To win the battle against rare diseases, drug developers must work hand-in-hand with patients. By Sharon KingMy colleagues and I were privileged earlier this year to meet with a caregiver and a young patient living with a rare form of pediatric cancer, who shared their stories and inspiring words of encouragement for the biopharmaceutical industry. Their message resonated with all who were there: Together we are working toward a world where every cure is possible. It’s a big vision. Every day, more than 400 million people around the world are battling a rare disease. Many of those people are facing a scarcity of treatment options, often limited to drug regimens that haven’t been updated in decades and that do little to cure the illness or even slow down the progression of symptoms. Patient communities have long known that working together is the key to making progress, and they have created ...
The nanocage technology combines both mRNA and traditional virus-based vaccines to create virus-like efficacious vaccines A new technology developed by researchers from King’s College London (KCL) could provide a new way of delivering drugs to advance next-generation treatments to prevent and treat a variety of diseases. Published in the nanotechnology journal Small, the ‘plug and play’ technology could also lead to new therapeutics that act as both a vaccine and a drug, preventing disease and symptoms. For over two decades, a common protein called ferritin, which manages iron in all organisms, has been used to create vaccines and deliver anticancer drugs, as well as other medicines, to the body. Until now, scientists have been unable to develop a universal approach to delivering a wide range of medicines due to the protein’s unique method of self-assembly. “The inability to easily control the assembly of natural protein nanocages like ferritin has been ...
The 2024 ESMO Gastrointestinal Cancers Congress (ESMO GI) is set to take place from June 26 to June 29 in Munich, Germany. Henlius will share the latest clinical data of phase 2 study (HLX22-GC-201) of its novel anti-HER2 monoclonal antibody (mAb), HLX22, combined with HANQUYOU (trastuzumab, HLX02, trade name: HERCESSI™️ in U.S. and Zercepac® in Europe) and chemotherapy for the first-line treatment of HER2-positive gastric/gastroesophageal junction (G/GEJ) cancer with Professor Jin Li of Shanghai East Hospital, School of Medicine, Tongji University as the leading principal investigator of this study. The results from HLX22-GC-201 were first released at the 2024 ASCO Gastrointestinal Cancers Symposium (ASCO GI) in January 2024, which showed that adding HLX22 to HLX02 (trastuzumab) and chemotherapy prolonged progression-free survival and enhanced antitumour response in patients with HER2-positive G/GEJ cancer in the first-line setting, with a manageable safety profile. The data released at 2024 ESMO GI are as follows: ...
Recently, the team of Chiatai Tianqing and WuXi AppTec disclosed for the first time in Bioorganic & Medicinal Chemistry Letters the molecular design thinking of the novel CDK2/4/6 inhibitor – TQB3616 (Culmerciclib) and R&D history. Small molecule CDK4/6 inhibitors (Palbociclib, Ribociclib, and Abemaciclib) have been successively approved for the treatment of metastatic breast cancer, of which Abemaciclib has shown fewer adverse effects in the clinic attributed to its superior inhibitory activity on CDK4 kinase than on CDK6. Therefore, the research team worked to discover pyrimidine-indazole molecules that are biased to inhibit CDK4 kinase and have some inhibitory activity against CDK2 and CDK6, and TQB3616 was the preferred molecule among such small molecules obtained by SAR screening. It was found that comparing Palbociclib and Abemaciclib, TQB3616 demonstrated different degrees of inhibitory effects on CDK2, CDK4, and CDK6 kinases, and a higher inhibitory capacity for CDK4 kinase [1]. The inhibition of tumor ...
The network will support the development, evaluation and adoption of innovative health and care technology The National Institute for Health and Care Research (NIHR) has awarded an NIHR HealthTech Research Centre (HRC) Network £5m to provide national coordination and leadership for NIHR HRCs to advance health innovations. Hosted by Sheffield Teaching Hospitals NHS Foundation Trust from 1 September 2024, the network will support the development, evaluation and adoption of innovative health and care technology. In November 2023, the NIHR announced £42m in funding to support 14 new NIHR HRCs to work with businesses to support the development of medical devices, diagnostics and digital technologies to allow people to better monitor their health, make earlier diagnoses and improve the management of conditions such as cancer, dementia, cardiovascular and respiratory disease. The HRCs are located in leading NHS organisations across England, including Guy’s and St Thomas’ and King’s College London’s HRC for ...
