Imagine reading this

[biopearl123]

Imagine reading this knowing you had to wait until 2024?

https://ashpublications.org/blood/artic ... y-Received

What does this say about our FDA

[biopearl123]

So after you read that, consider this. A little less than four years to approval and a lot more than 1 million interim blood transfusions, that's right more than 1 million at least, over that time period (this is probably an underestimate, someone check my math please--see previous posts). Well, I suppose that's one definition of insanity. Unmet medical need--four years is a long time to leave it unmet. Unmet by Imet. A horrible four years if you are a patient. So patients can enroll in studies and get Imetelstat you say? Yes all 170 (minus the control group who won't get Imetelstat). Not the thousands who desperately need therapy and face iron overload, higher risk of AML and just plain feeling terrible. A sustained 3 g rise in hemoglobin not achievable with transfusions or alternative therapies is dramatic. It's never been achieved in any meaningful way before. Its the difference between feeling like you are carrying a backpack full of cinderblocks all day or instead being able to enjoy a round of golf on a beautiful day. Four years is a lot of miserable days. So I ask myself can this be possible with all the FDA talk about unmet medical need and a commitment to changing the glacial system to approval? I just can't accept it, can you? I applaud Dr. Scarlett and team for adding the extra 40 sites. Each site needs only to enroll one or two patients to fill the study and many high enrollees will enroll many more. Will these sites have to be places like New Zealand (no Covid!!) but imagine the administrative burden dealing with even more far flung sites than Asia and the Middle East. It's a challenge. Many of you have written to FDA directors etc and think you words have no weight but I disagree. Keeping this information in the public domaine will help to highlight just how difficult the approval process remains. No to mention how MF approval will lag MDS. We are talking years. In the meantime, impressive part I MDS data continues onward, to mature, to use the words of the investigators. Actually, I think its time for the FDA to show some maturity. Regards, bp

[rccola335]

i would of liked to hear the presenters remarks at ASH - the KOL will be interesting on the 17th - I do not think we will be waiting until 2024 for approval

[huntingonthebluffs]

bp, your focus on the number of RBC transfusions (RBCT) seems to me to be a relatively new and un-vetted line of thought for this board and for anyone focusing on the benefits of Imetelstat. I think you are maybe shining a bright light on an issue that should be difficult to ignore and it maybe the canary.

MF and MDS are most common in people in their 60’s, 70’s and 80’s which I think, diverging from politically correct comments, may limit the urgency of the powers to be to be pressured on accelerating the process and advancing approval. In other words, in a callas way while it is unwritten it might be said that since these are diseases of the aged, it doesn’t have the importance of something addressing an unmet need across all age groups and after all there is palliative medicine available. Of course, it can also be said that FIC drugs focusing on oncology diseases generally are the best example of where the regulators drag things out.

While I am not opposed to calling something a spade, in our defense many, likely a majority of us in those upper age groups are technically, partly or possibly hardcore fully retired, many are not and either way we are at a point in our lives where our experience and wisdom and resources are often peaking and depending on our health have immense contributions to make as long as we have energy and good health as quality time is available. Whatever the part of the issue this concern might play in approvals dragging out endlessly or not, I think it is of utmost importance to find benefits of Imetelstat that goes beyond the older generations and can benefit the majority of the human race.

I would hope that the analysis on what impact Imetelstat could have in the US as well as worldwide with regards to reducing the RBCTs would be done and sitting on the shelf somewhere in a journal. However, since we have not to my knowledge seen this vetted by anyone, including Geron, you have likely hit a critical nail on the head. In my opinion, your out of the box and broad scope thinking on so many things Imetelstat does not seem to be a common skill held by the pharma industry leaders or the regulators, in fact narrow minded focus seems to be the norm and maybe there is some justification. However, I think we all know that Imetelstat will blow the doors off once approved and a myriad of combinations begin to flow though in treating many cancers and other conditions suffered by all age groups.

