Prof. Helen Lachmann
PP-26825
So before the availability of IL-1 blocking drugs, for most of these patients there was no effective treatment. The only treatments that worked were high dose corticosteroids at a dose of either a milligram per kilogram or half a milligram per kilogram, so essentially at extremely toxic doses that couldn't be maintained. And so either these patients came to us with severe corticosteroid damage or they'd never been treated. And to some extent, the patients who had not been treated were in better states. But the availability of IL-1 blocking drugs and the recognition that they were useful from the early 2000s onwards has been completely transformative. And particularly for the diseases for which there was no previous treatment and that is CAPS in this case. It has totally transformed patient outlooks. And since this is a dominant disease, this, when you get a family, you will get quite a large number of affected family members. And because this presents in childhood, it's not just the affected family members, it's the unaffected family members as well, because disease in childhood affects the whole family really severely. So this has been, from the point of view of a treating physician, it has been essentially a medical fairy tale. The ability to effectively control disease has been transformative for everybody involved in the patient pathway. And it's been a really nice story (Lachmann H, 2024).