Tsallake zuwa content

ADSTEM (2018)

Fahimtar yadda allogeneic mesenchymal stromal sel zasu iya canza tsananin cutar a cikin RDEB (ADSTEM).

Game da kudaden mu

Jagoran Bincike Farfesa John McGrath, Farfesa na Kwayoyin cututtukan fata da kuma mai ba da shawara ga likitan fata
Institution St John's Institute of Dermatology, Division of Genetics and Molecular Medicine
Nau'in EB RDEB
Hanyar haƙuri Mutane 10 da ke da RDEB a gwajin asibiti
Adadin kuɗi £432, 496 - An Kammala (Cure EB Ya Ba da Kuɗi - wanda aka fi sani da Asusun Bincike na Sohana)

 

Bayanan aikin

Game da Mesenchymal Stem Cells

Mesenchymal stromal Kwayoyin (MSC) kwayoyin halitta ne masu yawa masu iya girma zuwa nau'ikan tantanin halitta iri-iri, amma kuma suna da abubuwan hana kumburi. Jiko na MSCs na cikin jijiya, (gabatar da sel kai tsaye cikin magudanar jinin mutum) ya nuna yana amfanar marasa lafiya waɗanda suka sami bugun zuciya, gazawar koda, bugun jini, ciwon sukari, Crohn's da cutar Parkinson. Duk da haka, ba mu fahimci cikakken yadda aka cimma hakan ba. Kwayoyin ba sa shiga cikin nama mai karɓa - a zahiri kamar an cire su a cikin kwanaki, kuma suna ɓacewa ba tare da lahani ba, amma da alama suna ƙarfafa martanin rigakafin kumburi a cikin mai karɓa wanda ke dawwama tsawon kwanaki ko makonni.

Mutanen da ke da recessive dystrophic epidermolysis bullosa (RDEB) suna da wasu matsalolin da ke da alaƙa da halayen kumburi; waɗannan sun haɗa da anemia, rage ƙashi (osteoporosis), raunin waraka mara kyau, ƙaiƙayi, rashin ci da rashin ci gaba gaba ɗaya. Wannan rukunin ya yi fatan ƙara bincika ayyukan hana kumburin MSC a cikin manya tare da RDEB. Bayan fara bincike a cikin yara, Farfesa McGrath da tawagar sun yi shirin ba da infusions na MSC na ciki ga manya 10 tare da RDEB don bincika dalla-dalla mahimman alamomi a cikin jini da kyallen takarda. An kuma rubuta martanin su na asibiti (bokan fata, warkar da rauni, itching da zafi).

EBSTEM – Gwajin Jiki na Baya tare da MSCs

Sakamakon farko daga EBSTEM ya nuna cewa infusions sun bayyana lafiya kuma yana iya samun tasiri mai amfani a cikin yara. Wani muhimmin abin lura shi ne cewa mutane na iya ba da amsa daban-daban ga MSCs, yana jagorantar masu bincike don yin imani cewa mutane na iya samun wasu dalilai a cikin halittar halittarsu na halitta wanda ke sa su zama masu amsa masu kyau ko marasa kyau. ADSTEM ya shirya don tantance amincin MSCs a cikin manya. Ana buƙatar wannan binciken saboda idan aka kwatanta da yara, tabo a cikin manya tare da RDEB sau da yawa ya fi girma kuma akwai haɗarin ciwon daji na fata, don haka yana da mahimmanci a yi nazarin bayanin lafiyar MSC na ciki a cikin tsofaffin batutuwa kuma.

Wannan aikin yana da nufin ƙara fahimtar amsawar kumburi a cikin RDEB, taimakawa wajen kimanta ko MSCs suna da damar zama magani mai amfani, da kuma ƙarfafa yadda za a iya ba da irin wannan magani da kuma taimakawa wajen bunkasa karatun gaba a RDEB.

ADSTEM Abokin Bincike na Clinical, Dr Ellie Rashid, yana shirya MSCs don gwajin asibiti.

ADSTEM Abokin Bincike na Clinical, Dr Ellie Rashid, yana shirya MSCs don gwajin asibiti

Farfesa McGrath da tawagar sun gayyaci mutanen da ke da nau'in dystrophic na EB wadanda suka haura shekaru 18 don shiga gwajin asibiti na wannan nau'i na maganin tantanin halitta. Wannan ya haɗa da karɓar jiko na sel daga wani mutum marar dangantaka wanda ba shi da EB. Ƙungiyar yanzu ta bincika ko bayar da waɗannan ƙwayoyin cuta ba shi da lafiya kuma idan irin wannan magani zai iya inganta yanayin EB a cikin manya.

Manya goma masu fama da EB dystrophic recessive sun shiga cikin gwajin asibiti. Masu binciken sun gano cewa ba da kwayoyin halitta hanya ce ta "lafiya" ta yadda babu wanda ke da wani mummunan sakamako ko illa.

Sun kuma gano cewa ba da MSCs ya haifar da wasu gyare-gyare na asibiti kamar rage ƙaiƙayi, samun ƙarancin blish da inganta rayuwa. Waɗannan fa'idodin na sirri sun dade na 'yan watanni kafin su ƙare. Sun kuma nemi canje-canje a cikin fata da jini kafin da kuma bayan karbar MSCs kuma sun gano wasu kwayoyin halitta da sunadaran da ke da alaƙa da warkar da raunuka waɗanda MSCs suka canza.

Matakai na gaba

Wadannan binciken sun ba da sababbin ra'ayoyi game da yadda za a rage kumburi da kuma hanzarta warkar da raunuka a cikin fata na EB. Shirin yanzu shine ƙoƙarin gabatar da maganin MSC cikin kulawa na yau da kullun tare da yin amfani da sabbin bayanan dakin gwaje-gwaje don haɓaka har ma mafi kyawun jiyya ga mutanen da ke da EB.

“Wannan gwaji na asibiti ya ba mu babbar dama don duba aminci da yuwuwar fa'idodin maganin tantanin halitta a cikin mutanen da ke da EB dystrophic. Muna fatan waɗannan ƙwayoyin za su inganta warkar da raunuka tare da rage ƙaiƙayi da zafi, kuma cewa a cikin dogon lokaci gwajin zai haifar da canje-canje masu kyau a yadda muke bi da EB a asibitin "

Farfesa John McGrath, Babban Jami'in Bincike na ADSTEM

John McGrath MD FRCP FMedSci

Hoton kai na Farfesa John McGrath sanye da farar riga kuma yana murmushi a kyamarar

John McGrath MD FRCP FMedSci Farfesa ne na Kwayoyin cututtukan fata a Kwalejin King London kuma Shugaban Sashin Cututtukan Fatar Halittu, da kuma mai ba da shawara ga likitan fata a St John's Institute of Dermatology, Guy's da St Thomas' NHS Foundation Trust a London. A baya ya kasance ɗan ƙaramin ɗan bincike na EB mai tallafi na DEBRA kuma ya yi aiki akan binciken EB sama da shekaru 25. Yanzu yana jagorantar da haɗin kai akan ayyuka da yawa na ƙasa da ƙasa don haɓaka kwayoyin halitta, tantanin halitta, furotin da magungunan ƙwayoyi waɗanda zasu iya haifar da ingantattun jiyya ga mutanen da ke zaune tare da EB.

Masu neman hadin kai

Farfesa Francesco Dazzi, Dr Jemima Mellero, Dr Emma Wedgeworth, Dr Gabriela Petrof