Note: I would like to acknowledge Barbara's hard work. She is constantly seeking out experts in the stem cell field to answer our many questions. Without her hard work, the monthly Ask The Doctor column would not be possible.

Q & A
Q: My oldest brother is 65. He is ravaged by 30+ years of diabetes, legally blind and on dialysis 4 times a week. Is stem cell therapy an option for him?
A: There are several kinds of “stem cell” strategies currently under evaluation for diabetes, depending if he is Type 2 Diabetes Mellitus (T2DM) or Type 1 (T1DM). For T2DM there are clinical trials in progress (Mesoblast). For type 1 it is more complicated because you can not just promote regeneration or differentiation of new insulin producing cells but you also have to address autoimmunity that would destroy any new insulin producing cell. Embryonic stem cell differentiation into insulin producing cells has progressed a lot and pilot clinical trials are planned, probably for 2015. This will require immune protection because they are both allogeneic and to protect from autoimmune recurrence. There are several the approaches under investigation for differentiation of autologous stem cells and progenitor cells into insulin producing cell, but none at the clinical level so far.

Q: You are the founder of islet transplantation for Type 1 diabetes. What is your interest in stem cells in general and how do you see stem cells to help diabetes - will it help islet transplantation work better so there is no rejection?
A: I am not the founder of islet transplantation but I have been working on it since medical school … We are interested in stem cells and regenerative medicine in general because so many approaches, from anti-inflammatory and immunomodulatory strategies, to tolerance induction and providing an unlimited source of insulin producing cells will rely on stem cells

Q: Can a person's diet play a role in how successful stem cell treatment is?
A: Diets play a critical role in everything and therefore I would assume also in the success of stem cell therapies. For example in T2DM if you restore the mass of insulin producing cells but you do not modify diet and lifestyle you will lose again your insulin producing cells for exhaustion following, insulin resistance and hyperglycemia. In T1DM an anti-inflammatory diet could help to avoid triggering autoimmune response or immune rejection, but there are not a lot of studies in this direction.

Q: Do you have any knowledge about nerve regeneration with adult stem cells mixed in biomatrix gel transplanted through nerve conduits? When will something like this be in clinical trials?
A: I don’t know, but I would not be surprised if MSC could promote nerve regeneration I know there are trials in Spinal Cord Injury and many more pre-clinical model systems ben explored.

Q: Patients have been working hard to enter the debate on adult stem cell therapy regulation since our lives hang in the balance. Thank you for including our articles in the latest issue of CellR4. Any suggestions on how we can focus our efforts for greatest effect?
A: We all have to better communicate and leverage to achieve our common goal to develop cures in the fastest, most efficient and safest way possible. The Cure Alliance has a proposal and some objectives in this direction. Please see The Cure Alliance page and group on Facebook or www.TheCureAlliance.org). We have a proposal to create Fast Track Center for Testing innovative clinical trials and novel strategies. Arnold Caplan and Mike West have another white paper in this direction. Awareness of the public on the current barriers and impediments to the development of cures should be something we all work together. CellR4 (http://www.CellR4.org) is a start to provide a forum and a discussion opportunity on all of these topics, but we should do more, and when we raise enough consensus and awareness we should begin discussions with regulatory and government leaders. For now we do not have enough visibility but when we’ll have a couple of million of supporters, politicians will listen more carefully. As Warren Buffett said, when the majority of the people agree on a path then laws can change very quickly, in less than one year, even in the US

Q: I am a chronic inflammatory demylinating polyneuropathy with axonal degeneration patient. Would stem cells help improve my condition? What would be the best protocol?
A: I don’t know, unfortunately, but you could ask Dr. Dalton Dietrich, scientific director of the Miami Project to Cure Paralysis, or invite him to the next forum.

Q: University of Miami seems to be leading the country in clinical trials of autologous stem cell therapy for cardiac conditions and they have funding now to begin mesenchymal stem cell trials for diabetes. What do you think the future holds for this therapy at the University of Miami?
A: I know there are several trials in progress under the leadership of Dr. Joshua Hare for cardiac applications and the DRI is involved in MSC Trials for T2DM. Pilot trials in T1DM will become a reality probably in 2015. An a regenerative medicine institute will be soon announced ay UM to further promote and synergize all stem cell therapies and regenerative medicine clinical applications.

