Uphando lwexesha elizayo kunye novavanyo lweklinikhi lokuqhubela phambili kwi-MS

Umxholo

• Ushwankathelo
• Iindidi zeMS
• Ukuqonda ukuqhubela phambili kwe-MS
• Unyango lwe-PPMS
• I-Ocrevus (Ocrelizumab)
• Uvavanyo lweklinikhi oluqhubekayo lwe-PPMS
• Unyango lwe-stem yeseli ye-NurOwn
• Biotin
• Masitinib
• Ukugqitywa kwezilingo zeklinikhi
• Ibudilast
• Idebenone
• I-Laquinimod
• IFampridine
• Uphando lwePPMS
• Ukuthatha

Ushwankathelo

I-Multiple sclerosis (MS) yimeko engapheliyo yokuzimela. Kwenzeka xa umzimba uqala ukuhlasela iinxalenye zenkqubo ye-nervous system (CNS).

Uninzi lwamayeza kunye nokunyanga kugxile ekubuyiseni i-MS kwaye hayi kwi-MS ephambili ye-MS (PPMS). Nangona kunjalo, iimvavanyo zeklinikhi zihlala zigcinwa ukunceda ukuqonda ngcono i-PPMS kunye nokufumana unyango olutsha, olusebenzayo.

Iindidi zeMS

Ezi ndidi zine ziphambili zeMS zezi:

• Isifo esizimele sodwa (CIS)
• Ukubuyela kwakhona kwi-MS (RRMS)
• Inkqubela phambili ye-MS (PPMS)
• Inkqubela phambili yesibini ye-MS (SPMS)

Ezi ntlobo ze-MS zenzelwe ukunceda abaphandi bezonyango ukuba bahlele abo bathatha inxaxheba kuvavanyo lwezonyango. La maqela avumela abaphandi ukuba bavavanye ukusebenza kunye nokukhuseleka konyango oluthile ngaphandle kokusebenzisa inani elikhulu labathathi-nxaxheba.

Ukuqonda ukuqhubela phambili kwe-MS

Kuphela li-15 leepesenti okanye kubo bonke abantu abafunyenwe bene-MS abane-PPMS. I-PPMS ichaphazela amadoda nabasetyhini ngokulinganayo, ngelixa i-RRMS ixhaphake kakhulu kwabasetyhini kunamadoda.

Uninzi lweentlobo ze-MS zenzeka xa amajoni omzimba ehlasela i-myelin sheath. I-myelin sheath yinto enamafutha, ekhuselayo ejikeleze imithambo-luvo kwintambo yomqolo kunye nengqondo. Xa le nto ihlaselwa, ibangela ukudumba.

I-PPMS ikhokelela ekonakaleni kwemithambo-luvo nakwizicubu ezibomvu kwiindawo ezonakeleyo. Esi sifo siphazamisa inkqubo yokunxibelelana kwemithambo-luvo, sibangela ukungalindeleki kwempawu kunye nokuqhubela phambili kwesifo.

Ngokungafaniyo nabantu abane-RRMS, abantu abanamava e-PPMS baya besiba mandundu ekusebenzeni ngaphandle kokubuyela umva kwangoko okanye uxolelo. Ukongeza ekunyukeni ngokuthe ngcembe kokukhubazeka, abantu abane-PPMS banokufumana ezi mpawu zilandelayo:

• uvakalelo lokuba ndindisholo okanye ukurhawuzelela
• ukudinwa
• ingxaki ngokuhamba okanye ngokulungelelanisa iintshukumo
• imiba ngombono, njengombono kabini
• iingxaki kwimemori nasekufundeni
• ukudumba kwezihlunu okanye ukuqina kwemisipha
• utshintsho kwiimvakalelo

Unyango lwe-PPMS

Ukunyanga i-PPMS kunzima ngakumbi kunonyango lwe-RRMS, kwaye kubandakanya ukusetyenziswa kweyeza lokunyanga umzimba. Olu nyango lunika kuphela uncedo lwethutyana. Zingasetyenziselwa kuphela ngokukhuselekileyo nangokuqhubekekayo iinyanga ezimbalwa ukuya kunyaka ngexesha.

