Abstract:
OBJECTIVE:The high cost of orphan drugs limits their access by many patients, especially in low- and middle-income countries. Many orphan drugs are off-patent without alternative generic or biosimilar versions available. Production of these drugs at the point-of-care, when feasible, could be a cost-effective alternative. METHODS:The financial feasibility of this approach was estimated by setting up a small-scale production of recombinant human acid alpha-glucosidase (rhGAA). The commercial version of rhGAA is Myozyme™, and Lumizyme™ in the United States, which is used to treat Pompe disease. The rhGAA was produced in CHO-K1 mammalian cells and purified using multiple purification steps to obtain a protein profile comparable to Myozyme™. RESULTS:The established small-scale production of rhGAA was used to obtain a realistic cost estimation for the magistral production of this biological drug. The treatment cost of rhGAA using bedside production was estimated at $3,484/gram, which is 71% lower than the commercial price of Myozyme ™. CONCLUSION:This study shows that bedside production might be a cost-effective approach to increase the access of patients to particular life-saving drugs.
journal_name
Curr Pharm Biotechnoljournal_title
Current pharmaceutical biotechnologyauthors
Aldosari MH,den Hartog M,Ganizada H,Evers MJW,Mastrobattista E,Schellekens Hdoi
10.2174/1389201021666200217113049subject
Has Abstractpub_date
2020-01-01 00:00:00pages
467-479issue
6eissn
1389-2010issn
1873-4316pii
CPB-EPUB-104561journal_volume
21pub_type
杂志文章abstract::Chronic inflammation and oxidative stress increase with advancing age and appear to be involved in the pathogenesis of coronary heart disease, the leading cause of death worldwide. There is a need for animal models that reflect the increases in pro-inflammatory cytokines and oxidative damage observed during aging in h...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/138920110793262006
更新日期:2010-12-01 00:00:00
abstract::CNS disorders are the third major problem of health in developed countries, with approximately 10% of direct costs associated with a pharmacological treatment of doubtful cost-effectiveness. There is an alarming abuse of psychotropic drugs worldwide and only 20-30% of patients with CNS disorders appropriately respond ...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920112799857576
更新日期:2012-04-01 00:00:00
abstract::Clostridium perfringens alpha-toxin is thought to be an important agent in gas gangrene, which is a lifethreatening infection with fever, pain, edema, myonecrosis, and gas production. The toxin (370 residues) is composed of an N-terminal domain (1-250 residues, N-domain) in which the catalytic site is found and a C-te...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/1389201014666131226124348
更新日期:2013-01-01 00:00:00
abstract:AIMS:Candida species is the common cause of opportunistic fungal infections all over the world with increased mortality and morbidity especially in immunosuppressed patients. Fluconazole is the first line therapy for candidiasis. The antifungal resistance pattern in high-risk patients is a major concern. The present st...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/1389201019666180515104800
更新日期:2018-01-01 00:00:00
abstract::Better understanding of protein higher order structures (HOS) is of major interest to researchers in the field of biotechnology and biopharmaceutics. Monitoring a protein's HOS is crucial towards understanding the impact of molecular conformation on the biotechnological application. In addition, maintaining the HOS is...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/1389201011314020010
更新日期:2013-01-01 00:00:00
abstract::It is now widely believed that tumors originate from normal cells as a result of accumulated genetic/epigenetic changes. These alterations affect the signaling pathways at transcriptional and post-transcriptional level that drive cells into uncontrolled cell division, growth, and migration. Recent advancement of molec...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920108785915102
更新日期:2008-10-01 00:00:00
abstract::Several of the 28 mammalian transient receptor potential (TRP) channel subunits are expressed throughout the alimentary canal where they play important roles in taste, chemo- and mechanosensation, thermoregulation, pain and hyperalgesia, mucosal function and homeostasis, control of motility by neurons, interstitial ce...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920111793937862
