Dr. Bennett: My name is John Bennett from the University of Rochester, and my co-chair, Gary Gilliland from the Brigham Hospital, Harvard Medical School.
I would like to welcome you here this morning to a State of the Science meeting on myeloproliferative disorders, or myelo-signaling disorders.

The organizing committee is sitting here on my right, Drs. Cheson, Tallman, and Larson, who were bright enough themselves that they would like to get the responsibility shifted onto someone else's shoulders. So, they asked Gary and myself to assume responsibility for organizing this meeting.
Basically, what we are going to try to accomplish today is through the vehicle of breakout workshops this morning and summations this afternoon, and then this will all be transcribed and Dr. Gilliland and myself will meet, put this together, and it will then appear on the NCI website.

Also, we will submit portions of it to Leukemia Research as an overall state-of-the-art review on myeloproliferative disorders.

The purpose is to provide guidance to the leadership at the NCI, to indicate what steps they should take in regard to this group of fascinating disorders that, by and large, have been ignored, not formally by intent, by the Cooperative Groups and the NCI since the termination of the Polycythemia Vera Study Group, which goes back two or more decades. Dr. Silver, who is in the audience, and myself can remember that group very well.

The questions really to be addressed are: have we reached a point with these various disorders where the NCI can then take some policy measures to encourage, through the mechanism of RFAs or grant applications, more basic research, more translational research.;or can we move into phase II or phase III studies, out of individual institutions and into either the Cooperative Groups directly, or forming individual study groups -- funded, competed for -- to look at the therapy, management, and also the biology of these diseases. Certainly, the model exists and is currently being done in leukemia, AML, ALL, myelodysplasia in the Cooperative Groups, in which molecular genetic, cytogenetic, flow cytometry are being packaged together along with definitive studies and funded through subcontracts made to individuals or to the Cooperative Groups themselves.

So, the format that we set up, then, leads naturally from basic diagnosis to mechanisms, since these really are signaling disorders, the common links between these, and then into innovative therapeutic strategies as well as intensive strategies already being done by several of you in this room, including allogeneic and autologous bone marrow transplantation.

We have also included mast cell disorders because, no surprise, there is a link between mast cell proliferative disorders and AML, MDS, and myeloproliferative disorders. I think you are all aware of that.

We have eliminated from discussion today, except as it may be pertinent, chromosome-positive - BCL-ABL positive - CML, since that is a disease that has been well studied within the Cooperative Group mechanism, for which a variety of therapeutic strategies are now being rapidly mobilized, including the use of tyrosine kinase inhibitors and even Philadelphia-chromosome-positive ALL.
Obviously, it will come up selectively, particularly in chronic myelomonocytic leukemia, where a modest number of patients may respond -- depending on whether they have one of the specific translocations that involves the platelet-derived growth factor.

We do have, in the room -- I would just like to introduce them briefly -- two patient advocates, which is a responsibility that NCI shares with the other institutes in these types of meeting formats.

Mala Hermon and Robert Tollen, if they could both -- Robert Tollen on the right, Ms. Hermon on the left. I am very appreciative to Dr. Richard Silver for providing me with their names

They will be attending the meeting and are free to make comments as they see fit, in any of the sessions that they attend today. Gary is just pointing out one item. About a week and a half ago, I asked our representative from EMMES Corporation if they could scan through the various protocols approved at NIH, including the National Cancer Institute and other institutes. They provided me with those, and I went through those and tried to isolate those studies that focused on myeloproliferative disorders. Believe it or not, we are down to one page, consisting of eight or so approved intramural and extramural studies that involve primarily, although not exclusive, myeloproliferative or mast cell proliferative disorders.

Clearly, there is a huge gap out there in the United States. Hopefully, out of this meeting, will come some ideas and the generation of studies that can focus on these very interesting disorders So, we have assembled an interesting group of investigators, some of you with expertise in maybe only one idea, but others who have expertise in all or most of the myeloproliferative disorders, including diagnosis as well as research investigations and therapy. We welcome you all here, and we are delighted that you chose to give up some of your valuable time to join with us, and we look forward to an exciting and provocative day.

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