Knock out blood protein drug signals possible new treatment for Alzheimer's and diabetes

May 15, 2002

LONDON 15 MAY - Scientists at UCL have developed a drug to treat a serious medical condition linked to Alzheimer's disease and type 2 diabetes. The research, supported by the Medical Research Council (MRC) and reported in NATURE, is the culmination of 25 years of basic and clinical research and the UCL scientists hope that it may be the key to the treatment of other serious diseases.

Crucially, Professor Mark Pepys and his team have managed for the first time to remove from the human body a naturally occurring blood protein - known as SAP - that is linked to development of the disease.

Amyloidosis - a disorder in which the body's own proteins accumulate as abnormal fibres that damage organs and tissues leading to disease - claims the lives of 1,000 people in the UK every year. All parts of the body can be affected. Also amyloid deposits localised to the brain are associated with Alzheimer's disease and those in the pancreas with type 2 diabetes.

The research team, based at the UCL Centre for Amyloidosis and Acute Phase Proteins at the Royal Free Hospital, first showed that SAP from the blood contributes to amyloid deposition by sticking to amyloid fibres. They then set out to produce a drug to prevent this. Working with Roche, a new drug called CPHPC was developed that blocks the sticking of SAP to amyloid.

The NATURE research reveals how the new drug was developed and outlines the mechanisms that cause SAP to be removed from the blood. The drug glues pairs of SAP molecules tightly together and the resulting clump of protein is then promptly removed from the blood by the liver and destroyed. Disappearance of SAP from the blood greatly speeds up the removal of SAP from the amyloid deposits in the tissues. Importantly, the research means that CPHPC can also be used to remove SAP from amyloid deposits in the brain in Alzheimer's disease. The first clinical studies in Alzheimer patients are about to start.

Patients with systemic amyloidosis have already been treated with CPHPC for almost a year. There have been no side effects and their clinical condition has remained encouragingly stable.

Speaking today, Professor Pepys said:

'Our experimental studies identified SAP as a key suspect in the development of amyloidosis and we aimed to find a drug that could block its effects.

'Remarkably our drug produces a complete knockout of the single protein in the blood that we targeted. It's the first time that this has been achieved with a small molecule drug. Understanding the way the drug works suggests applications of the mechanism we have discovered to other proteins in the blood that contribute to many different diseases.'

Professor Pepys continued:

'Although amyloid deposits are closely associated with Alzheimer's disease and maturity onset diabetes, it is not known whether they actually cause these diseases. But there is no doubt that our work offers real hope for systemic amyloidosis, a very serious conditions which, until now, has been difficult and dangerous to treat.'
-end-


University College London

Related Diabetes Articles from Brightsurf:

New diabetes medication reduced heart event risk in those with diabetes and kidney disease
Sotagliflozin - a type of medication known as an SGLT2 inhibitor primarily prescribed for Type 2 diabetes - reduces the risk of adverse cardiovascular events for patients with diabetes and kidney disease.

Diabetes drug boosts survival in patients with type 2 diabetes and COVID-19 pneumonia
Sitagliptin, a drug to lower blood sugar in type 2 diabetes, also improves survival in diabetic patients hospitalized with COVID-19, suggests a multicenter observational study in Italy.

Making sense of diabetes
Throughout her 38-year nursing career, Laurel Despins has progressed from a bedside nurse to a clinical nurse specialist and has worked in medical, surgical and cardiac intensive care units.

Helping teens with type 1 diabetes improve diabetes control with MyDiaText
Adolescence is a difficult period of development, made more complex for those with Type 1 diabetes mellitus (T1DM).

Diabetes-in-a-dish model uncovers new insights into the cause of type 2 diabetes
Researchers have developed a novel 'disease-in-a-dish' model to study the basic molecular factors that lead to the development of type 2 diabetes, uncovering the potential existence of major signaling defects both inside and outside of the classical insulin signaling cascade, and providing new perspectives on the mechanisms behind insulin resistance in type 2 diabetes and possibly opportunities for the development of novel therapeutics for the disease.

Tele-diabetes to manage new-onset diabetes during COVID-19 pandemic
Two new case studies highlight the use of tele-diabetes to manage new-onset type 1 diabetes in an adult and an infant during the COVID-19 pandemic.

Genetic profile may predict type 2 diabetes risk among women with gestational diabetes
Women who go on to develop type 2 diabetes after having gestational, or pregnancy-related, diabetes are more likely to have particular genetic profiles, suggests an analysis by researchers at the National Institutes of Health and other institutions.

Maternal gestational diabetes linked to diabetes in children
Children and youth of mothers who had gestational diabetes during pregnancy are at increased risk of diabetes themselves, according to new research published in CMAJ (Canadian Medical Association Journal).

Two diabetes medications don't slow progression of type 2 diabetes in youth
In youth with impaired glucose tolerance or recent-onset type 2 diabetes, neither initial treatment with long-acting insulin followed by the drug metformin, nor metformin alone preserved the body's ability to make insulin, according to results published online June 25 in Diabetes Care.

People with diabetes visit the dentist less frequently despite link between diabetes, oral health
Adults with diabetes are less likely to visit the dentist than people with prediabetes or without diabetes, finds a new study led by researchers at NYU Rory Meyers College of Nursing and East Carolina University's Brody School of Medicine.

Read More: Diabetes News and Diabetes Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.