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Antibody-Based Therapies
Published in David E. Thurston, Ilona Pysz, Chemistry and Pharmacology of Anticancer Drugs, 2021
Tagraxofusp (Elzonris™) is a fusion protein consisting of interleukin 3 (IL-3) fused to diphtheria toxin that is used for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). It gained FDA approval in 2018 and it is considered to be a first-in-class medication. Elzonris™ is a novel targeted therapy directed to CD123 of the tumor cells. The IL-3 binds to the CD123-expressing tumor cells and delivers the cytotoxic diphtheria toxin to the cells. This causes the blockage of protein synthesis in the cell and causing cell death. The approval of Elzonris™ was based on the positive results of two multi-center, open-label, single-arm clinical trials, which were performed in two groups of patients. The rate of complete response was the primary endpoint of the trial. The primary endpoint was reached by approximately 54% of the patients. One of the patients achieved a complete response and one clinical complete response in the study. Most common adverse events include capillary leak syndrome, nausea, weight gain, fatigue, peripheral edema, and an increase in body temperature.
Current and emerging pharmacotherapeutic strategies for Tourette syndrome
Published in Expert Opinion on Pharmacotherapy, 2022
Lurasidone is a dopamine and serotonin receptor antagonist used for the treatment of schizophrenia and bipolar affective disorder. In a recently published small case series, this medication was added on to either risperidone or aripiprazole for the treatment of refractory TS with co-morbid obsessive symptoms and aggressive behavior, with encouraging results in terms of control of behavioral symptoms and tolerability (both motor and metabolic adverse effects) [70]. Ecopipam is a first-in-class medication characterized by selective D1 receptor antagonism demonstrated significant tic reduction and good tolerability in both an open-label study [71] and a follow-up randomized, placebo-controlled crossover study of young patients with TS [72]. A phase IIb trial of ecopipam in young patients with TS (D1AMOND study) includes an ongoing open-label extension following the randomization period. The results of the randomized controlled trial component of the D1AMOND study were recently made available: these encouraging findings suggested a 30% reduction in tic severity [73].
An evaluation of mitapivat for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency
Published in Expert Review of Hematology, 2022
Andrew B. Song, Hanny Al‐Samkari
Although mitapivat was initially investigated for potential use in cancer patients as a PK-M2 activator [25,26], the focus of its development shifted early on to the treatment of PKD via activation of PK-R. As a first-in-class medication, it is the only currently available treatment that directly targets the underlying pathophysiologic defect in PKD. Results of recent successful phase 2 and phase 3 clinical trials ultimately led to the approval of mitapivat by the U.S. Food and Drug Administration (FDA) for treatment of PKD-associated anemia in adults on February 17th, 2022 [27]. Mitapivat is also currently being studied for use in patients with alpha- and beta-thalassemia, sickle cell disease, congenital dyserythropoietic anemia and hereditary spherocytosis.
Role of ivabradine and heart rate lowering in chronic heart failure: guideline update
Published in Expert Review of Cardiovascular Therapy, 2018
Sheryl L Chow, Robert L. Page, Christophe Depre
The concept of heart rate lowering to improve outcomes is a novel approach to HF management. Beta-blockers are the cornerstone of therapy, but reaching target doses cannot always be achieved in patients with severe asthma, chronic obstructive pulmonary disease (COPD), or hypotension. Unlike other heart rate lowering medications, which cause negative inotropy or hypotension, ivabradine can be used safely in select HF patients. New guideline updates recommend adding ivabradine to standard guideline-directed management and therapy in patients with resting heart rate ≥70 bpm and sinus rhythm. Ivabradine is a first-in-class medication used to improve patient symptoms and reduce hospitalizations.