What is the difference between cell therapy and regenerative medicine

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Roles and remits of the regulators in regenerative medicine Each regulator has a clear remit and regulates distinct areas of the regenerative medicine process. The role of each of the regulators in regenerative medicine is set out below: Health Research Authority HRA have a remit to provide an ethics opinion on clinical trials.

More information about research approvals Please refer to the Approvals and Amendments area on our website for information about the approvals for research studies, or alternatively please visit the guidance on IRAS to find out more information about how to apply to individual review bodies.

Working in partnership We work closely with others and will continue to engage with those involved in regenerative medicine, including researchers, the British Society for Gene and Stem Cell Therapy , and the Cell Therapy Catapult to help clarify the regulatory requirements that apply.

Useful links Toolkits Clinical Trials Toolkit - this toolkit provides practical advice to researchers in designing and conducting publicly funded clinical trials in the UK.

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 — 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. The objective of cell therapy is to restore the lost function rather than produce a new organ, which could cause duplicity and undesirable effects.

Several resources of cells can be used to restore the damaged tissue, such as resident stem cells, multipotent adult progenitor cells or embryonic stem cells. Some cell therapies have been established and approved for clinical use, such as artificial skin derived from keratinocytes, derived from chondrocyte, cells of the corneal limbus or pancreatic islet transplantation. As ESCs are pluripotent they retain the ability to self-renew and to form any cell in the body.

ESCs have the advantage of versatility due to their pluripotency, but the use of embryos in the development of therapeutic strategies raises some ethical concerns.

Induced pluripotent stem cells iPSCs. A differentiated adult somatic cell, such as a skin cell is reprogrammed to return to a pluripotent state. These cells offer the advantage of pluripotency but without the ethical concerns of embryonic stem cells. While the efficiency of the process has been greatly improved since inception, the relatively low rate of reprogramming remains a concern. Nuclear transfer embryonic stem cells ntESCs.

These pluripotent cells are produced by transferring the nucleus from an adult cell obtained from the patient to an oocyte egg cell obtained from a donor. The process of transferring the nucleus reprograms the egg cell to pluripotency.

As with iPSCs, the derived cells match the nuclear genome of the patient and are unlikely to be rejected by the body. However, the major advantage of this technique is that the resulting ntESCs carry the nuclear DNA of the patient alongside mitochondria from the donor, making this technique particularly appropriate for diseases where the mitochondria are damaged or dysfunctional. A drawback of ntESCs is that the process of generation is cumbersome and requires a donor oocyte.

At the time of writing stem cell production using this technique has only been shown in lower mammals. Parthenogenetic embryonic stem cells pES. The final option for obtaining pluripotent cells is from unfertilized oocytes. Here the oocyte is treated with chemicals that induce embryo generation without the addition of sperm parthenogenesis and ESCs are harvested from the developing embryo. This technique generates ESCs that are genetically identical to the female patient.

However, this method is in the early stages of development and it is not known if cells and tissues derived from parthenogenesis develop normally. Hematopoietic stem cells HSCs are multipotent blood stem cells that give rise to all types of blood cells.

HSCs can be found in adult bone marrow, peripheral blood, and umbilical cord blood. Mesenchymal stem cells MSCs are multipotent cells present in multiple tissues including umbilical cord, bone marrow, and fat tissue. MSCs give rise to bone, cartilage, muscle, and adipocytes fat cells which promotes marrow adipose tissue. Neural stem cells NSCs. Adult neural stem cells are present in small number in defined regions of the mammalian brain.

These multipotent cells replenish neurons and supporting cells of the brain. However, adult neural stem cells cannot be obtained from patients due to their location in the brain. Epithelial stem cells. Epithelial cells are those that form the surfaces and linings of the body including the epidermis and the lining of the gastro-intestinal tract. Multipotent epithelial stem cells are found in these areas along with unipolar stem cells that only differentiate into one type of cell.

Epithelial stem cells have been successfully used to regenerate the corneal epithelium of the eye. Immune cell therapy. Cells that rapidly reproduce in the body such as immune cells, blood cells or skin cells can usually do so ex vivo given the right conditions. This allows differentiated, adult immune cells to be used for cell therapy.

The cells can be removed from the body, isolated from a mixed cell population, modified and then expanded before return to the body. A recently developed cell therapy involves the transfer of adult self-renewing T lymphocytes which are genetically modified to increase their immune potency to kill disease-causing cells.

Risks of any medical treatment depend on the exact composition of the therapeutic agent and its route of administration. Different types of administration, whether intravenous, intradermal or surgical, have inherent risks. Risks include the outcome that gene therapy or cell therapy will not be as effective as expected, possibly prolonging or worsening symptoms, or complicating the condition with adverse effects of the therapy.

