02:24 , Mar 29, 2019 |  BC Innovations  |  Targets & Mechanisms

Next-wave targets at AACR 2019 start filling the blanks in the tumor microenvironment

New and emerging targets at AACR 2019 show cancer researchers are gaining granularity on how to manipulate the tumor microenvironment, a top priority goal for the field. The targets point to emerging strategies for modulating...
17:54 , Nov 1, 2017 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: October 2017

New Therapeutic Targets and Biomarkers: October 2017 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during October 2017. Therapeutic targets are defined as any protein, gene or other...
19:42 , Oct 3, 2017 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Alzheimer's disease (AD) Mouse and cell culture studies suggest inhibiting PCDHGC5 could help treat AD. In cortical samples from a mouse model of AD, levels of PCDHGC5 were higher than in samples from normal...
21:08 , Jan 27, 2017 |  BC Week In Review  |  Clinical News

Ganaxolone: Preliminary Ph II data

Preliminary data from 4 patients with cyclin dependent kinase like 5 (CDKL5) epilepsy in an open-label, U.S. and Italian Phase II trial showed that once-daily doses of up to 1,800 mg/kg oral ganaxolone for an...
07:00 , Oct 24, 2016 |  BC Week In Review  |  Clinical News

Ganaxolone: Additional Phase II data

Additional data from 11 female patients ages 4-15 with protocadherin 19 (PCDH19) epilepsy and uncontrolled seizures despite antiepileptic pharmacotherapy in an open-label, U.S. and Italian Phase II trial showed that adjunct treatment with up to...
07:00 , Jun 16, 2016 |  BC Innovations  |  Translation in Brief

Dysfunction junction

Researchers from Memorial Sloan Kettering Cancer Center have shown brain metastases build gap junctions to flood neighboring astrocytes with pro-inflammatory signaling molecules, causing the cells to secrete cytokines that fuel tumor growth via a paracrine...
07:00 , Jun 2, 2016 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: Gap junction protein α 1, 43kDa (GJA1; CX43; connexin-43); protocadherin 7 (PCDH7)

Cancer INDICATION: Breast cancer; non-small cell lung cancer (NSCLC); cancer Patient sample and mouse studies suggest inhibiting GJA1 or PCDH7 could help treat brain metastases of breast cancer, NSCLC and other solid tumor types. In...
08:00 , Nov 2, 2015 |  BC Week In Review  |  Clinical News

Ganaxolone: Phase II data

An open-label, U.S. and Italian Phase II trial showed that 5 of 8 PCDH19 female pediatric epilepsy patients ages 2-18 who received once-daily oral ganaxolone for up to 26 weeks achieved a >50% reduction in...
07:00 , Apr 6, 2015 |  BC Week In Review  |  Clinical News

Ganaxolone regulatory update

FDA granted Orphan Drug designation to ganaxolone from Marinus to treat epilepsy in females with a mutation in the protocadherin 19 (PCDH19) gene. Ganaxolone is in Phase II testing for the indication in girls ages...
08:00 , Feb 23, 2015 |  BC Week In Review  |  Clinical News

Ganaxolone: Phase II started

Marinus began an open-label, U.S. Phase II trial to evaluate daily ganaxolone given as either oral liquid suspension or capsules for up to 26 weeks in about 10 girls ages 2-10 with confirmed mutations in...