Cell therapies: realizing the potential of this new dimension to medical therapeutics

Abstract

Stem cells promise to treat conditions poorly served by conventional therapeutics. Cells from both embryonic and somatic tissues are being used to create cell therapies for genetic, traumatic and degenerative conditions. The current human, healthcare and fiscal costs of these conditions are significant. This review summarizes the use of stem cells for neurological and cardiac disorders and diabetes to determine the requirements for generic translational research to assist such therapies to be a reality. While there are multiple strategies in each disease area, with no clear favourite, there are clear opportunities in treatments that use a single cell type. A key requirement is to work with pluripotent progenitor cells to cultivate and differentiate a sufficiently large population of functioning cells. Challenges also arise in determining and achieving timely delivery of the correct dose of cells to where they can most effectively treat the disease and best benefit individual patients.

Concise Review: Mind the Gap: Challenges in Characterizing and Quantifying Cell- and Tissue-Based Therapies for Clinical Translation

“…They evaluated angina frequency, nitroglycerine use, exercise tolerance, single‐photon emission computed topography imaging, and quality of life testing [43]. Other groups have used enhanced myocardial contractility and left ventricular ejection fractions, both in relation to normal limits, as suitable endpoints for their cardiac applications [44]. However, due to the complexity of several clinical applications, optimal efficacy measurements may evolve over time due to improved clinical information becoming available to inform the decision.…”

Thermo-responsive poly(N-isopropylacrylamide)-grafted hollow fiber membranes for osteoblasts culture and non-invasive harvest

“…It's important for regenerative medicine including tissue engineering and cell transplantation to harvest a large number of therapeutic cells, usually in the order of 10 10 cells for each patient [1,2]. Owing to the large scale area-to-volume ratio involved, hollow fiber membranes (HFMs) provide a tremendous amount of surface area for high cell number in relatively small volume of media.…”

Precision manufacturing for clinical-quality regenerative medicines

“…Researchers globally regard stem cell therapies as a treatment option with the potential to alter the face of contemporary medicine, and ultimately give a new and effective dimension to medical therapeutics. Recent work in RM (see the review [3]) has provided the proof of principle for cell-based replacement for a number of structures ranging from skin, musculoskeletal and neuronal tissue to osteochondral grafts and complex organs such as the kidney, which may supplant more conventional therapeutics to revolutionize current medical practice.…”

“…Clinical endpoints have to be defined at an early stage to allow for proper evaluation of cell-based therapies in patient trials. For cell-based treatments, the endpoints will have to be patient-specific, relative to the age of the patient, as well as being related to when the disease was diagnosed, to account for how any existing complications will affect the treatment outcome [3]. However, owing to the complexity of several clinical applications, optimal efficacy measurements may evolve over time due to improved clinical information being available to inform the decision.…”