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Ali vsa avtoprotitelesa IgG vodijo do avtoimunskih bolezni?

Ali vsa avtoprotitelesa IgG vodijo do avtoimunskih bolezni?


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Če je v naši krvi prisotnost avtoprotiteles, ali to pomeni, da bomo imeli avtoimunsko bolezen? Ali obstajajo nekatera avtoprotitelesa, ki ne povzročajo poškodb tkiv in organov?


Kot običajno pri biologiji, ni tako preprosto. Avtoprotitelesa so prisotna tudi pri ljudeh brez znakov avtoimunske bolezni. Imunski sistem je zapletena mreža pozitivnih in negativnih regulatornih celic in molekul – pogled na eno samo komponento verjetno ne bo določil stanja sistema. To je glavni razlog, da je bilo tako težko ugotoviti, kako selektivno delovati na imunski sistem, da bi zaustavili ali preprečili avtoimunsko bolezen, ne da bi pri tem ogrozili normalno delovanje imunskega sistema.

Tukaj je članek, ki razširja nekatere od teh vidikov: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2703183/

Na primer, opomba "Kljub fiziološki eliminaciji (negativna selekcija) ali funkcionalni inaktivaciji (anergiji) visoko afinitetnih, samoreaktivnih limfocitov T in B v timusu oziroma kostnem mozgu, obstajajo prepričljivi dokazi, da je nizka afiniteta potencialno avtoreaktivne celice vztrajajo in da je reaktivnost z nizko afiniteto na lastne antigene potrebna za preživetje limfocitov T in verjetno B v perifernem imunskem sistemu."

Pomembno je tudi upoštevati, da je afiniteta abs in receptorjev za antigene običajno odvisna od dodatnih molekul tretjih oseb, ki sodelujejo v interakciji, in da se lahko posamezna molekula v različnih situacijah pojavi zelo različno (na primer poškodba ali vnetje lahko spremeni nekaj molekule v prej nevidno konfiguracijo, ki spodbuja samoreaktivnost).


Nov svinec za avtoimunske bolezni iz kitajske medicine

Zdravilo, pridobljeno iz korenine hortenzije, ki se stoletja uporablja v tradicionalni kitajski medicini, je obetavno pri zdravljenju avtoimunskih motenj, poročajo raziskovalci iz Programa za celično in molekularno medicino in Inštituta za imunske bolezni pri otroški bolnišnici v Bostonu (PCMM/IDI), skupaj z Harvard School of Dental Medicine. V izdaji 5. junija znanost, kažejo, da spojina z majhno molekulo, znana kot halofuginon, zavira razvoj celic Th17, imunskih celic, ki so bile nedavno priznane kot pomembne akterje pri avtoimunskih boleznih, ne da bi pri tem spremenile druge vrste T celic, vključenih v normalno imunsko funkcijo. Nadalje dokazujejo, da halofuginon zmanjšuje patologijo bolezni v mišjem modelu avtoimunosti.

Trenutno ni dobrega zdravljenja avtoimunskih motenj, izziv je bil zatiranje vnetnih napadov imunskega sistema na telesna tkiva brez splošnega zatiranja imunske funkcije (s čimer se povečuje tveganje za okužbe). Glavno zdravljenje so protitelesa, ki nevtralizirajo citokine, kemične prenašalce, ki jih proizvajajo T celice, ki uravnavajo imunsko funkcijo in vnetne odzive. Vendar pa so protitelesa draga, dajati jih je treba intravensko in ne obravnavajo temeljnega vzroka bolezni, zato morajo bolniki preprosto vpijati citokine in ne ustaviti njihove proizvodnje, zato morajo prejemati pogoste intravenske infuzije, da zadržijo vnetje. Močna zdravila za zaviranje imunskega sistema se včasih uporabljajo kot zadnja možnost, vendar so bolniki ogroženi zaradi smrtno nevarnih okužb in drugih resnih stranskih učinkov.

Z vrsto poskusov so raziskovalci pokazali, da halofuginon preprečuje razvoj Th17 celic tako pri miših kot pri ljudeh, ustavi proces bolezni, ki ga sprožijo, in je selektiven pri svojih učinkih. Ima tudi možnost, da se jemlje peroralno. "To je res prvi opis majhne molekule, ki moti avtoimunsko patologijo, vendar ni splošni imunski supresor," pravi dr. Mark Sundrud iz PCMM/IDI, prvi avtor študije.

Celice Th17, ki so bile priznane šele od leta 2006, so bile vpletene v različne avtoimunske motnje, vključno z vnetno črevesno boleznijo, revmatoidnim artritisom, multiplo sklerozo, sladkorno boleznijo tipa 1, ekcemom in psoriazo. Genetsko se razlikujejo od drugih glavnih kategorij T-celic (Th1, Th2 in T-regulacijskih celic).

Th17 celice se običajno razlikujejo od "naïve" CD4+ T celic, ko pa so Sundrud in sodelavci gojili mišje CD4+ T-celice skupaj s citokini, ki običajno inducirajo razvoj Th17, je prišlo do izrazitega zmanjšanja celic Th17 &ndash, ne pa tudi regulatorja Th1, Th2 ali T. celice &ndash, ko je bil dodan halofuginon. Podobno je v kultiviranih človeških CD4+ T-celicah halofuginon selektivno zaviral proizvodnjo IL-17, glavnega citokina, ki ga proizvajajo celice Th17.

In pri miših z eksperimentalnim avtoimunskim encefalitisom (EAE), umetno povzročeno imunsko boleznijo, ki spominja na multiplo sklerozo pri ljudeh, za katero je značilna infiltracija celic Th17 v osrednji živčni sistem, je zdravljenje z nizkimi odmerki halofuginona znatno zmanjšalo tako razvoj EAE kot njegovega resnost. (Pri miših z drugo obliko EAE, ki ne vključuje celic Th17, halofuginon ni imel učinka.)

Raziskovalci so se spraševali, kako deluje halofuginon, so opravili študije mikromrež celic, zdravljenih s halofuginonom, da bi preučili vzorce izražanja genov kot odziv na zdravilo. Nepričakovano so se vklopili številni geni, ki sodelujejo pri odzivih na stres. Sčasoma so ugotovili, da halofuginon deluje tako, da aktivira biokemično pot, znano kot "odziv stradanja aminokislin" ali AAR, ki običajno ščiti celice, ko aminokislin, bistvenih gradnikov beljakovin, primanjkuje. Ko smo gojenim T-celicam, izpostavljenim halofuginonu, dodali presežek aminokislin, se AAR ni vklopil in celice Th17 so se lahko razvile. Nasprotno pa so raziskovalci lahko zavirali diferenciacijo Th17 preprosto z izčrpanjem aminokislin in s tem sprožili AAR.

Zakaj bi AAR preprečil nastanek celic Th17? Raziskovalci predlagajo, da ima AAR funkcijo varčevanja z energijo, saj upočasnjuje gradbene dejavnosti celice za ohranjanje aminokislin. "Ko celica zazna pomanjkanje aminokislin, poskuša ohraniti aminokisline s preprečevanjem specifičnih vrst odzivov, ki so energetsko dragi," pravi Sundrud. "V vnetih tkivih veliko celic proizvaja veliko beljakovin, zato bi bilo smiselno, da bi celica s pomanjkanjem aminokislin želela blokirati signale, ki spodbujajo vnetje."

Halofuginin je eno od 50 temeljnih zelišč tradicionalne kitajske medicine in se uporablja kot sredstvo proti malariji. Pred desetletji je ameriška vojska poskušala izboljšati svoje protimalarične lastnosti, vendar brez uspeha. Bil je v kliničnih preskušanjih za sklerodermo, a ker je zdaj v javnosti, farmacevtska industrija ni pokazala zanimanja za nadaljnji terapevtski razvoj.

Toda halofuginon ali neka derivativna spojina, ki še ni razvita, bi se lahko potencialno uporabila za obravnavo katere koli avtoimunske ali vnetne bolezni, povezane s celicami Th17, z aktiviranjem AAR, pravijo raziskovalci.

"Izjemno je, da halofuginon sproži AAR v vseh celicah, vendar selektivno zavira vnetne odzive T-celic," pravi dr. Anjana Rao iz PCMM/IDI, višji raziskovalec v študiji. "To spominja na delovanje ciklosporina A in FK506, dveh drugih imunosupresivnih zdravil, ki blokirata delovanje kalcineurina. Kalcineurin je prisoten v vseh celicah, vendar selektivno preprečuje zavrnitev presadkov srca, pljuč, jeter in kostnega mozga, ko jih dajemo bolnikom. Ti zdravila so revolucionirala transplantacijsko medicino, ko so bila uvedena pred več kot 20 leti, halofuginon pa lahko napoveduje revolucijo pri zdravljenju nekaterih vrst avtoimunskih/vnetnih bolezni."

