OK, here we go:
3 things strike me (other than the general shock of how little testing pharmaceuticals need to do for an orphan drug before they move into phase 2... , but I go through every time I look into this)
1. No other adverse reactions shared other than the jump in the blood pressure just like in the animal trial.
2. Then the "We are presently evaluating options for further development in various forms of achondroplasia." I presume this probably means hypochondroplasia and thanatophoric dwarfism.
3. The reference to the "non-growth endpoints" - that is an interesting consideration I haven't thought of before, lucky I am not in the business of developing drugs...
Petite yet fierce - Ramblings of a pathologically information hungry mother about hypochondroplasia, about rearing 3 girls in a foreign country and about all the random thoughts my sleep deprived and over-stimulated brain can come up with.
Please help me out here - what is the purpose of this drug? Growth? If so, how does it differ from GH?
ReplyDeleteThis drug specifically targets FGFR3 which regulates bone growth and is over-active in ACH and HCH thus leading to not enough growth. If it works it is THE cure. They are positioning it to say that it will help prevent issues with the foramen magnum, etc, as it would impact that growth too,but ultimately it would also help with growth tremendously.
ReplyDeletehttp://www.biomarinclinicaltrials.com/Achondroplasia.html
GH may help with some growth, but our kids our not GH deficient so it is not a real solution.
It is still very early stages. The animal trials were very promising. Phase 1 is really about toxicology in healthy adult humans... They will do Phase 2 for ACH children next year - but still no official word about HCH, although they patented their molecule for HCH as well.
As for me, I would wait at least until Phase 3 for HCH kids before I would consider enrolling, but in all honesty, I think if this succeeds it may be a well tested solution for our grandkids...
I went out for a walk and got thinking abut the choice of my words above... The word cure is misleading, it's not the cure, it is a treatment that targets the source of the problem. Where they stand today, it would mean a daily injection as long as the kids are growing. They are trying to find some agent that would allow for the molecule to work if taken orally... Hope that makes sense.
ReplyDeleteYes, it does make sense. Thank you. Sounds like an amazing project. I imagine timing of the drug would be important, in that it won't work after a child has finished growing? So it wouldn't work in adults?
ReplyDeleteYes, it wouldn't work in adults, only as long as kids are growing.
ReplyDeleteWith all these new drugs, I always wonder about long-term side affects that may not show up until later in life. So many weighty choices- take a chance on immediate aid, but risk worse effects later... how to decide, especially when it's for someone else?
ReplyDelete