Hat tip to Rori YMB

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biopearl123
Posts: 1665
Joined: Fri Jul 20, 2018 5:13 pm

Hat tip to Rori YMB

Post by biopearl123 » Wed Mar 16, 2022 9:45 pm

Hello all,

I posted here about IMetelstat in 2018 and very impressed to see this board is very alive and active!

I practice Hematology-Oncology in a large private practice in Florida and lead the clinical research program in affiliation with one of the largest research network companies in the US .

I would like to give my prospective here as a clinician and a researcher and NOT as a financial advisor. I will go further to say that I don’t pretend to know more than the average joe about stocks and investment.

MDS is a disease that affect the bone marrow productions lines. The bone marrow is where we produce three different types of cells, Red blood cells (to carry and deliver O2), White blood cells ( to fight infections) and Platelets (to start coagulation) .

MDS happens when an acquired mutation affects the normal order in the marrow which leads to production of less and dysplatic (abnormal) cells. There are different subtypes of MDS but almost always anemia is the first and most common symptoms. For example some patients even with advanced MDS may only develop anemia but still maintain normal or close to normal WBC and Plt production, (MDS with single linage dysplasia) while other patients may develop pancytopenia (low counts in all three lines).

Anemia is the first and most common symptom in MDS. Some patients have indolent disease and may maintain low but yet acceptable level of Hemoglobin (10-12) for years before they require any medical intervention while some patients progress faster and/or have more adverse features (poor risks) as determined by FISH and cytogenetics studies at the time of diagnosis.

As of today, our first line of treatment for MDS patients with anemia is ESA ( Erythropoietin stimulating agents) procrit, Epogen, or Aranesp to name a few.

Patients with a unique type of MDS called 5q syndrome (10-20%) respond very well to an oral chemo, Revlimid.

Some patients don’t respond well to ESA and Almost all will eventually stop responding after weeks to months or even few years and become progressively more dependent on blood transfusions.

Once a patient is declared refractory to ESA, he could be tried on a class of medications called hypomethylating agents ( Dacogen or Vidaza) which works in around 30% in improving Hb levels and some patients become transfusion independent again but eventually months later most fall back to needing transfusions again.

Allogeneic stem cell transplant is the only cure but most patients who develop MDS are 65 plus and would not be deemed eligible and not very motivated to undergo such an invasive and “aggressive” treatment with a mortality rate upwards 15- 20%.

Once you fail ESA, there is a HUGE unmet need for treatment to reduce the burden of blood transfusion in the MDS population, mostly elderly with co-morbidities.

I have followed Imetelstat story for few years. I was not surprised that Geron was told by the FDA to show more data based on a large p3 trial.

I was surprised when J2 dropped them off but again the financial decisions of big business is not my area of expertise nor I care to learn about it.

If P3 study data comes out as strong as the published data and is verified by independent investigators, IMT could become a new SOC (Standard of care) for a substantial number of patients who suffer from this terrible disease.

Hematologists/Oncologists are very quick to adopt new treatments upon approval. Most of the targeted and Immunotherapies were approved in the last 10-15 years. More emphasis on NEW and UNIQUE mechanism of action and less on traditional cytoxic chemotherapy.

I may post again about MF and will be happy to answer any medical questions related to MDS or MF. Again, I am not a financial advisor and I am not promoting any drugs. Just stating the facts.

Best,

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