Where can one with MF go to get in line for Imetelstat treatments?

[huntingonthebluffs]

A rhetorical question certainly, as we know the answer is, there is no line unfortunately. Statistics have been shared in various, indicating thousands of people in the US and around the world are suffering (and of course rapidly dying) from MF and many other hemo diseases as well. Many with MF are aware of Imetelstat and of those, most would likely want to be in the queue to receive Imetelstat asap or at least be able to determine if it could help them reverse, stop or slow the disease’s progression (e.g. CI, PR, CR). We are not dealing with a headache here, and it is all very complicated except for the patients and their families. JNJ/Jansen are playing the “long game” while the patients must play the “short game”, it seems critical that in this case, JNJ/Jansen/Geron/FDA/EMA should be playing both the long and the short games.

My question: Is it justified or rational for the FDA, EMA, JNJ/Jansen/Geron stand-by or stand firm on singularly conducting a lengthy process while thousands die each year with MF? Literally thousands are dying, while JNJ/Jansen/Geron assess every option, squeeze out every detail, test and document every treatment strategy, consider every competitive option, etc. Yet, there are interim options and designations that are available and likely could be obtained quickly to greatly speed the process while seemingly protecting the sponsors’ investments. I know I am over simplifying but I wouldn’t want that hanging over my head.

If this by chance is obvious then, I wonder if anyone on this board has appealed this or similar questions to the FDA or EMA or product sponsors and if so, what might have been the response? I’m likely one of the few laymen posting on ImetChat, and so I know it is possible that everything is proceeding as it should and must, just incredibly sad for those standing in line.

In addition, some related thoughts I believe to be correct:

1. The demise is fairly rapid for those suffering from MF.
2. No potential cure for MF exists with the exception of Imetelstat, a much studied and tested, but unavailable drug.
3. JNJ & Jansen appear rigid in their control of the CTs and allowing no use of Imetelstat beyond the CTs.
4. JNJ & Jansen are tracking “all results” coming from the trial in a very secretive manner.
5. All publicly known details regarding Imetelstat as well as business case fundamentals have been positive since the hold was lifted.
6. Expectations are very high that Imetelstat will bring transformative results in at least some of the hemo diseases.
7. Significant uncertainty exists in the public regarding the intentions of Geron or JNJ regarding when or even if Imetelstat will make it to market, an acquisition could occur, additional clinical trials will occur, etc.
8. Most of the preceding points reflect the need for exceptional urgency given the life and death situation at stake and yet apparently nothing budges from the norm.

And what I can only surmise:

1. No one outside the CTs have received or will receive Imetelstat in any form until the products are brought to the market through an exhaustive and lengthy formal process.
2. JNJ / Jansen have no intention of investing time or resources to aid patients outside the CTs or obtain the other designations (e.g. BTD, FTD, etc.).
3. Neither the FDA or EMA will intervene to speed any aspect of the process.
4. It is largely known which patients will respond to Imetelstat with MF and to a large degree, the scope of Imetelstat’s applicability to treating various cancers.
5. Combo’s using Imetelstat are being studied in the Jansen lab to determine which will provide the best results for MF and other diseases.
6. Testing strategies are being studied / developed to determine the 20-30% of patients with MF that can be significantly helped, prior to actually being treated with only Imetelstat and likely which could be helped using selected combination drugs.
7. Timing for a marketable Imetelstat drug and / or combo treatment strategies may (likely be) years away.
8. Thousands more will die in waiting and of course, many more thousands will eventually be helped with Imetelstat products and treatment strategies as they come to market.
9. Seems that all of the points above might be shortened, timewise due to the life and death situation at stake. Why not use the framework that exists for both a long and a shorter timeframe process on providing the drugs to those most in need.

I guess the bottom line is that, it’s easy to get excited about the science, the dynamics of the study, all the patents, our investments, etc. but for the sick and dying patients who have families and many contributions they can and want to make, this seemingly never ending process is a nightmare.

[cheng_ho]

We passed a "right to try" law last year in NH. Last weekend the NH GOP added FDA nullification to the party platform.

The only way medicine can progress is if the terminally ill are allowed to try new treatments.

[irishtrader52]

Hunting - Applause for your post. Exactly. I can't believe John has to travel 2000 miles every 3 weeks for more than 3 years - when imetelstat is being infused in others (just because they took jakafi and john took hydroxyurea) 20 minutes down the road. And, that is a minor complaint compared to those who cannot access imetelstat at all. C'mon - this game is getting old from a patient's perspective anyway. He is headed out again next Tuesday.

[huntingonthebluffs]

Thanks, Irishtrader52. This, in my mind at least, is exactly the situation that brings in activitists, professional activists (medical, government, investment, etc.). There is so much at stake here and so many things that are not making simple common sense. Makes me wonder if this chat board could bring to bear our collective weights / voices on these areas, medical and investment, somehow to shine light on what is becoming very frustrating and critical to so many on these various fronts.