My Regeneration

What can today’s generation hope for from regenerative medicine?

It’s a frontier for business, the medical profession and society. Regenerative medicine aims to shift the focus from palliative care to the development of treatments that address diabetes, Parkinson’s disease, stroke and heart disease. The hope is that we’ll live longer, healthier lives and have greater independence in old age, thereby reducing the burden on healthcare systems. Given global population demographics, this is essential: according to the World Health Organisation by 2050 there will two billion people aged 60 years and over.

Regenerative medicine already has a rich history of achievement, as illustrated in the timeline. Today, researchers and doctors are making inroads via the use of cell therapies, tissue scaffolds and growth factors; and new technologies, such as 4D bioprinting, all promising to dramatically increase the speed of progress. A number of companies present at BIO 2014, including AntiCancer Inc., RegenLab, Sanford Health, Merck, Lilly and Janssen Pharmaceutical Companies of Johnson & Johnson are pioneering regenerative medicine projects.

To get a better grasp of the achievements, challenges and potential of regenerative medicine, we talked to four of the world’s leading experts in the field. Here’s what they told us.

de GreyAubrey de Grey is the biomedical gerontologist who founded SENS (Strategies for Engineered Negligible Senescence) Research Foundation. He received his BA in Computer Science and Ph.D. in Biology from the University of Cambridge in 1985 and 2000, respectively. Dr de Grey is Editor-in-Chief of Rejuvenation Research, is a Fellow of both the Gerontological Society of America and the American Aging Association, and sits on the editorial and scientific advisory boards of numerous journals and organizations.

What do you think are the greatest achievements in regenerative medicine so far?

I think it would be difficult to argue against two breakthroughs published in 2006 and 2008 respectively: induced pluripotent stem cells (Shinya Yamanaka) and decellularisation of organs (Doris Taylor). Both these techniques proved to be quite easy to reproduce in other laboratories, so their potential is moving towards the clinic far more quickly than usual.

What are the biggest challenges in this research and development space?
It remains highly challenging to generate sufficiently pure stem cells of a desired type to ensure safety and efficacy. Also, while the decellularisation approach is a great breakthrough, we still need to get better at reseeding a scaffold with desired cells. As a solution to the vascularisation issue that has stalled solid-organ tissue engineering for so long, a promising alternative to decellularisation is organ printing, but it still faces daunting obstacles in terms of creating sufficiently tough blood vessels.

Where do you think the field will be in 10 years?
I think regenerative medicine will be at the heart of medicine for the elderly by 2024, having reached a level of sophistication that allows the genuine cure of relatively simple age-related conditions such as Parkinson’s Disease and the alleviation of a wide range of aspects of age-related ill-health.

 

LayrollePierre Layrolle is coordinator of the European regenerative medicine project Reborne (Regenerating Bone Defects using New biomedical Engineering approaches – www.reborne.org). Bone is the most frequently transplanted tissue with about 1 million procedures annually in Europe. The worldwide market of bone replacement material is currently estimated at $6.8 billion with 10 percent annual growth.

What do you think are the greatest achievements in regenerative medicine so far?
The production, characterization, storage and transportation of cells for therapeutic uses now meet pharmaceutical standards.
There are many advanced therapy medicinal products in clinical development. As participants in Reborne, we are performing 4 clinical trials for bone regeneration using autologous stem cells and advanced biomaterials in orthopedic and maxillo-facial surgeries.

What are the biggest challenges in this research and development space?
Regenerative medicine is very costly; roughly $27,472 for each patient. Allogeneic stem cells, automated bioreactors and standard culture media may decrease the costs of this personalized medicine.

Where do you think the field will be in 10 years?
If Phase 3 comparative clinical trials are successful, regenerative medicine could become a standard of care. In Europe, ATMPs (Advanced Therapy Medicinal Products) should be produced by public blood transfusion units because they already have experience in handling blood derivatives and cell therapy products, not by private companies. In situ reprogramming of cells and regeneration of tissues may take longer time as safety is a concern.

 

SerranoManuel Serrano obtained his PhD in 1991 for his research at the Centro de Biología Molecular “Severo Ochoa” under the supervision of M. Salas and J.M. Hermoso. From 1992 to 1996 he worked as a Postdoctoral Fellow in the laboratory of D. Beach at the Cold Spring Harbor Laboratory, New York, USA. In 1997, he returned to Spain to start his own research group at the Centro Nacional de Biotecnología. He moved to CNIO in 2003 to lead the Tumour Suppression Group.

What do you think are the greatest achievements in regenerative medicine so far?
In my opinion, cell reprogramming is the single most important achievement. Regenerative medicine is based on the curative potential of cells, and induced pluripotent stem cells are the only realistic source to obtain large amounts of functional, normal, patient-matched cells. This does not solve all the technical problems of regenerative medicine, but it certainly solves one of them.

What are the biggest challenges in this research and development space?
A huge challenge is to obtain functional engraftment and functional regeneration. Tissues are formed by many cell types organized in a very specific and sophisticated manner. At the moment it is unclear whether the simple injection of a single cell type will be effective in recovering the function of a diseased tissue. We have solved the problem of generating patient-matched cells. The next challenge is how to put these cells back into the patient in a manner therapeutically valuable.

Where do you think the field will be in 10 years?
Honestly, I have no idea. Ten years ago, the idea of obtaining embryonic stem-like cells from a patient was totally unthinkable. In 10 years from now I expect many surprises. Two areas that can make big advances are: tissue engineering, and in situ regeneration.

 

AtalaAnthony Atala, MD, is the Director of the Wake Forest Institute for Regenerative Medicine, and the W.H. Boyce Professor and Chair of the Department of Urology at Wake Forest University. Dr. Atala is a practicing surgeon and a researcher in the area of regenerative medicine. His current work focuses on growing new human cells, tissues and organs.

What do you think are the greatest achievements in regenerative medicine so far?
The fact that regenerative medicine therapies have been successfully applied in patients with a variety of medical conditions is significant and indicates the field’s potential.

What are the biggest challenges in this research and development space?
These range from the scientific hurdles inherent in engineering complex organs such as kidneys and livers to regulatory, financial and scale-up challenges.

Where do you think the field will be in 10 years?
Within ten years, I believe an increased number of patients will have benefitted from regenerative medicine therapies and that the treatments will be applied to a broader range of conditions.

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