Drug Delivery Systems for Metabolic Disorders

527 Pages·2022·13.763 MB·English

by  Harish Dureja, Narasimha Murthy, Peter Wich, Kamal Dua

Drug Delivery Systems for Metabolic Disorders
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Drug Delivery Systems for
Metabolic Disorders
Edited by
Harish Dureja
DepartmentofPharmaceuticalSciences,MaharshiDayanandUniversity,Rohtak,
Haryana,India
S. Narasimha Murthy
PharmaceuticsandDrugDelivery,UniversityofMississippi,Mississippi,UnitedStates
Peter R. Wich
Bioorganic&MacromolecularChemistry,SchoolofChemicalEngineering,
UniversityofNewSouthWales,UNSWSydney,Australia
Kamal Dua
DisciplineofPharmacy,GraduateSchoolofHealth,UniversityofTechnologySydney,
Ultimo,NSW,Australia
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Contents
Listofcontributors xv 1.4.7 Hypoparathyroidism 12
Abouttheeditors xix 1.4.8 Hyperparathyroidism 13
1.4.9 Pseudohypoparathyroidism 13
1. Introduction to metabolic disorders 1 1.4.10 Hyperthyroidism 14
1.4.11 Hypothyroidism 14
FoziyahZakir,SradhanjaliMohapatra,
1.5 Metabolicdisorderduetoderegulationin
UzmaFarooq,Mohd.AamirMirzaand
lysosomalstoragedisorders 14
ZeenatIqbal
1.5.1 Schindlerdisease 14
1.1 Metabolicdisordersduetoderegulation 1.5.2 Faberdisease 15
ofprotein/aminoacidmetabolism 2 1.5.3 Sandhoffdiseases 15
1.1.1 Proteinmetabolismdisorders 2 1.5.4 Pycnodysostosis 16
1.1.2 Aminoacidmetabolismdisorders 4 1.6 Metabolicdisorderduetoderegulationin
1.2 Metabolicdisordersduetoderegulation mitochondrialdisorders 16
oflipidmetabolism 5 1.6.1 Wilsondisease 16
1.2.1 Atherosclerosis 5 1.6.2 Pearsonmarrowsyndrome 16
1.2.2 Cerebrotendinousxanthomatosis 5
1.6.3 Leighsyndrome 16
1.2.3 Sitosterolemia 6 1.6.4 Alpersdisease 17
1.2.4 Gaucherdisease 6
1.6.5 Battendisease 17
1.2.5 Tay-Sachsdisease 6 References 17
1.2.6 Niemannpickdisease 6 Furtherreading 20
1.2.7 Letterer-Siwedisease 7
1.3 Metabolicdisorderduetoderegulation
incarbohydratemetabolism 7
2. Cellular and molecular mechanisms
1.3.1 Galactosemia 8
involved in metabolic disorders 21
1.3.2 Hereditaryfructoseintolerance 8
1.3.3 Fructose1,6-diphosphatasedeficiency 8 LovedeepSinghandRajbirBhatti
1.3.4 Glycogenstoragedisorders 8
2.1 Introduction 21
1.3.5 HurlerandScheiesyndrome 8
2.2 Alterationsinsingle-mindedprotein-1:
1.3.6 Huntersyndrome 9
downstreammediatorof
1.3.7 Sanfilipposyndrome 9
leptin-melanocortinpathway 21
1.4 Metabolicdisorderduetodisturbancein
2.3 Linkbetweenosteocalcindeficiency
hormonemetabolism 10
andinsulinresistance 22
1.4.1 Hypoglycemia 10
2.4 AberrationsintheJAK-STATpathway 22
1.4.2 Diabetes(hyperglycemia) 10
2.5 AlterationinAMPKactivity:
1.4.3 Hypopituitarismand insulinresistanceandcardiovascular
hyperpituitarism 11 problems 23
1.4.4 Sheehansyndrome 11 2.6 Conclusion 26
1.4.5 Pituitaryapoplexy 12 Acknowledgments 26
1.4.6 Hyperpituitarism 12 References 26
v
vi Contents
3. Current practices in drug delivery for 5.4 Symptomsanddiagnosis 68
