Thank you, Bozo. Thank you, Jenny. Thank you, all.
Tonight I drop Bozo off to be boarded for five days. Jenny goes to my cousin. And in the morning I head off to Orlando and the American Society of Hematology meeting.
Abandoning my parrot and dog to report on MPN therapeutic advances isn’t easy.
I hope it pays off. Often it seems we already know the preliminary results of most drug research affecting MPNs. I don’t anticipate a surprise like the unveiling of a new CAL-R-type discovery.
This year we have all lost more friends, close and far, to MF and we have seen friends restored to health through stem cell transplant. Despite the pain and suffering there is a reason for great hope. It seems possible the end of this long and deadly road is in sight…and shortly, many more of us will come safely through SCT.
This is the year gene therapy grabbed the headlines and emerged as our best therapeutic hope. That alone is worth the trip. Just a few weeks ago we learned Dr. Waseem Kasim and his group beat back a little girl’s leukemia using gene therapy…and, so far, it seems very much like a full cure.
Friday morning, at the Renaissance Hotel in Orlando, a small group of physicians and scientists will assemble for a conversation about gene therapy and its prospective role in cleaning our blood to permit stem cell transplant with our own cleared blood. If that can be done – and much of the technology is already in place — it will be huge for all of us. It will spell the end of delays as our physicians search for a matched donor. And it might totally end or largely eliminate Graft Versus Host Disease.
More than that, this meeting, this CREATE seminar is a coming together of genetic engineers, research hematologists, clinical hematologists and transplant specialists to start exploring the possibilities.
Gene therapy is not limited to Stem Cell Transplant since repair and replacement of mutated genes is already far advanced in other blood conditions.
This work has been funded by the MPN Research Foundation. The organization and launch of MPN Genetics Network, the recruitment, advertising, and production work, the hotel, travel, materials… all paid for by the Foundation. That funding, along with more than a half-million dollars in new CRISPR research grants and the whole-hearted MPNRF staff support of the CREATE Seminar – special thanks to Raquel Nunez and Michelle Woerhle — has enabled this vital initiative.
In recognition of the significance of gene therapy, the MPN community’s good friends, Dr. Richard Silver (CR&T), Dr. Srdan Verstovsek (MD Anderson, and Dr. Ruben Mesa (Mayo Clinic/Scottsdale) contributed funds to help defray costs of the CREATE Seminar.
Meantime, if you’re looking for a place to make a substantial yearend donation, invest in our common future and give to our MPN Research Foundation..
Thank you…back to you in a few weeks with a full report on ASH and CREATE.