Cellprothera said its decision to run clinical trial of heart attack cell therapy itself rather than in collaboration with a CRO reflects the complex regulations and logistics involved in testing such patient-specific therapies.
The Mulhouse, France-headquartered firm published results from a Phase I/IIb trial of its post-heart attack cell therapy – ProtheraCytes - this month, explaining the product was associated with improvements in “multiple efficacy endpoints” in subjects.
ProtheraCytes is composed of stem cells derived from CD34+ cells harvested from the peripheral blood of the patient being treated. The aim of the therapy is to regenerate damaged tissues, in this case cardiac tissue.
CellProthera opted to run much of the study – which involved 49 patients at 13 sites in France and the UK – itself according to CEO Matthieu de Kalbermatten, who told Clinical Insider supply chain control was key.
“We have been working with a French CRO called Multihealth Group, in charge of data management and Pharmacovigilance, and partial site monitoring.
“Regulatory activities were internalized. Monitoring activities were assigned to freelance senior CRA in the UK. MRI analysis was assigned to an independent corlab. E-CRF was assigned to a French digital e-solution company.”
“However,” Kalbermatten added “CellProthera wanted to keep control over the clinical operations, maybe more than usual. It is an autologous therapy which requires fresh material and a smooth transfer from production centres to cardiology centres, back and forth. It was key for us to manage the whole process, solve bottlenecks to improve feasibility and accumulate along the way precious know-how.”
And the know-how covered a range of areas, from study design through to regulatory approvals according to Kalbermatten.
“Due to our specific product and protocol, the regulatory timelines were longer than usual. Dealing with biologic material, the timing for regulatory approval is extend as it requires, on top of national authorities’ approval - MHRA for UK, ANSM for France -, also to have a human tissue license from the HTA in the UK and transfer agreements in place between production centers and heart centers, which can take time.
“Similarly, the use of SPECT nuclear imaging in the protocol requires specific authorization from ARSCA in UK for example, which can also extend time before starting the trial,” he said.
Stem cell potential
In a press statement Faiez Zannad, professor of therapeutics at the Université of Lorraine, who was chairman of the study’s steering committee and emeritus, was similarly positive about potential of the approach for cardiac regenerative therapy.
“[This] data suggests patients at high risk following a heart attack may benefit from treatment with ProtheraCytes, to help regenerate post-ischemic myocardial damage and prevent progression to further cardiovascular illnesses. It should be further evaluated in a larger Phase III trial.”
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