China 
Africa 
Explore chapters and articles related to this topic
Doxil® — The First FDA-Approved Nano-Drug: From an Idea to a Product
Published in Dan Peer, Handbook of Harnessing Biomaterials in Nanomedicine, 2021
The first clinical use of liposomes dates back to 1974, when the fate of radiolabeled liposomes was examined in three cancer patients (Gregoriadis et al., 1974). This was followed by Gregoriadis and coworkers’ use of liposomes as a carrier for the enzyme glucocerebroside (a beta-glucosidase) for enzyme replacement therapy for Gaucher’s disease treatment (Belchetz et al., 1977). More extensive studies in humans with radiolabeled “fluid”-phase sonicated liposomes indicated that the major tissues of liposome uptake are the liver and spleen (Richardson et al., 1979). A similar study in which the distribution of technetium-99m-labeled MLV in patients with Hodgkin’s disease was performed by Perez-Soler et al. (1985). The earliest report in the literature on the therapeutic use of liposomes as drug carriers given systemically to a sizable group of patients described the experience gained after administration of amphotericin-B in liposomes. It showed clear beneficial antifungal activity without apparent toxicity at doses in which serious toxic effects of the free drug occurs (Lopez-Berestein et al., 1985). A similar study with another (sonicated) liposomal amphotericin-B formulation was performed later in cancer patients with fungal infections by Sculier and coworkers (1988). A pilot human study with the water-insoluble cytotoxic drug NSC 251635 was also performed (Sculier et al., 1986).
Biocatalytic Nanoreactors for Medical Purposes
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2019
Oscar González-Davis, Chauhan Kanchan, Rafael Vazquez-Duhalt
Gaucher’s disease is an inherited disorder caused by deficient activity of the enzyme β-glucocerebrosidase, found mainly in lysosomes. The lack of glucocerebrosidase activity results in an accumulation of glucocerebroside in the lysosomes of macrophages, especially in the reticuloendothelial system. The accumulation of glucocerebroside leads to hepatomegaly, splenomegaly, anemia, and thrombocytopenia causing fatigue, discomfort, infections, bleeding and bruising. In addition, it induces bone related problems such as pain, bone crises, and avascular necrosis. Other problems such as lung disease, impaired growth, and delayed puberty are also associated with Gaucher’s disease (Grabowski et al., 1998; Stirnemann et al., 2017). The clinical effectiveness of the ERT has been reviewed (Grabowski et al., 1998; Connock et al., 2006). A recombinant and glycoengineered glucocerebrosidase containing mannosyl-ended oligosaccharides has been designed in order to be recognized by the specific receptors for α-mannosyl on macrophages (Oh, 2015). Also, a plant-derived variant of glucocerebrosidase has been mannosyl-targeted to disease-affected cells (Tekoah et al., 2013). Thus, the targeted delivery of enzyme for the specific treatment of Gaucher’s disease is feasible.
Pulmonary hypertension induced by drugs and toxins
Published in Philippe Camus, Edward C Rosenow, Drug-induced and Iatrogenic Respiratory Disease, 2010
Kim Bouillon, Yola Moride, Lucien Abenhaim, Marc Humbert
Gaucher’s disease is an autosomal-recessive disorder that is caused by a deficiency of the enzyme glucocerebrosidase. PAH has been detected in patients with type 1 Gaucher’s disease. This may be due at least in part to perivascular infiltration by Gaucher’s cells with vascular obliteration and plugging of the capillaries with Gaucher’s cells. However, three cases of PAH have been reported to be associated with the use of alglucerase after eliminating the possible involvement of Gaucher’s cells.61,62
Progress in spray-drying of protein pharmaceuticals: Literature analysis of trends in formulation and process attributes
Published in Drying Technology, 2021
Joana T. Pinto, Eva Faulhammer, Johanna Dieplinger, Michael Dekner, Christian Makert, Marco Nieder, Amrit Paudel
Enzymes are catalyst proteins essential for the biochemical reactions in living organisms.[41] Therapeutic enzymes are mainly applied as replacement therapies in genetic disorders, such as Gaucher disease.[42] Other approved enzymes are applied in blood clotting diseases, cancers, immunodeficiency, etc.[42] The accessibility and the ease of functional analysis[43] of enzymes made them the most extensively studied group of protein therapeutics via spray-drying.[21,24,28,44–75]