metabolic disorders 31 5.4.1 5-ALA-dehydratase-deficient
porphyria 68
PoojaJain,ThomsonSantoshAlex, 5.4.2 Acuteintermittentporphyria 68
ManviSingh,NaziaHassan,Mohd.AamirMirza
5.4.3 Porphyriacutaneatardaand
andZeenatIqbal
hepatoerythropoieticporphyria 68
3.1 Introduction 31 5.4.4 Hereditarycoproporphyria 69
3.2 Oraldrugdeliveryapproaches 32 5.4.5 Variegateporphyria 69
3.3 Site-specificdrugdeliveryapproaches 34 5.4.6 Congenitalerythropoieticporphyria 69
3.4 Targeteddrugdelivery 38 5.4.7 Erythropoieticprotoporphyriaand
3.5 Intelligentdrugdelivery 39 X-linkedprotoporphyria 69
3.6 Conclusion 41 5.5 Treatmentstrategies 70
Acknowledgments 41
5.5.1 Acuteintermittentporphyria 70
References 41
5.5.2 5-ALA-dehydratase-deficient
porphyria 70
5.5.3 Porphyriacutaneatardaand
4. Recent developments in the
hepatoerythropoieticporphyria 70
treatment of amyloidosis 47
5.5.4 Hereditarycoproporphyria 70
NabamitaHalder,SushmaTalegaonkarand 5.5.5 Variegateporphyria 70
MonalisaMishra 5.5.6 Congenitalerythropoieticporphyria 70
5.5.7 Erythropoieticprotoporphyriaand
4.1 Introduction 47
X-linkedprotoporphyria 70
4.2 Thehistoryofamyloidosis 47
5.6 Overviewoftreatmentdrugsandcurrent
4.3 Amyloidfibril 48
treatmentproblems 71
4.3.1 Amyloidfibrillogenesis 49
5.6.1 Intravenoushemin(panhematin) 71
4.3.2 Pathogenicityofamyloidfibril 51
5.6.2 Chloroquineandhydroxychloroquine 71
4.3.3 Classificationofamyloidosis 51
5.6.3 Afamelanotide 72
4.3.4 Hereditarysystemicamyloidosis 55
5.6.4 Beta-carotene 72
4.3.5 Methodsusedfordiagnosisof
5.6.5 Givosiran(GIVLAARI) 72
amyloidosis 56
5.7 Noveltherapies 72
4.3.6 Treatmentofamyloidosis 58
5.8 Summaryandconclusion 74
4.4 Conclusion 59
References 76
Acknowledgments 59
References 59
6. Advanced drug delivery systems
5. Advanced drug delivery systems targeting kwashiorkor involving a
targeting to improve therapeutic disturbance in protein metabolism 77
outcomes in porphyria 65
PoojaMittal,RamitKapoorand
VindhyaBasetty,JackDeruiter,SuhrudPathak, BrahmeshwarMishra
KamalDuaandMuralikrishnanDhanasekaran
6.1 Introduction 77
5.1 Introduction 65 6.2 Physiologicalchangesassociatedwith
5.2 Prevalence 65 kwashiorkordisease 78
5.3 Pathophysiology 65 6.3 Biochemicalmanifestationsand
5.3.1 5-ALA-dehydratase-deficient metabolicdisordersassociatedwith
porphyria 65 kwashiorkordisease 78
6.4 Signs,symptoms,andclinical
5.3.2 Acuteintermittentporphyria 66
manifestationsinkwashiorkor 79
5.3.3 Congenitalerythropoieticporphyria 66
6.5 Currenttreatmentofkwashiorkor 80
5.3.4 Porphyriacutaneatardaand
6.6 Metabolicdisturbancesassociatedwith
hepatoerythropoieticporphyria 66
kwashiorkor 80
5.3.5 Hereditarycoproporphyria 66
6.7 Roleofnanotechnologyindrug-delivery 81
5.3.6 Variegateporphyria 68 6.8 Differenttypesofdrugdeliveryvehicles 82
5.3.7 Erythropoieticprotoporphyria 68 6.9 Advanceddrugdeliverysystemtargeting
5.3.8 X-linkedprotoporphyria 68 kwashiorkor 82
Contents vii
