Cell Therapy News/Articles, page-16789

Whytee..... I really don't understand why you read MSB posts. I think it's beyond the majority of the posters here. You need to do your research without your blinkers on.


Why did mesoblast not do an adult trial years ago? Here are a bunch of reasons:

1. The last adult trial failed - so why do another?

• I need to mention though - at the time this trial was almost entirely made up of skin GvHD adults, which the company has ( for years now ), realized that MSC IV injection is not possible to migrate to cover all the skin without massive doses. Skin being the largest organ in the body.

2. The last trial pre GvHD001 did show promising signs in children for response - so why do a trial in adults ?

• I also need to mention, this was around the time where the company were also discovering the treatment was not good for skin, but also they did not have long term mortality figures yet.

3. On the basis the last adult trial failed, and the most recent treatment data for adults was almost all skin GvHD with the new Remestemel formula, and previous to that ( unfortunatley ) , the product was Osiris which had something like 1/3rd the potency of the current product. The company would be utterly insaine to follow your advice and run another adult trial.

4. Post the GvHD 001 trial CRL 3 years ago.

• The FDA told MSB not to run any more trials until the potency assay was fixed
• The company had a new tool being the validated MAGIC biomarker scores, and post hoc were able to confirm in the GvHD001 trial - particularly for patients with high MAGIC scores, high risk patients that the treatment worked extremely well - although this data was only in children.
• Last year, they were also able to confirm long term survival data for children, using the new Remestemcel formula which they also did not have, and which supports the notion of the cells having greatest benfit on the sickest children.

5. Let's wait and see what the FDA say this month. You never know they may just accept mesoblasts data and approve for children, and then in that case great decision not to waste " hundreds of millions" on the children trial plus an adult trial.