Dr. Malcolm Whitman in dr. Tracy Keller s Harvardske šole za dentalno medicino in Anjana Rao, doktorica PCMM/IDI, sta bila višja raziskovalca študije. Študijo so financirali štipendije Nacionalnega inštituta za zdravje, Fundacije za raziskovanje mladostniške sladkorne bolezni in Inštituta za raziskave raka.

Vir zgodbe:

Materiali, ki jih zagotavlja Otroška bolnišnica Boston. Opomba: Vsebino je mogoče urediti glede na slog in dolžino.


Argenx poroča o finančnih rezultatih za prvo četrtletje 2018 in zagotavlja posodobitev poslovanja

9. maja 2018

Breda, Nizozemska / Gent, Belgija
- argenx (Euronext & Nasdaq: ARGX), klinično biotehnološko podjetje, ki razvija globoko linijo diferenciranih terapij na podlagi protiteles za zdravljenje hudih avtoimunskih bolezni in raka, je danes objavilo finančne rezultate in zagotovilo posodobitev poslovanja za prvo četrtletje, ki se je končalo 31. marec 2018.

"V tem četrtletju smo dosegli odličen napredek pri izvajanju naše strategije cevovoda in smo dobro pripravljeni na še eno leto, bogato z mejniki v letu 2018. Za začetek našega vodilnega programa efgartigimod (ARGX-113) smo predstavili celoten niz podatkov iz faze 2 klinično preskušanje pri miasteniji gravis na letnem srečanju Ameriške akademije za nevrologijo (AAN), ki je pokazalo zmanjšanje rezultatov bolezni, ki je povezano z našim razumevanjem mehanizma kandidata za zdravilo. Napredek smo dosegli tudi v Evropi, saj smo na tem prvem mestu prejeli oznako zdravila sirota Indikacija. Še naprej ostajamo na pravi poti, da v drugi polovici leta sporočimo podatke o dveh dodatnih indikacijah za efgartigimod, vključno z imunsko trombocitopenijo in pemfigusom vulgaris,« je komentiral Tim Van Hauwermeiren, izvršni direktor argenxa. "Prav tako napreduje preostali del našega sistema, vključno z ARGX-110, kjer smo prešli v 2. fazo kliničnega preskušanja pri na novo diagnosticiranih bolnikih z AML, ki niso primerni za kemoterapijo, in pričakujemo, da bomo do konca leta sporočili nove podatke o odzivu. še naprej iščejo vznemirljive cilje med našimi partnerji raziskovalnih institucij in to predstavili v prvem četrtletju z dodatkom ARGX-117 v naš cevovod, ki ponuja novo ciljno pot za argenx in potencialno način za dodajanje sinergijske vrednosti našemu cevovodu efgartigimod- pristop v izdelku."

PRVO ČETRETJE 2018 IN ZADNJI POUDARKI

FINANČNI POUDARKI (na dan 31. marca 2018) (v primerjavi s finančnimi poudarki na dan 31. marca 2017)

PODROBNOSTI OPERATIVNIH REZULTATOV

Izdelki v kliničnem razvoju:

Izdelki v predkliničnem razvoju:

PRIHAJAJOČI PRIČAKOVANI MEJNIKI

KLJUČNE ŠTEVILKE (PREČIŠČENO)

(*) Družba je 1. januarja 2018 sprejela MSRP 15 z uporabo spremenjenega retrospektivnega pristopa. Učinek sprejetja MSRP 15 znaša 0,9 milijona evrov za tri mesece, ki so se končali 31. marca 2018.

PODROBNOSTI FINANČNIH REZULTATOV

Poslovni prihodki so dosegli 6,9 milijona evrov v treh mesecih, ki so se končali 31. marca 2018, v primerjavi s 7,2 milijona evrov v treh mesecih, ki so se končali 31. marca 2017. Zmanjšanje prihodkov za 1,1 milijona evrov je predvsem posledica zmanjšanja pripoznanja prihodkov, povezanega z bližajoči se zaključek predkliničnih aktivnosti v okviru našega stalnega sodelovanja z LEO Pharma. Drugi poslovni prihodki so se povečali za 0,8 milijona evrov, kar je predvsem posledica (i) povečanja popustov davka na izplačane plače za zaposlovanje določenega raziskovalno-razvojnega osebja in (ii) povečanja prihodkov iz državnih podpor po odobritvi v marcu 2018 2,5 milijona evrov VLAIO odobritev za identifikacijo novih terapevtskih protiteles.
Za tri mesece, ki so se končali 31. marca 2018, so stroški raziskav in razvoja znašali 15,1 milijona evrov v primerjavi z 12,2 milijona evrov za tri mesece, ki so se končali 31. marca 2017. Povečanje odhodkov za raziskave in razvoj v letu 2018 za 2,9 milijona evrov je bilo v glavnem povezano do (i) stroškov, povezanih z načrtovanim povečanjem števila zaposlenih v raziskavah in razvoju in (ii) povečanih stroškov nadomestil na podlagi delnic, povezanih z dodelitvijo delniških opcij našim zaposlenim v raziskavah in razvoju (vključno s povečanjem stroškov socialne varnosti za 1,1 milijona evrov o delniških opcijah, podeljenih nekaterim belgijskim in nebelgijskim rezidenčnim uslužbencem).


Argenx napoveduje razširitev svojega cevovoda z dodatkom ARGX-117, usmerjenega na komplementa, za zdravljenje hudih avtoimunskih bolezni

Breda, Nizozemska/Gent, Belgija - argenx (Euronext & Nasdaq: ARGX), biotehnološko podjetje na klinični stopnji, ki razvija globok cevovod diferenciranih terapij na podlagi protiteles za zdravljenje hudih avtoimunskih bolezni in raka, je danes objavilo dodajanje novega kandidata za cevovod, ARGX-117, ciljanje na kaskado komplementa s terapevtskim potencialom pri indikacijah, ki jih posredujejo avtoprotitelesa.

ARGX-117 je visoko diferencirano terapevtsko protitelo, opremljeno z argenxovo lastniško Fc inženirsko tehnologijo NHance®, ki obravnava novo tarčo v klasični poti kaskade komplementa. S potencialno diferenciranim mehanizmom delovanja ARGX-117 predstavlja široko paleto možnosti za več indikacij, ki jih posreduje avtoprotitelesa, in ima lahko sinergistični učinek s svinčevo avtoimunsko spojino ARGX-113.

"Pravice za ARGX-117 smo pridobili kot del našega programa inovativnega dostopa, s katerim smo z Broteio Pharma identificirali revolucionarno delo na tem protitelesu. Mehanizem delovanja ARGX-117 nam lahko omogoči, da raziščemo njegove potencialne koristi v različnih indikacije, posredovane s komplementom. Poleg tega lahko ARGX-117 sinergizira z našo svinčevo avtoimunsko spojino ARGX-113, ki cilja na FcRn, da odstrani patogena protitelesa imunoglobulina G (IgG), medtem ko lahko naše novo protitelo, usmerjeno na komplement, obravnava tudi imunoglobulin M (IgM). )-posredovane avtoimunske bolezni," je komentiral Tim Van Hauwermeiren, izvršni direktor argenxa. "Še naprej izvajamo našo strategijo cevovoda, da iščemo kandidate, ki izhajajo iz vznemirljive znanosti, jih izboljšamo z našimi platformskimi tehnologijami in jih vpeljemo v razvoj v indikaciji sirote. ARGX-117 predstavlja potencialni produkt zdravljenja. za indikacije sirote."

argenx in Broteio sta leta 2017 začela sodelovanje za izvedbo raziskave s podporo Univerze v Utrechtu, da bi prikazala predklinični dokaz koncepta mehanizma ARGX-117. Kot del pogodbe o sodelovanju je argenx izkoristil ekskluzivno možnost licenciranja programa in prevzel odgovornost za nadaljnji razvoj in komercializacijo. Finančni pogoji ne bodo razkriti.

O programu inovativnega dostopa
Z našim programom inovativnega dostopa (IAP) prinašamo naše tehnologije odkrivanja protiteles v središče novih ciljnih raziskav s tesnim sodelovanjem z akademskimi strokovnjaki in majhnimi biotehnološkimi podjetji. IAP našim sodelavcem omogoča uporabo naših tehnologij za odkrivanje funkcij novih beljakovin pri bolezni. V zameno prejmemo zgodnji dostop do ciljev s terapevtsko pomembnostjo in potencialom, da postanemo naslednji programi terapevtskih protiteles v našem načrtu.

Vloga komplementa pri avtoimunskih boleznih
Klasična pot sistema komplementa je sestavljena iz vrste beljakovin, ki se aktivirajo, ko se avtoprotitelesa IgG ali IgM vežejo na svoje tarče. Ta mehanizem prispeva k poškodbam tkiva in disfunkciji organov pri številnih avtoimunskih vnetnih boleznih. Cilj ARGX-117 je ključnega pomena pri lizi celic, okrašenih s protitelesi, in je aktiven, ko poteka imunska reakcija. ARGX-117 ima terapevtski potencial pri avtoimunskih indikacijah sirote in velike populacije. Predklinično razvojno delo na ARGX-117 poteka v tesnem sodelovanju s prof. E. Hackom (UCM Utrecht, Laboratorij za translacijsko imunologijo).

