Feasibility Study for Bedside Production of Recombinant Human Acid α-Glucosidase: Technical and Financial Considerations.

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 Biotechnol

authors

Aldosari MH,den Hartog M,Ganizada H,Evers MJW,Mastrobattista E,Schellekens H

doi

10.2174/1389201021666200217113049

subject

Has Abstract

pub_date

2020-01-01 00:00:00

pages

467-479

issue

6

eissn

1389-2010

issn

1873-4316

pii

CPB-EPUB-104561

journal_volume

21

pub_type

杂志文章
  • New developments of clinical trial in immunotherapy for Alzheimer's disease.

    abstract::Active or passive immunotherapy is expected to slow or stop the pathological process of Alzheimer's disease (AD). Immunotherapy for AD has demonstrated that targeting beta-amyloid (Aβ) or tau protein with vaccines or antibodies can reduce AD pathologies. Active anti-Aβ immunization for AD includes using AN1792 and sec...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/138920101606150407112319

    authors: Yang C,Xiao S

    更新日期:2015-01-01 00:00:00

  • General principles of photodynamic therapy (PDT) and gastrointestinal applications.

    abstract::The purpose of this review article is to describe the history of photodynamic therapy (PDT), its current medical applications, the mechanism of action, contraindications of the method, and different types of photosensitizers used. The second part of the article deals with applications for gastrointestinal diseases. Th...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/1389201043376805

    authors: Wiedmann MW,Caca K

    更新日期:2004-08-01 00:00:00

  • Synthesis, Self-assembly, and in vitro Biocompatibility of a Novel Steroidal Oxime.

    abstract::A novel steroidal oxime (SO) was synthesized using an economic method and then characterized by Fourier transform infrared (FTIR) and 1H nuclear magnetic resonance (NMR). When dispersed in pure water, SO can self-assemble into a nano-scale circular structure which was verified by dynamic light scattering (DLS) and tra...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:10.2174/1389201016666150817094116

    authors: Chou TH,Chen CW

    更新日期:2015-01-01 00:00:00

  • Wound Healing Effect of Curcumin: A Review.

    abstract::Wound healing is a complex process that consists of several phases that range from coagulation, inflammation, accumulation of radical substances, to proliferation, formation of fibrous tissues and collagen, contraction of wound with formation of granulation tissue and scar. Since antiquity, vegetable substances have b...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:

    authors: Tejada S,Manayi A,Daglia M,Nabavi SF,Sureda A,Hajheydari Z,Gortzi O,Pazoki-Toroudi H,Nabavi SM

    更新日期:2016-07-21 00:00:00

  • A Study on Ecotoxicological Effects of Nano-copper Oxide Particles to Portunus Trituberculatus.

    abstract::As an important nano-material, nano-copper oxide particles (CuO-ENPs) harbor a vast range of characteristics, including electronic correlation effect, thermal stability, catalytic activity, sterilization and other properties. At present, the mechanism of ecotoxicological effects of CuO-ENPs is not yet clear, and has b...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:10.2174/1389201021666201229111251

    authors: Li T,Hu H,Yang C,Zhang B,Ma L

    更新日期:2020-12-28 00:00:00

  • Some peculiar effects of NO-synthase inhibition on the structure and function of cardiovascular system.

    abstract::Long-term increase of blood pressure represents one of the most important risk factors triggering many cardiovascular diseases, and via counter-regulatory mechanisms it is itself modulated by them. Adequate perfusion of the respective areas with nutrients requires appropriate production of vasodilatory and vasoconstri...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/138920111798281072

    authors: Kristek F

    更新日期:2011-09-01 00:00:00

  • Tinospora cordifolia (Giloy): Phytochemistry, Ethnopharmacology, Clinical Application and Conservation Strategies.

    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

    authors: Kumar P,Kamle M,Mahato DK,Bora H,Sharma B,Rasane P,Bajpai VK

    更新日期:2020-01-01 00:00:00

  • Intra-uterine Growth Retardation as a Risk Factor of Postnatal Metabolic Disorders.

    abstract::Intra-uterine growth retardation (IUGR) represents one of the major problems in perinatal medicine. IUGR is one of main causes of perinatal mortality and morbidity. A huge number and variety of established and possible causes of IUGR have been described. There are currently no data about effective treatment of this fe...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/1389201017666160301104323

    authors: Mierzynski R,Dluski D,Darmochwal-Kolarz D,Poniedziałek-Czajkowska E,Leszczynska-Gorzelak B,Kimber-Trojnar Z,Agnieszka-Wankowicz,Oleszczuk J

    更新日期:2016-01-01 00:00:00

  • Adulteration Practices of Psychoactive Illicit Drugs: An Updated Review.

    abstract:BACKGROUND:Powdery drugs such as cocaine and heroin are frequently adulterated or diluted predominantly to obtain more doses and to increase the drug dealer's profits, but also to enhance, to modify or to oppose drug effects. The aim of this report is to provide an overview of the recent scientific literature on medici...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/1389201018666170710184531

    authors: Solimini R,Rotolo MC,Pellegrini M,Minutillo A,Pacifici R,Busardò FP,Zaami S

    更新日期:2017-01-01 00:00:00

  • Gallic Acid Protects from Acute Multiorgan Injury Induced by Lipopolysaccharide and D-galactosamine.

    abstract:BACKGROUND:Secondary metabolites of plants, the polyphenols, play a vital role in protection from many health problems in human beings. Structurally favored phytochemicals may be studied to protect multiorgan injury. At pharmacological doses, gallic acid is nontoxic to mammals and is generally absorbed in the intestine...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:10.2174/1389201021666200615165732

    authors: Rakshit S,Nirala SK,Bhadauria M

    更新日期:2020-01-01 00:00:00

  • Vancomycin Therapeutic Regime Adjustment in Newborns and Infants with Bacterial Infection: Case Series.

