I just finished attending the ANR parents day and information day.
One of the talks was by Holger Lode who is in charge of the LTI trials.
Under SIOPEN both LTI and STI are using the same drug, ch14.18/CHO. The main focus around the LTI trial is to look at reducing toxicity while maintaining efficacy.
A huge reason this may become very important is the recent discontinuation of GM-CSF and Interleukin 2 under SIOPEN. When reviewing the SIOPEN data recently the board found that adding cytokines (IL2/GM-CSF) to immunotherapy did not improve outcomes. The result of this recent finding is that SIOPEN discontinued the cytokine test arm.
However, the data had a reasonable flaw. In particular, adding cytokines to immunotherapy is so painful that a much larger group of patients simply stopped immunotherapy prior to finishing the course. The graphs were looking at ALL the patients even the ones that did not complete immunotherapy. Personally, I am surprised that in such a setting outcomes were so close.
So the big question still remains. Does adding cytokines improve outcomes on cohorts that complete the full immunotherapy course?
SIOPEN are going to test this hypothesis with an LTI trial. Since is it significantly less painful, adding cytokines becomes feasible without introducing the same huge levels of pain.
So, I guess, the big promise of LTI, beyond reduced pain is that there is a possibility that they can administer cytokines in a much less painful setting.
Beyond the question of pain there are some other concerns with LTI that need lots of attention.
Not every parent is comfortable taking their child home with a pump. What if something goes wrong? What if you are 50km from the hospital? What if the complications of sending kids home with a pump are deemed too high and you are stuck for 2 weeks in hospital every immunotherapy round for 6 months with a mostly fine kid. That is gruelling psychologically.