Thanks FDA….you made our REF2021 impact statement even better!!!!…… Only joking.. but today signals a new chapter in our journey with cladribine.
The MDs became CLAD lads way back in December 2004, when we did our first experiment with cladribine.
We saw the potential of the intravenous and subcutaneous variant in MS and had spotted that we could develop an oral variant, if we could have got it off the shelf in Boston (Probably parked so it wouldn’t complete with alemtuzumab).
As to our experiment with cladribine in Mice…..It’s didn’t work :-(.
I presented the daaaata (a clue here is where it was presented:-) for the first time and only yesterday . But hey ho who cares if it is not an EAE “cure of the week”. It’s all biology and mice have the wrong biology
Makes a change that it wasn’t “cure of the week” from animal experiments
But little did we know that ProfG was secretly working with the the oral cladrine tablet team to aquire the rights to develop a new oral variant of cladribine, where the patent was filed in 2004.
ProfG was intimately involved with the development of the first trial of oral cladribine tablets. Dr M&M treated our first person with oral cladribine. This was published in 2010.
Sadly in 2011 the FDA put a spanner in the works and they wanted more trial data. The company spat the dummy (an Australian term) and refused to do it and a bit like a spoilt kid…cut off their nose to spite their face.
They were before their time…a treatment effect with a long-term efficacy off a short-term treatment. It worked by depleting lymphocytes. The FDA didn’t get it, that it was a mechansim of action and not a side effect.
In the rush to do the studies, they had forgotten to do a second trial and a safety signal spoiled the party
Amazingly the European Mediciens Agency still don’t get it as they made the similar comments when oral cladribine was approved in europe in 2017.
Anyway MD and DrK picked up the pieces realising that the men in grey suits (actually that’s neuros in UK, in Australia apparently it’s the men in Beige and the US it’s men in blue blazers, beige chinos and histoically loafers…no joke but now it’s clunky running shoes or two tone comedy shoes…you think I’m joking…Go to a meeting in the US:-)
DrK and his team realised that the safety signal was probably a fluke. The FDA wanted more information from the European company, but seemed happy to ignore an even greater signal from an American -made drug:-(.
Anyway today the Food and Drug Association has seen sense and has today approved cladribine tablets for the treatement of MS. This is approved for relapsing and secondary progressive MS.
So two treatments for our American Friends in one Week.
So in a nutshell
Could I say it in a sound bite?
A new agent that gives selective reduction, replacement and resetting of the imune response to target a core disease biology of relapsing (and progressive) MS…A platform for neuroprotection and repair that give long term effect from a short term treatment
P.S. Cladribine tablets has yet to be shown to work in progressive MS, but the biolgy tells me what will happen…..
COI: Multiple but am currently presenting at a meeting