    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

    authors: Bidu NS,Fernandes BJD,Dias EJC,Filho JNC,Bastos REA,Godoy ALPC,Azeredo FJ,Pedreira JNR,Couto RD

    更新日期:2019-01-01 00:00:00

  • Characterization of protein higher order structure using vibrational circular dichroism spectroscopy.

    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

    authors: Nagarkar RP,Murphy BM,Yu X,Manning MC,Al-Azzam WA

    更新日期:2013-01-01 00:00:00

  • Molecular, Cellular and Pharmaceutical Aspects of Synthetic Hydroxyapatite Bone Substitutes for Oral and Maxillofacial Grafting.

    abstract::Bone grafts are widely used for augmentation procedures in oral and maxillofacial surgery, with autogenous bone being the gold standard. Recently, the focus of research has shifted towards synthetic bone substitutes, as no second surgery is needed and large quantities of graft can easily be provided. Within the broad ...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/1389201017666161202103218

    authors: Gotz W,Papageorgiou SN

    更新日期:2017-01-01 00:00:00

  • The Effect of a Common Antibiotics Doxycycline on Non-Healing Chronic Wound.

    abstract:BACKGROUND:Up to 25% of diabetic patients will develop a diabetic foot ulcer. Chronic wounds such as diabetic foot ulcers often fail to heal with conventional therapies. In recent years, it has been identified that chronic wounds are usually associated with elevated level of matrix metalloproteinases (MMPs). Doxycyclin...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/1389201018666170519095339

    authors: Xu DH,Zhu Z,Fang Y

    更新日期:2017-01-01 00:00:00

  • Natural Products Mediated Regulation of Oxidative Stress and DNA Damage in Ultraviolet Exposed Skin Cells.

    abstract::Data obtained through high-throughput technologies have gradually revealed that a unique stratified epithelial architecture of human skin along with the antioxidant-response pathways provided vital defensive mechanisms against UV radiation. However, it is noteworthy that skin is a major target for toxic insult by UV r...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:10.2174/1389201016666150731111308

    authors: Farooqi AA,Li RN,Huang HW,Ismail M,Yuan SS,Wang HM,Liu JR,Tang JY,Chang HW

    更新日期:2015-01-01 00:00:00

  • Counting and behavior of an individual fluorescent molecule without hydrodynamic flow, immobilization, or photon count statistics.

    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

    authors: Földes-Papp Z,Baumann G,Demel U,Tilz GP

    更新日期:2004-04-01 00:00:00

  • Structures of TGF-β receptor complexes: implications for function and therapeutic intervention using ligand traps.

    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

    authors: Hinck AP,O'Connor-McCourt MD

    更新日期:2011-12-01 00:00:00

  • Synergistic Effect of Tazobactam on Amikacin MIC in Acinetobacter baumannii Isolated from Burn Patients in Tehran, Iran.

    abstract:BACKGROUND:Burn is still an important global public health challenge. Wound colonization of antibiotic resistant bacteria such as Acinetobacter baumannii can lead to high morbidity and mortality in burn patients. The aim of this study was to evaluate the inhibitory effect of tazobactam on efflux pump, which can cause a...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:10.2174/1389201021666200220121122

    authors: Azimi L,Tahbaz SV,Alaghehbandan R,Alinejad F,Lari AR

    更新日期:2020-01-01 00:00:00

  • Green and Simple Synthesis of Silver Nanoparticles by Aqueous Extract of Perovskia abrotanoides: Characterization, Optimization and Antimicrobial Activity.

    abstract:BACKGROUND:Herein, we report the biosynthesis procedure to prepare silver nanoparticles as reduction and capping agents with the aqueous plant extract of Perovskia abrotanoides. METHODS:The therapeutic application of silver nanoparticles entirely depends on the size and shape of the nanoparticles therefore, their cont...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:10.2174/1389201020666190618121218

    authors: Mirsadeghi S,Koudehi MF,Rajabi HR,Pourmortazavi SM

    更新日期:2020-01-01 00:00:00

  • Effect of oil extract from microalgae (Schizochytrium sp.) on the viability and apoptosis of human osteosarcoma cells.

