Biotechnology company Bioheart has commenced its FDA-approved clinical trial REGEN to assess the safety and efficacy of its gene-modified stem cells, MyoCell, on patients who suffer from congestive heart failure (HF).
The phase I clinical trial, conducted by lead investigator Imad Alhaddad, MD, co-director of the Jordan Cardiovascular Center in Plymouth, Mass., will investigate the gene-modified Myocell, a regenerative cell therapy that uses myoblasts/muscle cells genetically modified to produce a higher quantity of SDF-1 proteins, as a prospective treatment for congestive HF patients.
The REGEN trial will enroll 15 patients in a multicenter, randomized, dose escalation study to assess the safety and cardiovascular effects of the implantation of MyoCell SDF-1 in congestive HF patients post myocardial infarction.
According to the Sunrise, Fla.-based Bioheart, four preclinical trials have shown that the gene-modified MyoCell can be more effective than treatment with the non-modified MyoCell during tissue regeneration and repair.
Bioheart said that REGEN trial participants will receive a biopsy of the leg muscle. Cells acquired from the biopsy will then be sent back to Bioheart where they will be expanded and induced to produce myoblasts. There, the myoblasts will be segregated from the muscle until one billion cells are grown and then transferred back to the patients’ treatment center where the cells will be injected via a needle-tipped injection catheter into the patient’s heart.
Myoblasts will be created with use of an adenovirus vector of a non-viral vector and their increased levels of the SDF-1 protein will stimulate angiogenesis and promote the regeneration of tissue.
According to the company, the heart muscle cannot release enough SDF-1 protein to attract the number of stem cells that would self-heal congestive HF patients and this can result in scar tissue and impaired heart function. Use of SDF-1 proteins facilitates a more rapid repair of the heart’s tissue.
During preclinical animal studies, 54 percent of the animals injected with the gene-modified MyoCell had improved heart function, compared to 27 percent of the animals treated with MyoCell without gene-modification.
After completion of the REGEN trial, Bioheart said it will conduct a further Phase II and III MARVEL study. The REGEN trial is being funded by Ascent Medical Technology Funds and jointly conducted by the Philadelphia BioMed Produce Development Center and research organization.