August 2026 Research Update

This month added longer-term, real-world information that octreotide long-acting release (octreotide-LAR), a long-acting form of a medicine that reduces insulin release, was linked with improved blood sugar control for most children whose congenital hyperinsulinism did not respond to diazoxide. The study also described a safety picture that was mostly manageable, with some children developing gallstones or short-lived liver enzyme increases. For families, this helps put expectations around what long-term treatment and monitoring might involve when this medicine is used over years. [1]

In 23 children treated after diazoxide failure, the researchers reported that most reached normoglycemia (blood sugar in the target range) while on octreotide-LAR. Growth during treatment was described as acceptable, and the report did not describe serious treatment-related side effects during follow-up that averaged multiple years. This supports the idea that, for many children, long-acting octreotide can be used for sustained control, although the study did not compare it against other options. [1]

Side effects reported during octreotide-LAR use included gallstones in 3 children and mild, temporary liver enzyme elevations in 6 children. The study also noted neurodevelopmental delay in 10 children, but it did not show whether this was caused by the medicine, the severity or timing of low blood sugars, or other factors. For patients and caregivers, this is a reminder that outcomes like development can reflect many parts of the overall disease course, not just one treatment. [1]

For families already familiar with octreotide, this study mainly adds longer follow-up in children using a monthly (long-acting) form and describes what problems were most often seen during ongoing care. It may matter now in the sense that it clarifies what this treatment looked like in practice for diazoxide-unresponsive children, including common monitoring issues like gallstones and liver tests. What remains uncertain is how well these results apply to every type of congenital hyperinsulinism and how octreotide-LAR compares with other approaches, because the report did not include a direct comparison group. [1]

This month also brought early, lab-based evidence for a new direction in treating severe KATP-channel congenital hyperinsulinism, using medicines that activate the ghrelin receptor (a hormone pathway involved in glucose balance). In a mouse model carrying human stem cell-derived islets with a KCNJ11 knockout, ghrelin-related drugs raised blood sugar, and pairing one of them with octreotide had a stronger effect than either alone. For patients, this does not show a proven new treatment yet, but it helps explain how combination approaches might be designed for harder-to-control forms of the condition. [2]

The researchers built a human cell model meant to mimic KCNJ11-related congenital hyperinsulinism and observed that these islets released much more insulin even at low glucose than control islets. In that setting, acyl-ghrelin and relamorelin increased fasting blood glucose in mice carrying the CHI-like grafts. This supports the idea that turning on the ghrelin pathway can push glucose upward, at least in this experimental system. [2]

Relamorelin plus octreotide raised blood glucose more than expected from the two drugs alone and reduced insulin output from the human graft, including a reported reduction in human C-peptide (a marker of insulin release). The study also suggested the effect may involve somatostatin signaling within the islet (a natural "brake" hormone that can reduce insulin release). For families, this explains why researchers are interested in pairing drugs that act through different pathways to better control low blood sugar in severe forms. [2]

This is early research in lab-made human islets and in mice, so it mainly matters for what it could lead to in the future rather than changing care now. It helps clarify that ghrelin receptor agonists (including relamorelin) can raise blood sugar in a model of KCNJ11-related congenital hyperinsulinism, and that combining relamorelin with octreotide could have a larger effect on both glucose and insulin release in that setting. What remains uncertain is whether the same balance of benefits and risks would occur in children, because animal and lab findings do not reliably predict real-world patient outcomes and the study did not test long-term use. [2]