Ngelixa uLawulo lwezoKutya kunye noLawulo lweziyobisi (i-FDA) luvumile amayeza amaninzi e-RRMS, ayizizo zonke ezifanelekileyo iintlobo eziqhubela phambili ze-MS. Amayeza e-RRMS, ekwabizwa ngokuba ziziyobisi eziguqula izifo (ii-DMDs), athathwa ngokuqhubekayo kwaye ahlala eneziphumo ebezingalindelekanga.

Ukuchithwa ngokunyanisekileyo kwezilonda kunye nomonakalo wentsholongwane kunokufumaneka nakubantu abane-PPMS. Izilonda zivutha kakhulu kwaye zinokubangela umonakalo kwi-myelin sheath. Okwangoku akucaci nokuba amayeza anciphisa ukuvuvukala angazicothisa na ii-MS.

I-Ocrevus (Ocrelizumab)

I-FDA yamkele i-Ocrevus (ocrelizumab) njengonyango lwe-RRMS kunye ne-PPMS ngo-Matshi 2017. Ukuza kuthi ga ngoku, kuphela kwesiyobisi esele sivunyiwe yi-FDA ukunyanga i-PPMS.

Uvavanyo lwezonyango lubonakalise ukuba luyakwazi ukucothisa ukuqhubela phambili kweempawu kwi-PPMS ngeepesenti ezingama-25 xa kuthelekiswa ne-placebo.

I-Ocrevus ikwavunyiwe kunyango lwe-RRMS kunye ne "kwangoko" i-PPMS eNgilane. Akukavunywa kwezinye iindawo zase-United Kingdom.

IZiko leLizwe leZempilo eliGqwesileyo (i-NICE) laqala lamlahla u-Ocrevus ngesizathu sokuba indleko yokuyibonelela ingaphezulu kwezibonelelo zayo. Nangona kunjalo, i-NICE, iNkonzo yezeMpilo yeSizwe (i-NHS), kunye nomenzi weziyobisi (iRoche) ekugqibeleni baphinda baxoxa ngexabiso lazo.

Uvavanyo lweklinikhi oluqhubekayo lwe-PPMS

Eyona nto iphambili kubaphandi kukufunda ngakumbi malunga neefom ze-MS. Amachiza amatsha kufuneka aye kuvavanyo olungqongqo lweklinikhi ngaphambi kokuba i-FDA ivume.

Uninzi lwezilingo zeklinikhi zihlala iminyaka emi-2 ukuya kwemi-3. Nangona kunjalo, ngenxa yokuba uphando lilinganiselwe, kwanokuzama ixesha elide kuyafuneka kwi-PPMS. Olunye uvavanyo lweRRMS luyenziwa kuba kulula ukugweba ukusebenza kwamayeza ekubuyeni kwakhona.

Jonga iwebhusayithi yeNational Multiple Sclerosis Society kuludwe olupheleleyo lwezilingo zeklinikhi eUnited States.

Ezi zilingo zikhethiweyo zilandelayo ziyaqhubeka ngoku.

Unyango lwe-stem yeseli ye-NurOwn

I-Brainstorm Cell Therapeutics yenza uvavanyo lweklinikhi yesigaba II ukuphanda ukhuseleko kunye nokusebenza kweeseli ze-NurOwn kunyango lwe-MS eqhubela phambili. Olu nyango lusebenzisa ii-stem stem ezivela kubathathi-nxaxheba abaye bakhuthazwa ukuba bavelise izinto ezithile zokukhula.

Ngo-Novemba ngo-2019, uMbutho weSizwe we-Multiple Sclerosis Society wanikezela ngeBrainstorm Cell Therapeutics isibonelelo sophando esiyi- $ 495,330 yokuxhasa olu nyango.

Ityala kulindeleke ukuba liphele ngoSeptemba 2020.

