Monthly Archives: October 2017

Regenerative medicine may be defined as the process of replacing or "regenerating" human cells, tissues or organs to restore or establish normal function. This field holds the promise of regenerating damaged tissues and organs in the body by replacing damaged tissue or by stimulating the body's own repair mechanisms to heal tissues or organs. Regenerative medicine also may enable scientists to grow tissues and organs in the laboratory and safely implant them when the body is unable to heal itself. Current estimates indicate that approximately one in three Americans could potentially benefit from regenerative medicine.

Regenerative Medicine refers to a group of biomedical approaches to clinical therapies that may involve the use of stem cells. Examples include cell therapies (the injection of stem cells or progenitor cells); immunomodulation therapy (regeneration by biologically active molecules administered alone or as secretions by infused cells); and tissue engineering (transplantation of laboratory grown organs and tissues). While covering a broad range of applications, in practice the latter term is closely associated with applications that repair or replace portions of or whole tissues (i.e., bone, cartilage, blood vessels, bladder, skin). Often, the tissues involved require certain mechanical and structural properties for proper functioning. The term has also been applied to efforts to perform specific biochemical functions using cells within an artificially-created support system (e.g., artificial pancreas or liver).

Cord blood stem cells are being explored in several applications including Type 1 diabetes to determine if the cells can slow the loss of insulin production in children; cardiovascular repair to observe whether cells selectively migrate to injured cardiac tissue, improve function and blood flow at the site of injury and improve overall heart function; and central nervous system applications to assess whether cells migrate to the area of brain injury alleviating mobility related symptoms, and repair damaged brain tissue (such as that experienced with cerebral palsy). Cord blood stem cells likely will be an important resource as medicine advances toward harnessing the body's own cells for treatment. Because a person's own (autologous) stem cells can be infused back into that individual without being rejected by the body's immune system, autologous cord blood stem cells have become an increasingly important focus of regenerative medicine research.

Regenerative medicine has made its way into clinical practice with the use of materials that are able to assist in the healing process by releasing growth factors and cytokines back into the damaged tissue (e.g., (chronic) wound healing). As additional applications are researched, the fields of regenerative medicine and cellular therapies will continue to merge and expand, potentially treating many disease conditions and improving health for a variety of diseases and health conditions.

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Regenerative Medicine - AABB

Resource page

For the purposes of the information provided on this page we have adopted the definition of the term regenerative medicine that was used in the House of Lords Regenerative Medicine Report (see below). This was:

regenerative medicine is used to refer to methods to replace or regenerate human cells, tissues or organs in order to restore or establish normal function. This includes cell therapies, tissue engineering, gene therapy and biomedical engineering techniques, as well as more traditional treatments involving pharmaceuticals, biologics and devices.

Each regulator has a clear remit and regulates distinct areas of the regenerative medicine process. However, we work closely together to provide effective advice and guidance to support establishments through the regulatory requirements. Each regulator has a core set of standards that apply depending on where you are in the process, from cell derivation to treatment. We are all focused on ensuring that the standards that are applied at one stage of the process do not act as a barrier at another.

The role of each of the regulators in regenerative medicine is set out below:

Health Research Authority (HRA) has a remit to provide an ethics opinion on clinical trials. Those involving gene therapy regenerative medicines are reviewed through the Gene Therapy Advisory Committee (GTAC). Other regenerative medicine studies may be reviewed by other appropriately flagged RECs. It also provides the Integrated Research Application System (IRAS) through which applications and approvals from GTAC/RECs and MHRA for clinical trials involving regenerative medicines can be made.

Human Fertilisation and Embryology Authority (HFEA)[external link] regulates the use of human embryos or human admixed (human-animal) embryos to derive stem cells for use in the treatment of patients.

Human Tissue Authority (HTA) [external link] remit includes regulation of organisations that remove, store and use of human tissue or cells; this includes where they are used as starting materials for Advanced Therapy Medicinal Products (ATMPs). Under the European Union Tissues and Cells Directives (EUTCD), it licenses establishments that remove, test, process, store, and distribute tissues or cells that will (or may) be used to treat patients.

Medicines and Healthcare products Regulatory Agency (MHRA)[external link] remit includes responsibility for granting the appropriate authorisation for the manufacturing site of ATMPs, which are prepared and used under the hospital exemption, and for ATMPs made and supplied under the specials scheme under the relevant provisions in medicines legislation. In the area of clinical trials, the MHRAs remit includes assessment of applications for clinical trial authorisation and the associated manufacturers licence for investigational ATMPs. The National Institute for Biological Standards and Control (NIBSC) [external link], which houses the UK National Stem Cell bank, is part of the MHRA.

Please refer to the Research Community area of the website for information about the approvals for research studies and how to apply to individual review bodies. Further information about GTAC is also provided on this site. Additionally the Stem Cell Toolkit [external link] provides regulatory routemaps that are specific to individual stem cell projects.

Department for the Environment Food and Rural Affairs (DEFRA) [external link] has an Advisory Committee on Releases to the Environment (ACRE) [external link], which advises government on requests for permission to release genetically modified organisms (GMO) into the environment. In 2013, this committee published advice on gene therapy clinical trial for heart disease.

Health and Safety Executive (HSE) [external link] has the Scientific Advisory Committee on Genetically Modified Organisms (Contained Use) SACGM (CU) [external link]. This committee provides technical and scientific advice to the UK Competent Authorities on all aspects of the human and environmental risks, and is responsible for maintaining guidance on the contained use of GMOs.

