April 25, 2013 6:53pm

Regenerative Medicine

Regenerative medicine  repairs, replaces or creates living tissue or organ function damaged by the effects of age, disease, damage, or congenital defects. Its promise is to overcome limitations of current medications and treatments with the ability to heal previously irreparable organs or to grow new ones when the body cannot heal itself.  

Regenerative medicine is an emerging field of therapeutic product development that may allow all human cell and tissue types to be manufactured or made available from a patient's own natural stores of cells on an industrial scale.  This new technology is made possible by the isolation of human cells from a variety of sources in the human body using  “reprogramming” into cells to enhance their healing properties.  Unlike pharmaceuticals that require a molecular target, therapeutic strategies in regenerative medicine are generally aimed at regenerating affected cells and tissues, and therefore may have broader applicability. 

Regenerative medicine represents a revolution in the field of biotechnology with the promise of providing therapies for diseases previously considered incurable. The current wave of regenerative medicine includes cell-based therapies, fully formed artificial tissue and stem cell therapies being introduced to the body in order to repair or heal ailments. Novel cell-based therapies promise to target challenging chronic conditions by arresting degeneration or restoring functionality. Regenerative medicine also has the potential to solve the problem of the shortage of organs available for donation compared to the number of patients that require life-saving organ transplantation.  

The regenerative medicine market will grow exponentially from its current base of $1.8B to potentially more than $10B by 2020. The regenerative medicine field reverberates with potential implications for the $750B pharmaceutical and $200B medical device markets creating significant growth opportunities.  Commercial efforts in regenerative medicine go beyond cells as therapies or manufacture of replacement organs. It also includes the development and sale of products designed for research applications in the near term as well as products that may be used for diagnostic purposes, or to improve upon the design and discovery of traditional pharmaceutical pills.  

Research products can be marketed without regulatory or other governmental approval, and thus offer relatively near-term business opportunities, especially when compared to therapeutic products. 

 

Stem Cells

Stem cells have the remarkable potential to develop into many different cell types in the body during early life and growth. In addition, in many tissues they serve as a sort of internal repair system, dividing essentially without limit to replenish other cells as long as the person or animal is still alive.

When a stem cell divides, each new cell has the potential either to remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

Stem cells can now be grown and transformed into specialized cells with characteristics consistent with cells of various tissues such as muscles or nerves through cell culture. Highly plastic adult stem cells from a variety of sources, including umbilical cord blood and bone marrow, are routinely used in medical therapies.

Embryonic cell lines and autologous embryonic stem cells generated through therapeutic cloning have also been proposed as promising candidates for future therapies. Their ability to grow into virtually any of the body's specialized cells is giving drug developers new ways to test drugs in the lab. In many ways, this may be viewed as an extension of personalized medicine, although we believe we are the first to do so.

Research on stem cells continues to advance knowledge about how an organism develops from a single cell and how healthy cells replace damaged cells in adult organisms.

Stem cell research is one of the most fascinating areas of contemporary biology, but, as with many expanding fields of scientific inquiry, research on stem cells raises scientific questions as rapidly as it generates new discoveries.

Autologous stem cell transplantation is distinguished from allogenic stem cell transplantation where the donor and the recipient of the stem cells are different people. Autologous stem cell (undifferentiated cells from which other cell types develop) are removed from an individuals from your own blood or bone marrow, stored, and later given back to the same person. In allogeneic stem cell therapy the donor is a different person to the recipient of the cells.  In pharmaceutical manufacturing, the allogenic methodology is promising because unmatched allogenic therapies can form the basis of "off the shelf" products;

Human embryonic stem cell research is controversial, and regulation varies from country to country, with some countries banning it outright. Nevertheless, these cells are being investigated as the basis for a number of therapeutic applications, including possible treatments for diabetes and Parkinson's disease.

Many cells types have the capacity to release soluble factors such as cytokines, chemokines, and growth factors which act in a paracrine or endocrine manner. These factors facilitate self-healing of the organ or region. This includes cells that naturally secrete the relevant therapeutic factors, or which undergo epigenetic changes or genetic engineering that causes the cells to release large quantities of a specific molecule.  

Some types of stem cells include:

  • Neural stem cells (NSCs) are the subject of ongoing research for possible therapeutic applications, for example for treating a number of neurological disorders such as Parkinson's disease and Huntington's disease;
  • Mesenchymal Stem Cell Therapy or MSCs are multipotent and fast proliferating and these unique capabilities mean they can be used for a wide range of treatments;
  • Hematopoietic stem cell or HSCs possess the ability to self-renew and differentiate into all types of blood cells, especially those involved in the human immune system. HSCs therapy can also render its cure by reconstituting damaged blood-forming cells and restoring the immune system after high-dose chemotherapy to eliminate disease.

