Drug to delay Type 1 Diabetes approved by NICE

Diabetes experts from across Birmingham Health Partners have welcomed the approval of teplizumab for use on the NHS, following an announcement by the National Institute for Health and Care Excellence (NICE).

The immunotherapy drug can delay the progression to symptomatic type 1 diabetes in people who are in stage 2 of the condition, and will now be available through the NHS for children over 8 years and adults with Stage 2 Type 1 Diabetes.

The NICE announcement follows work by academics and clinicians to improve the support available for children and young people who go onto develop type 1 diabetes. At BHP – via founding members the University of Birmingham, Birmingham Women’s and Children’s Hospitals and University Hospitals Birmingham NHS Foundation Trusts – research is ongoing through a screening programme which can help to identify young people at risk of the condition.

The ELSA (EarLy Surveillance for Autoimmune diabetes) study, led by Professor Parth Narendran, has seen thousands of young people screened for antibodies that act as markers for whether type 1 diabetes will develop later in life.

Today’s announcement recognises the crucial role that screening programmes, including ELSA, will have for identifying young people who will benefit from Teplizumab.

ELSA has also enabled patients to have early access to Teplizumab, with the first young person in the UK, 14-year-old Sam from Kings Norton, receiving the drug at Birmingham Children’s Hospital under the care of Dr Renuka Dias and her specialist team at the Clinical Research Facility. Now, with the approval for funded use on the NHS it is estimated that around 1,100 people could be eligible for teplizumab in the first year-based data provided from the ELSA study.

Parth Narendran, Professor of Diabetes Medicine at the University of Birmingham said: “Today’s NICE recommendation for teplizumab marks a significant milestone in the UK for people at the very earliest stages of type 1 diabetes.

“As the first disease-modifying therapy shown to delay progression to clinical, insulin-requiring type 1 diabetes, teplizumab has the potential to transform the treatment paradigm from reacting to disease onset to intervening earlier in the disease process. This means that patients identified early, for example through the ELSA study led by the University of Birmingham, will have benefit from treatment that can give valuable additional years free from the daily burden of managing type 1 diabetes.

“This decision will go a long way to help the development of screening, monitoring and prevention pathways that will underpin the future of type 1 diabetes care.”

Type 1 diabetes is an autoimmune condition that causes the body to attack and destroy insulin-producing cells in the pancreas. Without insulin, blood glucose levels rise dangerously, and, without treatment, death. Diagnosis often comes suddenly and in crisis – one in four children in the UK are diagnosed in diabetic ketoacidosis (DKA), a life-threatening emergency caused by extremely high blood glucose levels and requires emergency treatment in hospital.

Teplizumab targets the immune system’s attack on insulin-producing beta cells in the pancreas. By modulating the immune response, it can delay the onset of clinical type 1 diabetes in people who are in stage 2.

Clinical trials have shown that a single course of teplizumab, which is administered in hospital daily for 14 days, can halve the progression rates to symptomatic type 1 diabetes.

Following today’s announcement, NHS will need to develop new testing and treatment pathways to make teplizumab available in practice.

Work is already under way to help build this infrastructure. The ELSA study, which is  co-funded by Diabetes UK and Breakthrough T1D, demonstrated what childhood screening for type 1 diabetes might look like in the real world.

A second phase, ELSA 2, was launched with £1.5 million in funding and has expanded screening to all children aged 2 to 17 across the UK and aiming to recruit a further 30,000 children.

ELSA 2 will also establish new NHS Early-Stage Type 1 Diabetes Clinics, providing families with clinical and psychological support and creating a clear pathway from screening through to diagnosis, monitoring and treatment.

Dr Renuka Dias, a Consultant Paediatric Endocrinologist working at Birmingham Women and Children’s Hospital and is an Honorary Associate Clinical Professor at the University of Birmingham and the Lead Paediatrician for the ELSA study.

Dr Dias said: “The approval of teplizumab by NICE offers people with early-stage Type 1 Diabetes to delay the need for insulin potentially for several years and represents a genuine step-change in how we think about the management of this relentless condition and hope that in time we can move closer to managing Type 1 diabetes without insulin as the first-line therapy.”

The announcement that teplizumab will be available on the NHS will give young people and adults more time to prepare for living with type 1 diabetes, with trial data suggesting that the drug can delay the onset of symptomatic diabetes by an average of 2.5 years.

As well as teplizumab, academics at the University of Birmingham are researching other ways to preserve the cells that produce insulin in the pancreas, and which are lost in type 1 diabetes.

Colin Dayan, Professor of Clinical Diabetes and Metabolism is collaborating with colleagues across the UK in the Type 1 Diabetes Immunotherapy Consortium to conduct clinical trials in adults and children with new-onset type 1 diabetes and develop new approaches to beta cell preservation and ultimately aim to find ways to manage type 1 diabetes without the need for insulin dependency.

Professor Dayan said: “This is such an exciting moment – it is the first step towards treating type 1 diabetes without insulin. Teplizumab delays the need for insulin by more than 2 years. Many other drugs have also shown promise in slowing the damage to insulin making cells by the immune system. As we pick up more cases early by screening and combine treatments to retain the body’s insulin making cells for longer and longer, we can foresee the day that insulin treatment in children becomes a thing of the past.”