From 13 October 2014, the MHRAs Innovation Office is the portal for all regulatory queries concerning regenerative medicines. A one stop shop service provides a single point of access from the four regulators in the field, the Human Tissue Authority (HTA), the Human Fertilisation and Embryology Authority (HFEA), Health Research Authority (HRA) and the Medicines and Healthcare products Regulatory Agency (MHRA), who will provide a co-ordinated single response service for free regulatory advice.

Any query relating to the regulation of regenerative medicines, including Advanced Therapeutic Medicinal Products (ATMPs) can be submitted to the MHRAs Innovation Office and will be answered by the relevant experts from the four regulatory bodies.

Individuals or companies who have regulatory questions concerning regenerative medicines and who are unsure which agency to direct their inquiry to, or have a query that impacts several regulators, should use the Innovation Office advice form.

The HRA and others work closely together and will continue to engage with those involved in regenerative medicine, including researchers, the British Society for Gene and Stem Cell Therapy [external link], and the Cell Therapy Catapult [external link] to help clarify the regulatory requirements that apply.

The HRA recently held a regenerative medicine event hosted by the Cell Therapy Catapult to look at changes and discuss issues with the sector, regulators and representative bodies. Additionally in 2012, the MHRA hosted an event on the regulation of regenerative medicine [external link].

As set out in the Government Response to the House of Lords Inquiry [external link] a Regenerative Medicine Expert Group (RMEG) is being established to develop an NHS regenerative medicine delivery readiness strategy and action plan. This group will build on existing initiatives so that the NHS is fully prepared to deliver these innovative treatments. The group will be supported by the Department of Health; members will be drawn from a number of groups and organisations, including the HRA. The remit of the Regenerative Medicine Expert Group will include a role to monitor the effect of regulation on the development of regenerative medicines in the UK.

More generally, the HRA is working in partnership with a range of organisations to improve the environment for research in the UK. Please refer to our projects and plans pages for more information.

During 2012-13, the House of Lords Science and Technology Committee held an inquiry into regenerative medicine in the UK. For more information about the inquiry, the resulting report and the HRAs responses please use the links below:

UK Stem Cell Toolkit [external link]This toolkit is intended to be a reference tool for those who wish to develop a programme of human stem cell research and manufacture, including clinical applications. It applies only to the regulation of human stem cells and their use in the laboratory and clinical settings. The toolkit provides regulatory routemaps that are specific to individual stem cell projects. It does this by using your responses to questions when you start using the toolkit.

Clinical Trials Toolkit [external link]This toolkit provides practical advice to researchers in designing and conducting publicly funded clinical trials in the UK. It provides information on best practice and outlines the current legal and practical requirements for conducting clinical trials. The toolkit is primarily focused on Clinical Trials of Investigational Medicinal Products (CTIMPs) and the regulatory environment and requirements associated with these. However researchers and R&D staff working on trials in other areas will also find useful information and guidance of relevance to the wider trials environment.

Cell Therapy Catapult [external link]The Cell Therapy Catapult was established in 2012 to grow the UK cell therapy industry. It was set up to help businesses take innovative ideas through to commercialisation. The website has specific regulatory resource pages, which include an overview of the relevant regulations for cell therapy.

MHRA Innovation Office [external link]The MHRA Innovation Office helps organisations that are developing innovative medicines, medical devices or using novel manufacturing processes to navigate the regulatory processes in order to be able to progress their products or technologies. Examples of innovative products include Advanced Therapy Medicinal Products (ATMPs), nanotechnology, stratified medicines, novel drug/device combinations, and advanced manufacturing.

UK Regenerative Medicine Platform (UKRMP) [external link]The Medical Research Council (MRC), Biotechnology and Biological Sciences Research Council (BBSRC) and the Engineering and Physical Science Research Council (EPSRC) have established the UKRMP to address the challenges associated with translating scientific discoveries towards clinical impact.

UK Stem Cell Bank [external link]The UK Stem Cell Bank was established to provide a repository of human embryonic, foetal and adult stem cell lines as part of the UK governance for the use of human embryos for research. Its role is to provide quality controlled stocks of these cells that researchers worldwide can rely on to facilitate high quality and standardised research. It also prepares stocks of EUTCD-Grade cell lines for use as starting materials for the development of cellular therapies. The UK Stem Cell Bank is hosted by NIBSC [external link], which is part of the MHRA.

UK Trade & Investment (UKTI) Life Science Investment Organisation (LSIO) [external link]This dedicated unit within UKTI is intended to support overseas companies to invest and expand in the UK from the earliest research and development collaborations through to clinical trials, commercial operations and partnerships.

Knowledge Transfer Network (KTN) Regenerative Medicine Priority Area [external link]This is an official group within the Healthtechnologies and Medicine Knowledge Transfer Network (KTN). Knowledge Transfer Networks have been set up by the Technology Strategy Board (TSB) to facilitate collaboration and stimulate innovation by bringing together people from a range of organisations with a variety of expertise.

DEFRA Advisory Committee on Releases to the Environment (ACRE) [external link]

HSE Scientific Advisory Committee on Genetically Modified Organisms (Contained Use) (SACGM (CU) [external link].

MHRAs Clinical Trials, Biologicals and Vaccines Expert Advisory Group[external link]

Regenerative Medicine Expert Group [external link]

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Regenerative Medicine - Health Research Authority