ABSTRACT
Advanced Therapy Medicinal Products (ATMPs) comprising cell, gene, and tissue-engineered therapies have demonstrated enormous therapeutic benefits. However, their development is complex to be managed efficiently within currently existing regulatory frameworks. Legislation and regulation requirements for ATMPs must strike a balance between the patient safety while promoting innovations to optimize exploitation of these novel therapeutics. This paradox highlights the importance of on-going dynamic dialogue between all stakeholders and regulatory science to facilitate the development of pragmatic ATMP regulatory guidelines.
ABSTRACT
Osteoarthritis (OA) is the most common degenerative joint disorder worldwide and to date no regenerative treatment has been established in clinical practice. This review evaluates the current literature on the clinical translation of mesenchymal stromal cell (MSC)-based therapy in OA management with a focus on safety, outcomes and procedural specifics. PubMed, Cochrane Library and clinicaltrials.gov were searched for clinical studies using MSCs for OA treatment. 290 articles were initially identified and 42 articles of interest, including a total of 1325 patients, remained for further examination. Most of the included studies used adipose tissue-derived MSCs or bone-marrow-derived MSCs to treat patients suffering from knee OA. MSC-based therapy for knee OA appears to be safe and presumably effective in selected parameters. Yet, a direct comparison between studies was difficult due to a pronounced variance regarding methodology, assessed outcomes and evidence levels. Intensive scientific engagement is needed to identify the most effective source and dosage of MSCs for OA treatment in the future. Consent on outcome measures has to be reached and eventually patient sub-populations need to be identified that will profit most from MSC-based treatment for OA.