    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

    authors: Sahin D

    更新日期:2020-09-27 00:00:00

  • Immunomodulatory drugs (IMiDs): a new treatment option for myelodysplastic syndromes.

    abstract::The IMiDs represent a new proprietary class of thalidomide analogues that possess greater potency and less toxicity than the parent compound. As a group, these agents share the pharmacologic property of modulating cellular response to ligand activation, the precise biologic effect of which is cell lineage and stimulan...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/138920106778521587

    authors: Kale V,List AF

    更新日期:2006-10-01 00:00:00

  • Biological Aspects and Clinical Applications of Mesenchymal Stem Cells: Key Features You Need to be Aware of.

    abstract::Mesenchymal stem cells (MSCs), a form of adult stem cells, are known to have a self-renewing property and the potential to specialize into a multitude of cells and tissues such as adipocytes, cartilage cells, and fibroblasts. MSCs can migrate and home to the desired target zone where inflammation is present. The uniqu...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:10.2174/1389201021666200907121530

    authors: Saeedi M,Nezhad MS,Mehranfar F,Golpour M,Esakandari MA,Rashmeie Z,Ghorbani M,Nasimi F,Hoseinian SN

    更新日期:2020-09-07 00:00:00

  • Advances in biotechnology for tissue engineering of bone.

    abstract::Tissue engineering is a rapidly developing field applying the disciplines of cell biology, developmental biology, molecular biology and biomimetic engineering to regenerate new tissues for replacement therapies in clinical contexts. To aid in the elicitation and reiteration of the processes of morphogenesis of tissues...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/1389201003379031

    authors: Ripamonti U,Tasker JR

    更新日期:2000-07-01 00:00:00

  • Food with Influence in the Sexual and Reproductive Health.

    abstract:BACKGROUND:Sexual dysfunction and infertility are conditions with high prevalence in the general population. Nutritional factors have been reported to have an impact on sexual and reproductive health. OBJECTIVE:The aim of this review is to summarize the data on nutritional factors that have influence on male and femal...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/1389201019666180925140400

    authors: Silva T,Jesus M,Cagigal C,Silva C

    更新日期:2019-01-01 00:00:00

  • The Role of the Coagulase-negative Staphylococci (CoNS) in Infective Endocarditis; A Narrative Review from 2000 to 2020.

    abstract::Coagulase-negative staphylococci (CoNS) are part of the microbiota of human skin and rarely linked with soft tissue infections. In recent years, CoNS species considered as one of the major nosocomial pathogens and can cause several infections such as catheter-acquired sepsis, skin infection, urinary tract infection, e...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/1389201021666200423110359

    authors: Noshak MA,Rezaee MA,Hasani A,Mirzaii M

    更新日期:2020-01-01 00:00:00

  • Nanosuspension as an Efficient Carrier for Improved Ocular Permeation of Voriconazole.

    abstract:BACKGROUND:The present limitations related to the ocular administration of antifungal drugs for the treatment of fungal keratitis include poor ocular bioavailability, limited retention time, and low ocular tissues penetration. METHODS:This study aimed to prepare a novel ophthalmic voriconazole-loaded nanosuspension ba...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:10.2174/1389201021999200820154918

    authors: Qin T,Dai Z,Xu X,Zhang Z,You X,Sun H,Liu M,Zhu H

    更新日期:2020-08-20 00:00:00

  • Cardiac oxidative stress and inflammation are similar in SAMP8 and SAMR1 mice and unaltered by curcumin and Ginkgo biloba extract intake.

    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

    authors: Schiborr C,Eckert GP,Weissenberger J,Müller WE,Schwamm D,Grune T,Rimbach G,Frank J

    更新日期:2010-12-01 00:00:00

  • Imaging adoptive cell transfer based cancer immunotherapy.

    abstract::The prospect of mobilizing the power of the immune system in the fight against cancer has fascinated generations of immunologists. After decades of sporadic successes scattered amongst frequent failures, the field of tumor immunology is now poised to take advantage of a greatly improved understanding of the molecular ...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/138920110792246528

    authors: Rabinovich BA,Radu CG

    更新日期:2010-09-01 00:00:00

  • Mechanisms underlying the hepatotoxic effects of ecstasy.

    abstract::3,4-Methylenedioxymethamphetamine (MDMA or ecstasy) is a worldwide illegally used amphetamine-derived designer drug known to be hepatotoxic to humans. Jaundice, hepatomegaly, centrilobular necrosis, hepatitis and fibrosis represent some of the adverse effects caused by MDMA in the liver. Although there is irrefutable ...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章,评审

    doi:10.2174/138920110791591535

    authors: Carvalho M,Pontes H,Remião F,Bastos ML,Carvalho F

    更新日期:2010-08-01 00:00:00

  • Therapeutic potential of non-coding RNAs and TLR signalling pathways in Rheumatoid arthritis.

    abstract:BACKGROUND:Rheumatoid arthritis (RA) is a common connective tissue disease, characteristic of chronic and invasive synovitis in single or multiple joints and vasculitis. RA is a heterogeneous disease with unclear pathogenesis. Therefore, exploring the etiology and pathogenesis of the disease is essential for identifyin...

    journal_title:Current pharmaceutical biotechnology

    pub_type: 杂志文章

    doi:10.2174/1389201021666201001142829

    authors: Liu J,Wang X,Wang S,Liu F

    更新日期:2020-10-01 00:00:00