Type 1 diabetes is a T-cell mediated autoimmune disease. For more than 40 years, researchers have tried to intervene in the autoimmune process to halt or perhaps even reverse the slow destruction of insulin producing beta cells [1]. This has proven an elusive goal, but very recently FDA granted a breakthrough therapy designation to teplizumab, an anti-CD3 monoclonal antibody  [2]. The drug modifies CD8+ T lymphocytes, which are thought to be the key effector cells that kill beta cells.

Waiving Bridging studies under certain circumstances for biosimilar applications?

A bridging study is a study performed in a new region to provide pharmacodynamic or clinical data on efficacy, safety, dosage and dose regimen that will allow extrapolation of foreign clinical data to the population in the new region [1]. However, in most cases bridging studies between an original product versus a so called foreign reference or a local reference product generate costs without providing any notable benefit for the specific patient, nor notable scientific output. 

Treatment inertia calls for an integrated personalized diabetes management: iPDM

Diabetes represents a huge and multidimensional challenge for European societes. It not only leads to premature ageing and frailty but also promotes chronic conditions like cardiovascular disease, blindness, dementia and even cancer. Despite the availability of numerous treatment options treatment inertia is still a common problem: many patents still fail to reach their treatment goals. According to the UK National Diabetes Audit data 2016- 2017, only 30% of people with type 1 diabetes and 67% of people with type 2 diabetes achieved a HbA1c target of not more than 58 mmol/l (7.5%).

Introduction

The euglycemic, hyperinsulinemic glucose clamp is the gold standard for the determination of pharmacokinetic and pharmacodynamic (PK/PD) effects of new anti-diabetic drugs, in particular insulins. In a typical glucose clamp experiment, a drug-induced decline in blood glucose (BG) concentrations is prevented by infusing glucose at a variable rate, so that BG is "clamped" at a pre-determined target level.

KomIT - the new Center of Competence for Innovative Diabetes Therapy

During the next three years KomIT, the new Center of Competence for Innovative Diabetes Therapy, will be established at the German Diabetes Center (DDZ) based on a total funding of about 3.5 Mio Euro. This funding is jointly provided by the Land North Rhine-Westphalia and the European Union. KomIT is coordinated by Professor Michael Roden, the scientific Managing Director of the DDZ.

Unmet medical needs in NAFLD and NASH

NAFLD (non-alcoholic fatty liver disease) comprises a spectrum of conditions starting with adiposity of the liver (fatty liver, steatosis hepatis) and ranging up to NASH (non-alcoholic steatohepatitis). Currently about 30% of the general population and 60-80% of people with type 2 diabetes are considered to be affected by NAFLD [1].

Premature mortality from NAFLD comes from the development of diabetes and cardiovascular disease and progression of the fatty liver to the more irreversible and aggressive NASH which again predisposes for incident liver cirrhosis and hepatocellular carcinoma [2]. Despite the potentially disastrous outcome of NAFLD and an increasingly rich knowledge concerning the underlying molecular pathogenesis no rational drugs and therapies specifically dedicated to the treatment of NAFLD are available yet. 

In this blog we first provide a brief overview on the key pathogenetic drivers in NAFLD. Then we highlight the inherent opportunities and challenges for developers of lifestyle interventions and drugs tackling NAFLD. And last but not least we discuss some key issues related to the design and implementation of clinical trials in NAFLD and NASH.

If you want to learn more on Profil’s general perception of the liver’s role in health and disease you are invited to read our „Liver first? The liver in diabetes and cardiovascular disease“ series of blogs (part I, part II, part III).

Personalized Diabetes Management in Europe

In the year 2018 Profil joined the team creating the iPDM-GO (integrated Personalized Diabetes Management Goes Europe) consortium for implementing integrated Personalized Diabetes Management in Europe. Following a thorough and competitive evaluation the iPDM-GO project at the beginning of 2019 became part of the Innovation portfolio curated by EIT Health. EIT Health is a public-private knowledge and innovation community (KIC) of best-in-class health innovators backed by the European Union. The KICs Innovation ecosystem enables a collaborative multi-stakeholder approach to implementing a more effective diabetes management in Europe.

Close the loop in diabetes care

Artificial pancreas systems are medical products that use algorithms informed by continuous glucose monitoring (CGM) data of a given patient, thereby regulating the rates of a continuous subcutaneous insulin infusion through an insulin pump. In this way, artificial pancreas systems are taking over control of the patient’s blood glucose levels.

Background

Diabetes is a multidimensional challenge for global societies. Despite the availability of drugs and technology many patients don’t reach their treatment goals.  According to the UK National Diabetes Audit data 2016-2017 only 30% of people with type 1 diabetes and 67% of people with type 2 diabetes achieved a HbA1c target of ⩽58 mmol/l (7.5%). When considering also blood pressure and cholesterol targets, these figures dropped to 19% and 41% respectively.

The good news is that diabetes leaves a huge room for Innovation. Risk factors are modifyable, type 2 diabetes is potentially reversible and the role of the patient’s self-management is of outstanding importance. Diabetes could serve as a paradigm for cracking down treatment inertia and narrowing the gap between the efficacy of investigational medicinal products seen in well controlled clinical trials and the lower than expected effectiveness of drugs observed in real-world chronic care (E2E gap) [1].

The glucose clamp is a method for the determination of pharmacokinetic and pharmacodynamic (PK/PD) effects of anti-diabetic drugs (e.g. insulin) where the blood glucose (BG) concentration lowering effect is antagonized by variable glucose infusion rates (GIR).