O Broteiu
Broteio Pharma B.V. (Broteio) s sedežem v Utrechtu na Nizozemskem je skupno podjetje med Prothix B.V. in Bioceros Holding B.V., ustanovljeno za razvoj monoklonskih protiteles proti komplementu.

Glede argenxa
argenx je biotehnološko podjetje na klinični stopnji, ki razvija globok cevovod diferenciranih terapij na podlagi protiteles za zdravljenje hudih avtoimunskih bolezni in raka. Osredotočeni smo na razvoj kandidatov za izdelke s potencialom, da bodo bodisi prvi v razredu proti novim tarčam ali najboljši v razredu proti znanim, a kompleksnim tarčam za zdravljenje bolezni s pomembno neizpolnjeno medicinsko potrebo. Našo zmožnost izvajanja na tem fokusu omogoča naš nabor diferenciranih tehnologij. Naša platforma SIMPLE Antibody TM, ki temelji na močnem imunskem sistemu lame, nam omogoča izkoriščanje novih in zapletenih ciljev, naše tri tehnologije za inženiring protiteles pa so zasnovane tako, da nam omogočajo razširitev terapevtskega indeksa naših kandidatov za izdelke.
www.argenx.com

Za dodatne informacije se obrnite na:

Joke Comijn, vodja korporativnih komunikacij in IR
+32 (0)477 77 29 44
+32 (0)9 310 34 19
[email protected]

Beth DelGiacco (US IR)
Stern odnosi z vlagatelji
+1 212 362 1200
[email protected]

Izjave za prihodnost
Vsebina te objave vključuje izjave, ki so ali se lahko štejejo za "izjave, usmerjene v prihodnost". Te izjave, usmerjene v prihodnost, je mogoče prepoznati z uporabo terminologije, usmerjene v prihodnost, vključno z izrazi "verjamem", "ocena", "predvideva", "pričakuje", "namerava", "lahko", "bo" ali " bi moral," in vključuje izjave, ki jih daje argenx o predvidenih rezultatih svoje strategije in argenxov napredek ter pričakovani klinični razvoj ter regulativni mejniki in načrti, povezani z ARGX-117. Izjave za prihodnost po svoji naravi vključujejo tveganja in negotovosti, bralce pa opozarjamo, da kakršne koli takšne napovedne izjave niso jamstvo za prihodnjo uspešnost. Dejanski rezultati argenxa se lahko bistveno razlikujejo od tistih, ki jih napovedujejo izjave o prihodnosti kot posledica različnih pomembnih dejavnikov, vključno s pričakovanji argenxa glede njegovih prirojenih negotovosti, povezanih s konkurenčnim razvojem, predkliničnimi in kliničnimi preskušanji ter dejavnostmi razvoja izdelkov in regulativnimi zahtevami za odobritev, zanašanje argenxa na sodelovanje s tretjimi osebami, ki ocenjujejo komercialni potencial argenxa Kandidati za izdelke argenxova sposobnost pridobitve in vzdrževanja zaščite intelektualne lastnine za svoje tehnologije in zdravila argenxova omejena zgodovina delovanja in zmožnost argenxa, da pridobi dodatna finančna sredstva za operacije ter dokonča razvoj in komercializacijo svojih kandidatov za izdelke. Nadaljnji seznam in opis teh tveganj, negotovosti in drugih tveganj je na voljo v dokumentih in poročilih ameriške komisije za vrednostne papirje in borzo argenxa (SEC), vključno v končnem prospektu v zvezi z javno ponudbo argenxa v ZDA, vloženem pri SEC v skladu s členu 424(b) Zakona o vrednostnih papirjih iz leta 1933, kakor je bil spremenjen, kot tudi naknadne vloge in poročila, ki jih je argenx vložil pri SEC. Glede na te negotovosti bralcu svetujemo, naj se ne zanaša pretirano na takšne napovedne izjave. Te napovedne izjave veljajo samo na dan objave tega dokumenta. argenx se ne zavezuje, da bo javno posodabljal ali spreminjal informacije v tem sporočilu za javnost, vključno s kakršnimi koli izjavami o prihodnosti, razen če to zahteva zakon.

To obvestilo distribuira Nasdaq Corporate Solutions v imenu strank Nasdaq Corporate Solutions.

Izdajatelj te objave jamči, da je izključno odgovoren za vsebino, točnost in izvirnost informacij, ki jih vsebuje.
Vir: argenx SE preko GlobeNewswire

OBJEM#2178132


Abcuro zbere 42 milijonov dolarjev za financiranje serije A-1, imenuje Johna B. Edwardsa za izvršnega predsednika upravnega odbora in Davida de Graafa, dr. kot glavni izvršni direktor

Abcuro, Inc., biotehnološko podjetje na klinični stopnji, ki razvija terapije za avtoimunske bolezni in raka z natančno modulacijo citotoksičnih T in NK celic, je danes objavilo zaključek financiranja serije A-1 v vrednosti 42 milijonov dolarjev in imenovanje Johna B. Edwardsa za izvršni predsednik upravnega odbora in David de Graaf, dr. kot glavni izvršni direktor. Financiranje sta vodila Mass General Brigham Ventures in Sanofi Ventures ter vključevala vlagatelje Pontifax Venture Capital, Hongsen Investment Group, RA Capital Management in Samsara BioCapital ter druge.

Sočasno s tem financiranjem je Jason Hafler, dr. iz Sanofi Ventures, Iyona Rajkomar iz Pontifax Venture Capital, Ryan Berry, dr. iz RA Capital Management in Donald McCarthy, dr. iz Samsara BioCapital so se pridružili upravnemu odboru Abcuro, ki vključuje tudi Julius Knowles iz Mass General Brigham Ventures in Yajun Xu, Ph.D., predsednik Hongsen Investment Group.

"Abcuro je v preteklem letu razvil prepričljiv podatkovni paket za podporo temu krogu financiranja za napredovanje našega vodilnega programa v kliniko," je povedal g. Edwards, izvršni predsednik. "Spodbudila nas je stopnja podviga in farmacevtskega zanimanja za Abcurovo pionirsko delo, usmerjeno v natančno ciljanje citotoksičnih T in NK celic. Še posebej smo veseli, da lahko pozdravimo Davida kot izvršnega direktorja, ko vstopamo v to ključno obdobje rasti za Abcuro."

Izkupiček tega financiranja bo porabljen za napredek ABC008, protitelesa proti KLRG1, zasnovanega za izčrpavanje citotoksičnih T celic, ki napadajo zdravo tkivo pri različnih avtoimunskih boleznih. Pri bolnikih s sporadičnim miozitisom inkluzijskega telesa (IBM) bo izveden prvi dokaz mehanizma in varnosti pri ljudeh, medtem ko so dodatne indikacije v preiskavi. IBM je kronično in izčrpavajoče vnetno stanje skeletnih mišic brez razpoložljivih farmacevtskih terapij. Prihodki iz serije A-1 bodo podpirali tudi tekoči predklinični razvoj onkološkega programa podjetja ABC015, protitelesa, ki blokira anti-KLRG1, ki lahko obnovi efektorske citotoksične T in NK celice, kar ima za posledico močan protitumorski odziv. To vključuje finančno in znanstveno podporo Fundacije za raziskave multiplega mieloma za oceno ABC015 pri multipli mielomu.

"KLRG1 je prepričljiva tarča pri imunski modulaciji tako pri avtoimunskih boleznih kot pri raku, saj nam omogoča natančno ciljanje na klinično pomembne citotoksične T in NK celice," je dejal dr. de Graaf. "Zelo sem zadovoljen s skupnim navdušenjem Abcurovih vlagateljev pri iskanju kliničnega potenciala ciljanja na KLRG1."

G. Edwards v Abcuro prinaša več kot trideset let izkušenj pri odkrivanju, razvoju in globalni komercializaciji biofarmacevtskih izdelkov. Sodeloval je pri razvoju ali komercializaciji desetih bioloških zdravil, ki jih je odobrila FDA, in je imel uspešne izstope v osmih od desetih biotehnoloških podjetij, pri katerih je pomagal zgraditi, vključno s Tilosom, Siamabom, Exonics, Adnexusom, F-star in Genetics Institute. Ustvaril je hematološko terapevtsko podjetje, ki je na koncu doseglo več kot 1 milijardo dolarjev letnega prihodka za Wyeth/Genetics Institute in je bil odgovoren za vodenje razvoja najhitrejšega biološkega zdravila, da bi napredoval od faze I do odobritve FDA.


