Reality check

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biopearl123
Posts: 299
Joined: Fri Jul 20, 2018 5:13 pm

Reality check

Post by biopearl123 » Fri Apr 03, 2020 3:25 am

Today's PR is hardly surprising and I have mentioned in previous posts on SA and maybe here too that major contributing sites like Italy, Israel, South Korea will be literally shut down regarding ongoing clinical studies. I think we need to take a worse case look and assume that no new patients are being enrolled in the MDS study and some if not all of the patients currently enrolled may not wish to venture out or be limited by lock down rules. This basically rolls the study back to day one. The AML study won't start this year. Just as bad is that it would be foolhardy to assume the virus can not be transmitted by transfusion, making treatment of MDS patients difficult. The virus could be more selective regarding mortality in patients with heme disorders so patients will not want to come to hospitals where they might be exposed. So the news in not good on many fronts. The MF study on the other hand is closed and there is a reasonably high likelihood of some form of approval this year. That would be good news. One would hope the FDA would recognize the extraordinary situation that high transfusion requirement patents with MDS/MF anreven AML face, and allow compassionate use of Imetelstat in these patients especially given the very impressive results from part one of the MDS study. Everyone is understandably focused on the virus but the blood supply will suffer and patients who need blood will suffer. I say to the FDA: you need to approve now or at least allow a study that could lead to approval for MDS and maybe AML, that can be completed on a much shorter timeline. bp

bucbeard
Posts: 34
Joined: Wed Jul 25, 2018 12:30 am

Re: Reality check

Post by bucbeard » Fri Apr 03, 2020 2:31 pm

absolutely correct assessment on all points.

regarding approval, the FDA must do this, or they are absolutely failing their responsibility for the good of these patients, the good of the healthcare system and the good of the people of our nation.

Secret Third Arm
Posts: 31
Joined: Tue Aug 28, 2018 3:26 pm

Re: Reality check

Post by Secret Third Arm » Fri Apr 03, 2020 6:44 pm

BP-

I don't think that patients who are transfusion dependent have any choice but to venture out. That's how they get their transfusions (or die), so I think that the opportunity to venture out LESS which is afforded by being on Imetelstat and therefore needing fewer transfusions, becomes more important than ever.

Stay safe, stay healthy, stay home.

- STA/BTS

biopearl123
Posts: 299
Joined: Fri Jul 20, 2018 5:13 pm

Re: Reality check

Post by biopearl123 » Fri Apr 03, 2020 10:04 pm

STA, agree but there has to be blood available and blood from good donors which is getting scarcer.

Secret Third Arm
Posts: 31
Joined: Tue Aug 28, 2018 3:26 pm

Re: Reality check

Post by Secret Third Arm » Fri Apr 03, 2020 10:48 pm

Even more to my point BP. With blood being scarce then recipients will be looking into alternatives to transfusions. My guess is that Scarlett will hit this point hard with the FDA. Having Imetelstat available for MDS patients right now would alleviate some of the strain on the healthcare supply chain (i.e. blood).

Too bad the 2Q meeting is for MF only, although I think that they will be able to make up for some of the clinical trial patients we lose overseas with more US patients realizing they should be staying away from hospitals.

biopearl123
Posts: 299
Joined: Fri Jul 20, 2018 5:13 pm

Re: Reality check

Post by biopearl123 » Sat Apr 04, 2020 12:25 am

STA, anemia amelioration is an issue not only for MDS but also AML and MF (a secondary endpoint in the MF study). Your point is relevant, important and right on. The FDA, with low risk to them and to patients could allow compassionate use in high TR, lower risk patients based on the current data. It is unlikely that they will but it would be a logical response to this drastic, unique and awful situation. At the very least since the most mature data is in MF, approval would help anemia treatment for those patients and help protect the blood supply. As it is, transfusion donors will be chosen on a less selective basis and their blood will enter the pool of available product. bp

biopearl123
Posts: 299
Joined: Fri Jul 20, 2018 5:13 pm

Re: Reality check

Post by biopearl123 » Sat Apr 04, 2020 6:32 am

If there are 40,000 MDS cases in the US and Imet can be applied to 85% of them per Geron's slides, and if each patients Tx requirements goes down by 2 units every 2 months (12 units/year--this is very conservative but allows for not all MDS patients having a tx requirement), then 408,000 units of blood would not be required to support the failing bone marrow's of these patients. Almost half a million transfusions. These assumptions do not take into consideration the wide range of tx requirements in these patients or the probability that some may not require tx at all. It's just a very broad guess. Please feel free to modify and critique. bp

biopearl123
Posts: 299
Joined: Fri Jul 20, 2018 5:13 pm

Re: Reality check

Post by biopearl123 » Sun Apr 05, 2020 6:50 pm

Blood tax services listed here at about 3600.

https://www.hopkinsmedicine.org/the_joh ... harges.pdf

I have seen other estimates closer to 7 to 8 hundred per unit. Cost has to consider collection, screening for blood borne disease (including it appears COVID19), preparation for storage, storage, preparation for administration and delivery including bags, tubing, facility costs etc etc. It is crazy that theFDA, especially now is not recognizing Geron’s potential to uniquely help. We are talkin hundreds of millions here not to mention the potential to alter the actual natural history of this disease. Stay strong, stay health, stay home wear a mask, wash your hand etc. Best wishes, and I really mean that, bp

cheng_ho
Posts: 128
Joined: Sun Apr 03, 2016 11:27 pm

Re: Reality check

Post by cheng_ho » Mon Apr 06, 2020 1:05 am

I agree with everyone about the urgency of conserving blood (after all, we GERNiatrics need it for parabiosis ;)

However, please stop saying things like: "...the FDA must do this, or they are absolutely failing their responsibility for the good of these patients, the good of the healthcare system and the good of the people of our nation."

Either read a history of the FDA and learn what its purpose and incentives are, or stop posting about them. Trying to build your model of regulatory agencies out of old movies to make yourself "feel better" is the worst kind of intentional self-delusion.

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