6.10 Conclusion 83 8.5.5 GRASstatusofthematerial 130
References 83 8.5.6 Regulatoryclearance 130
8.5.7 Nanoparticles 130
7. Advanced drug delivery systems 8.5.8 Liposomes 130
targeting metabolic disorders: 8.5.9 Dendrimer 130
erythropoietic protoporphyria 85 8.6 Conclusion 130
References 131
NitinVerma,KomalThapa,NehaKanojia,
HarishDurejaandKamalDua 9. Advanced drug delivery systems
in the management of Gaucher
7.1 Introduction 85
7.2 Etiology 85 disease 137
7.3 Clinicalfeatures 86
GufranAjmalandBrahmeshwarMishra
7.3.1 Dermatologicinvolvement 86
7.3.2 Hematologicalinvolvement 87 9.1 History 137
7.4 Diagnosis 87 9.2 Epidemiology 137
7.5 Pathophysiology 87 9.3 Etiology 137
7.6 Genotype-phenotypecorrelations 88 9.4 Pathogenesis 138
7.7 Currentandemergingtherapies 88 9.5 ClinicalclassificationofGaucherdisease 139
7.7.1 Modulationofironsupply 88 9.6 ManagementofGaucherdisease 140
7.7.2 Antisenseoligonucleotidetherapy 89 9.6.1 Baselinemanagement 140
7.7.3 Increasedlighttolerancewith 9.6.2 Disease-specificmanagement 140
antioxidantsorstimulatorsof 9.6.3 Otherspecifictreatments 143
skinmelaninsynthesis 89 9.7 Conclusion 145
7.7.4 Surgery 89 References 146
7.7.5 Consultations 90
7.8 Conclusionandfutureperspective 91 10. Advanced drug delivery systems
References 91 focusing the metabolic disorders
due to deregulation of lipid
8. Combating atherosclerosis with
metabolism: Niemann Pick
nanodrug delivery approaches: from
Disease and Letterer-Siwe disease 149
bench side to commercialization 97
NimishaSrivastavaandRishabhGupta
SubhNaman,SrishtiNaryal,RishiPalliwal,
ShivaniRaiPaliwalandAshishBaldi 10.1 Introduction 149
10.1.1 Lipidmetabolicdisorder 149
8.1 Introduction 97
10.2 Neimann’sPickdisease 149
8.2 Atherosclerosis 98
10.2.1 Commonsymptomsof
8.2.1 Etiology 98
Neimann’sdisease 150
8.2.2 Pathogenesis 98
10.3 Etiology 150
8.2.3 Clinicaleffects 100
10.3.1 Themolecularbiology
8.2.4 Pharma-armamentariumand
underlyingNP-C
mechanisticinsights 100
pathophysiology 151
8.3 Nanoformulationsforthetreatmentof
10.3.2 LipidtraffickingandNPC1:
atherosclerosiswithimplementationof
cholesteroltransport 151
qualitybydesign 104
10.3.3 NPCproteinfunction:NPC1&
8.3.1 Nanoparticles 104
NPC2 152
8.3.2 Liposomes 111
8.4 Dendrimers 120 10.3.4 Diagnosis 152
8.4.1 ImplementationofconceptQbD 10.3.5 Historytakingandclinical
duringtheformulationofdendrimer 122 examination 152
8.5 Challengesintranslatingnanodrug 10.3.6 Laboratoryfindings 152
deliverysystemsfrombenchtobedside 128 10.3.7 Genetictesting 152
8.5.1 Scalability 128 10.3.8 Pathophysiologyof
8.5.2 Safety 128 sphingomyelinasedeficiency
8.5.3 Processoptimization 129 Niemann’spickdisease 153
8.5.4 Stabilityoftheproduct 129 10.3.9 Niemann’sPickdiseasetypeA 153
viii Contents
10.3.10 Niemann’sPickdiseasetypeB 153 12. Novel 3D printing drug delivery
10.3.11 Niemann’sPickdiseasetypeC 153 system for the prevention and
10.3.12 Regularlaboratorytesting 153 treatment of Atherosclerosis 177
