Results 141 to 150 of about 4,926,176 (337)

'Fast-track' drug approvals hit speed bumps in Japan [PDF]

, 2004
Douglas Sipp
openalex   +1 more source

Adaptaquin is selectively toxic to glioma stem cells through disruption of iron and cholesterol metabolism

Molecular Oncology, EarlyView.
Adaptaquin selectively kills glioma stem cells while sparing differentiated brain cells. Transcriptomic and proteomic analyses show Adaptaquin disrupts iron and cholesterol homeostasis, with iron chelation amplifying cytotoxicity via cholesterol depletion, mitochondrial dysfunction, and elevated reactive oxygen species.
Adrien M. Vaquié, Davod R. Shah, Eliane E. S. Brechbühl, Michael McNicholas, Zhaoyang Xu, John H. Stockley, Laura Morcom, Diana Gold Diaz, Gemma C. Girdler, Rachel V. Seear, Gabriel Balmus, Rajiv R. Ratan, Harry Bulstrode, James A. Nathan, Manav Pathania, Kevin M. Brindle, David H. Rowitch   +16 more
wiley   +1 more source

Despite surge in orphan drug designations, approvals still lag [PDF]

, 2011
Monica Heger
openalex   +1 more source

Patient‐specific pharmacogenomics demonstrates xCT as predictive therapeutic target in colon cancer with possible implications in tumor connectivity

Molecular Oncology, EarlyView.
This study integrates transcriptomic profiling of matched tumor and healthy tissues from 32 colorectal cancer patients with functional validation in patient‐derived organoids, revealing dysregulated metabolic programs driven by overexpressed xCT (SLC7A11) and SLC3A2, identifying an oncogenic cystine/glutamate transporter signature linked to ...
Marco Strecker, Keren Zohar, Martin Böttcher, Thomas Wartmann, Henry Freudenstein, Maximilian Doelling, Mihailo Andric, Wenjie Shi, Or Kakhlon, Katrin Hippe, Beatrix Jahnke, Dimitrios Mougiakakos, Franziska Baenke, Daniel Stange, Roland S. Croner, Michal Linial, Ulf D. Kahlert   +16 more
wiley   +1 more source

Implications of Evolving Disease Classification for Drug Approval in Juvenile Idiopathic Arthritis. [PDF]

Paediatr Drugs, 2022
Case SM, Nigrovic PA.
europepmc   +1 more source

Predictors of response and rational combinations for the novel MCL‐1 inhibitor MIK665 in acute myeloid leukemia

Molecular Oncology, EarlyView.
This study characterizes the responses of primary acute myeloid leukemia (AML) patient samples to the MCL‐1 inhibitor MIK665. The results revealed that monocytic differentiation is associated with MIK665 sensitivity. Conversely, elevated ABCB1 expression is a potential biomarker of resistance to the treatment, which can be overcome by the combination ...
Joseph Saad, Rhiannon Newman, Elmira Khabusheva, Sofia Aakko, Eric Durand, Mahesh Tambe, Heikki Kuusanmäki, Alun Parsons, Juho J. Miettinen, Komal Kumar Javarappa, Ezgi June Olgac, Nemo Ikonen, Mika Kontro, Kimmo Porkka, Heiko Maacke, Janghee Woo, Ensar Halilovic, Caroline A. Heckman   +17 more
wiley   +1 more source

Regulatory Disparities Between US Accelerated Approval and Japanese Authorization of Oncology Drugs: An Analysis of Evidence Quality

Clinical and Translational Science
While the accelerated approval (AA) program in the United States expedites the availability of drugs based on preliminary evidence to fulfill unmet medical needs, it has also raised significant concerns, including a lack of robust evidence of efficacy ...
Maho Iwata, Anju Murayama
doaj   +1 more source

I-ACT for Children: helping close the gap in drug approval for adults and children. [PDF]

Pediatr Res, 2023
Coppes MJ, Jackson C, Connor EM.
europepmc   +1 more source

Data from Real-World Evidence in Support of Oncology Product Registration: A Systematic Review of New Drug Application and Biologics License Application Approvals from 2015–2020

, 2023
Bhakti Arondekar, Mei Sheng Duh, Rachel H. Bhak, Maral DerSarkissian, Lynn Huynh, Kelsey Wang, John Wojciehowski, Melody Wu, Bryon Wornson, Alexander Niyazov, George D. Demetri   +10 more
openalex   +1 more source

Gene-silencing technology gets first drug approval after 20-year wait

Nature, 2018
Heidi Ledford
semanticscholar   +1 more source