Q: If the FDA called you tomorrow and asked for your recommendations on regulating our own stem cells, what would you say?
A: I would tell them to please stay out of autologous stem cell therapies, unless the cells are genetically modified or dramatically expanded to the point of genetic instability and risk for cancer. Simple product release criteria should be sufficient to assure safety of autologous stem cell products. In addition, they should establish advisory committees that include patient advocates for every clinical application and be even more permissive for “no option” patients as it has been discussed in several recent papers in CellR4 (http://www.CellR4.org) where you can find patients’ perspectives and even the opinion of an ethic expert from UM.

Q: Some patients are trying to fund clinical trials themselves. There is one for Parkinson's and another for cerebral palsy. Do you think this is an idea that universities could embrace and help to move forward as long as funds were mostly generated by the patients?
A: I never charged a patient in my life and hope will not have to change that … In my view there could be Foundations set up for that specific disease treatment that could do fundraising for pilot, demonstration trials and then the transplants could be performed for free to the patients selected independently from weather they can pay for their treatment or not. Some argue that if you would allow patients to pay for their experimental treatment then you would privilege people that can afford those treatments versus those who cannot. On the other side think if that would be the only option to support expenses for a clinical trials then why shouldn’t we let who is willing and can afford it to support those pilot trials and then if it will work for them the treatment will become established and accessible also to those who could not pay for it at the beginning. You could argue that in the absence of those wealthier patents who could afford to pay for the pilot trial it would have been impossible to develop the treatment and also that they took the risk of failure or side effects associated with an unknown treatment. Not an easy decision though. The best scenario would still be to be able to raise the funding necessary even from patients and philanthropy but independently from who will receive the treatment, or that NIH would have a budget asides for such high risk high return translational pilot trials, with special study section to select them composed of scientists, ethic experts and patients/patient advocates, so that the decision on what to fund is not based exclusively on scientific merit.

Q:Why is there so much resistance to change in this industry in your opinion? It seems like we are dealing with so many who are blocking patient access to their own stem cells. It's disheartening and unrealistic to think patients will not go offshore and yet those who cannot are left at the mercy of the antiquated regulatory system and those who are protecting their own interests. It is so unfair. Is your Cure Alliance group working to bring about change?
A: The Cure Alliance is trying to change this but to do so we need more than scientists, we need patients, patients advocates, business leaders philanthropy leaders, communication and media leaders, the message and the awareness campaign must go viral in social media, publications such as CellR4 (http://www.CellR4.org), TED Conferences, TV and radio shows. We should have millions of followers on Facebook: The Cure Alliance (page and group), and on the website: http://www.TheCureAlliance.org and not just 15,000.