10.3.13 Diagnosticbiomarkers 153
10.3.14 Plasmaoxysterols 153 RachelParise,R.JayachandraBabu,JackDeruiter,
10.3.15 Plasmalysosphingolipids 154 KamalDuaandMuralikrishnanDhanasekaran
10.3.16 ManagementofNPDs 154
12.1 Importanceof3Dprintingdrug
10.3.17 Letterer-Siwedisease 154 deliverysystems 177
10.3.18 Signandsymptoms 154 12.2 Pathophysiologyofatherosclerosis 177
10.3.19 PathophysiologyofLetterer 12.3 Drugsusedforthepreventionand
Siwedisease 155 treatmentofatherosclerosis 179
10.4 Stage1:earlylesion’sphase 155 12.4 Problemswithcurrentoraldrug
10.5 Stage2:fibrousphase 155 delivery 185
10.6 Stage3:extralesionphase 155 12.5 Customized3Dprinteddosageformsfor
10.7 DiagnosisLettererSiwedisease 155 atherosclerosisandassociateddiseases 186
10.7.1 Physicaldiagnosis 155 12.6 Conclusion 187
10.7.2 Laboratorydiagnosis 155 References 187
10.7.3 Epidemiology 155
10.7.4 Treatment 156 13. Advanced drug delivery systems to
10.7.5 Noveldrugdeliverysystems treat Huntington’s disease:
fortreatmentofNiemann’s challenges and opportunities 189
PickdiseaseandLetterer-Siwe
SukritiVishwas,MonicaGulati,Rubiya
disease 156
Khursheed,K.R.Arya,SachinKumarSingh,
10.7.6 Resealederythrocytes 156
NirajKumarJha,ParteekPrasher,
10.7.7 Liposomes 156
DeepakKumarandVijayKumar
10.7.8 Microcapsules 156
10.7.9 Nanocarriers 156 13.1 Introduction 189
10.8 Prospects 156 13.2 Globalprevalenceandeconomic
10.9 Conclusion 159 burden 190
References 159 13.3 Pathophysiology 190
13.3.1 Geneticfactors 190
11. Genetic disease and Niemann-Pick
13.3.2 Mitochondrialdysfunction 190
disorders: novel treatments and 13.3.3 Oxidativestress 190
drug delivery systems 161 13.3.4 Neurodegeneration 190
13.4 Pharmacologicalmanagementof
JosephLawson,ErinHarrell,JackDeruiter, Huntington’sdisease 191
SuhrudPathak,SatyanarayanaPondugulaR, 13.5 Herbaltherapy 191
KamalDuaandMuralikrishnanDhanasekaran 13.6 Challengesassociatedwithconventional
therapyandtheneedforadvanceddrug
11.1 Introductiontogeneticdisorders 161
deliverysystems 191
11.2 Classificationofinheritedmetabolic
13.6.1 Nanoemulsions 195
disorders 163
11.3 Physiologyoflipidmetabolismand 13.6.2 Self-emulsifyingdrugdelivery
diseasesassociatedwithlipid system 195
metabolism 163 13.6.3 Liposomes 199
11.4 EssentialaspectsofNiemann-Pick 13.6.4 Polymericmicelles 200
disease:prevalence,pathophysiology, 13.6.5 Nanostructuredlipidcarriers 201
diagnosis,andsymptoms 167 13.6.6 Solidlipidnanoparticles 201
11.5 Currenttherapeuticstrategiestotreat 13.7 Conclusionandperspectives 201
Niemann-Pickdisease 171 References 202
11.6 Problemsassociatedwiththecurrent
therapeuticstrategiestotreat
14. Advanced drug delivery systems
Niemann-Pickdisease 172
for targeting obesity 207
11.7 Possiblenoveldrugdeliverysystemsto
improvetherapeuticefficacyin
NishaGulati,KamalDuaandHarishDureja
Niemann-Pickdisease 172
References 174 14.1 Introduction 207
Contents ix
