Trials, page-14

We patiently wait for the official 008 study to give us evidence of potential DMOAD effects at trial level...that's in regards to OA...MPS again has it's own pathway and it's own set of observations but we already know about some of the evidence....

Here is a refresher on the back of Gan Gan's evidence:



Rat - MPS trial conducted back in June 2014, Icahn School of Medicine at Mount Sinai

Mate, to me it reads like the nominations for a few of those Academy Awards in Hollywood:

This is what OUR iPPS does:

• Markedly improved mobility.
• Improved tracheal morphology.
• Increased trabecular bone in the femurs and vertebrae.
• Improved dentition, including tooth mineral densities.
• Reduced levels of inflammatory markers in the serum.

I didn't make that up...this is Not Mozz words about what I think or what I hope...this is from the PLoS ONE paper (see reference below).



This line also did it for me:

"we did observe positive effects on skull/snout lengths with both sc and oral treatment".


...and one more statement in the final discussion of results:


"As noted above, microCT analysis revealed improvements in femoral and vertebral trabecular bone with PPS treatment."



There was also highly relevant data for MPS GAG levels as evidenced by this statement from the researchers:


"One of the surprising, positive outcomes from this study was that PPS treatment led to a reduction of GAG storage in the MPS VI rats. A notable difference between the oral and sc treatments was that in the latter significant reductions in tissue GAGs were observed."



These reduced GAG levels were noticed even in organs such as the Liver and Spleen due to iPPS's action.

All very good Mozz...but was it safe?

Again I turn to the paper:

"Throughout the 6-month study we monitored liver enzymes and various clotting factors weekly. There was no elevation of liver enzymes above baseline, and no abnormalities seen in clotting factor levels. No adverse effects of sc PPS treatment were observed."




I've asked you in the past, do we really know what we truly own? One of the final paragraphs sums up just how compelling this iPPS actually is:



"When considering the clinical utility of PPS in MPS patients, we predict that it may be used alone or in combination with other therapies, including ERT or HSCT. When used alone we anticipate that it will reduce inflammation systemically, as well as locally in joint tissues including the synovium and articular cartilage. This could result in reduced pain, increased mobility, and/or increased joint range of motion. Over time we also predict that it may slow the progression of tracheal deformities, enhance trabecular bone and bone strength, and perhaps result in reduced pulmonary infections and/or enhance lung function."


Wait, so thats:

Reduce Inflammation systematically
Reduce Pain
Increase mobility
Increase joint range of motion
Slow progression of tracheal deformities (!)
Enhance bone strength
and MAYBE, just maybe, reduce pulmonary infections as a bonus for us.

I rest my case.




Access to the answers before the test? Amazing.




REFERENCE
https://www.researchgate.net/publication/263432054_Dose_Responsive_Effects_of_Subcutaneous_Pentosan_Polysulfate_Injection_in_Mucopolysaccharidosis_Type_VI_Rats_and_Comparison_to_Oral_Treatment