---

A LightCure on the Horizon: With Congenital Hyperinsulinism International as Advocacy Partner, European Scientists are Developing a Potential New Photodynamic Therapy Treatment for Congenital Hyperinsulinism

Read the CHI LightCure Press Release here.

Congenital Hyperinsulinism International (CHI) is excited to announce we are part of a new research consortium called LightCure. The LightCure Consortium is developing a targeted photodynamic therapy, that one day could be a promising treatment for congenital hyperinsulinism (HI). HI is the most frequent cause of severe, persistent hypoglycemia in newborn babies and children, occurring in approximately 1/28,000 births. HI can be life-threatening with few treatment options; complications of the condition can include brain injuries. developmental disorders and epilepsy.

The scientific researchers who are a part of the LightCure consortium hypothesize that via a minimally invasive process, light can be used to target and eliminate improperly working pancreatic cells. This project is made possible by a grant of just over €8.2M from the European Union research arm Horizon Europe and encompasses CHI’s work to strengthen patient engagement and raise worldwide awareness of HI and its myriad challenges.

“If this approach is successful, it would be an incredible breakthrough,” says Julie Raskin, CEO of CHI. “We love working side by side with scientists on this important project. By including our organization, the consortium has demonstrated its recognition of the important role the patient and caregiver community play in raising awareness of the consequences of late diagnosis and in developing patient-reported outcome measures, which are needed in the treatment development process.”

As the second highest-funded LightCure partner at just above €1.4M over 72 months, CHI’s role in the project is to raise awareness of the need for universal newborn screening so that in the future babies born with HI do not develop neurologic and/or developmental secondary and avoidable conditions. As part of this work, CHI will develop and launch a Glucose as a Vital Sign campaign, which will be a graphic and text awareness campaign for the general public and medical professionals. CHI will also produce a documentary film about living with HI that will be translated into multiple languages. CHI will galvanize the global community of affected individuals and families to participate in communicating to medical professionals the consequences of late diagnosis. The grant will also add to CHI’s ability to educate medical professionals on the condition and related clinical research at conferences and meetings. CHI will also disseminate information to the CHI Community and general public about the LightCure project, in all its phases.

The grant will also support an expansion of the global HI Global Registry (HIGR) patient registry. The HI Global Registry (HIGR) is the only patient-powered hyperinsulinism (HI) registry. HIGR contains a series of surveys designed to capture information about various aspects of being diagnosed with and living with hyperinsulinism. The LightCure will allow CHI to invest in the growth and development of HIGR including expanding languages, types of data collected, and ways for researchers to access and utilize the data. Together with other LightCure consortium partners, CHI is also tasked with developing validated patient-reported outcome measures (PROMs) which will be used to better understand how HI affects a person’s quality of life, perceptions of overall health, symptoms, and others aspects of health and well being. This work will contribute to a better understanding of the burden of disease for people with HI and their families. This initiative will also provide researchers with a way to more effectively measure, not only the current impact of the condition, but also any benefits conferred by more effective future treatments.

Finally, as a true indicator of the centricity of the patient community in the LightCure project, CHI is tasked with creating an active patient advisory board to enhance shared therapy decision-making, and to advise the consortium partners on the project.

The 11-member consortium, led by Martin Gotthardt, of Stichting Radboud Universitair Medisch Centrum (Radboud) in the Netherlands hopes that by the end of this decade, physicians will have full human use of the photodynamic therapy, which would be applied shortly after birth and tailored for individual patients to best eliminate the side effects of existing treatments. Preclinical testing has produced extremely encouraging results. “We can foresee a day when, as a newborn is found to have HI, that baby’s quality of life will not be compromised,” Gotthardt says. “This development means that within little more than a handful of years, families may no longer have to choose between evils, faced with the replacement of HI with another disease characterized by severe secondary morbidity.” Currently, to treat HI, many patients undergo pancreatic surgery, which results in patients developing diabetes and pancreatic insufficiency.

To develop this cutting-edge therapy, scientists at the consortium member research centers have successfully isolated an extended amino acid chain that can be modified to bind only to the pancreatic cells responsible for the overproduction of insulin, the root of HI and its complications. The modification entails equipping the amino acid chain, a polypeptide called EX, with a photosynthesizer shown to inhibit the problematic beta cells. The photosensitizer, 700DX, can be activated by light of a specific wavelength to produce radical oxygen species leading to damage – or, if necessary, inducing total destruction – of only the cells in question; hence, the principle of targeted photodynamic therapy (tPDT).

Further, by dosing the photosynthesizer-equipped polypeptide, or EX700DX, to a particular patient and then activating it by exposure to light of a predefined wavelength, the treatment provides double specificity. This will allow physicians injecting their patients with EX700DX to normalize the patients’ blood-glucose levels, thereby avoiding the morbidity associated with current HI management.

HI requires complex management to maintain stable blood sugar. This can include multiple treatments, strict feeding schedules, or G-tubes to ensure continuous feeding. This can put a burden not only on the individual living with HI, but also on their family, especially their caregivers.

In addition to CHI and Radboud, the participating LightCure organizations are:

• Erasmus Universitair Medisch Centrum Rotterdam, the Netherlands
• Charité Universitaetsmedizin Berlin, German
• Hochschule Fuer Angewandte Wissenschaften Hamburg, Germany
• Medizinische Universitaet Wien, Austria
• piCHEM Forschungs- und Entwicklungs GmbH, Austria
• TRACER Europe B.V., the Netherlands
• Queen Mary University of London, United Kingdom
• Great Ormond Street Hospital, United Kingdom
• University College London Hospital, United Kingdom

You can read more about the LightCure project in the two articles linked below:

Prevent life-threatening low sugar in babies with laser light

How laser therapy prevents babies’ life-threatening disease