14.2 Reasonforweightgain 207 16.1.2 Galactoseandfructose
14.3 Obesitymanagement 208 metabolismdisorders 233
14.4 Conventionalanti-obesitytreatments 209 16.1.3 Glycogenstoragedisorders 234
14.5 Advanceddrugdeliverysystems 209 16.1.4 Glucosetransportdisorder 234
14.5.1 Nanoparticle 209 16.1.5 Congenitaldisordersof
14.5.2 Metallicnanoparticles 209 glycosylation 234
14.5.3 Solidlipidnanoparticles 211 16.1.6 Glucosemetabolismdisorders 235
14.5.4 Liposomes 211 16.2 Conventionaltreatmentforglucose
14.5.5 Microneedles 211 metabolismdisorders 235
14.5.6 Nanoemulsion 212 16.2.1 Insulintherapies 235
14.5.7 Gene-basedtherapy 212 16.2.2 Herbaltreatmentfordiabetes
14.5.8 Interleukins 212 mellitus 238
14.6 Conclusionandfutureprospectus 212 16.3 Alternativeapproachestodiabetes 240
References 213 16.3.1 Dietaryfibers 240
16.3.2 Mineralsusedinthetreatment
ofhyperglycemia 241
15. Advanced drug delivery systems 16.4 Nanomaterialsfortreatmentof
for treatment of diabetes mellitus 217 diabetes 242
16.4.1 Oraladministration 242
SakshiPriya,AnchalTyagi,MahaveerSingh,
16.4.2 Inhalations 243
SushamaRawat,AbhayRaizaday,
16.4.3 Nano-pumps 243
SantoshKumarSinghandGauravGupta
16.4.4 Artificialpancreas 243
15.1 Introduction 217 16.5 Advancesininsulindelivery 244
15.2 Antidiabeticmedication-related 16.6 Conclusionandfutureperspectives 245
complications 218 References 245
15.3 Possibilitiesprovidedbythevarious
Noveldrugdeliverysystems 218
17. Emerging drug delivery in the
15.4 Developmentanddesigningofvarious
advanceddrugdeliverysystemsfor treatment of hyperpituitarism and
diabetestreatment 219 hypopituitarism 249
15.4.1 Particulatetransportsystem 220
VimalArora,AtulKabra,RitikaPuri,
15.4.2 Microparticulatesystems 220
GovindVyas,KamalDuaandHarishDureja
15.4.3 Nanoparticulatesystems 221
15.4.4 Vesicularsystem 222 17.1 Introduction 249
15.4.5 Transdermaldrugdelivery 17.1.1 Pituitarygland(hypophysis) 249
systems 223 17.1.2 Hormonesofpituitarygland 249
15.4.6 Transdermaldeliverysystems 17.1.3 Humangrowthhormoneor
incorporatedwithbiosensor 224 somatotropin 249
15.4.7 Pointofcaretherapy 225 17.1.4 Thyroid-stimulatinghormone
15.4.8 Employmentoftechnologiesfor orthyrotropin 250
carrier,entrapment,penetration, 17.1.5 Prolactinormamotrophin
andrelease 226 hormoneorluteotrophic
15.5 Prospectsandconclusion 228 hormone 250
References 229
17.1.6 Follicle-stimulatinghormone 251
17.1.7 Luteinizinghormone 251
17.1.8 Adrenocorticotropichormone 251
16. Advanced drug delivery system in
17.1.9 Melanocyte-stimulating
the treatment of hyperglycemia and
hormone 251
hypoglycemia 233 17.1.10 Antiduretichormoneor
vasopressinorpitressin 251
VimalArora,AtulKabra,RamBabuSharma,
17.1.11 Oxytocin(OT,Pitocin) 251
HarishDurejaandKamalDua
17.2 Treatmentofhypopituitarismand
16.1 Introduction 233 hyperpituitarism 252
16.1.1 Carbohydratemetabolism 17.2.1 Treatmentofhypopituitarism 252
disorders 233 17.2.2 Treatmentofhyperpituitarism 253