Precision in diagnosing and categorizing tumors is essential for delivering effective treatment to patients. Currently, the gold standard for identifying various types of brain tumors involves DNA methylation-based profiling. DNA methylation functions as a regulatory mechanism to control gene activity, essentially turning genes on or off. However, the time required for such testing can be a significant hindrance, often taking several weeks—a delay that can be critical when patients need prompt decisions regarding their treatment. Additionally, these tests are not widely available in most hospitals around the world. Now, a new artificial intelligence (AI) tool has been developed to classify brain tumors more quickly and accurately. Researchers at The Australian National University (ANU, Canberra, Australia) have created DEPLOY. This new method predicts DNA methylation patterns to classify brain tumors into 10 major subtypes. DEPLOY utilizes histopathology images, which are microscopic images of patient tissue samples. The model was trained and ...
A new editorial paper was published in Oncoscience (Volume 11) on April 25, 2024, entitled, “The price of hope: CAR-T therapy in pediatric leukemia.” We stand at the crossroads of medical innovation, where cutting-edge scientific discoveries intersect with the resilience of the human body, providing hope to families grappling with a diagnosis of pediatric leukemia. In this new editorial, researcher Alex Hoover from the University of Minnesota Medical School’s Division of Pediatric Blood and Marrow Transplantation discusses the chimeric antigen receptor T-cell (CAR-T) therapy tisagenlecleucel (tisa-cel) -; a groundbreaking development in the treatment of B-cell lineage acute lymphoblastic leukemia (B-ALL) (the most common childhood cancer). Following the pivotal ELIANA trial, tisa-cel was approved in the United States for the treatment of refractory or second or greater relapse of B-ALL in patients under age 25. This innovative therapy involves genetically modifying a patient’s native T-cells – immune cells with the ability ...
Spread through Anopheles mosquitoes, the disease affected around 249 million people globally in 2022 Researchers from the Antimicrobial Resistance (AMR) Interdisciplinary Research Group (IRG) at Singapore-MIT Alliance for Research and Technology (SMART), in collaboration with the Massachusetts Institute of Technology, Columbia University Irving Medical Center and Nanyang Technological University (NTU), Singapore, have identified a link between malaria parasites developing resistance to antimalarial drugs. Affecting around 249 million people globally in 2022, malaria is a mosquito-borne disease that occurs when parasites spread to humans through the bites of infected Anopheles mosquitoes. Specifically focusing on an antimalarial drug known as artemisinin (ART), researchers used a cellular process called transfer ribonucleic acid (tRNA) modification – a mechanism which allows cells to respond rapidly to stress by altering RNA molecules within a cell. ART-based combination therapies are a first-line treatment for patients with uncomplicated malaria and help reduce the number of parasites during the ...
Mike Hollan Industry experts gather to discuss the current investor market and how the IRA may impact the industry in the coming years. Financial Times US Pharma and Biotech Summit 2024After the post-Covid crash, investors are seeing promising trends in the pharma and biotech industry in 2024. The following speakers presented a panel discussing the industry from an investors perspective at the Financial Times’ US Pharma and Biotech Summit 2024 in New York City: Harmeet Dhillon, head of public policy at GSK; Amit Sinha, chief investment officer at Goldman Sachs; Darius Lakdawalla, quintiles chair in pharmaceutical development at the University of Southern California; and Johanna Grossman, head of healthcare and US life sciences at the US Stock Exchange. Oliver Barnes, a US pharmaceutical and biotech correspondent for the Financial Times moderated the event. The conversation focused on both the current state of the industry and where each of the speakers ...
Don Tracy, Associate Editor Roche’s human papillomavirus solution is reportedly one of the first of its kind to be available in the United States. Roche announced that the FDA has approved its human papillomavirus(HPV) self-collection solution, one of the first of its kind approved in the United States. According to the company, the new tool allows individuals to collect their own samples in a healthcare setting for HPV testing, supporting early detection and prevention of cervical cancer. Analyzed through Roche’s Cobas molecule instrument, positive results require follow-up care with a healthcare provider. Roche’s fully automated Cobas 5800/6800/8800 Systems can provide up to 96 results in approximately three hours, 384 results for the Cobas 6800 System, and 1,056 results for the Cobas 8800 System in eight hours.1 “With vaccinations, innovative diagnostic tools and screening programs, achieving the WHO’s goal of eliminating cervical cancer by 2030 is within reach,” said Matt Sause, ...
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