Abcuro zbere 42 milijonov dolarjev za financiranje serije A-1, imenuje Johna B. Edwardsa za izvršnega predsednika upravnega odbora in Davida de Graafa, dr. kot glavni izvršni direktor

NEWTON, Mass.--(BUSINESS WIRE)--Abcuro, Inc., biotehnološko podjetje na klinični stopnji, ki razvija terapije za avtoimunske bolezni in raka z natančno modulacijo citotoksičnih T in NK celic, je danes objavilo zaprtje serije A v vrednosti 42 milijonov dolarjev -1 financiranje in imenovanje Johna B. Edwardsa za izvršnega predsednika upravnega odbora in Davida de Graafa, dr. kot glavni izvršni direktor. Financiranje sta vodila Mass General Brigham Ventures in Sanofi Ventures ter vključevala vlagatelje Pontifax Venture Capital, Hongsen Investment Group, RA Capital Management in Samsara BioCapital ter druge.

Sočasno s tem financiranjem je Jason Hafler, dr. iz Sanofi Ventures, Iyona Rajkomar iz Pontifax Venture Capital, Ryan Berry, dr. iz RA Capital Management in Donald McCarthy, dr. iz Samsara BioCapital so se pridružili upravnemu odboru Abcuro, ki vključuje tudi Julius Knowles iz Mass General Brigham Ventures in Yajun Xu, Ph.D., predsednik Hongsen Investment Group.

»V preteklem letu je Abcuro razvil prepričljiv paket podatkov za podporo temu krogu financiranja za napredovanje našega vodilnega programa v kliniko,« je povedal g. Edwards, izvršni predsednik. »Spodbudila nas je stopnja podviga in farmacevtskega zanimanja za Abcurovo pionirsko delo, usmerjeno v natančno ciljanje citotoksičnih T in NK celic. Še posebej smo veseli, da lahko pozdravimo Davida kot izvršnega direktorja, ko vstopamo v to ključno obdobje rasti za Abcuro.

Izkupiček tega financiranja bo porabljen za napredek ABC008, protitelesa proti KLRG1, zasnovanega za izčrpavanje citotoksičnih T celic, ki napadajo zdravo tkivo pri različnih avtoimunskih boleznih. Pri bolnikih s sporadičnim miozitisom inkluzijskega telesa (IBM) bo izvedeno prvo pri človeku preskušanje mehanizma in varnosti, medtem ko so dodatne indikacije v preiskavi. IBM je kronično in izčrpavajoče vnetno stanje skeletnih mišic brez razpoložljivih farmacevtskih terapij. Prihodki iz serije A-1 bodo podpirali tudi tekoči predklinični razvoj onkološkega programa podjetja ABC015, protitelesa, ki blokira anti-KLRG1, ki lahko obnovi efektorske citotoksične T in NK celice, kar ima za posledico močan protitumorski odziv. To vključuje finančno in znanstveno podporo Fundacije za raziskave multiplega mieloma za oceno ABC015 pri multipli mielomu.

"KLRG1 je prepričljiva tarča pri imunski modulaciji tako pri avtoimunskih boleznih kot pri raku, saj nam omogoča natančno ciljanje na klinično pomembne citotoksične T in NK celice," je dejal dr. de Graaf. "Zelo sem zadovoljen s skupnim navdušenjem Abcurovih vlagateljev pri iskanju kliničnega potenciala ciljanja na KLRG1."

G. Edwards v Abcuro prinaša več kot trideset let izkušenj pri odkrivanju, razvoju in globalni komercializaciji biofarmacevtskih izdelkov. Sodeloval je pri razvoju ali komercializaciji desetih bioloških zdravil, ki jih je odobrila FDA, in je imel uspešne izstope v osmih od desetih biotehnoloških podjetij, pri katerih je pomagal zgraditi, vključno s Tilosom, Siamabom, Exonics, Adnexusom, F-star in Genetics Institute. Ustvaril je hematološko terapevtsko podjetje, ki je na koncu doseglo več kot 1 milijardo dolarjev letnega prihodka za inštitut Wyeth/Genetics in je bil odgovoren za vodenje razvoja najhitrejšega biološkega zdravila do napredka od faze I do odobritve FDA.


Argenx pritegne ogromno 250 milijonov evrov naložbe po pozitivnem preizkusu faze II

Podjetje zdaj načrtuje napredovanje efgartigimoda, fragmenta protitelesa, ki ga navdihujejo protitelesa lame, v preskušanja faze III za zdravljenje primarne imunske trombocitopenije (ITP) avtoimunske krvne bolezni.

Pri ITP imunski sistem napade in uniči telesne trombocite, zaradi česar so bolniki nagnjeni k krvavitvam in modricam. V preskušanju so ugotovili klinično pomembne izboljšave ravni trombocitov v krvi pri bolnikih z ITP, če so prejemali efgartigimod. Zdravljenje je imelo tudi dober varnostni profil.

Efgartigimod se testira tudi za zdravljenje drugih avtoimunskih bolezni. Lani je imel uspešno klinično preskušanje faze II za nevromuskularne bolezni miastenijo gravis, testirajo pa se tudi za zdravljenje kožnih stanj, kot je pemphigus vulgaris.

Ta ogromna javna ponudba je eden največjih evropskih krogov zbiranja sredstev za biotehnologijo v letu. Sledi podoben uspeh Argenxove IPO v vrednosti 115 milijonov dolarjev (103 milijona evrov) v letu 2017 in še 231 milijonov dolarjev (195 milijonov evrov) javne ponudbe lanskega decembra, obe pa sta daleč presegli svoj cilj.

Zdravljenje s protitelesi, ki ga navdihuje imunski sistem lame, uporabljajo tudi druge biotehnologije v Evropi, kot sta Isogenica, ki ima knjižnični sistem sintetičnih protiteles, in Ablynx, ki ga je Sanofi kupil s pogodbo v vrednosti 3,9 milijarde evrov v začetku tega leta.

Argenx ima tudi druge projekte v delu, vključno z velikim sodelovanjem z ameriškim podjetjem AbbVie za nova zdravljenja raka in z dansko biotehnološko LEO Pharma, ki razvija zdravljenje za vnetne kožne bolezni.


Ali vsa avtoprotitelesa IgG vodijo do avtoimunskih bolezni? - Biologija

Forum ponuja vrhunski program

Diamantina Health Partners praznuje uspeh svojega drugega letnega foruma, Srečanje umov: prevedi-prenesi-preobrazi.

Dvodnevni forum na Translational Research Institute v kampusu bolnišnice Princess Alexandra Hospital je od 30. maja do 2. aprila 2014 privabil več kot 315 prijav, s čimer je utrdil svoj položaj vodilnega dogodka za organizacijo.

Forum je bil osredotočen na to, kako prevesti znanost v oskrbo, prenos znanje v praksi in preoblikovati zagotavljanje zdravstvenega varstva na področjih zdravljenja raka in kroničnih bolezni ter staranja – dve temi DHP.

Prvič je dogodek vključeval predforumski simpozij, Raziskave in inovacijske ideje, ki je vključeval nagrado v višini 10.000 $ iz PA Research Foundation.

Nagrado je prejela dr. Fiona Simpson za svojo predstavitev o pristopu k premagovanju epitelnega raka. Drugouvrščeni so bili dr. John A Duley z univerze v Queenslandu, dr. Selena Bartlett s tehnološke univerze Queenslanda in dr. Ingrid Winkler z univerze Mater.

Program foruma je vključeval 47 govorcev, vključno z dvema uglednima kliničnima akademikoma iz Združenih držav.

Forum je odprl minister za znanost, informacijsko tehnologijo, inovacije in umetnost Queenslanda Ian Walker.

Majhno število predstavitev je bilo objavljenih v glasilu.

Predsednik Diamantina Health Partners dr. David Theile je dejal, da sta kaliber predavateljev in kakovost informacij povzročila program svetovnega razreda.

"Radi bi se zahvalili govornikom, predsednikom, sodnikom in udeležencem za njihove prispevke in podporo sporočilu foruma o pomenu prenosa raziskav v resnične izboljšave rezultatov zdravstvenega varstva in potrebi po spodbujanju in podpori tega procesa s sodelovanjem, « je dejal dr. Theile.

»Posebna zahvala gre našim mednarodnim glavnim govornikoma dr. Victorju Montoriju, profesorju medicine na kliniki Mayo, in dr. Davidu Albertsu, profesorju medicine, farmakologije, prehranskih znanosti in javnega zdravja ter zaslužnem direktorju Centra za raka Univerze v Arizoni.

"Forum je zagotovil odlične priložnosti za večje povezave in sodelovanja, ki bodo zagotovila večjo integracijo raziskav, izobraževanja in oskrbe za boljše zdravje."

Dr. Theile se je zahvalil tudi sponzorjem Pathology Queensland (sklad SERTF), PA Research Foundation, illumina, QFAB in Translational Research Institute.

