Radboud and UMCG awarded major European grant for rare children’s disease

Researchers from the RadboudUMC and the UMCG in a European consortium have been awarded funding of € 7.3 million to carry out research into rare diseases as part of the European Framework Programme FP7. The grant is for an international research project (BetaCure) among children with congenital hyperinsulinism. The project will be headed by nuclear physician Martin Gotthardt (RadboudUMC) in association with surgeon Go van Dam (UMCG).

BetaCure is developing specific radioactive and optical fluorescent contrast agents to enable surgeons to discern insulin-producing cells (beta cells) in the pancreas. In addition, the ‘targeting’ concept will be used to make the beta cells more visible during operations on children with hyperinsulinism, or to enable them to be destroyed selectively without the need for major surgery.

Hyperinsulinism

Approximately 1 in every 50,000 babies is born with the congenital disease hyperinsulinism. A genetic defect means that the beta cells in the pancreas of these children produce too much insulin. As a result, the children have a constant risk of a low blood glucose level, which can lead to serious brain damage. Without prompt intervention, most children would die. But even when treatment is available and on time, it is not always the perfect solution. More than three-quarters of patients do not respond to medication, making surgical removal of the pancreas the only option. This major procedure (which is only performed in a few centres in Europe) can save their lives. However, it causes life-long diabetes and chronic digestive problems, and carries the usual risk of potential complications for very young children who undergo major surgery.

The aim of the BetaCure project is to use innovative nuclear and optical imaging to give an impression of the extent of beta cell overproduction. The next step would be selective elimination of the overproductive cells by means of a minimally invasive procedure, such as laparoscopic surgery or photoimmunotherapy.

Contrast agents

The consortium will work alongside centres in Germany, Switzerland and Great Britain to develop contrast agents in the Department of Nuclear Medicine and Molecular Imaging (Elsinga and Brouwers) and the GMP facility (Lub-de Hooge) of the UMCG. Industrial partners and paediatric surgeons in London and Berlin will help to develop a special laparoscope, which will detect fluorescent signals and allow surgeons to remove the cells selectively.

The method also shows promise for treating other abnormalities, such as insulin-producing tumours in the pancreas in adults, or monitoring the function of insulin-producing cells after a pancreas transplant or beta cell transplant.