更新日期:2011-01-01 00:00:00
abstract::MicroRNAs are small non-coding RNA molecules which do not code for proteins, but control the expression of a significant proportion of the genome. The microRNA expression differences of normal and malignant tissue, as well as the differences in various tumor entities qualify this RNA species as biomarker for cancer pa...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/1389201015666140519124909
更新日期:2014-01-01 00:00:00
abstract::In this review, the compartments of the vasculature will be discussed with respect to their potential contribution to the build-up of a differentiated and functionally meaningful vessel system. Chronic ischemia of muscle tissue results in microvessel rarification, which represents a potential therapeutic target, given...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920107779941390
更新日期:2007-02-01 00:00:00
abstract::Many theoretical models of molecular interactions, biochemical and chemical reactions are described on the single-molecule level, although our knowledge about the biochemical/chemical structure and dynamics primarily originates from the investigation of many-molecule systems. At present, there are four experimental pl...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/1389201043376986
更新日期:2004-04-01 00:00:00
abstract::Assessment of immunogenicity is a major aspect in evaluating the safety of biological therapeutic proteins. It is important to evaluate the immunogenic potential of the biologics in an appropriate fashion using clearly defined strategy and clinical trials. The studies must include the appropriate risk assessment proce...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920109788488860
更新日期:2009-06-01 00:00:00
abstract:BACKGROUND:Bacteriophages are viruses, which are obligate parasites of specific bacteria for the completion of their lifecycle. Bacteriophages could be the possible alternative to antibioticresistant bacterial diseases. With this objective, extensive research in different fields is published which are discussed in this...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/1389201021666191217111156
更新日期:2020-01-01 00:00:00
abstract:BACKGROUND:Osteosarcoma is a malignant tumor type that starts in bone and occurs most frequently in adolescents. Traditional techniques are insufficient, especially for metastatic ones. As alternative treatment techniques, natural products are worthy to examine due to being safe and innovative. Essential polyunsaturate...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/1389201021666200928101029
更新日期:2020-09-27 00:00:00
abstract::TGF-β isoforms (TGF-β1, -β2, and -β3) are secreted signaling ligands that stimulate the expression of protein components of the extracellular matrix, regulate the growth and differentiation of epithelial cells, modulate immune cell function, and play roles in the development of several essential organs, including the ...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920111798808383
更新日期:2011-12-01 00:00:00
abstract::There is a growing trend of herbal medicines in India as well as other parts of the world. The use of herbal medicines alone or alongside prescription drugs for disease management is quite common now. We hereby carry out a review of dominant species of Selinum mainly S. vaginatum C.B. Clarke and S. wallichianum (DC.) ...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/1389201020666181227150829
更新日期:2018-01-01 00:00:00
abstract::The aim of the present article was to review the role of the placenta function in the transfer of immunosuppressants and monoclonal antibodies used for the treatment of inflammatory bowel disease, as well as the available data regarding the safety of these therapies during pregnancy. Methotrexate can cause congenital ...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920111795470903
更新日期:2011-05-01 00:00:00
abstract:OBJECTIVE:The goals of this study were to observe how luteolin protects endothelial cells (ECs) from injury stimulated by Angiotensin II (Ang II), investigate the role of vascular endothelial dysfunction in vascular smooth muscle cell proliferation and migration in vitro and investigate its primary mechanism of action....