Hvala za vaše mnenje

Diamantina Health Partners se zahvaljuje udeležencem foruma, da so si vzeli čas za povratne informacije o dogodku.

Izvršni svetovalec Diamantina Health Partners Areti Gavrilidis je dejal, da so bile povratne informacije izjemno dragocene za načrtovanje foruma 2015.

Od tistih, ki so se odzvali, jih je več kot polovica dogodek ocenila kot odličen (49,45 %) ali izjemen (15,38 %). Prvih pet poudarkov je bilo:

  • predforumski simpozij in program nagrad
  • predstavitvi slavnostnih govornikov dr. Victorja Montorija in dr. Davida Albertsa
  • raznolikost predstavitev in tem
  • prevod raziskav v prakso s predstavitvami
  • dobro zasnovan program – dobro upravljanje časa.

Anketiranci so bili tudi povabljeni, da prispevajo ideje za forum 2015. Med odgovori je bil poziv k večji osredotočenosti na današnje klinične izzive in več interaktivnih srečanj.

Diamantina Health Partners would like to congratulate the Princess Alexandra Hospital on its success in achieving a Magnet facility designation for the third time. The Magnet designation is the highest international recognition for nursing excellence and the Princess Alexandra Hospital is the only facility outside of the United States to achieve it for a third time. This is an exemplary achievement!

Dr David Theile
predsednik

Areti Gavrilidis
Executive Consultant

Inaugural symposium attracts quality presentations

The inaugural Pre-Forum Research and Innovation Awards symposium at the Meeting of Minds attracted 62 submissions with 30 presentations chosen to showcase the potential human benefits of translational research.

A $10,000 Research and Innovation Ideas Award was sponsored by the PA Research Foundation was awarded to Dr Fiona Simpson for her presentation “Stop the traffic: a way to conquer epithelial cancer”.

Dr Simpson, a researcher at UQ Diamantina Institute, located within the Translational Research Institute at the Princess Alexandra Hospital Campus, said the win would enable the research team to continue their research into cancer therapy.

“Our lab is just four years old and $10,000 makes a huge difference to us,” she said.

The project has enormous relevance for Queenslanders, who have the highest incidence of squamous cell cancers (SCC) in the world.

Project lead Dr Fiona Simpson said advanced stage patients were presently treated with a combination of radiation and new targeted molecular therapies such as Cetuximab, an antibody which binds to the epidermal growth factor receptor (EGFR). EGFR is a driver in many epithelial tumours and monoclonal antibody therapies such as Cetuximab are designed to inhibit the receptor.

However, at present a high percent of advanced SCC patients fail treatment and suffer significant side-effects.

Dr Simpson said there was no test for determining which patients would respond to treatment.

Dr Fiona Simpson
accepting her Award

“We took a novel approach. With the consent of the patients at the PAH, live tumour samples were collected and an imaging technique developed to look at what the EGFR does in actual patient samples,” she said.

“We were surprised by our findings, as the EGFR was supposed to sit on the surface of the tumour cells and, when activated, should be taken up into the cells for down-regulation and degradation.

“Approximately 35 percent of the patients had EGFR which stayed on the tumour cell surface and was not taken in when activated.

“While expectations were that this would worsen patient outcomes, we found the opposite was true.”

Dr Simpson said the UQDI scientists, who were working with immunologists Drs James Wells and Stephen Mottorollo, have shown that the antibody therapy gets stuck with the receptor on the tumour cell surface and recruits the immune system to kill the tumour cells.

Having EGFR stuck on the tumour surface allows patients’ tumours to not just stop growing (a response to EGFR signalling inhibition by Cetuximab) but to also shrink or regress as the immune system is recruited.

Dr Simpson said the group had filed two patents—one to test whether patient EGFR location status predicts patient outcomes in monoclonal antibody therapy and the other to test a small molecule inhibitor in combination with Cetuximab.

“The idea is to inhibit the uptake of EGFR from the cell surface for a short time to allow the immune system to be recruited,” she said.

“This approach should also be helpful in increasing responses to many other monoclonal therapies, including Herceptin therapy in breast cancer and Rituximab therapy in leukemia.”

Old is new again for cancer drug

A project to improve the efficacy of older cancer drugs has received recognition at the pre-forum Research and Innovation Ideas symposium.

Dr John Duley
accepting his Award

University of Queensland Pharmacology senior lecturer Dr John Duley’s project “Fluorouracil toxicity: evidence that a transport defect explains the majority of cases” was announced as one of the three runners-up of the symposium’s Research and Innovation Ideas Award.

Also awarded runner-up was Dr Selena Bartlett from the Queensland University of Technology for her presentation “A novel treatment approach for obesity—targeting brain nicotinic receptors with ChampixTM”, and Dr Ingrid Winkler from Mater Research Institute for the presentation “Vascular bone marrow niches protect Acute Myeloid Leukaemia Stem Cells from chemotherapy”.

Dr Duley informed the impetus for the project, which was on the NHMRC Best of Ten award for 2013, was to make “old” drugs work more efficiently.

In his presentation, Dr Duley indicated that about one third of severe side-effects during cancer chemotherapy with 5-fluoro-uracil (5FU) or capecitabine result from partially deficient dihydropyrimidine dehydrogenase (DPD), the major rate-limiting step for 5FU catabolism.

The underlying cause of the remaining two thirds of toxicity remains unknown.

“Our aim is to develop a test to predict 5FU or capecitabine toxicity.” Dr Duley said the health outcomes for the patients were lower rates of injury or death, a better response rate to the drug and cost savings for the health system.

He indicated the project is a collaboration between The University of Queensland, Mater Research Institute, St Vincent’s Hospital Sydney, Monash Medical Centre in Melbourne, GenesFX Health in Melbourne and the Laboratory of Genetic and Metabolic Diseases, Academic Medical Centre in the Netherlands.

Diamantina Comprehensive Cancer Centre

With Comprehensive Cancer Care as one of the 2014 themes for the forum, presentations focussed on:

  • translation stories
  • transfer of knowledge to practice
  • transformation of delivery of healthcare.

Is breast cancer treatment too harmful?

One Queensland researcher is asking whether breast cancer screening does more harm than good.

Diamantina Comprehensive Cancer Centre Theme Leader Associate Professor Euan Walpole in his presentation Ductal Carcinoma in situ of the breast – a case for change in strategy said there was worldwide interest in the results of mammographic screening and its real impact.

Dr Walpole said his study follows controversy in the medical literature about the possible over-diagnosis of cancer and the subsequent aggressive treatments.

izredni profesor
Euan Walpole

“Screening for breast cancer has been a major public initiative across the western world for the last 20 years,” Dr Walpole said.

Studies suggest breast cancer screening reduces the incidence of breast cancer but also increases the diagnosis of some cancers that would not have progressed or killed the patient in their life expectancy.

“Questions have been raised about the screening of breast cancer and the treatment of precancerous conditions.”

Dr Walpole said women underwent significant intervention to protect themselves from breast cancer when it was possible that some of those cancers were not a threat to their life expectancy.

The essential outcome from Ductal Carcinoma in situ, which in the past was diagnosed only after mastectomy, is uncertain.

“Are we potentially hurting more people than we’re helping? Everyone believes that on the balance we are helping more people…but we are trying to get a better measure of what’s happening in reality in our population.”

The team at Princess Alexandra Hospital, Queensland Cancer Control Analysis, The University of Queensland, Queensland University of Technology and Metro South, are looking at historical data with a view to positively impact on the diagnosis and treatment of Queensland patients.

“It’s important not to assume that because of past information, everything is okay,” he said.

“And I think this is a very nice example of how you use statistics to show lots of different things to make sure you are asking the right question.”

Selfies may stop cancer - iPhones into scanners

Queensland researchers are turning iPhones into personal skin scanners so patients can detect suspicious spots.

Epithelial Malignancy Stream Leader within the Diamantina Health Partners Comprehensive Cancer Centre, Professor Peter Soyer’s presentation focused on the trial of an iPhone accessory and app that people can use when they scan their skin for suspicious moles.

Professor Soyer said iPhone accessory Handyscope was an easily attachable optical device and would be more effective than the naked eye for melanoma detection.

The project is being run by the University of Queensland’s Dermatology Research Centre, Queensland University of Technology (QUT) and Queensland Institute for Medical Research.

“Routine skin self-examination can save lives but its effectiveness is relatively low,” Professor Soyer said.

“With the Handyscope patients can scan their skin, take photos of suspect skin lesions, record them in an application, and then send the data onto health professionals for examination.

“This development might change the way health care is delivered in the future and ultimately improve skin cancer treatment outcomes.”

HandyScope attaches to the iPhone camera, has a 20x magnification capacity and a polarised light that goes deeper into the skin to show lesions more clearly.

Professor Soyer said high-risk melanoma and recovering skin cancer sufferers who visit a specialist every few months or live in rural and remote areas could benefit the most from this innovation.

