MDS foundation - first post I read

[rccola335]

I think we all can read this and think what would probably be helpful and it is shameful it was not approved on a priority basis -

"My mother’s is diagnosed for Myelodysplastic syndrome (MDS) and Erythropoietin was the treatment. In the beginning, Erythropoietin seems to work for my mother but now we are back to the same condition, her Hb is dropping in 2-3 months and she needs blood transfusion. Below are the Hb levels and the treatment (medicines and Injections she was advised).

Then from last 6 months, she is on luspatercept 50 mg injection, however its also not working, the blood transfusion needs to happen every 25-30 days now a days.

Now doctors have confirmed that luspatercept 50 mg injection is not working as well, and so my mother had to be on blood transfusion as long as she lives

Reaching out to this forum to see if there is any treatment available for her, my mom is in India"

[kmall]

RC - did you get a date on that post?

[biopearl123]

January 16, 2024

[biopearl123]

If the MDS foundation is true to their mission, at the very least a link to the EAP on clinical trials should be posted, preferably by the hosts of their website. That is unless there are other censorial forces at play.

[rccola335]

i did not register and reply to it - I thought it might be inappropriate considering my position - I have a feeling that story will be a common one

[rccola335]

there was the post the other day about the guy on imtetelstat and feeling better than he had in the last 5 years ( I was also suspicious of his start day on Jan 23) - and it reminded me of the lady in australia that was on the news who received imetelstat for AML from Dr Lane and said how great she felt on it (I was surprised that went public) and I can not find that video anymore (i can find a partial video but the part where she talks about how great she feels and wished she could make a donation is nowhere to be found)

did Geron ask for standard review so they could also look at their data for Impress with the FDA? that data is supposed to be available this month - I think imetelstat will be in for big days when you look at how the KOL's seem so excited about it , the few patient responses we have seen - and Dr Lane in australia saying the were seeing positive results for AML back in October (remember JNJ had imetelstat on its list for AML, and MDS pricing manager back in 2019)

[biopearl123]

RC, good question about standard review. I doubt Geron would pass up the possibility for priority review as they could always go back to the FDA with new AML data later. I think priority was not granted because, well I just don’t know why. In my mind it was a mistake, or driven by other factors, perhaps the protection of the Luspatercept franchise for another few months, or because it is currently regarded as second line therapy. Re the Dr. Lane, Dr. Bruedigam, nice Australian lady, AML, Imetelstat video, I never saw anything like a testimonial as to whether she felt better or had a clincal response to treatment. That surprised me as its absence was glaring. I am glad you were able to see it and relate it to us so at least we know it was there initially. Perhaps it was edited out for regulatory reasons. I was struck by the absence of any mention of how the drug affected her but was glad to hear Dr. Lane say the results were promising and see how a justifiably beaming Dr. Bruedigam was clearly delighted at having elucidated a clear previously undefined MOA (non canonical) for Imetelstat. And that after five years of committed non stop work. Sounds like Blackburn/Greider quality work. As Kmall has pointed out the clinical trials site lists Jan 2024 as the release for top line data in the single agent HR/AML study. But I don’t think we are going to see that data this month. (Weren’t we promised an update on the oral agent too? Come on John.) Love to be wrong about that one! Regards, to all bp

[huntingonthebluffs]

Members probably know this but the EAP is not available in India or anywhere outside of USA.

Per the Geron website:
The imetelstat expanded access protocol (EAP) is a U.S.-only protocol for eligible adult participants diagnosed with lower risk (very low, low, intermediate risk by IPSS-R) myelodysplastic syndromes (MDS) who are red blood cell (RBC) transfusion dependent, have failed to respond or have lost response or are ineligible for erythropoiesis-stimulating agents (ESAs), had not received prior treatment with either a hypomethylating agent or lenalidomide and were non-del(5q). Additional information to be available on www.clinicaltrials.gov.

U.S.-based healthcare professionals seeking more information about the imetelstat EAP can email GeronEAP@wepclinical.com.

Lower risk MDS patients who are interested in enrolling to the imetelstat EAP should speak with their physician to understand if imetelstat is an appropriate treatment option.

[biopearl123]

Hunt, good point, thanks for the clarification.