Cell Therapy News/Articles, page-13517

Let me pick up on a few concerning points @DocMcstuffins

1. It is my strong opinion that your risk to benefit profile for direct injection of Rex-L into the myocardium is some way off. You just need to see the amazing MACE results achieved with an outstanding safety profile in the DREAM-HF trial. I stress again, Dr Perin and the many other Cardiologists across the many trial sites would certainly agree that the benefits far outweigh the risks. Even the Ethics Committee would have come to this opinion before the trial commenced (green lighting a SHAM procedure for the non intervention group to neutralise biases).

2. It is my strong opinion that you do not fully understand the benefits of a direct injection over a systemic infusion for treating conditions that are more localised like CHF or CLBP or perianal fistula. Direct injection allows for more cells to be directed to the exact place of need with an increased exposure time. This direct route also bypasses the pulmonary first-pass effect which occurs during intravenous administration of MSCs where it is proposed that the cells tend to first localise in the lungs.

3. What I do agree on is that a systemic IV infusion is easier to perform and less specialised staff may administer (but this also has risks that you may be aware of). However IMO CHF is a somewhat more localised condition and would benefit from a more localised treatment approach (i.e. localised injection performed by a Cardiologist). In the DREAM-HF trial this procedure only required an overnight stay. On top of this we were also able to reduce strokes due to the homing effects of the cells. So why would you mess with a good thing? There are many thousands of patients right now that would greatly from our therapy (even a chance the FDA sees it as unethical to deny such patients access to this treatment in the short term - the upcoming FDA decision will be interesting and welcomed soon). I would not hold up progress here on the very very very very off chance that a more convenient IV mode of delivery could provide superior results. IMO changing the delivery method to IV infusion just won't be nearly as easy to get a high enough concentration of cells to the inflammed parts of the myocardium and it would also likely require much larger doses and/or more than one infusion which in itself may pose addition safety concerns. For me the risk to benefit ratio strongly favours localised injections directly into the inflammed myocardium - as per the DREAM-HF trial protocol).

4. Of course an IV infusion may be more beneficial for multi-organ inflammation as occurs in aGvHD (liver, gut and skin) or conditions affecting the lungs such as ARDS (in this instance the pulmonary first-pass effect is advantageous because we are targeting inflammation in the lungs); but as discussed above it would be far less beneficial for the localised conditions of CHF, CLBP, perianal fistula (I am sure the work of Dr Amy Lightner will reveal localised injection for perianal fistual works best).

My point in short, it depends on the extent and location of the inflammation as to whether a localised injection or a systemic infusion or both will be most beneficial. I just strongly disagree with you that a systemic infusion will outperform a localised injection for CHF; as did the experts in the field before and after the DREAM-HF trial. Do you think that the FDA asked us for further analysis if they were concerned about, or were at odds, with their direct injection risk to benefit analysis? So simply put, the Phase 4 confirmatory trial (or second phase III) trial will remain a direct injection - so there is no point discussing further - it will not change anything - so no need to further clog up the boards to throw shade/doubt.

5. Yes I am happy with my holdings. I have profited from trading MSB technically (one trade making over $20K in two days). Vastly more important I have taken advantage of the recent lows to bring my average down to under $2 (because I firmly believe in the science; the SP is trading at a significant discount IMO; the markets we are targeting are HUGE - upside galore).

* Disclosure for the Doc - I am not a doctor, I do not work in a hospital; however I do have an undergraduate degree in medical science, a masters degree in public health and health economics, have worked extensively in the life science research and pharmaceutical industry primarily in sales, management and and health outcomes positions - which included working on submissions to the Pharmaceutical Benefits Advisory Committee - the committee that informs the government whether a certain medication is cost-effective and therefore to be recommended (or not-recommended) for inclusion on the PBS. So I do have expertise in reading clinical trials and determining whether a medicine is cost-effective to the health system. What's more I also have a share trading and investment diploma. *

So enough of your comments like: you don't know what its like to prescribe drugs that are not in the drug cupboard, you have never done a cardiac catheterisation, you only know what you google nonsense. As you are aware there are many informed people on this forum in this space (you don't need to be a doctor or work in a hospital to have a grounded view).