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Cell therapies for cardiovascular-related conditions is a closely watched, much studied, oft-discussed, and hotly contested segment of the cell therapy industry.
The data to-date are admittedly confusing. From a clinical perspective, the studies for which we have data have been relatively small involving a mish-mash of indications, endpoints, eligibility criterion, methods and/or route of administration, as well as the time of administration relative to event or disease progression.
Further compounding any interpretation of the data, from a technical perspective, is the fact the products have been widely varied in terms of being autologous vs allogeneic, expanded and not, genetically modified and not, from a plethora of different sources, and utilizing a wide variety of cell types from skelatal myoblasts, cardiomyocytes, mesenchymal stromal cells, mononuclear cells, etc.
All this makes it extremely difficult to draw any conclusions with respect to what's working and what's not. We will not attempt to do so.
All we do below is attempt to give a snapshot of the industry-sponsored cell therapy trials currently ongoing for cardiovascular-related conditions. So here it is:
Commercial:
Pharmicell's Heartcelligram is the only cell therapy to have received regulatory approval for commercial distribution for the treatment of a cardiac-related indication. Heartcelligram is an autologous cell therapy approved in 2011 by the Korean Food and Drug Administration (KFDA) for the treatment of Acute Mycardial Infarction (AMI). The price is reportedly $19,000 and the trial data behind the approval has not yet been published in a peer-reviewed journal.
Phase III or II/III:
There are currently only 3 active and recruiting cardiac-related, industry-sponsored cell therapy trials. Interestingly they all involve autologous products, two involve devices, two involve centralized manufacturing, two involve bone marrow cells as a source, two are only in European clinical sites, and two are targeting ischemic-related conditions.
- Baxter Therapeutics' Auto-CD34+ cells
- phase III trial actively recruiting
- Indication: refractory angina and chronic myocardial ischemia
- Estimated enrollment: 444
- Estimated primary completion: June 2016
- Cytori
- phase II/III trial actively recruiting
- Indication: ST-elevation acute myocardial infarction
- Estimated enrollment: 360
- Estimated primary completion: July 2014
- Miltenyi Biotec
- phase III trial actively recruiting
- Indication: myocardial ischemia or coronary artery disease
- Estimated enrollment: 142
- Estimated primary completion: July 2012
Two companies warrant particular mention at this stage as they appear to be in transition between phases II and III.
Cardio3 Biosciences initially designed a trial of their autologous C-Cure in heart failure secondary to ischemic cardiomyopathy to be a phase II/III trial enrolling 240 patients. While the trial began in late 2008 and is still registered as active but no longer recruiting on ClinicalTrials.gov the entry has not been updated for almost a year.
In 2010 the company announced that after enrolling 45 patients - of which 21 were in the treatment arm (24 in the control arm) - they decided to close the study to future enrollment and prepare for a phase III trial. This decision was reportedly based on "very encouraging data".
Dr. Christian Homsy, CEO of Cardio3 BioSciences provided the following guidance: “The highly promising data we report today build on the favourable safety profile we have observed through this Phase II trial and documents in patients our belief that we have with C-Cure a product candidate with the potential to make a real difference in the treatment of heart failure... As noted in the company’s press release of 29 June 2010, with the Phase II stage completed and to allow for potential modifications to the trial protocol, Cardio3 BioSciences has not proceeded to Phase III recruitment into the trial but has continued to gather all data for the six month analysis. Through the Phase II trial, we gained significant experience in working with a highly innovative stem cell therapy in a clinical setting, and we are using this acquired knowledge in the design of our planned Phase III programme." The phase III trial of C-Cure is expected to commence in the second half of 2012.
Mesoblast has also announced with its strategic partner, Teva, that they are proceeding with plans to conduct a phase III study of its allogeneic cell therapy product, Revascor, in chronic heart failure. Most anticipate this clinical trial application to be filed sometime in late 2012.
Mesoblast has also announced with its strategic partner, Teva, that they are proceeding with plans to conduct a phase III study of its allogeneic cell therapy product, Revascor, in chronic heart failure. Most anticipate this clinical trial application to be filed sometime in late 2012.
Phase I or II:
There are over 20 active, industry-sponsored earlier-stage trials (phase I, I/II or II) for cardiovascular-related conditions. At least 5 of these are expected to have clinical readouts this year.
Hope this is useful.
