaningful deterioration compared with investigator’s choice among platinum-refractory patients being treated for recurrent or metastatic head and neck cancer.

On Monday, June 26, 2017, the Biden Foundation announced the launch of the Biden Cancer Initiative, their new venture to continue the fight to make progress in cancer prevention, detection, treatment, and care.

Data from multiple studies have demonstrated the efficacy of Venclexta/Venclyxto in treating relapsed or refractory chronic lymphocytic leukemia, according to Roche.