Regenerative medicine

Guaranteeing the safety and efficacy of regenerative biotherapies, such as stem cells and tissue engineering, is challenging because they often involve complex biological systems and processes that are not yet fully understood. Furthermore, they may involve the use of living cells, which can have unpredictable responses in the body, and whose long-term effects are difficult to predict or measure. Third, there is a lack of standardized clinical protocols and regulatory, and given the costs of clinical trials, it can be difficult to gather sufficient data to assess their safety and efficacy.

How to ensure safety of these therapeutics? What kinds of risks are associated and how to manage such risks?

The immune system is designed to recognize and attack foreign cells or organs that it perceives as a threat, including transplanted cells. If a threat is perceived, the body may reject transplanted cells, even if they are derived from the patient’s own body (autologous transplantation). Additionally, an embryonic stem cell or engineered tissue may be modified or manipulated in ways that make them more likely to be recognized as foreign by the immune system.

What kinds of clinical data exist today that measure rejection of transplanted cells? What is the state-of-the-art on the immune rejection of transplanted issues?

Regenerative biotherapeutics often involve living cells requiring specialized growth conditions and handling procedures, which can be difficult to reproduce on a large scale. For instance, a cell or tissue used in these treatments may be highly sensitive to changes in their environment, and it may be very difficult to scale up manufacturing while maintaining compliance with regulatory requirements. In this sense, real-time monitoring may be challenging, as requires the development of sensors and monitoring systems that can provide accurate and reliable data in time. Regenerative therapies, like stem cells and tissue engineering, are linked to unsteady supply-chains, where the costs of raw materials and time-sensitive logistic requirements complexify procurement tasks. Moreover, the absence of homogeneous control measures for the manufacturing of regenerative therapies can lead to inconsistencies in quality.

How to develop an appropriate protocol for scalable manufacturing of regenerative biotherapies? To what extent will the energy and raw materials crisis impact them? How to ensure consistent quality in the manufacturing of these therapeutics?

Whenever integrating embryonic stem cells into target tissues it is challenging to control the differentiation and integration of the cells. While, embryonic stem cells have the ability to differentiate into multiple cell types, it can be difficult to recreate the complex signals and interactions that occur in the native tissue. Furthermore, the behaviour of embryonic stem cells can be influenced by factors such as age, disease status, and genetic background, making it difficult to predict their behaviour in different individuals or diseases contexts. Researchers have progressively highlighted the a need for non-invasive methods to track the fate and behaviour of transplanted embryonic stem cells over time.

What is the scientific state-of-the art with respect to differentiation and integration of stem-cells into target tissues? How will non-invasive methods shape regenerative therapies?

The clinic use of human cells has been raising several ethical concerns related to safety, equity, and unintended consequences of these ground-breaking innovations. Over the past few years, the need for clear regulatory frameworks has become pivotal, to ensure the safety and efficacy of regenerative medicine, while balancing the necessity for research. The global nature of regenerative medicine research and development can make it challenging to harmonize ethical and regulatory standards across different jurisdictions and cultural contexts and there is a need for a joint effort to overcome this important aspect of healthcare innovation.

How to engage different stakeholders, including patients, researchers, policymakers and industry, in the regulation of stem-cells? What are the current and future main roadblocks to the regulation of these therapeutics?

Regenerative medicine therapies are often expensive, and the cost can be a barrier to access for many patients. Additionally, there may be a limited number of centers that offer these therapies, which can make it difficult for patients to access treatment. Finally, in countries where insurance plans do not cover regenerative medicine therapies, access to such treatments is even more limited for patients. Addressing these challenges will be essential to ensure that patients have equitable access to the benefits of regenerative medicine.

How to facilitate the access to regenerative treatments? What key factors will play a role in the reimbursement?