LUM-201 for Children Living with Moderate Pediatric Growth Hormone Deficiency

Andrew Dauber, MD, Chief of Endocrinology, Children's National Medical Center, Washington, DC, discusses LUM-201 for children living with moderate pediatric growth hormone deficiency
Transcript:
My name is Andrew Dauber. I am the chief of endocrinology at Children's National Hospital in Washington, DC. As a pediatric endocrinologist, I see kids with all different types of endocrine disorders, but my major area of personal interest and research is in growth disorders and short stature, so I myself do research on rare genetic forms of growth disorders, but I also do a lot of clinical trials work in the whole arena of growth disorders with novel therapeutics, and various diagnostic techniques.
Great question. Actually, short stature is the most common reason for referral to a pediatric endocrinologist outside of diabetes. It's a very common reason that we're seeing kids for an evaluation, and one of the significant causes of short stature that we don't wanna miss is pediatric growth hormone deficiency.
Growth hormone is made by the pituitary glands, a gland that sits, I always describe it as, back between your eyes under it's going right under your brain. It's the master regulator of a number of different hormones, but including secreting growth hormone, and growth hormone is responsible for one of the major players obviously in causing children to grow normally.
You can have growth hormone deficiency for a number of different reasons. You can either be born with it, in a congenital form or you can acquire it a little bit later in childhood. The acquired growth hormone deficiency can happen due to a brain tumor, rarely something like the most common one being a tumor called a craniopharyngioma, but anything that impacts on the pituitary gland itself.
There's some other rare causes in children who have had radiation therapy for various cancers, severe head trauma, other things can cause growth hormone deficiency, but then there are children who are born, and it becomes more clear over time. They might have anatomic abnormalities in the way the pituitary gland forms, so not all of the connections were made correctly, and as a result, the child has growth hormone deficiency, either alone or sometimes as part of other, multiple pituitary hormone deficiencies.
There are also rare genetic causes that can lead to congenital growth hormone deficiency, or children who had brains that didn't a 100% formed correctly can also have associated growth hormone deficiency. It becomes obvious over the years of life as the child's not growing normally falling off of their growth curve, and then when we start to do a hormonal evaluation, we see that markers of how growth hormone's working, are low, suggestive of growth hormone deficiency.
Up until really the last year or two, the only available treatment for individuals with growth hormone deficiency was daily growth hormone injections, and recombinant human growth hormone has been available since the mid-1980s, and now there are, hundreds of thousands, and millions of people who have received it over the years, and it is a daily injection therapy where essentially you're replacing the hormone that has been missing.
In the last year or two, there have now been once-a-week forms of growth hormone. In the US at least, there are now three different weekly formulations of growth hormone that are approved for children with growth hormone deficiency, just a longer-acting version of that daily growth hormone.
Sure. These two trials focus on a new approach, a medication called LUM-201, and LUM-201 instead of being an injection therapy, it's an oral growth hormone secretagogue, so it's a medicine that binds to those cells in the pituitary glands and at the level above that at the hypothalamus as well. It binds to a receptor called the growth hormone secretagogue receptor. This receptor's job is to tell the brain and the pituitary gland to make more growth hormone, to secrete more growth hormone.
This new medication, LUM-201, the medication actually has been studied for a number of years, but in these new phase 2 trials, the idea was, can this oral medication help restore the growth hormone secretion in children with mild or growth hormone deficiency back to normal levels. As I said, this medicine was studied a number of years ago, but what in a later analysis, the data, what they realized was that in patients with severe growth hormone deficiency, where maybe the pituitary gland's just not going to be able to restore. There's some, permanent abnormality or something where it doesn't have the capacity. This medicine is not appropriate for those patients.
These new phase 2 trials, what they did was they selected patients who were likely to respond or have the ability to respond to an oral growth hormone secretagogue, and they did that using a strategy called a predictive enrichment marker or a PEM.