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/1389201015666140113113843
更新日期:2014-01-01 00:00:00
abstract:PURPOSE:The work was performed to investigate the feasibility of preparing ocular inserts loaded with Poly-ε-Caprolactone (PCL) nanoparticles as a sustained ocular delivery system. METHODS:First, Atorvastatin Calcium-Poly-ε-Caprolactone (ATC-PCL) nanoparticles were prepared and characterized. Then, the optimized nanop...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/1389201021666200519133350
更新日期:2020-01-01 00:00:00
abstract::Plants are rich in a wide variety of secondary metabolites with antimicrobial properties. Phytochemistry studies on the plants extracts in general, and on essential oils (EOs) in particular are focused on the isolation and identification of the components of complex mixtures, in order to determine structure - activity...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/138920101602150112151549
更新日期:2015-01-01 00:00:00
abstract::Spirulina, a filamentous cyanobacterium, possesses diverse biological activities and nutritional significance due to high concentration of natural nutrients, having bio-modulatory and immuno-modulatory functions. Different Spirulina preparations influence immune system viz. increase phagocytic activity of macrophages,...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920105774370607
更新日期:2005-10-01 00:00:00
abstract:AIM OF THE STUDY:To assess tramadol and O-desmethyltramadol (M1) concentrations and their correlations with analgesia in patients with cancer pain. PATIENTS AND METHODS:Thirty opioid-naive patients with nociceptive pain intensity on VAS (visual analogue scale) > 40 received controlled-release tramadol as the first (15...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/138920111794295738
更新日期:2011-02-01 00:00:00
abstract:BACKGROUND:Biologic drugs, introduced in clinical practice almost twenty years ago, represent nowadays a prominent treatment option in patients with chronic inflammatory arthritis, such as Rheumatoid Arthritis, Psoriatic Arthritis and Spondyloarthritis, that include ankylosing spondylitis and non-radiographic axial spo...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/1389201019666171226151852
更新日期:2017-01-01 00:00:00
abstract::In recent years, many systems biology approaches have been used with various cancers. The materials described here can be used to build bases to discover novel cancer therapy targets in connection with computer-aided drug design (CADD). A deeper understanding of the mechanisms of cancer will provide more choices and c...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/1389201017666161019160606
更新日期:2016-01-01 00:00:00
abstract::The innate immune system protects against infectious microbes by the recognition of pathogen- associated molecular patterns, which serve to detect pathogens on the host cell surface or in endosomes by pattern recognition receptors such as Toll-like receptors, nucleotide-binding oligomerization domain-containing protei...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920112804583087
更新日期:2018-01-01 00:00:00
abstract::To better identify patients at high risk for cardiovascular events, several markers of risk have been proposed for use in screening. Recently, oxidative stress and inflammation have been evaluated as potential tools for prediction of the risk of cardiovascular events. Among them, we have measured reactive oxygen speci...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920106776597612
更新日期:2006-04-01 00:00:00
abstract:BACKGROUND:Carbon nanotubes (CNTs) have been considered highly successful and proficient in terms of their mechanical, thermal and electrical functionalization and biocompatibility. In regards to their significant extent in bone regeneration, it has been determined that CNTs hold the capability to endure clinical appli...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章
doi:10.2174/1389201018666170127105555
更新日期:2017-01-01 00:00:00
abstract::Tinospora cordifolia (Giloy) is a medicinal plant used in folk and Ayurvedic medicines throughout India since ancient times. All the parts of the plant are immensely useful due to the presence of different compounds of pharmaceutical importance belonging to various groups as alkaloids, diterpenoid lactones, glycosides...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/1389201021666200430114547
更新日期:2020-01-01 00:00:00
abstract::Mammalian cell lines are important host cells for the industrial production of pharmaceutical proteins owing to their capacity for correct folding, assembly and post-translational modification. In particular, Chinese hamster ovary (CHO) cells are the most dependable host cells for the industrial production of therapeu...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920110791111960
更新日期:2010-04-01 00:00:00
abstract:BACKGROUND:Vancomycin is used mostly to overcome infections caused by methicillinresistant microorganisms. There are no well-established administration protocols for neonates and infants, so the leak of a specific administration regime in that population may lead to serum concentrations beyond the specified range. OBJ...
journal_title:Current pharmaceutical biotechnology
pub_type:
doi:10.2174/1389201020666190319161511
更新日期:2019-01-01 00:00:00
abstract::Polyphenols are a widely distributed group of natural products found in fruits, vegetables, nuts, seeds, stems and flowers. Such compounds, especially dietary flavonoids and tannins, have been shown to exert antioxidant, antiinflammatory, anti-cancer and antibacterial effects and may have beneficial effects on human h...
journal_title:Current pharmaceutical biotechnology
pub_type: 杂志文章,评审
doi:10.2174/138920101504140825121142
更新日期:2014-01-01 00:00:00