MSB Trading 2020 - a new dawn, page-717

Hi Joannie!

They were referring to this picture, used in the presentation.



I have absolutely no qualms reposting the photo.

This is the 2nd slide from Dr Joanne Kurtzberg's presentation about the 'unmet need' from the recent proceedings.

This is the reality of Acute GVHD as you can see from the slide. However, all we can see is what's happening on the surface - skin rash.

Never mind the cramps. Never mind the painful diarrhea. Never mind all the emotions flowing in the parents' and care providers' minds as they see a helpless child being ravaged by this complication as they try to change out the cloth netting every hour.

Here are the stats. Not my own opinion, pure, peer-reviewed stats, paraphrased from her slides.

1. ANNUALLY, there are about 1300 allogenic HSCTs (Hematopoietic stem cell transplantation) that are done in kids in the USA. (CIBMTR, 2019)
2. Despite SOC prophylaxis, 25-80% of them will develop acute GVHD (CIBMTR, 2019)

25-80% of 1300

= 325 to 1040 children.

Read that again.

325 to 1040 children.

3. On the onset, SOC is corticosteroids. Think methyl-pred.
4. About 50% respond well. Thank God.

But wait... what happened to the other 50%?

They DO NOT respond well, and another way of calling it would be 'Steroid-refractory'

That brings our range to -

~160 to 520 children EVERY YEAR.

Who have developed acute GVHD after a HSCT (which they already were hoping for an improvement) and DO NOT respond to steroids.

5. The mortality for steroid-refractory Acute GVHD is as high as 70-90% (Macmillan et al., 2003, Jagasla et al., 2012, Aral et al., 2002)

70-90% of 160-520

= 112 (low side) to 468 (high side)

Yes. 112-468 deaths.

With Rem-L, that % chance of mortality drops to ~36% (Day 28 Overall Response) and ~21% (Day 100 Survival)

Let's not lose sight of how incredible this treatment is, and the hope it will bring.

This is JUST in peds acute GVHD!

Let's not even consider the ugly reality of COVID-19 and the ARDS complication!!

Come on FDA! Lets get a move on and SAVE LIVES!