Column: Reviving optimism for regenerative medicine

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A scientific researcher extracts the RNA from embryonic stem cells in a laboratory. Photo by Getty Images

A scientific researcher extracts the RNA from embryonic stem cells in a laboratory. Photo by Getty Images

Grim stories about the abuse of regenerative medicine and stem cell therapies have been in the headlines. Hundreds of international stem cell clinics now hawk unproven, unregulated therapies to desperate people. Such stem cell tourism often does more harm than good. But while these stories — and there are many — deserve our attention and regulatory oversight, we shouldn’t let them detract from the positive forward motion of regenerative medicine and the very real potential for individual patients and the national economy.

With the public storyline dominated by unproven stem cell therapy disasters, the FDA has moved to curb these unproven therapies and recently wrapped up hearings on increased regulation to protect patients from the latest generation of snake oil purveyors. The life science community should embrace the discrediting of unproven therapies promoted without data for economic gain, and instead focus on the promise of research held to the highest standards.

The next wave of regenerative medicine research is tackling head-on enormous health care issues: AIDS, Alzheimer’s disease, diabetes, heart disease, blood cancers, blindness — the list goes on. Despite the complexity of the investigative issues, regenerative medicine, which encompasses stem cell research, tissue engineering, and gene therapy, has the potential to positively affect many clinical areas.

Academic institutions and responsible companies are working hard to unlock the potentially transformative impact of stem cells. In the process, they could validate and secure the next branch of the biomedical industry.

Many academic institutions, private organizations, and state governments have dedicated resources to the development of responsible regenerative medicine. Along with accountable, funded companies, they are the responsible stewards of regenerative medicine. Their successes as they find new ways to tackle the immense, costly diseases that stifle our economy and affect our communities personally, socially, and fiscally will mean a more sustainable health care system.

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In the past few months alone, academia has uncovered a host of potential new applications for regenerative medicine with the potential for widespread economic and clinical impact. A few worth noting:

Accelerated drug discovery can lead to faster drug development timelines and increased patient and societal benefit. Last month, researchers announced a muscle-on-a-chip platform, built using stem cells from patients with Duchenne muscular dystrophy. This is a boon to researchers struggling to understand how this disabling disease works and to pinpoint treatments. This is a potential model for diseases where the lack of a large patient base and the challenges of animal models create limitations in bench and clinical research.

New focus on economically crippling diseases can take aim at conditions that weigh down our economic growth and depress populations. In October, researchers announced they are closer to a “truly effective treatment” for type 1 diabetes using embryonic stem cells as a starting point to produce the massive quantities of cells needed for cell transplantation. Rockefeller University scientists hailed this as “one of the most important advances to date in the stem cell field.” Though it will take plenty of work to translate this discovery into clinical efficacy, after many years of efforts in this area we are now seeing the fruit of extensive scientific labors.

Lower costs to treat disease may be achieved through precision diagnostics and better understanding of disease progression. Last month, researchers at the Stanford University School of Medicine and the University of Tokyo announced research showing that withholding the amino acid valine depleted blood stem cells in mice, making it possible to perform a blood stem cell transplantation. In the lab, human cells also responded to valine restriction, suggesting the potential for this approach in humans to circumvent the need for radiation and chemotherapy in advance of bone marrow transplantation. This is just a single promising example of stem cell-related research which could have an impact on cost, safety, and efficacy compared to our current standards of care.

Every day, positive advances are quietly being put forth by academia and industry: Stanford researchers repaired the gene that causes sickle cell disease; researchers at Brigham and Women’s Hospital in Boston have identified a human lung stem cell that is self-renewing; an international team of top-notch researchers identified a new mechanism to sharpen memory during aging.

The list of successes should, I hope, eclipse the painful stories of rogue cell stem clinics taking advantage of vulnerable patients. We must prevent charlatans from clouding the picture and undermining the future of biomedical research just as we must prevent false therapies from entering the marketplace.

Medical and scientific advances often take decades from initial concept to proof of clinical efficacy. Success requires perseverance, funding, and continual insight and hard work. We have been hearing about the potential of cell-based therapeutics and regenerative medicine for many years. Now, recent advances and the collective efforts being made that parallel the remarkable scientific progress in many other fields give us reason to hope and believe that the years of research are closing in on a meaningful clinical and societal contribution.

Jonathan Gertler, MD, served for many years as an academic vascular surgeon and investigator. He is the managing partner and CEO of Back Bay Life Science Advisors in Boston and is a frequent speaker and writer on strategic issues facing life science companies at all stages. This article is reproduced with permission from STAT. It was first published on Nov. 18, 2016. Find the original story here.

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