 

Gene Therapy

Gene therapy is an evolving field of medicine in which faulty genes are corrected in cells. Genes control heredity and provide the basic biological code for determining a cell's specific functions.

The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a defective gene. In gene therapy, the healthy copy of a defective gene is packaged within a vector, a biological delivery mechanism which is used to transport the genetic information into the diseased cells within the body. Once the gene is delivered into the correct cell, a therapeutic protein is naturally made by the cell from the therapeutic gene.

Drugs work by binding with proteins that are the underlying cause of a specific disease. But a number of therapies in development seek to work by acting directly on genes that are responsible for producing the deleterious protein in the first place. There are several technologies for accomplishing this, whether it is the introduction of a gene through a viral vector, RNAi, antisense or the use of so-called zinc finger proteins.

 

Gene therapy has the potential to cure disease, not just treat it chronically. While expectations are that the approach will carry a high price tag, in the realm of $1 million or more, insurers will be likely to pony up if the theory that it saves money in the long run bears out.

 

Immunotherapy

Immunotherapy is treatment that uses certain parts of a person’s immune system to fight diseases such as cancer, auto-immune disorder or viral disease.

Immunotherapy is a type of treatment designed to boost the body's natural defenses. It uses materials either made by the body or in a laboratory to improve, target, or restore immune system function. It is not entirely clear how immunotherapy treats cancer. However, it may work in the following ways:

  • Stopping or slowing the growth of cancer cells;
  • Stopping cancer from spreading to other parts of the body by training the immune system to attack cancer cells specifically;
  • Helping the immune system work better at destroying cancer cells, virus or other foreign bodies

In the last few decades immunotherapy has become an important part of treating some types of cancer. Newer types of immune treatments are now being studied, and they’ll impact how we treat cancer in the future. This field has been labeled immune-oncology.

Cell-based immunotherapies are proven to be effective for some cancers. Immune effector cells such as lymphocytes, macrophages, dendritic cells, natural killer cells (NK Cell), cytotoxic T lymphocytes (CTL), etc., work together to defend the body against cancer by targeting abnormal antigens expressed on the surface of the tumor due to mutation.

 

Immunomodulation

Immunomodulation involves the alteration of immune responses known to alter cellular or humoral immunity used to adjust immune responses to a desired level.

The active agents of immunotherapy are collectively called immunomodulators. They are a diverse array of recombinant, synthetic and natural preparations, often cytokines. Some of these substances, such as granulocyte colony-stimulating factor (G-CSF), interferons, imiquimod and cellular membrane fractions from bacteria are already licensed for use in patients. Others including IL-2, IL-7, IL-12, various chemokines, synthetic cytosine phosphate-guanosine (CpG) oligo-deoxynucleotides and glucans are currently being investigated extensively in clinical and preclinical studies.

Immunomodulatory regimens offer an attractive approach as they often have fewer side effects than existing drugs, including less potential for creating resistance in microbial diseases.

 

Monoclonal Antibodies

Have already become an important tool in biochemistry, molecular biology, drug discovery and medicine; the idea of a magic bullet was first postulated that if a compound could be made that selectively targeted a disease-causing organism, then a toxin for that organism could be delivered along with the agent of selectivity. It is now possible to create monoclonal antibodies that specifically bind to that substance; they can then serve to detect or purify that substance. The second generation of MAB technology will be evolutionary but productive and profitable for those with the wherewithal and IP to take advantage of it.

 

Functional Genomics

Functional genomics advances will eventually impact healthcare to an unprecedented extent, not only by helping to enable the widespread practice of personalized medicine, but also by leading the way toward the treatment of learning and memory disorders, the cure of hereditary diseases, and the retardation of aging. This example of cutting-edge technology will both increase and decrease risk. The increase is obvious. A gigantic opportunity, such as the retardation of aging, will entail many years of research, costly false leads, and huge outlays of capital. The decrease in risk is due to the coming of age of personalized medicine. Customized therapeutics are likely to provide greater efficacy with fewer side effects if targeted to population subsets. Clinical trials should require lower enrollments and shorter periods of time to complete and result in reduced failure rates and fewer product recalls following FDA approval. The costs of R&D should drop considerably and lead to high rates of return from such niche market-directed therapeutics.