The Promise of Stem Cell Research: Recent Advancements and Setbacks

Few technologies spark as much fascination, hype, and controversy as stem cell technology. One of the most interesting medical applications of stem cell research is in regenerative medicine, where stem cells are being developed to regenerate tissue and repair failing organs. Perhaps the most befitting symbol for this emerging technology is the Greek god, Prometheus, whose immortal liver was feasted on day after day by Zeus’ eagle and day after day was able to regenerate. The leading minds in science and medicine today hope to make this legendary concept of regeneration into reality, but hurdles abound.

The excitement about stem cell research and its potentially transformative therapeutic applications is evidenced by the large investments into research that have been made by companies, governments, and universities around the world. Significant unmet needs associated with chronic diseases have catalyzed this investment. In contrast to the symptomatic-focused treatment offered by conventional therapeutics, stem cell therapies offer potentially curative treatments for many diseases that arise as a result of damage to terminally differentiated cells. High market potential for both embryonic and adult stem cell therapies has resulted in strategic partnerships between large pharmaceutical companies and stem cell research-based companies, such as the agreement between Athersys and Pfizer to develop and market MultiStem for the treatment of Inflammatory Bowel Disease. Pharmaceutical companies are also interested in exploring the various methods in which stem cells could be utilized in the drug discovery process to accelerate the discovery of novel and safe drugs. Illustrative of this interest, GlaxoSmithKline, AstraZeneca, and Roche teamed together to form a consortium with the UK government to develop stem cells for safety testing of new drugs.

Reflecting on a year of highs and lows in 2010, we at Scientia Advisors believe that while significant scientific and clinical advances propelled the field forward, key challenges still remain firmly in place. In October 2010, 12 years after human embryonic stem cells were first isolated, a therapy derived from such cells was tested in humans for the first time by Geron. The company’s trial is designed to treat spinal-cord injury with an injection of differentiated neural cells into the injury site and is the first clinical trial of its kind. A second company, Advanced Cell Technology, won FDA approval in November 2010 to begin human tests of its embryonic-stem-cell-derived therapy for an inherited form of blindness. While these two human trials represent major steps forward in stem-cell research, federal judge Royce Lamberth stunned the stem-cell community by issuing an injunction blocking federal funding for any research involving embryonic stem cells in August 2010. Embryonic stem cell researchers in the field say that Lamberth’s decision will have a deleterious effect, stunting promising medical research.

Amidst ongoing efforts to promote scientific discovery and avoid the ethical controversies that have consistently gripped the field, advancements in the development of induced pluripotent stem cells (iPS), which can be derived from adult tissue without the use of embryos, have been promising. In 2010, Derrick Rossi and his team at Harvard developed a new method for making iPS cells using RNA instead of DNA to produce the four proteins needed to reprogram the cell, effectively decreasing the risks of triggering tumor growth when creating these cells. ModeRNA Therapeutics, a new startup in Cambridge, MA, is focusing on this promising research.

Given the tremendous promise of stem cell technology, with the possibility of products that could revolutionize the treatment of diseases and conditions, the potential for profit is staggering. Tempering this exuberance, however, are the myriad ethical, legal, political, and financial challenges that face this field of research. The eventual resolution of these conflicts will determine the success of the research and potentially the face of medicine in the future. We encourage players in the stem cell research space to both examine their current approaches for participating in this emerging field and understand the strategic opportunities that exist. Such opportunities include potentially lucrative partnerships with global healthcare companies and highly specialized startup companies, optimization of drug development and toxicity screening strategies, and the creation of innovative corporate strategies and profitable business models that could transform the treatment paradigm.

Joe McWilliams is a senior analyst at Scientia Advisors, a Cambridge, Massachusetts-based healthcare and life sciences consultancy. This post first appeared on the Scientia advisors blog.