“Patients could monitor their lesions wherever they are, reducing the required face-to-face examination time with their health professional. Melanoma diagnosis by One Click is becoming a reality.”

Tests - Tissues - Trials and Translational Research

A successful half day meeting Tests – Tissues – Trials, coordinated by Diamantina Health Partners (DHP) and Professor Ken O’Byrne, was held 22nd May 2014 at the Translational Research Institute (TRI) Auditorium.

The focus of the meeting was the integration of tests, tissues and clinical trials for translational research on the PAH campus. Registrants from major Brisbane institutions attended the meeting to hear from twelve local and interstate experts on key topics including:

  • Genomics and molecular diagnostics
  • Biobanking and data management
  • Clinical trials

Chronic Disease and Ageing

The first day of the two-day forum was dedicated to chronic disease and ageing, with 19 presentations addressing:

  • translation: the challenges and rewards of science to clinical capacity
  • transfer: knowledge dissemination into practice
  • transform: changing practice
  • transform: system development—Safety Efficacy Economics.

Lessons in the journey to commercialisation

The road from discovery to commercialisation can be long and expensive, according to UQ Diamantina Institute lead autoimmune researcher Professor Ranjeny Thomas.

Professor Thomas mapped her “challenging yet rewarding” ten-year journey from the discovery of an antigen specific therapy for rheumatoid arthritis to commercialisation as part of her presentation at the forum.

She said there were some key messages for researchers considering the same path.

She highlighted that it can take a very long time between discovery and commercialisation, for various reasons.

“It’s expensive to commercialise and produce and maintain a patent. If no investment is forthcoming the patent may be dropped.

“It could be that the idea – while transformative - is well ahead of its time or difficult for investors to understand or conceptualise.”

Professor Thomas highlighted the importance of confidentiality at least until protection was in place.

“It is also good to collaborate outside your area of expertise to get a better product or outcome for your research.”

In 2003, Professor Thomas and Dr Brendan O’Sullivan discovered that the body’s immune response could be “re-educated” to turn off, rather than react to a self-antigen responsible for an autoimmune disease, such as rheumatoid arthritis.

By targeting the cause of autoimmune disease, rather than treating the symptoms, the team realised that this type of immunotherapy offered the potential for long-lasting treatment by working with the body’s own immune system to fight disease. In 2006, with Dr Nigel Davies from UQ School of Pharmacy, they developed a targeting system using liposome particles for delivery of antigen-specific immunotherapy.

After clinical trials demonstrating proof-of-concept of this idea, the US pharmaceutical company Janssen Biotech came on board in 2013 to co-develop antigen-specific immunotherapy for RA patients.

“I think I followed the journey of many inventors,” Professor Ranjeny said.

“Inventors throughout history are often not understood until late in the piece.

“Once you can demonstrate enablement of your idea, the rest of the world eventually catches on. The challenge is to keep the money flowing in the meantime!”

Professor Thomas said UniQuest had played an essential role in the process by filing a patent, sourcing non-traditional sources of funding for commercialisation and introducing her to potential investors. For more information about the collaboration with Janssen Biotech, visit www.uq.edu.au/news

Researchers are on the verge of translating a cure for diabetes in mice into a treatment for patients.

In his presentation, Deputy Director (Research) at the Mater Research Institute Professor Mike McGuckin said the discovery that cytokine IL-22 suppresses oxidative stress in pancreatic beta cells could revolutionise the treatment of Type 2 diabetes.

An estimated five percent of the Australian population is affected by diabetes.

“In diabetes, beta cells experience oxidative stress and this is thought to be central to the inability to produce sufficient insulin to control blood glucose,” Professor McGuckin said.

“Treatment of diabetic mice with IL-22 restores glycaemic control, suppressing hyperinsulinaemia, hyperproinsulinaemia and insulin resistance.”

Diabetics could potentially replace their insulin injections with the protein.

“This is completely different to anything that has been done before,” Professor McGuckin said.

“It is early days—we’ve done it in mice and it works and we’ve done it in human cells and they have responded appropriately.

“Whether it revolutionises diabetes treatment depends on what happens when we do clinical trials. It’s a biological therapy that might have side-effects that we are not seeing in the mice that could make it inappropriate for human therapy.

“However, it does open up a whole new pathway for treating diabetes that might not include this cytokine but the pathways that it drives.”

Professor McGuckin said the project, which has been run at Mater Research in collaboration with Professor John Prins, Associate Professor Jon Whitehead and Professor Josephine Forbes, was an example of the concept behind Diamantina Health Partners.

“We are using knowledge and translating it into health outcomes by collaborating with appropriate people,” he said.

“It was because we had these connections with diabetes researchers and because we saw the ramifications for diabetes that we were able to do this work.

“As a piece of science it has broad implications and in terms of clinical medicine it’s got pretty broad implications. The story is not just confined to diabetes—it has relevance to other diseases that involve inflammation and stress in cells.”

Frailty index to improve the care of vulnerable older people

Work is underway to produce an index to help clinicians managing older patients.

Senior Lecturer in Geriatric Medicine at The University of Queensland Dr Ruth Hubbard spoke at the forum on research by the Centre for Research in Geriatric Medicine (CRGM) to develop a frailty index. The frailty index is being derived from data collected in the interRAI Acute Care assessment instrument, which is already widely used across Queensland.

“Older people in hospital are at high risk of prolonged hospital stays, institutionalisation and death,” Dr Hubbard said.

This increased vulnerability to adverse outcomes is commonly referred to as frailty. The team are motivated to improve the care of older people in hospital by coming up with a scoring system that will help us stratify risk. That scoring system is known as the frailty index. The higher your index, the more likely you are to have an adverse outcome in hospital.

Dr Hubbard informed that the index is designed to help specialists in their decision-making, as it will give an indication to the physiological reserves of a patient.

“I believe some specialists have an idea that some people are more frail than others but have difficulty quantifying that. This frailty index will enable them to put a number on their gut feeling about which patients are going to benefit and which aren’t.”

Dr Hubbard said the team includes Professor Len Gray and Dr Nancye Peel from CRGM and Professors Ken Rockwood and Arnold Mitniski from Dalhousie University in Nova Scotia. It is also looking at whether frailty status on admission was the most important factor related to outcomes.

“There is a theory that all that matters is current status,” Dr Hubbard said.

“Other people believe it may be your baseline status.

“The third school of thought is that it is your trajectory, which is the difference between your baseline and your current status, that is most important.

“We are going to do some mathematical modelling to tease out which of these three is most likely to be the most helpful for our patients.

”A better understanding of frailty could enable us to target health care resources more effectively and appropriately.”

Inflammation, Infection and Immunity

There is a wealth of medical research talent in the Metro South Health precinct, both present and hovering in the wings waiting to emerge. This came to light at a recent Diamantina Health Partners (DHP) Inflammation, Infection and Immunity in Digestive Disease research workshop held at the Translational Research Institute and hosted by the Department of Gastroenterology and Hepatology, Princess Alexandra Hospital (PAH) on 16th May 2014.

In addition to bringing together the DHP to showcase current research and to forge new collaborations, the challenge was to stimulate and integrate clinicians and basic scientists. The workshop brought together clinicians and scientists sharing a keen interest in how to better understand and improve the management of highly prevalent disorders of the digestive tract, including liver disease.

To accommodate the time constraints that clinicians typically experience, the format of the workshop aimed to cover a broad spectrum of work in a very short period of time. The combination of Mini-State-of-the-Art presentations and very focussed research presentations provided valuable overall insight into ongoing and emerging medical research in the Metro South.

While the meeting was based on a strong research interest this DHP meeting took the collaborative approach to a completely different level by not only involving clinicians and clinical scientists, but reaching out to basic scientists from a variety of disciplines. The meeting leveraged not only on the skills and expertise available at the TRI and the PAH, but also those of scientists from the basic sciences and neurosciences based at The University of Queensland St. Lucia campus, and the Queensland Institute of Medical Research.

If success can be judged by comments of attendees, then this was truly a successful and valuable workshop, with attendees leaving with a spring in their step as they contemplated how they can collectively with new collaborators, undertake quality medical research and truly make a difference as they strive towards targeting and controlling chronic disease.

Integrated Trauma and Recovery

First “ROADMAP to RESEARCH” Course in Australia at TRI

The first course “Roadmap to research” for 20 surgical residents and registrars was held over two evenings, on the 3rd and 4th June 2014, at the Translational Research Institute (TRI). Professor Michael Schuetz with assistance from Professor Mark Smithers organised this event for young doctors interested in surgery, through the PAH Division of Surgery and the Diamantina Health Partners. The course was run by Dr Beate Hanson, Director at the AO Clinical Investigation and Documentation Centre – Zurich / Switzerland and Adjunct Professor for Epidemiology at the University of Seattle, who has extensive experience in running similar courses in Europe and the USA.