Biotin

IMedDay Pharmaceuticals SA okwangoku iqhuba inqanaba lesithathu kuvavanyo lweklinikhi ngokusebenza kwethamo eliphezulu le-biotin capsule ekunyangeni abantu abane-MS eqhubela phambili. Ityala likwajolise ekugxileni ngokukodwa kubantu abaneengxaki zokuhamba.

I-Biotin yivitamin ebandakanyekayo ekuphembeleleni ukukhula kwamaselula kunye nemveliso ye-myelin. I-capsule ye-biotin ithelekiswa ne-placebo.

Ityala alisaphindi lithathe abathathi-nxaxheba abatsha, kodwa akulindelwanga ukuba liphele kude kube nguJuni 2023.

Masitinib

Inzululwazi ye-AB iqhuba uvavanyo lweklinikhi yesigaba sesi-3 kwi-masitinib yeziyobisi. I-Masitinib sisiyobisi esivimba impendulo yokuvuvukala. Oku kukhokelela kwimpendulo yomzimba yokuzikhusela esezantsi kunye namanqanaba asezantsi okudumba.

Ityala livavanya ukhuseleko kunye nokusebenza kwe-masitinib xa kuthelekiswa ne-placebo. Iirejimeni ezimbini zonyango lwe-masitinib zithelekiswa ne-placebo: Iirejimeni yokuqala isebenzisa idosi efanayo kulo lonke, ngelixa enye ibandakanya ukunyuka komthamo emva kweenyanga ezintathu.

Ityala alisaphindi lifumane abathathi-nxaxheba abatsha. Kulindeleke ukuba igqitywe ngoSeptemba 2020.

Ukugqitywa kwezilingo zeklinikhi

Ezi zilingo zilandelayo zigqityiwe kutsha nje. Uninzi lwabo, iziphumo zokuqala okanye zokugqibela sele zipapashiwe.

Ibudilast

IMediciNova igqibe inqanaba lesibini lokuvavanywa kweklinikhi kwi-ibudilast yeziyobisi. Injongo yayo yayikukuchonga ukhuseleko kunye nomsebenzi weziyobisi kubantu abane-MS eqhubekayo. Kolu phando, i-ibudilast ithelekiswa ne-placebo.

Iziphumo zokuqala zophando zibonisa ukuba i-ibudilast ikucothisile ukuqhubela phambili kwengqondo ye-atrophy xa kuthelekiswa ne-placebo kwisithuba seeveki ezingama-96. Iziphumo ebezingalindelekanga zixhaphakileyo ezichaziweyo yayizimpawu zesisu.

Nangona iziphumo zithembisa, iimvavanyo ezongezelelweyo ziyafuneka ukubona ukuba iziphumo zolu vavanyo zinokuphinda zenziwe kwakhona kunye nendlela ibudilast enokuthelekiswa ngayo ne-Ocrevus kunye nezinye iziyobisi.

Idebenone

IZiko leLizwe lokuLwa nokwaliwa kunye nezifo ezosulelayo (i-NIAID) kutsha nje ligqibe inqanaba loku-I / II lonyango lokuvavanya isiphumo se-idebenone kubantu abane-PPMS. I-Idebenone yinguqulelo eyenziweyo yecoenzyme Q10. Kukholelwa ukunciphisa umda kwinkqubo yemithambo-luvo.

Ngalo lonke ixesha lokugqibela leminyaka emi-2 yolu vavanyo lweminyaka emi-3, abathathi-nxaxheba bathathe iyeza okanye indawo ye-placebo. Iziphumo zokuqala zibonise ukuba, ngaphezulu kwesifundo, i-idebenone ayibonisanga nzuzo ngaphezulu kwe-placebo.

I-Laquinimod

Amashishini eTev Pharmaceutical axhase isifundo sesigaba II kumzamo wokuseka ubungqina bengcinga yokunyanga i-PPMS ngelaquinimod.

