Richard Boyd

Australian researchers have developed an advanced method of growing 3D models of children’s tumours — ‘tumouroids’ containing living cancer cells from the children — to enable high-throughput drug testing to find the best treatment for each child, quickly and efficiently. Published this week in the international journal EMBO Molecular Systems Biology, this development could prove a game-changer for precision medicine programs around the world — including Australia’s own Zero Childhood Cancer Program (ZERO), jointly run by Children's Cancer Institute and Kids Cancer Centre at Sydney Children’s Hospital — which aim to match patients to treatments in an effort to achieve the best possible clinical outcomes. Professor Maria Kavallaris AMHead of the Translational Cancer Nanomedicine Group at Children’s Cancer Institute, who led the research, said that the new method overcomes a number of hurdles that have limited the use of tumour testing methods in the past. These include having insufficient patient tumour samples to develop a relevant model from, and the time involved in developing such a model, with traditional methods taking months and the new method taking just days. “Determining which treatment is best for which child in a clinically useful timeframe is a major challenge for precision medicine programs such as ZERO,” Professor Kavallaris commented. “The technology we’ve developed is a major advance because it allows us to rapidly grow tumours — including cancers that have been very difficult to grow in the lab in the past — that maintain the features of the original sample, meaning that they are truly representative of the patient’s tumour. This allows us to test for drug sensitivities not only quickly, but with confidence that the results are relevant.” “This development has the potential to significantly improve personalised cancer care, as well as accelerate the development of new effective therapies for children with cancer.” To develop the model, the researchers used advanced 3D bioprinting technology and a specially engineered matrix (hydrogel system) that mimics a tumour’s natural environment. Using cancer cells from children with two of the more commonly diagnosed childhood tumours, neuroblastoma and sarcoma, they then created patient-specific ‘tumouroids’. When the researchers tested their 3D bioprinting approach in the high-throughput drug screening platform used by ZERO, they found it to be both compatible and feasible. “Our approach represents a major advance in cancer modelling and drug testing, and provides a powerful new tool for precision oncology,” said Professor Kavallaris. “We believe it holds great promise for improving survival in children with high-risk cancers.” Key funding bodies for the research included Cancer Council NSW and the National Health and Medical Research Council (NHMRC). Read more at https://lnkd.in/gfvakqeM

231

3 Comments

🔍 Spotlight on a high-impact review in Antibody Therapeutics (IF = 4.5) 🧬 Next Generation of Multispecific Antibody Engineering This in-depth review by Daniel Keri, Matt Walker, Isha Singh, Kyle Nishikawa and Fernando Garces from Gilead Sciences outlines current challenges and strategic solutions in engineering multispecific antibodies — from molecular design to controlled quaternary assembly. 🔗 Free access to the full article: https://lnkd.in/gA5SEHSy #antibodies #antibody #antibodytherapeutics #mabs #mab #antibodydiscovery #antibodydevelopment #biologics

46

2 Comments

AKV9 Targets Aggregation, Showing Promise in Both ALS and AD | https://lnkd.in/gkA6Vvvw AKV9, a small molecule discovered in the Silverman lab and advanced by Akava Therapeutics, inhibits protein aggregation via an intracellular, lysosome-dependent mechanism. Originally developed for ALS, AKV9 also shows promise in Alzheimer’s disease by blocking Aβ oligomerization. With oral bioavailability, in vivo efficacy, and low toxicity, AKV9 represents a structurally simple, mechanistically intriguing candidate for multiple neurodegenerative diseases. Read it on Drug Hunter: https://lnkd.in/gkA6Vvvw

201

3 Comments

🌠 Rising star of eye research Dr Jiang-Hui (Sloan) Wang is bringing his cutting-edge gene therapy techniques from the United States to Australia. His new CERA lab will be one of only a few capable of developing new viral vectors to treat inherited retinal diseases (IRDs) involving large genes, like Usher syndrome. 🧬🔬 Usher syndrome type 1B, caused by mutations in the MYO7A gene, leads to profound hearing loss, progressive vision decline from teenage years and balance problems. IRDs with large genes were once considered untreatable, but gene therapies that can correct faulty genes and prevent vision loss are emerging. However, delivering the full corrected gene is challenging. "The MYO7A gene that causes Usher syndrome is too large for a single AAV to deliver to fix the mutations," says Dr Wang. While the AAVs are safe, the deep injections risk damaging the cells they aim to treat. Dr Wang's revolutionary approach uses less invasive injections to deliver the complete gene into the eye. By splitting MYO7A and reassembling it inside the retina using an engineered viral vector, he aims to reverse retinal degeneration in a mouse model of Usher syndrome. Dr Wang developed these cutting-edge techniques under world-renowned gene therapy expert Professor Guangping Gao at UMass Chan Medical School and will now advance this technology in Australia for future clinical use. Dr Wang is deeply appreciative his work is being supported by CERA’s donor community: “These generous gifts will make a meaningful difference in accelerating our efforts to develop gene therapy for Usher syndrome.” “If successful, this method could offer a safe and efficient way to treat other IRDs with large genes, using the same injection approach, and will benefit teams working to develop gene therapies across CERA.” Read the full story from our latest edition of Visionary: https://ow.ly/yRXr50X7xgJ

28

2 Comments