In the meantime, the side benefit of reducing the number of RBCTs on the scale possible with Imetelstat appears to be undeniable. Reduced medical costs, QOL, time saved/redirected on everyone’s part, etc. across the US and globe seems monumental. Maybe this is an important “competitive advantage” that JS is aware of and doesn’t want to discuss in public but in addition to having approval justification weight with the regulators, much value can be seen by all patients, Geron and investors, the proverbial win, win, win.

I assume bp, that you have flashed this in front of Geron’s IR department and probably others at Geron and have received very little feedback. However, this may be one of those areas that we as patients / investors might have an opportunity for real traction even fireworks and in addition to the analysis being done, we might get the bright minds on this board, YMB, SA, Twitter, etc to push this out in a major way to Geron, KOL’s, regulators and financial analysts to raise this up on a level with other justifications (e.g. RWD) for accelerating approval. Just maybe it could uncover that “beacon of reason” you refer to as MIA at the FDA.

[biopearl123]

huntingonthe bluffs, thank you for your comments. Here is what our FDA has done to make blood more available (remember that blood is perishable and can not be stockpiled):

https://www.fda.gov/news-events/press-a ... g-pandemic

After you read that, see if you feel reassured about the safety of our blood supply in the time of COVID. Regards, bp

[cheng_ho]

Actually, blood can be frozen quite efficiently... remember, I spent seven years in labs freezing and reanimating human cells

We've been freezing and reanimating cells at >95% efficiency since 1960. It's just a matter of either finding the right antifreeze (we used glycerol or DMSO, both a little harsh for transfusions), or various physics-based tricks for preventing crystals. One recent development:

https://www.bbc.com/news/uk-england-cov ... e-26087585

But NIH and FDA think that freezing organs or even blood is "socially mischievous" (yes, that's a quote, from the early 80's)... the people at the top aren't technically knowledgeable, their "ideas" come from fashions in social-science thought, plus a heavy dose of old horror movies from their childhood.

BTW, they also force transplant organs to be sent to community hospitals with no transplant teams. There is no one at the top, it's just empty suits placed there by the waves of mindless political faction fights.

[huntingonthebluffs]

Thanks, biopearl for that link regarding the FDA guidelines for allowing a donor to donate blood as it sheds some light on the criticality of the availability of blood for RBCTs and the demand for over 40K RBCTs per day in the US. I am very grateful not to require RBCTs and I believe anyone would have concerns about the discussion in the link you provided. I view the need as a necessary evil and yet it seems the FDA is focused only on meeting the need not reducing it given a way forward for that ability.

While some might think I am not aware of the RBCT discussions by various at Geron, KOLs, etc. but I have utilized this board from essentially the beginning as many here have as well and listened to most CCs and various presentations, etc. I followed the progress of the battle against MF for several years before that related to my brother-in-law who died from MF in June 2015. While he was receiving a couple transfusions a month for a long time and the last 6 months was receiving 1+/week . It was a “damned if you do and damned if you don’t” situation. If one is interested they might do a little reading on the many side affects and types of transfusion reactions, many serious and especially when experienced by someone in the later stages of MF. So the whole idea of lowering the hurdle to give blood, while apparently necessary, also has a level of risk (not quantified in the article) for someone with a compromised immune system and especially after having their spleen removed but all receivers are at some risk and that risk is going to increase regardless of what was stated in the article.

So bottom-line, the article highlighted the critical shortage of the RBC supply to justify backing the restrictions down. Apparently the only choice is to find a way to allow more blood to be donated, even when adding some level of risk versus finding ways to reduce RBCTs to bring demand into balance with supply during challenging times. And so I again say for those who are missing your point, basically a paradigm shift of its own, this needs to be vetted as regardless of the previous comments and discussions on how Imetelstat reduces the transfusion requirements for both MF and MDS patients, some of us and the powers to be only recognize that as a benefit on an individual basis while the big picture here is mysteriously hiding a key point and it likely has a major benefit to the world’s blood supply. I personally see this dilemma as another example of where the FDA has failed to anticipate and yet cannot see their inability to expedite a safe drug through to approval as contributing to this blood supply crisis.