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Compliance Requirements for Stem Cell Trials

BayBiotech.NET

Stem Cell Therapy although holds promises for the treatment of many diseases, this is an area not much explored yet and many countries worldwide are drafting the guidelines for conducting safer clinical trials. Therefore, it is accepted widely that prior to conducting clinical trials with stem cells a stronger than usual proof of concept may be required for the safety and efficacy of the therapy.

One of the major concerns is the rapid cell division cycles of such cells that may lead to tumor generation. Thus, it is recommended that the dose of administered cells to humans must be well adjusted to below the minimum number of cells observed to form tumors in animal models.
Due to many unanswered questions regarding the safe use of stem cells a long-term follow-ups and Biovigilance must be planned carefully before starting a trial.

In October, 2009,Center for Biologics Evaluation and Research (CBER), FDA, has issued a draft guidance to potential sponsors (cord blood banks, or registries, and individual physicians serving as sponsor-investigators) to assist in the submission of an investigational new drug application (IND) for certain unlicensed (not in accordance with 21CFR 601) hematopoietic progenitor cells, cord (HPC-C), that are needed for treatment of a patient with a serious or life-threatening disease or condition and there is no satisfactory alternative treatment.

The draft guidance is available at the following link: http://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Blood/UCM187146.pdf

Sponsors are encouraged to send in IND and BLA (Biological License Applications) as soon as possible to allow sufficient time for review, comment, and resubmission as needed to complete all actions by the end of this 2 yr period.

Since the cell/ gene therapies are emerging prime areas, a worldwide effort is ongoing for prospective harmonization and convergence of regulatory approaches. Several countries within European Commission as well as other part of the world are working closely via exchange of guidelines to evolve successful strategies for acceptance and harmonization of such therapies.

A world wide map on stem cell guidelines is available at: http://www.mbbnet.umn.edu/scmap.html.

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