Applications from regenerative medicine to gene therapy as well as antiviral therapeutic interventions emphasize self-healing possibilities.
Therapeutic Interventions Emphasize Self-Healing
Therapeutic interventions of various kinds try to improve the body’s capacity to defend, repair, and even cure itself. Interventions that attempt to enhance self-healing span cell-based therapy, gene therapy, small molecule drugs, biologics, and tissue engineering.
Advances in each of these areas are being followed by Allied Market Research. Which has concluded that stem cell technologies look especially promising. For example, stem cell technologies are set to revolutionize the human ability to produce neural cells in abundance.
Stem cells may overcome a limitation of human tissues that is as sobering as it is familiar. The retina and the central nervous system have extremely poor regenerative capabilities. This limitation seems all the more dire when one contemplates the prevalence of Leber congenital amaurosis, age-related macular degeneration. As well as other diseases that lead to visual impairment.
Interest in Stem Cell Therapy Is Rising
If anything, enthusiasm for stem cell technology can be too great. For example, earlier in the year, the U.S. Food & Drug Administration decided to take a closer look at clinics that provide stem cell treatments. The FDA hopes closer regulation may not only curb unproven therapies, but also enhance confidence in truly effective interventions.
Researchers are excited about a new stem cell approach to repairing heart damage. This approach, developed by Amit Patel, M.D., director, Clinical Regenerative Medicine, University of Utah, involves the selective expansion of bone marrow cells. In the IxCELL-DCM trial (to assess the efficacy of the Ixomyocel T cell therapy), which included Utah Senator Ralph Okerlund, researchers observed a 37% reduction in the number of hospitalizations or deaths related to heart disease and heart failure.
Other Therapeutic Approaches
Another promising approach is the possibility of combining regenerative medicine and organ transplantation. This approach is being developed by Maria Cristina Piña-Barba, Ph.D., a researcher at the Institute of Material Research, National Autonomous University of Mexico. Her team is concentrating on blending different stem cell techniques to regenerate the damaged parts of various organs like kidneys and heart. The number of kidneys required each year in Mexico alone for transplant is approximately 12,431. While the demand for hearts is about 58, there is an increasing need for 7,396 corneas, 3 lungs, and 404 livers each year in the country.
One way to begin is by “decellularizing bovine bone,” said Dr. Piña-Barba. “We have also decellularized tracheas, kidneys, livers, and hearts.” When calcium is stripped from hard bone tissue, the result is a sponge of collagen. This sponge may then be seeded with stem cells.
“We give [the seeded sponge] the form of the organ needed, and in two months there is tissue formation,” continued Dr. Piña-Barba. “This process is a possible solution to tracheal disorders caused by … cancer, stenosis (abnormal narrowing) and traumas that can lead to respiratory diseases and even death.” These innovations will open new avenues for growth in regenerative medicine.
Gene Therapy Pipeline is Swelling Fast
Another fast-growing segment is gene therapy. Recent research, including a preclinical trial, demonstrates the importance of gene therapy for the sickle cell disease. In fact, gene therapy is inspiring so much confidence that gene therapy warrantees are being contemplated. In fact, GlaxoSmithKline decided to offer a money-back guarantee on a gene therapy called Strimvelis.
GSK announced that if Strimvelis failed to cure a disorder called bubble boy disease, patients would be refunded the total amount. Sarah Spencer, a spokesperson for the drug maker, was quoted by CNN to say that in pricing Strimvelis, GSK “reviewed how best to make the one-time treatment cost for the medicine affordable to health care systems.”
“Given the extremely low patient numbers for Strimvelis and therefore low budget impact,” Spencer continued, “many payers have indicated that Strimvelis is likely to be funded on a case-by-case basis and does not warrant a potentially complicated pricing arrangement.” Startups in countries like Israel are also seen investing on gene therapy to discover a cure for cancer.
Where Research is Heading
The competitive landscape for gene therapy also looks bright. The much-awaited acquisition of the year was Allergan’s investment in Retrosense Therapeutics, a gene therapy company. The investment has helped Allergan access an advanced technology and expand its eye care pipeline. While many companies focus on possibilities, Novartis has shuttered its gene therapy experiment that promised a cure for cancer.
By: Swapna Supekar
09/12/2016
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