The translation of preclinical findings to effective therapies requires overcoming a set of hurdles. These challenges are faced by all entities engaged in therapeutic drug discovery. Whether they follow the traditional pharmaceutical chemistry approach or use biotechnology to manufacture new molecules, they need to identify and test promising drug candidates, and to progress them though preclinical and clinical trials to prove that they can effectively and safely meet medical needs ..read more

Finding a suitable human iPSC (hiPSCs) line is the first step of many disease modeling and cell therapy projects. Once a suitable donor has been found and hiPSCs have been generated, certain quality control (QC) measures of the cell culture to verify its identity and quality are crucial. If portions of the QC are missing or deviate from the standard, the results acquired with these cells may be unusable or may produce misleading conclusions. Below we address common QC tests and why these are important.  Here we focus on research-level projects, as clinical projects following regulatory g ..read more

CHIR99021, also known as CT99021, is a small molecule compound that has proven to be a potent and versatile tool in the world of stem cell biology and regenerative medicine. It acts as a selective inhibitor of glycogen synthase kinase 3 (GSK-3) with a high affinity for GSK-3β. GSK-3 is a serine/threonine kinase that plays a crucial role in various cellular pathways. (1)   CHIR 99021 – Mechanism of Action  The mechanism of action of CHIR-99021 involves inhibiting GSK-3 activity. GSK-3 has two isoforms, GSK-3α and GSK-3β, and CHIR99021 can inhibit both isoforms. By inhibiting GSK-3, C ..read more

Part one of this blog series, Bias in AI for Precision Medicine  discussed the racial and sex-specific bias that has plagued the developing world of artificial intelligence (AI) in precision medicine; diving into why finding ways to rectify that bias is of the utmost importance for ensuring the proper ethical evolution of AI in the world of research and medicine. In addition to those biases data privacy and data protection are further causes of apprehension in the continued transition towards a more AI rich medical research space. Here we highlight some of those concerns and potential so ..read more

In recent times, interest in the therapeutic use of cells derived from induced pluripotent stem cells (iPSCs) has grown exponentially. These iPSC-derived therapies are currently being developed for the treatment of numerous diseases, including amyotrophic lateral sclerosis (ALS) and corneal epithelial stem cell deficiency ..read more

In the ever-evolving landscape of healthcare and drug development, data analytics has emerged as a pivotal force in shaping clinical trials. Clinical trial analytics, a multifaceted discipline, describes the process of deciphering and harnessing the wealth of data accumulated during clinical studies. Its overarching objective is to distill raw data into actionable insights, thereby illuminating pathways to enhance drug efficacy, safety, and patient outcomes.1  In this exploration of clinical trial analytics, we delve into the core principles, the distinctions between primary and secondar ..read more

Members of the UK Parliament were impressed and somewhat surprised recently by an exchange between the Prime Minister and the leader of the opposition during Prime Minister’s questions.1 The opponents exchanged polite introductions and proceeded to debate the issues of the day in a calm and thoughtful manner, without raising their voice. The members were in fact watching an exchange with an AI chatbot, programmed to answer questions of the type often raised in the weekly shouting match. Although no insults were traded, the MPs agreed it could well have been mistaken for a genuine exchange. Ne ..read more

In the late 1970s, Dr Mike Mulvany and Dr William Halpern developed various new techniques to better investigate small blood vessel function, including the wire myograph system. This new laboratory apparatus was designed for investigations into the physiology and pharmacology of blood vessels that were too small to mount in a traditional organ bath, meaning researchers could readily investigate intact isolated arteries smaller than 1-2 mm in diameter in an in vitro system for the first time.1,2  Today, there are several companies that manufacture these systems, and one specialist contrac ..read more

A recent law passed in the USA means that new drugs do not need to be tested on animals before human studies. With support from over 200 organizations, the "FDA Modernization Act 2.0" expands the range of models that can be used to test a compound before clinical trials.1 Under this new law animal testing can still be used, but alternative testing methods now also represent a legitimate option. In this article, we will explore the ethical implications of this new legislation, what it might mean for the use of alternative models, and why some researchers are skeptical about its impact ..read more

The motilin receptor is a heterotrimeric G-protein-coupled receptor (GPCR38) involved with regulating stomach motility.1,2 Since its discovery in 1999 researchers have been keen to develop compounds that can modify this receptor.3 In this article, we will explore everything that is known about its function, physiology, and pharmacology ..read more