So when does common sense and morality come into play?

[huntingonthebluffs]

I have watched the Geron / Imetelstat story play out for several years now and have never heard of an issue. It has been progressively better news from CT readouts regarding treatment of hemo diseases, both safety and efficacy. Certainly in the hemo arena, side effects are known and manageable. Delivery of the drug in treatment is well understood and effective. Analyzing and qualifying patients is well understood based on their blood disease and health conditions. Doctors know rather quickly if Imetelstat is helping so treatment doesn’t need to continue where results aren’t occurring. Yet in all these years, patients keep dying by the tens of thousands without access to Imetelstat. So maybe I have left room for some to quibble on my overly generalized comments, however, if you are suffering from the diseases Imetelstat is able to impact, there are no warnings that will prevent the patient from trying this drug treatment assuming the care and costs can be mediated.

So what are we waiting for? Can anyone honestly say the FDA, EUA, JNJ, Janssen or Geron do not know what we have here? Imetelstat is in a league of its own showing stellar results, yet it is being handled and brought through the process as if it is radio-active. Many more exciting steps and levels of drug combinations are clearly in view but apparently nothing can go there until we get an approval on the singular drug treatment.

Right to try legislation, Orphan Drug Designation, Fast Track Designation, various patient advocate groups, etc. can’t begin to push through the resulting steel mesh of approval prevention employed by the FDA, EUA, government policy makers, legal system, insurance companies, all flavors of hospitals and care facilities and especially evident is the drug makers themselves not suiting up. Okay so obviously money is important here, a lot of fingers in the pie and making big money from the existing system. Millions of people making big salaries/bonuses, companies and individuals doing whatever is required to protect their turf and of course that is expected and not just a biotech thing. Sure getting drugs out there is incredibly complex and expensive but with all this firepower could not a solution short of going to the United Nations be found?

Question is does this all make sense, common sense? Does morality come into play here? While this system has served most of us acceptably well, I find it all inhuman and immoral on many levels for those without options. Watching this circus play out is really becoming the sadist and most frustrating part of what should and likely will be a fantastic advance of science for all of us. Thinking about what this would take to really streamline in time and costs, I find myself wishing I was 50 years younger to go with the passion I have now for wanting changes made to speed up the delivery / availability of new drugs to those that are R/R to all else. The development of the science get all the attention as it has a broad brush financially, however, making it available to humans in critical need is barely out of the dark ages, in my opinion. I have a lot more thoughts here but this is way too long already!

Thanks again Biopearl, for taking charge on keeping this forum working and for your great posts!

[biopearl]

Nicely said Hunt, I think it is a good summary of the frustrations patients are feeling as they wait. And wait. Clearly the FDA was not willing to approve on the basis of the Dr. Tefferi studies or even the ET studies. In some ways waiting for a critical juncture is strategic-- the longer we wait the better the data seems to get but at some point a critical mass is defined and no further response from Imetelstat as a single agent can reasonably be expected. It may be that by allowing the FDA more time to evaluate this drug (after all it is a completely new molecular entity), future studies and approvals might be truncated. Hard to say. I do think the journey for approval in in at least one of the areas we are most familiar will occur within a few months. And thank you for your kind words. I expect this site to heat up once we know more. bp