About Camillo Ricordi, M.D.
Camillo Ricordi, M.D. is the Stacy Joy Goodman Professor of Surgery, Distinguished Professor of Medicine, Professor of Biomedical Engineering, and Microbiology and Immunology, and serves as Scientific Director and Chief Academic Officer of the University of Miami Diabetes Research Institute. At the University of Miami Leonard M. Miller School of Medicine, Dr. Ricordi also serves as Chief of the Division of Cellular Transplantation, Department of Surgery, Director of the Diabetes Research Institute Cell Transplant Center and Responsible Head of the cGMP Human Cell Processing Facility, an NIH funded cGMP facility that has been providing Human Cell Products for research and clinical applications at UM, in Florida and across state barriers since 1993. Dr. Ricordi has also served as Co-Director of the Executive Office of Research Leadership (2001-2003) and as Senior Associate Dean for Research (2003-2006) at the UM Miller School of Medicine.
Dr. Ricordi completed high school with a perfect score and all graduate and post-graduate studies with the highest scores and honors in Milan, Italy. After medical school, board certification and military service as a medical officer in the Italian air force, he moved to Washington University in St. Louis, Missouri, where he received an NIH Research Trainee Award (1986-1988) working with islet cell transplant pioneer Prof. Paul E. Lacy. Dr. Ricordi subsequently spent four years (1989-1993) with transplant pioneer, Prof. Thomas E. Starzl, as Director of Cellular Transplantation at the University of Pittsburgh Transplantation Institute. Since 1993, he has been at the University of Miami, Leonard M. Miller School of Medicine (UM).
Acknowledged by his peers as one of the world's leading scientists in diabetes cure focused research and cell transplantation, Dr. Ricordi is well-known for inventing the machine that made it possible to isolate large numbers of islet cells (insulin-producing cells) from the human pancreas and for performing the first series of successful clinical islet transplants that reversed diabetes after implantation of donor purified islets into the liver of recipients with diabetes. The procedure is now used by laboratories performing clinical islet transplants worldwide. Dr. Ricordi has also developed highly innovative strategies in an attempt to transplant cells and organs without the continuous requirement for anti-rejection drugs. He coordinated the University of Miami Stem Cell, Cellular Therapies and Tissue Engineering Initiative, which resulted in the establishment of the W. H. Coulter Center for Translational Research at the University of Miami.
Dr. Ricordi was president of the Cell Transplant Society (1992-94), co-founder and chairman of the National Diabetes Research Coalition (Chairman 1997), co-founder and president (1999-2001) of the International Association for Pancreas and Islet Transplantation (IPITA), and a member of the council of The Transplantation Society (2002-2008). He also served on the council of the American Society of Transplant Surgeons (2000-2002), on the National Institutes of Health (NIH-NIAID) Expert Panel on clinical approaches for tolerance induction, on the FDA Biologic Response Modifiers Advisory Committee, on the NIH/NCRR Islet Cell Resources (ICRs) Executive Committee, on the NIH-NIDDK Strategic Planning Committee and on the NIH-NIAID Expert Panel on Transplantation Research. He is currently serving as Chairperson of the Clinical Islet Transplant Consortium (NIDDK-NIAID). He has also been serving on several NIH study sections, including Surgery, Anesthesia and Trauma, the General Clinical Research Centers, Small Business Innovative Research, the Immune Tolerance Network, in addition to serving as a reviewer for several international funding agencies.
Dr. Ricordi has received numerous honors and awards, including the 2001 Nessim Habif World Prize in Surgery (University of Geneva) for developing a technology that significantly contributed to the advancement of a surgical field. He was awarded the 2002 Outstanding Scientific Achievement Award and delivered the Lilly Lecture at the 2002 Congress of the American Diabetes Association. He also delivered the opening plenary (Galileo Lecture) at the European Association for the Study of Diabetes (EASD) Congress in Rome (2008). In 2009 Dr. Ricordi was Knighted by the President of the Republic of Italy in the highest Order of the Republic (the Order of Merit) with the Knighthood decoration of Cavaliere Ufficiale and in 2010 he was only surgeon and one of the few ever inducted into the Association of American Physicians (AAP). In 2011 Dr. Ricordi received the D-Life's Top Award for making the biggest difference in diabetes in 2012 (international web-based public vote competition).
Dr. Ricordi is currently serving on the editorial boards of Cell Transplantation (Editor-in-Chief), American Journal of Transplantation (Associate Editor), and Transplantation. He also served on the boards of Transplantation Proceedings, Tissue Engineering, and Graft (Editor-in-Chief, 1998-2002).
An Adjunct Professor at the Wake Forest Institute for Regenerative Medicine, Wake Forest University, and at the Karolinska Institutet in Stockholm, Sweden, he also serves as President of ISMETT (Mediterranean Institute of Transplantation and Advanced Therapies) in Palermo, Sicily and on the board of the DRI at H. San Raffaele Institute in Milan, Italy.
Dr. Ricordi serves as President of the Cure Focus Research Alliance and Chaiman of the Diabetes Research Institute Federation, coordinating and promoting cure focused research at over 24 leading institutions worldwide, while further developing the telescience platform technology to eliminate geographic barriers to scientific collaboration. These initiatives now allow scientists and project teams from around the world to synergize efforts and work together like if they are in the same physical space.
Dr. Ricordi has authored over 600 scientific publications, and as an inventor, he has been awarded 11 patents.

Contact Information
Camillo Ricordi, M.D.
Stacy Joy Goodman Professor of Surgery
Distinguished Professor of Medicine
Professor of Biomedical Engineering, Microbiology and Immunology
Director, Diabetes Research Institute and Cell Transplant Center, University of
Miami
1450 NW 10th Avenue, Miami, FL 33136
Phone: 305 243-6913; Fax: 305 243-4404
E-mail: ricordi@miami.edu
http://www.diabetesresearch.org