New potential PV treatment : abc.net.au... - MPN Voice
New potential PV treatment
Very interesting. It sounds like another approach that would target hepcidin to regulate erythropoiesis. It sounds like an approach that is targeting the gene that produces hepcidin rather than working as a hepcidin mimetic like rusfertide. It will be interesting to see if this enters clinical trials.
Thanks for posting it.
Albeit lots of medical phraseology that may well be gobbledygook to us lay people… ☺️
It was definitely a very technical article intended for a medical science audience. I wonder if Google translate speaks gobbledygook (AKA genetics, proteomics, physiology).
This also looks like it could be a low cost treatment compared to say Beresmi or Chemo. Hope it gets a good look at.
I would expect it will be rather expensive when it first comes out.
To compare the monthly costs
Hydroxyurea - $25.00/60 tabs
Pegasys - $4,177/ 4 syringes
Jakafi - $14,000 / 60 tabs
Besremi - $14,000/2 syringes
The cost of drug development is high. MPNs are rare disorders, so manufacturers cannot count on high volume. The organizations that develop and produce medications intend to make a profit. They need to recover the development and manufacturing costs and make a profit to keep investors happy. I would not be at all surprised to see the costs of any of the new hepcidin targeting medication closer to Beremi than hydroxyurea. That is how the economics of medicine work.
Regardless of the cost, seeing another avenue for treating PV is hopeful. We are very fortunate that so much is happening in MPN research.
Wow that's why the NHS has said no to Besremi.
This is related to Rusfertide as Hunter notes. As I understand it in very simplified hepcidin is a natural agent in our bodies and is good in PV for red blood cell (RBC) control. By forcing more hepcidin to be present, RBC and thus HCT/Hb is reduced.
-Rusfertide is a current approach in trials. It is "mimetic of hepcidin" meaning it is a sort of imitation or synthetic hepcidin. So taking it increases hepcidin directly. But it requires frequent shots.
The new idea here is to prevent the liver from capping hepcidin production. Member Physassist has posted the info below. I think this is the agent "SLN124 is a gene silencing therapy that works by inhibiting a gene responsible for hepcidin regulation in the liver" So it's sort of like our broken Jak2 that turns off blood cell regulation, but here it's modern gene therapy for good purpose. SLN124 should require fewer shots.
A catch to all these is many PV pts also have high PLT and/or WBC. Hepcidin is not directed to fixing these, so both these hepcidin treatments may be best for example when RBC is the only symptom or when IFN or other drug is working but not controlling RBC well enough.
In other words, these agents should help remove any need for PHLBs.
From PhysAssist re SLN124:
New PV diagnosis Dec 17
PV
New treatment 
Pv or NOT PV ?