The aim was to provide introductory training on the role of evidence based medicine, specifically focusing on how to critically analyze and appraise clinically based trials and studies. This also provided basic insights into how one might design and conduct their own studies.

The “Roadmap to research” course was the first course of this type in Australia. The six hour course structure was interactive with presentations and tasks performed by the participants. The tasks included:

  • The formulation of research questions
  • considering the best study design
  • sample size calculation
  • appropriate analysis of the data obtained when conducting a study.

Dr Beate Hanson explaining how
the Internet can be used to help
you find practical information on
how to run clinical studies

As a trained surgeon and epidemiologist, Dr Hanson offered a very practical, hands on course, which was highly valued by all participants. The two interactive evenings were an excellent starting point for young clinicians encouraging them to get involved with research projects in the future.

The organisers plan to run this workshop annually at the TRI. The course organisers are grateful to Dr Beate Hanson and to the participants “who will be the first of many groups that will attend similar courses, on our campus, in the future”.

Articles from high-impact journals were critically analysed and the weaknesses and or strengths were appraised.

Evidence and Innovation into Clinical Practice

Associate Professor Ian Scott, Leader of the DHP Evidence and Innovation Theme, recently published a paper in the Australian Health Review 2014 38: 125-133 – Ten clinician-led strategies for improving value of health care – which has attracted attention and resulted in being invited to attend workshops at the Grattan Institute and the Productivity Commission in Melbourne to discuss the strategies further.

These 10 clinician-led strategies are:

  1. minimise errors in diagnosis
  2. discontinue low- or no-value practices that provide little benefit or cause harm
  3. defer the use of unproven interventions
  4. select care options according to comparative cost-effectiveness
  5. target clinical interventions to those who derive greatest benefit
  6. adopt a more conservative approach nearing the end of life
  7. actively involve patients in shared decision making and self-management
  8. minimise day-to-day operational waste
  9. convert healthcare institutions into rapidly learning organisations
  10. advocate for integrated patient care across all clinical settings

Professor Scott is the inaugural chair of the recently established Australian Deprescribing Network: a national collaboration of doctors, clinical pharmacists and researchers interested in optimising quality use of medicines in patients with complex diseases, with focus on discontinuing inappropriate medications.

The Society of Hospital Pharmacists of Australia recently held a meeting on deprescribing in Brisbane. Associate Professor Scott presented “Evidence-based medicine applied to older populations – the case for deprescribing” and stated that “deprescribing processes need to identify patients at high risk of adverse drug events, evaluate drug utility with particular reference to expected life span and care goals, prioritise drugs for discontinuation and then implement discontinuation regimen with close monitoring”.

Associate Professor Ian Scott

Associate Professor Scott presented on 28 May 2014 at the Measuring Health Outcomes to Inform Policy conference in Melbourne on the topic Using outcome data to improve health service delivery. His presentation focussed on the application of research outcomes: using outcome data as well as clinical process data to evaluate the effectiveness and appropriateness of care, obtained from prospective registry or cohort studies which use data linkage, and which incorporate patient-reported measures of function and quality of life as well as discrete end-points of death or clinical events.

Ms Areti Gavrilidis, Executive Consultant DHP, attended the conference and a pre-conference workshop. The pre-conference workshop program on the 26 May run by Professor John McNeil. The following was highlighted: the purposes of outcome measurement what might be measured as the outcome of epidemiological pitfalls discussed examples of outcome measurement such as mortality rates, hospital readmission rates, unexpected return to theatre rates examples of clinical registries for outcome measurement filling in gaps outcome data and payment for performance.

Two full days of stimulating presentations and panel discussions followed on 27-28 May. Professor Fiona Stanley AC, Founding Director and Patron of the Telehealth Institute for Child Health Research, gave the opening keynote address highlighting the importance of good linked data for effective health system evaluation. Other areas covered the first day by recognised experts included: collecting data to inform policy and strategic planning what outcomes consumers expect from healthcare improving health outcomes through policy design with presentations on efficiency and effectiveness of health expenditure policy innovation medicare locals' role in system change. The second day of the conference focussed on developing policies to inform budget decisions using data to improve service delivery and improving patient outcomes.

The Australian Society for Medical Research (ASMR) Queensland Health & Medical research awards, 2014

The Queensland Health and Medical Research Awards, an initiative of ASMR, honour the quality of scientific endeavour in Queensland. The winners were announced at the ASMR Medical Research Week Gala dinner held Friday May 30, 2014. Congratulations to TRI and DHP members for their success in these awards.

Click here to download the full list of winners and finalists.

Prime Minister visits TRI

The significance of the Translational Research Institute (TRI) to Australian medical research was demonstrated by the fact that the Prime Minister, Tony Abbott, and the Federal Minister for Health, Peter Dutton, asked TRI to host discussions on the Medical Research Future Fund (MRFF) on Monday 19 May 2014.

As part of the visit, the Prime Minister and Federal Minister for Health toured the TRI research laboratories and took part in a round table discussion with leading members of the research and university communities.

Click here for more details of the Prime Minister’s visit and comments on Australian Medical Research.

PAH designated as a Magnet facility for the third time

On 18th June 2014, over 300 nursing and multidisciplinary staff were informed by Deborah Zimmerman, Chair of the Commission for the Magnet Recognition Program, through a live broadcast in the Russell Strong Auditorium, that PAH had been designated as a Magnet facility for the third time! The audience were elated with the announcement that the document submission and site visit which assessed PAH against stringent Magnet criteria had scored a level of excellence.

Receiving the Magnet credential is the highest recognition for nursing excellence that can be attained internationally and makes PAH one of only 400 organisations around the world to have received Magnet designation – with PAH the only facility outside of the United States to achieve it for a third time .

PAH Nursing Services not only met the stringent Magnet criteria but also achieved four exemplars for exceptional high standards of nursing care, identified as best practice within the profession. The exemplary areas commended by the American Nurses Credentialing Centre (ANCC) included PAH structures and process to develop, expand and advance nursing research nursing satisfaction results how the organisation is valued by the community and how nursing leaders have guided transition during periods of planned and unplanned change.

Ms Zimmerman acknowledged the overwhelming feedback received from staff regarding the leadership, vision and guidance of Veronica Casey, Executive Director Nursing Services PAH, who has supported nurses at all levels of the organisation to achieve this outcome. The ANCC made special commendations about the multidisciplinary collaboration and team work along with various quality initiatives which PAH is leading including pressure injury prevention state-wide nurse sensitive indicators dementia and blood management. In addition, the extensive work and recognition that is received by PAH nursing at state, national and international level was acknowledged.

This success is a testament to the ongoing support of the leadership team at PAH, a collaborative interdisciplinary approach to improving patient outcomes and PAH nurses for their commitment and dedication to best practice safety, quality and patient satisfaction.

Research for a better future…

3D melanoma model helps explain therapy evasion

Researchers based at TRI from the UQ Diamantina Institute have developed a 3D model of a melanoma tumour that enables them visualise the growth states of cancer cells in real-time. In a paper published in the journal Pigment Cell and Melanoma Research, they describe how they have used cutting-edge imaging technology to develop a 3D model that precisely measures cell cycles as a melanoma tumour grows.

The detailed findings challenge the idea that melanoma cells can either invade or proliferate. In fact, they can do both. Moreover, the results suggest that cells in stasis are poor invaders. Kliknite tukaj za več podrobnosti.

Congratulations to Professor Jenny Martin on her appointment to the Pharmacology and Therapeutics Advisory Committee (PTAC) announced on 17th June 2014 by the Pharmaceutical Management Agency (PHARMAC).

Professor Martin is Head of the Princess Alexandra Clinical School at The University of Queensland and senior staff specialist and clinical pharmacologist at Brisbane's Princess Alexandra Hospital. For further information visit the PHARMAC website.

Diamantina Health Partners now has a YouTube channel!

The 2014 Diamantina Health Partners Forum presentations will soon be available for viewing on YouTube: subscribe to the DHP YouTube channel to be notified when new videos are uploaded.

Diamantina Health Partners Forum 2015

Planning is underway for the 3rd Diamantina Health Partners Forum to be held from 29 April-1 May 2015.

The theme of the 2015 Forum will be on Meeting of the Minds: inspire – innovate – implement as we – individuals, institutions and industries – work together to deliver better health.

2nd Queensland Translating Research into Practice (TRIP) Symposium "Tackling the challenges of TRIP in busy clinical settings"

The TRIP Symposium aims to enhance collaboration across Queensland in implementation science and ensure that research based evidence of best available practice translates effectively into everyday clinical practice and improved health outcomes.