Akuqondwa ngokupheleleyo ukuba isebenza njani i-laquinimod. Kukholelwa ekutshintsheni indlela yokuziphatha kweeseli zomzimba, ke ngoko kuthintela ukonakala kwenkqubo yemithambo-luvo.

Iziphumo zolingo ezidanisayo zikhokelele umenzi wazo, i-Active Biotech, ukuba ayeke uphuhliso lwe-laquinimod njengechiza le-MS.

IFampridine

Kwi-2018, iYunivesithi yaseDublin yagqiba ulingo lwesigaba se-IV ukujonga isiphumo se-fampridine kubantu abanesifo esingasebenziyo somzimba kunye ne-PPMS okanye i-SPMS. I-Fampridine yaziwa ngokuba yi-dalfampridine.

Nangona olu vavanyo lugqityiwe, akukho ziphumo zixeliweyo.

Nangona kunjalo, ngophando lwase-Itali lwango-2019, ichiza linokuphucula isantya sokuqhubekeka kolwazi kubantu abane-MS. Uphengululo lwango-2019 kunye nohlalutyo lweemeta kugqityiwe ukuba kukho ubungqina obuqilileyo bokuba ichiza liphucule amandla abantu abane-MS ukuhamba imigama emifutshane kunye namandla abo okuhamba okubonwayo.

Uphando lwePPMS

I-National Multiple Sclerosis Society ikhuthaza uphando oluqhubekayo kwiindidi eziqhubekayo ze-MS. Injongo kukwenza unyango oluyimpumelelo.

Olunye uphando lujolise kumahluko phakathi kwabantu abane-PPMS kunye nabantu abasempilweni. Uphononongo lwakutsha nje lufumanise ukuba iiseli zeziqu kwingqondo zabantu abane-PPMS zijongeka zindala kuneeseli ezifanayo zabantu abasempilweni abakwiminyaka efanayo.

Ukongeza, abaphandi bafumanise ukuba xa ii-oligodendrocyte, iiseli ezivelisa i-myelin, zivelisiwe kwezi seli zinama-stem, baveza iiproteni ezahlukeneyo kunalezo zisempilweni. Xa le proteni ibivaliwe, ii-oligodendrocyte beziziphatha ngendlela eqhelekileyo. Oku kunokunceda ukucacisa ukuba kutheni i-myelin isesichengeni kubantu abane-PPMS.

Olunye uphando lufumanise ukuba abantu abane-MS abaqhubela phambili babenamanqanaba asezantsi eemolekyuli ezibizwa ngokuba yi-bile acid. Ii-acid zeebile zinemisebenzi emininzi, ngakumbi ekwetyisweni. Kananjalo banesiphumo sokuchasana nokudumba kwezinye iiseli.

Izamkeli ze-bile acid zafunyanwa nakwiseli ezikwiiseli ze-MS. Kucingelwa ukuba ukongezwa nge-bile acid kunokunceda abantu abane-MS eqhubekayo. Ngapha koko, ulingo lweklinikhi lokuvavanya kanye oku kuyaqhubeka ngoku.

Ukuthatha

Izibhedlele, iiyunivesithi kunye neminye imibutho e-United States isebenza ngokuqhubekayo ukuze ifunde okungakumbi malunga ne-PPMS kunye ne-MS ngokubanzi.

Ukuza kuthi ga ngoku lichiza elinye kuphela, i-Ocrevus, evunyiweyo yi-FDA kunyango lwe-PPMS. Ngelixa i-Ocrevus icothisa ukuqhubela phambili kwe-PPMS, ayiyeki inkqubela phambili.

Ezinye iziyobisi, ezinje nge-ibudilast, zibonakala zithembisa ngokusekwe kuvavanyo lwakwangoko. Ezinye iziyobisi, ezifana ne-idebenone kunye ne-laquinimod, aziboniswanga ukuba ziyasebenza.

Izilingo ezongezelelweyo ziyafuneka ukuchonga unyango olongezelelekileyo lwe-PPMS. Buza umboneleli wakho wezempilo malunga nolingo lwamva nje lwezonyango kunye nophando olunokukunceda.