Their administration may induce a strong immune response to the protein in the case of replacing proteins from genetic diseases. This immune response may become uncontrolled and lead to normal proteins or cells being attacked, as in autoimmune diseases.

High doses of some viruses can be toxic to some individuals or specific tissues, especially if the individuals are immune compromised. Gene therapy evaluation is generally carried out after birth.

There is little data on what effects this therapeutic approach might have on embryos, and so pregnant women are usually excluded from clinical trials. Risks of cell therapy also include the loss of tight control over cell division in the stem cells. Theoretically, the transplanted stem cells may gain a growth advantage and progress to a type of cancer or teratomas.

Since each therapy has potential risks, patients are strongly encouraged to ask questions of their investigators and clinicians until they fully understand the risks. Viral vectors and oncolytic viruses are designed to reduce the risk of adverse effects, and each viral vector is rigorously tested in cells and animals before it is considered for human use. The viral vectors used in human trials are prepared under strict guidelines to ensure purity and integrity.

However, every medicine has risks. Thus, it is essential that patients thoroughly discuss the potential risks of any new therapy with their physicians, patient advocate, family, and investigators of a clinical trial. Both approaches have the potential to alleviate the underlying cause of genetic diseases and acquired diseases by replacing the missing protein s or cells causing the disease symptoms, suppressing expression of proteins which are toxic to cells, or eliminating cancerous cells.

Gene therapy involves the transfer of genetic material into the appropriate cells. In genetic diseases, the stem cells of the afflicted tissue are often targeted. The adult stem cells of the tissue can replenish the specialized cells. Expressing the appropriate gene in the stem cells ensures that the subsequent specialized cells will contain the therapeutic protein. Introduction of genes into cells can be carried out in culture with subsequent administration to the patient, or by direct injection of vectors into the body.

Cell therapy is the transfer of cells to a patient. For treatment of most diseases by cell therapy, stem cells are chosen because their establishment in the patient leads to continual production of the appropriate specialized cells. Stem cells from the patient are altered by gene therapy in culture to express the relevant functional protein. The improved stem cells are administered or returned to the patient. Scientists and clinicians use the following four methods to carry genetic material into the targeted cells.

Non-vector methods such as electroporation, passive delivery, and ballistic delivery. This is a common technique in the lab. Naked DNA or RNA may also be taken up by target cells using a normal cellular process called endocytosis after addition to the medium surrounding the cells. Membrane-bound vesicles. Different types of liposomes are being developed to preferentially bind to specific tissues. Viral vectors. Viruses have an innate ability to invade cells.

Viral vectors for gene therapy are modified to utilize the ability of viruses to enter cells after disabling the capability of the virus to divide. Different types of viruses have been engineered to function as gene therapy vectors. For oncolytic viruses, such as adenovirus and herpes simplex virus, fewer viral genes are replaced and the virus is still able to replicate in a restricted number of cell types.

Different types of viral vector preferentially enter a subset of different tissues, express genes at different levels, and interact with the immune system differently. Gene therapy can be combined with cell therapy protocols. Cells are collected from the patient or matched donor and then purified and expanded in vitro. Scientists and clinicians then deliver the gene to the cells using one of the three methods described above.

Those cells that express the therapeutic gene are then re-administered to the patient. Viruses as gene delivery vectors. Modified viruses are used as carriers in gene therapy. These viral vectors protect the new gene from enzymes in the blood that can degrade it, and deliver it to the relevant cells. Viral vectors efficiently coerce the cells to take up the new gene, uncoat the gene from the virus particle, and transport it, usually to the cell nucleus.

The transduced cells begin using the new gene to perform its function, such as synthesis of a new protein. Viral vectors are genetically engineered so that most of their essential genes are missing, which prevents uncontrolled replication of the virus and makes room for insertion of the gene to be delivered.

Many different viral vectors are being developed because the requirements of gene therapy agents for specific diseases vary depending on the affected tissue, the level of gene expression, and the required duration of expression. Oncolytic Viruses. Oncolytic viruses are engineered to replicate only or predominantly in cancer cells and not in normal human cells. Once oncolytic viruses replicate in cancer cells they cause the cancer cells to burst, releasing more oncolytic viruses to infect surrounding cancer cells.

Put simply, gene therapy works by changing the genetic information of a population of cells in a way that alleviates or combats the cause or symptoms of a disease. The challenges of gene and cell therapists can be divided into three broad categories based on disease, development of therapy, and funding.

Note that there are many susceptible genes and additional mutations yet to be discovered. Gene replacement therapy for single gene defects is the most conceptually straightforward.