This year's keynote lectures will be given by TRIP experts Professor Paul Glasziou, Dr. Shelly Wilkinson and Philippa Middleton. This event promises to present a wealth of stimulating topics and will cater for a broad range of clinical interests. We hope the symposium will allow further understanding into the various research streams currently ongoing in Queensland to promote and foster collaboration.

datum: 25 July 2014 | Venue: Des O'Callaghan lecture theatre, Mater Hospital Campus

2014 Princess Alexandra Hospital Symposium

To commemorate the first anniversary of the Translational Research Institute and the second anniversary of Diamantina Health Partners, the 2014 Princess Alexandra Hospital Symposium program is aligned to the flagship themes of DHP and its mission to achieve better health globally through the integration of clinical care, research and education. These strategic developments bring together research, teaching and treatment, to consolidate the Princess Alexandra Hospital campus at the forefront of shaping the future of health in Queensland, nationally and internationally.

Central to the Symposium program is Professor Boris Bastian, University of California, San Francisco, this year’s International Fellow. As an eminent clinical scientist, he will illustrate in his keynote address how interdisciplinary and global partnerships have advanced the understanding of the causes of skin cancers and are translating this new knowledge to novel therapies for their treatment. Professor Bastian is also this year’s Kurt Aaron Orator.

Check the website for more information about the 2014 Princess Alexandra Hospital Symposium.

datum: 4 August – 8 August 2014 | Venue: Russell Strong Auditorium, PAH

TRI-QUT Conference for the Biology and Clinical Application of Mesenchymal Stromal Cells

TRI and QUT present the 2014 Stem Cell Conference. A host of international and national speakers will address the following topics:

  • cellular and molecular biology of mesenchymal stem cells
  • mesenchymal stromal cells
  • induced pluripotent stem cells
  • bioengineering applications for stem cell therapies and the results of clinical trials using stem cells and their progenitors.

Confirmed speakers include Professor Frank Barry, National University of Ireland and Professor Katarina Le Blanc, Karolinska Institute and Huddinge Hospital, Sweden.

Registracija je brezplačna. Complimentary morning tea, lunch and afternoon tea will be available during the Symposium.

Abstracts on any aspects of stem or progenitor cells are welcome. There will be prizes for the three best abstracts submitted by students and for the three best abstracts submitted by post-doctoral fellows.

Closing date for abstracts: 1 August 2014

For event enquiries please contact IHBI Events [email protected]

datum: 10 – 11 September 2014 | Venue: Translational Research Institute, Brisbane

TRX14 "Value Found in Translation"

TRX14 aims to enhance trans-disciplinary collaborations across human disease research, drug and diagnostic discovery and development, IT and bioinformatics.

The event will bring together key stakeholders from biopharma, biotech, research and development, academia and government, providing the opportunity to interact, and catalyse possibilities to establish partnerships between organisations from around the globe.

Such partnerships will work towards improved translation of data into knowledge to achieve enhanced research outcomes and value for all stakeholders.

For further information go to http://trx14.com.au/ or email [email protected]

datum: 24 October 2014 | Venue: Translational Research Institute, Brisbane

AusBiotech 2014 National Conference

The AusBiotech national conference is now the largest annual gathering of biotechnology experts in Australia and the Asia Pacific Region. The 2014 conference will gather Australian and international biotech leaders and stakeholders for another round of quality speakers and valuable networking. In addition to a stimulating conference program, AusBiotech’s Business Matching Program will again facilitate more than 2,500 meeting requests between participants from the biotechnology, biopharmaceutical, life sciences, business, investment, research and health industries.

For further information go to http://ausbiotechnc.org/

datum: 29 – 31 October 2014 | Venue: Gold Coast Convention and Exhibition Centre

Life Sciences Queensland (LSQ) will host the I-20 event on November 14, 2014. I-20 aims to advocate for the support and continued role that innovation plays, highlighting the growing importance of the life sciences industry as a key influence upon global economic development and sustainability.

Through I-20 LSQ hopes to achieve increased awareness of the role innovation plays and to foster opportunities for cooperation amongst G20 nations—particularly with regard to addressing global issues common to all members—as well as attempt to influence national and global innovation policy for Australia.

Join the I-20 mailing list to keep informed on event updates by contacting [email protected] or keep up to date via this website

“We are all faced with a series of great opportunities - brilliantly disguised as insoluble problems.” John W. Gardner

We welcome our members' contributions. If you have anything you wish to add to our next edition, please email your full name, contact details, name of organisation and attach a word document with your contribution of no more than 350 words to [email protected]

We also welcome your feedback and encourage you to contact us with any ideas, collaborations or queries you may have: [email protected]

Telefon: +61 7 3443 8063
Location: Level 7, Translational Research Institute
37 Kent Street, Woolloongabba QLD 4102, Australia
E-naslov: [email protected]
Website: http://diamantina.org.au/

Translational Research Institute

The Diamantina Connect newsletter is a free information service for the Diamantina Health Partners and broader community and is produced by the office of the Diamantina Health Partners.


If last week's action hadn't already sent you a clear enough signal that Tesla (TSLA) shares play by their own rules, maybe Thursday did the trick.

On Thursday morning, Tesla announced that it would be selling 2.65 million shares in a stock offering. This offering was worth just over $2 billion as of Wednesday’s closing price.

Stock offerings, for the uninitiated, are dilutive. This means existing shareholders will own a smaller piece of the company after a stock offering than before. As a result, the price of a company's stock tends to drop following these events.

This is a normal thing that happens. What is less normal is a stock rallying 4.8% after such an offering. But normal things don't happen to Tesla shares.

And that is because the zgodba for Tesla right now is also not normal. Collaborative Fund's Morgan Housel on Thursday tweeted a simple outline of how investors are seeing the Tesla story.

People think Tesla will do well --> stock surge.

Stock surge --> raise tons of cash.

Tons of cash --> will help Tesla do well. https://t.co/1Qzilouk48

— Morgan Housel (@morganhousel) February 13, 2020

Again, the negative read on stock offerings is largely because these events are viewed through the prism of what investors are losing — a portion of their stake in the company — rather than what the company is gaining.

But as Matt Levine at Bloomberg outlined on Thursday, Tesla’s equity raise also sees the company use the stock market in the way it was intended, but isn’t often used. Which is primarily to raise money.

The public market allows companies to raise money from a lot of investors all at once. Except large pools of money in private equity, venture capital, and direct lending has made this option less attractive and less of a necessity.

“Big public companies now mostly use the stock market as a way to return capital to investors U.S. public companies now buy back hundreds of billions of dollars more stock than they sell,” Levine wrote Thursday. “Once you accept the consensus that public companies don’t benefit directly from their stock prices, you start to wonder why a company would want to be public at all.”

Of course, Elon Musk himself agreed not too long ago. Except now, Tesla’s stock price is up a lot. And so Musk has made the fairly straightforward decision to benefit from that increase.

Now, in its fourth quarter earnings report, Tesla said the company has grown to the point of being self funding. In other words, Tesla doesn’t — or didn’t — potrebujejo any more investor capital to continue growing at the rate it hopes to grow.

But as Housel outlines above, a way to read the rally in Tesla’s stock — both before Thursday’s announcement and particularly po this news — is to see it as the market essentially asking the company to take on more capital to grow more quickly.


First human-monkey embryos created: A small step toward a huge ethical problem

Scientists have created the world’s first monkey embryos containing human cells in an attempt to investigate how the two types of cell develop alongside each other. The embryos, which were derived from a macaque and then injected with human stem cells in the lab, were allowed to grow for 20 days before being destroyed.


Apoptosis (programmed cell death)

Povzetek

Organizers: John A . Cidlowski and J . John Cohen March 5.11. 1995 Tamanon. Colorado Page Plenary Sessions March 6 Physiological Regulation of Apoptosis . 258 Cell Surface Activation of Apoptosis . 259 March 7 Signaling: Intracellular Pathways . 259 Apoptosis Genes: Genetic Approaches . 261 March 8 Enzymology of Apoptosis . 261 Anti-Apoptosis Genes . 261 March 9 Recognition of Apoptotic Cells . 262 Apoptosis/Cancer/Aids . 263 March 10 Apoptosis. Reprodukcija. Development. Aging . 263 Neural Apoptosis . 264 Late Abstracts . 265 Poster Sessions March 6 Poster Session I (B8- 100-1 55) . 266 March 7 Poster Session 2 (B8-200-253) . 280 March 8 Poster Session 3 (98-300-353) . 293 March 10 Poster Session 4 (98-400-452) . 307 Lute Abstrncts . 321 257 Apoptosis (Programmed Cell Death) Physiological Regulation of Apoptosis BE001 INTRACELLLL4R AN11 EXTRACEI.LULAR SIGNALS IN PROGR

I) CELL DEATII AND APOPTOSIS, Richard A. Lockshin,' Fheresa Latham,'.', Jana Jochovi,'.' and Zahra Zakeri'. 'Department of Biol. Sci.. St. John's University, Jamaica NY 1 I439 and 'Department of Biolop, Queens College and Graduate Center of CLW, Flushing. 11367. The programmed cell death that characterizes metamorphosis of mammalian secretory tissues and many insect tissues, such as those of muscle and


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