Athersys and their cardiac parter, Angiotech, announced today that they got the ok from the FDA to begin a Phase I clinical study using Athersys’ Multistem adult stem cell product in acute myocardial infarction (MI). This is the second IND approval for Athersys in just over a month (and a welcome Christmas present for the Cleveland-based company). In my last post on Athersys, I suggested that they were accepting some market risk by their choice of indications in order to minimize clinical risk. However, cardiac regeneration is the Holy Grail of cell therapy today, as this is one of the most substantial markets available for cell technologies. The first (and maybe second or third) cell therapy company on the market with a successful cardiac product that can be manufactured at the necessary scale will begin building a Genzyme-like market capitalization. This is a very important milestone for this company.

The two press releases (1, 2) actually provide some interesting details:

1) Athersys has partnered with Angiotech for this and other cardiovascular indications.

2) The cells have been independently tested at Angiotech. If Angiotech is going to stick their neck out for a cardiac trial, they must have wanted to verify that the preclinical model also worked for them. It is good news that this partnership has continued to this level.

3) The adult stem cells will be “injected directly into and around the zone of myocardial injury from an intra-coronary approach” using an infusion catheter. This is different than Osiris’ intravenous infusion administration of Provacel, and looks to be a device approach that optimizes delivery vs. a reagent approach that Cellcyte is taking.

4) The quote from the press release states that the “approach is designed to provide… efficient, localized delivery and enhanced cell retention in the area of greatest need.” This suggests that they have optimized cell retention via novel delivery strategies either via the device or that there is a biomaterial component to the formulation that enhances cell retention at the site – which would explain Angiotech’s role in the collaboration. Angiotech has many hydrogel-based materials (from their acquisition of Cohesion Technologies a couple years ago) that could be used for these types of applications.

5) Lonza is their contract manufacturer, and the press release suggests that Lonza has verified the possibility that a single donor could “produce hundreds of thousands or even millions of clinical doses.” Thus, raw materials for therapeutic cell production could be minimized (but I wonder if a really good donor could negotiate royalties on sales?).

Unfortunately, this trial is not listed on ClinicalTrials.gov, as I would be interested in checking out the exclusion criteria and see if they talk more about mode of delivery. A search of ClinicalTrials.gov does turn up over 30 stem cell cardiac trials – and the space is getting more and more crowded with time. However, the field is wide open and Athersys is staking their claim as the first allogenic, scalable, and directly injected cell therapy for cardiac regeneration.