Hi @Pharmagang,
I'm struggling to post a response to your earlier note: Post #:70894705
Thanks very much for your blog on BP's MSB review at BP Healthcare Conference 2023. Valuable points I wouldn't have had otherwise. You've inspired me to post something to redress some of the bullshit being posted about MSB's 'dire' situation.
Regarding your comment that ' Our old mate Steven Bauer is a director at the BMT group. ', I think I know why you say this, but I don't believe this is actually the case & I think you may have (quite unintentionally) referenced yet another stakeholder in the area who happens to share the name. I've done that myself often enough.
Dr 'Steven' Bauer MD is, as best I can tell from my searching, still resident as the Chief Regulatory Science Affairs Program Officer at Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina after his much-unlamented resignation from OTAT and the FDA in 2022.
These days 'Steven' amuses himself by attending events such as the 2023 World Stem Cell Summit, and "supervising" Wake Forest U scientific staff engaged in review of cell-based biological therapies and research 'relevant to their use in clinical trials'. Let's see how he gets on with the FDA when Wake Forest U try to get marketing approvals to make $$$ out of their research, which apparently now focuses on multipotent stromal cell biology and stromal cell-hematopoietic cell interactions that influence development of lymphocytes. Quelle surprise! It must have irked him terribly, at a personal level, that an Australian company had the temerity to actually be in front of him at the cutting edge of translational medicine for MSCs in the USA i.e. treating and curing a disease and an unmet need as vicious as aGVHD. So, I think you share my view that our old mate 'Steven's view of the vastly underrated - and yet overriding - importance of his own personal work (& that of the FDA's taxpayer- and Bigpharma-funded internal research unit under his supervision) was never in any doubt. He's on the record in the ODAC transcripts. But we digress...
I think you're possibly referencing Mr 'Steve' Bauer, a Director at the BMT Infonet, who is a very different animal. If I'm correct, (Mr) 'Steve' Bauer seems a genuine salt-of-the-earth character, one of whose key achievements in life - and no small one - is being a 38-year bone marrow transplant non-Hodgkin lymphoma disease survivor, now living in Denton, Texas. So, I don't believe MS has anything to worry about with 'Steven' Bauer MD in this particular space (but I do think he'll try to compete for $$$ later).
BMT Infonet, by the way, seems to perform a fantastic service in the USA, as a not-for-profit highly-regarded advocacy organization for transplant and CAR T-cell therapy recipients before, during and after treatment. Link here. This is genuinely paying it forward stuff that these good people do - informing & supporting patients, intending patients and carers. We can all empathise and agree with BMT Infonet as potential or current collaborators with MSB, because these are the very patients MSB treats with Ryoncil under EAP & these patients can only be the better for this selfless work.
But this got me thinking as you surely did @Pharmagang: what if the BMT mob we're looking for to help us with an adult aGVHD clinical trial, is actually conflicted?
What if they had someone like Bauer-exFDA so clearly conflicted & in a position to stymie things?
Or if they have their own agenda that doesn't work with ours?
Or if they simply aren't excited enough to drive this trial through to its necessary end despite the FDA's delaying tactics while it finally prepares long-overdue cellular therapy guidances, resources itself properly and spins its little political narratives?
Well, it turns out MSB shareholders are 'in luck', IMO.
And by 'in luck' I mean that the CEO and CMO of Mesoblast Ltd deserve (IMO) to be lauded for making the luck that Mesoblast Ltd and all its shareholders will enjoy over the next 2 years with BMT CTN.
BMT CTN (Link here) is the strategic network of clinical trial institutions which plans, oversees and facilitates the conduct of multi-institutional clinical trials in the USA to evaluate promising cellular therapies for patients needing Hematopoietic cell transplantation (HCT), or Cellular vaccines, or Chimeric antigen receptor T-cells (CAR-T) therapies to deal with life-threatening disease. They're setup and funded by the US government since 2001 i.e. they're funded by the National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute (NCI), and they see their work as facilitating participation of a large network of centers in trials available to patients in all regions of the United States of America.
But wait, there's much more... BMT CTN studies enroll patients from 20 Core Centers and 75 Affiliate Centers; which conduct more than 88% of US transplant and cell therapy procedures annually. Collaborations leverage a well-functioning BMT CTN infrastructure, developed over more than 20 years, including the BMT CTN Steering and Executive Committees, an NHLBI Protocol Review Committee, NHLBI Data and Safety Monitoring Boards, and BMT CTN Technical Committees.
...and the Data and Coordinating Center (DCC) at BMT CTN coordinates the patient enrolment from all of the Core members & through all of this the DCC works with CIBMTR, the research centre responsible for MSB's most recent survival studies put up for marketing approval to the FDA:
OK. That's all so very good for MSB.
And if we supply the trial product (which seems done, subject only to that FDA potency/ release assay signoff) the US taxpayer via the USA Public Health Sevice is picking up the tab for Mesoblast Ltd's adult aGVHD trial and recruitment is anticipated to be done by EOCY24, as @Pharmagang was able to find out.
The final touch is that BMT CTN works closely with CIBMTR - the real-world registry which carried out long-term survival studies for MSB and whose real-world data management is the gold standard in the USA atm in relation to bone marrow transplant disease and treatment data (and so, for independent GVHD data).
I'm getting goosebumps just thinking about the synergies here.
But where's the luck I mentioned? Well, y'know how we were talking about Steven Bauer sticking his head in & possibly not being a force for good... have a look again at the description above of where real responsibility & the corresponding authority lies in relation to the BMT CTN i.e. the responsibilities of the Chair and Chair Elect of the Steering Committee of the CMT CTN, & then have a look at this screenshot:
All good IMO.
See y'all at the AGM.
Cheers and GLTAH..
(20min delay)