A Few Quick Thoughts from the MPN Patient Symposium

I just got back home from the whirlwind trip to New York City for the 6th International Patient MPN Symposium sponsored by the MPN Research Foundation and the Cancer Research & Treatment Fund.  The day was filled with fascinating speakers (several of whom were new to me) and each of them taught me something new.

I'll organize the 25 pages of notes later, but I thought I'd tempt you with these nuggets. :)

Cool Terms I Picked up at the MPN Patient Symposium

Somatic mutation:  a change in a gene that occurs after birth (you aren’t born that way)

Molecular mimicry:  some genes mimic others

Epigenome:  the new area for research

We are all entitled to our own opinions, but not our own facts.

JAK2 and Exon 12 and TET2 – a few of many mutations under exploration

Genomic Instability:  affects the micro environment and what happens with MPNs

Specialized Niche Cell:  cells that are highly specialized (example:  Endothelial cells)

Endothelial cells:   lining of blood vessels;  they also instruct other cells and broadcast directions throughout the body.  They also instruct organ regeneration (eg, liver and lungs). 

Inductive angiogenesis:   uh, i forgot already!

Proliferative angiogenesis:   forgot this one, too!

Role of vascular niche cells in self-renewal of stem and tumor cells:

The micro environment that are created by the mutations have huge impact on what happens. 

Angiocrine Factors                

Niche cell:  activate endothelial cells

Leukemic initiating cells – they expand the vasculature in the bone marrow.

Factoids and A-Ha’s! 

• Platelet-Leukocyte aggregates and micro-particles promote thrombosis.  The different kinds of cells can act together to promote thrombosis. 

• We need drugs that target the endothelial cells.   Endothelial cells expand hematopoietic stem and progenitor cells by angiocrine expression of Notch ligands.

• The first hematopoiesis occurs with the endothelium. 

• The leukemia from MPNs is completely different from the normal leukemias.  We need different treatments because the biology is different.

• The Chronic Myeloid Leukemia is triggered by the Philadelphia chromosome, so it is regarded as a different disease process from MPNs.

• JAK2 is not a “switch” that can be flipped on or off; it has at least 10 different mutations, which cause different disease results in patients.

JAK2:  The One Mutation, Three Diseases problem.

Why do patients develop ET, PV, or PMF?

Level of JAK2 allele burden determine ET, PV or PMF – more so in men than women

How much is the disease versus the host predisposition to develop an MPN?

Are there other cooperating somatic mutations?

• Despite all the advances, we still have a long way to go – do not be misled by where we are.

• For those of us taking warfarin (Coumadin), Dr. Weksler said that the newer anti-coagulants are “not there yet”.  Some patients have tried Pradaxa and gone back to warfarin.  Pradaxa must be taken twice a day consistently and this is problematic for many patients.  She believes that we’ll have a better alternative in a few years.

• Much of the knowledge gained and progress made has occurred because patients before us and among us have shared their tissue samples, DNA, and whole selves for testing and clinical trials.

There was so much more covered.  I hope this tempts you to watch the video when it is available online at the MPN Research Foundation

Stay tuned!

peace,

Marina