WG RegMed

Regenerative Medicine addresses the repair, replacement or regeneration of damaged tissues or organs via a combination of technological approaches.

Regenerative Medicine addresses the repair, replacement or regeneration of damaged tissues or organs via a combination of technological approaches. They can be divided into two sub-areas: smart biomaterials and advanced cell therapy. Ultimately, it is envisaged as being able to cure specific diseases or repairing damaged tissues, such as cartilage, bone, teeth, muscle, or nerves.

 

Academia is the driver for regenerative medicine with industry still lagging behind in terms of translation of research findings into products. The academic push unfortunately often lacks the industrial knowledge of where the real market opportunities are with the result that comparatively few products are being developed by fully integrated larger companies. This mirrors the inevitably slow introduction of new modalities, such as biological therapeutics, to a heavily regulated sector. Despite the successful and exciting results in recent years, academic research often stops at the first step in product development- demonstration of a proof of concept in a small animal model. Long term safety, standardisation and cost-effectiveness of the proposed solution are often not investigated. This misalignment between knowledge generated in academia and knowledge needed for the clinical, industrial and therapeutic translation is partly responsible, for the lack of regenerative medicine products.

 

The key challenges that have to be addressed to aid translation into profitable products are:

  • The validation of product manufacturing processes to meet the high technical and quality standards required for regulatory approval
  • The proof of long term safety and efficacy
  • The minimization of costs through the scale-up and the automation of the manufacturing process itself (process optimization)
  • Understanding the profitability of these new drugs, e.g. the reimbursement schemes envisaged
  •  The training of professionals for a more effective clinical adoption

 

Example of use of nanotechnologies in Regenerative Medicine, extracted from an Article in the Nanomed Series (International Innovation Magasine) - Issue 133, April 2014.

 

The field of tissue engineering has advanced in the last 15 years, offering the potential for regenerating almost every tissue and organ of the human body. Among current clinical applications are the production of artificial skin for burn patients, tissue engineered trachea and blood vessels, cartilage for knee-replacement procedures, urinary bladder replacement, urethra substitutes and cellular therapies for the treatment of urinary incontinence. With the development of enabling technologies – especially in the field of micro- and nanotechnology – tissue engineering is now increasingly overlapping with nanomedicine, as they share common applications.


SYNTHESISING SCAFFOLDS
General strategies for tissue engineering involve the implantation of isolated cells, tissue-inducing biologics or cell substitutes into the organism, with or without the use of 3D synthetic constructs – commonly referred to as scaffolds or matrices. Scaffolds are porous solid biomaterials designed to perform some or all of the following functions:

  • Promote cell-biomaterial interactions, cell adhesion and extracellular deposition
  • Allow transport of gases, nutrients and factors for cell survival, proliferation and differentiation
  • Degrade at a controllable rate that is optimal for tissue regeneration, without inflammation or toxicity

 

Developing scaffolds that mimic the architecture of tissue at the nanoscale is one of the major challenges in the field. Luckily, the recent development of nanofibres has greatly enhanced the scope for fabricating scaffolds, as nanofibres can potentially mimic the architecture of natural human tissues and microporous structures to favour cell adhesion, proliferation, migration and differentiation.


SAFE AND EFFECTIVE
In regenerative medicine, the prior manipulation of individual cells in vitro and its combination with biomaterials that provide scaffolding support, highlight the complexity of safety and efficacy issues. Similar issues arise with direct in situ injections. Are the cells localised in the appropriate site? How might they survive, migrate and differentiate?


Novel nanocontrast agents, as well as their combined use with cell products, can help document quality control and therapeutic effect. In this context, the use of magnetic resonance imaging (MRI) may improve the monitoring of implanted cells. The addition of nanosystems to advanced therapy medicinal products (ATMPs) can be either an integral part of therapeutic interventions (eg. magnetic targeting; theranostics with drug) or a tool for efficacy control (imaging agent for cell tracking). The most commonly used MRI contrast agents are superparamagnetic iron oxide nanoparticles. Iron oxide nanoparticles may facilitate cell tracking while remaining inert. Nanoformulated materials are thus an active area of R&D.


ON THE HORIZON
Medicinal products based on tissue engineering are classified as ATMPs. At present, ATMPs present major scientific, regulatory and economic challenges. Numerous questions have to be answered before the engineering of tissue and organs can become routine practice.
Nanotechnologies clearly have an impact on tissue engineering. First, the natural niche of cells is structurally hierarchical and nanocomposite in nature. Understanding this niche and taking the biomimicking route should bring improvements to the field of tissue engineering. This progress is parallel to the advancement of tools that effectively probe, monitor, characterise and understand living constructs. As regulators have recognised the importance of cell tracking in the context of ATMPs, novel approaches should be developed to improve safety and detection, and allow for clinical translation.

 

Both fields have yet to produce profitable products. Although nanoparticlebased therapy is becoming more common in the treatment of cancer, integrating a nanosystem into the constructs of tissue engineering has so far proved challenging and has not yet been clinically validated. Both nanomedicine and tissue engineering – as quite recent emerging fields – are clearly having difficulty transitioning from academic research to industrial phases and successful products. However, they are bringing together various disciplines and clinically relevant approaches. This is currently one exciting challenge for academic and industrial developers.

Document Actions