Drug screening in a zebrafish model of SCN1A mutation has
identified lead compounds for development of novel therapies.
Through its SMA Project, a $ 32 million contract therapeutics development program that ran from 2003 until earlier this year, the agency
identified a lead compound, dubbed ALB - 111, that induces the read - through of stop codons during protein production.
Not exact matches
The objective of the research project
led by Marko Kallio, Principal Scientist at VTT, was to accelerate the drug development process by
identifying new
compounds that would possess similar binding properties and cellular phenotype, but a different chemical structure, as the selected drugs in clinical use or investigational
compounds in development.
«The
compounds identified in this study, when administered orally, both reduced the inflammation that is a hallmark of multiple sclerosis and protected against the nerve cell damage seen in mouse models of the disease,» said Jeffery Haines, PhD, a post-doctoral fellow at Mount Sinai and the study's
lead author.
«Our computer aided drug screening process has now
identified two new classes of anti-cancer agents, specifically targeting two distinct and novel mechanisms underpinning cancer,» said Dr Andrea Brancale from Cardiff University's School of Pharmacy, who
led on the
compound's design.
The problems prompted the university to suspend a clinical trial of an anticancer
compound Chen had
identified, and
led to his resignation last September.
If the
compound identified in this study successfully reduces tissue death and improves recovery in further experiments, it could
lead to new approaches for preserving brain cells after an ischemic stroke,» said Francesca Bosetti, Ph.D., Pharm.D., program director at the NIH's National Institute of Neurological Disorders and Stroke (NINDS).
«The majority of
compounds identified inhibit not just one but many kinases, and that can
lead to a number of side effects.»
The initial work
identified the precursor to GS - 5734, a small - molecule antiviral agent, which
led to the effort by Gilead and USAMRIID to further refine, develop and evaluate the
compound.
Next we use such assays to
identify LEAD chemical
compound (s) with therapeutic potential for HD or to validate drug or gene - based therapeutic approaches developed by our collaborators (Pr.
Curis Inc. in Cambridge has received a $ 5.4 million, three - year grant from the Spinal Muscular Atrophy Foundation to
identify therapeutic
compounds to treat spinal muscular atrophy, a debilitating neuromuscular disease that is the
leading genetic cause of infant and toddler death.
SCIEX is a
leading manufacturer of mass spectrometers used to
identify and characterize chemical
compounds in a variety of industrial and academic fields.
The Spinal Muscular Atrophy Foundation awarded the grant to Curis to
identify therapeutic
compounds that could be used to treat the neuromuscular disease, which is the
leading genetic cause of infant and toddler death.
«Phenotypic screens are probably underutilized for
identifying drug targets and potential
compounds,» says Daniel Tardiff, a scientist in the Lindquist lab and
lead author of one of the Science papers.
A collaboration with the Roche pharmaceutical company
led to the screening of 200 000 Roche
compounds on human neural stem cells in order to
identify proliferative drugs.
Coupled with a new gene - signature approach, this research project could
identify compounds that will ultimately
lead to the discovery of new neuropsychiatric drugs.
After developing and optimizing assays in - house, Gladstone scientists can transfer these assays to the SMDC or other external facilities to perform high - throughput screening with large
compound libraries to
identify potential drugs that may
lead to therapies.
Their goal is to
identify the
compounds that could lower levels of the protein tau in the brain, which is considered one of the most promising approaches in Alzheimer's research and could potentially
lead to new drugs to treat the disease.
Ultimately, two of the
identified genes
led to
compounds being studied in drug development projects with pharmaceutical partners.
To create the Hub, the researchers —
led by Broad chief scientific officer Todd Golub and postdoctoral scholar Steven Corsello, an oncologist at Dana - Farber Cancer Institute — scoured public and proprietary databases to
identify compounds representing a diverse range of targets and chemical structures.
Utilizing mass spectrometry and 1H - NMR (Fig. 2; Supplemental Figs. 1 — 3A), we confirmed > 95 % purity of our two
lead compounds and confirmed the structure of the
identified RIPK2 inhibitors 1 and 2.
Cosford had been investigating ULK1 using medicinal chemistry and chemical biology, and had
identified some promising
lead compounds using rational design.
Lead Optimization improvement of
compounds that have been
identified as «
leads» in early drug development.
We are pursuing both goals by studying the underlying biology, defining therapeutic targets,
identifying active molecules by high - throughput screening and developing
lead compounds.
Recalling the concern reflected in the outcome document of the United Nations Conference on Sustainable Development, entitled «The future we want», 1 that the health of oceans and marine biodiversity are negatively affected by marine pollution, including marine debris, especially plastic, persistent organic pollutants, heavy metals and nitrogen - based
compounds, from numerous marine and land - based sources, and the commitment to take action to significantly reduce the incidence and impacts of such pollution on marine ecosystems, Noting the international action being taken to promote the sound management of chemicals throughout their life cycle and waste in ways that
lead to the prevention and minimization of significant adverse effects on human health and the environment, Recalling the Manila Declaration on Furthering the Implementation of the Global Programme of Action for the Protection of the Marine Environment from Land - based Activities adopted by the Third Intergovernmental Review Meeting on the Implementation of the Global Programme of Action for the Protection of the Marine Environment from Land - based Activities, which highlighted the relevance of the Honolulu Strategy and the Honolulu Commitment and recommended the establishment of a global partnership on marine litter, Taking note of the decisions adopted by the eleventh Conference of the Parties to the Convention on Biological Diversity on addressing the impacts of marine debris on marine and coastal biodiversity, Recalling that the General Assembly declared 2014 the International Year of Small Island Developing States and that such States have
identified waste management among their priorities for action, Noting with concern the serious impact which marine litter, including plastics stemming from land and sea - based sources, can have on the marine environment, marine ecosystem services, marine natural resources, fisheries, tourism and the economy, as well as the potential risks to human health; 1.
[Poitou & Bréon] The first results published in Nature (2011 and 2013) then in Science (2014) have
identified some chemical
compounds that are present in the air and may
lead to cloud condensation nuclei (CCN) in quantities similar to those observed.
Anti-Terrorism Force Protections Specialist (2010 — 2011) • Responsible for force protection and physical security measures for 6
compounds, 100 Iraq reconstruction projects and 1600 personnel throughout Iraq • Research terrorist activities and conduct trend analysis to determine safe travel routes and safe havens • Coordinated the detection, prevention, and investigation of security vulnerabilities providing sound low cost recommendations of
identified vulnerabilities • Increase physical security for reconstruction projects and Forward Operating Base • Ensure the effective utilization of vehicle and pedestrian access control measures • Conduct ATFP level 1 training and briefings for 500 personnel •
Lead access control procedures to forward deployed camps • Extract, analyze, and distribute classified SIGACTS from CIDNE database
Anti-Terrorism Force Protections Country Manager (02/2011 — 07/2011) • Responsible for force protection and physical security measures for 7
compounds, 80 Iraq reconstruction projects, and 600 personnel throughout Iraq • Research terrorist activities and conduct trend analysis to determine safe travel routes and safe havens • Coordinated the detection, prevention, and investigation of security vulnerabilities providing sound low cost recommendations of
identified vulnerabilities • Increase physical security for reconstruction projects and Forward Operating Base • Ensure the effective utilization of vehicle and pedestrian access control measures • Conduct ATFP level 1 training and briefings for 200 personnel •
Lead access control procedures to forward deployed camps • Extract, analyze, and distribute classified SIGACTS from CIDNE database