Regulation of Cellular Therapies and the Stamina Foundation: The Position of The Cure Alliance, beyond Smoke and Mirrors

by Camillo Ricordi

There have been a few very opinionated and equally misleading commentaries recently accusing Camillo Ricordi and/or The Cure Alliance of supporting “deregulation” of cellular therapies and/or the Stamina foundation in Italy. The Cure Alliance has never supported either deregulation or the Stamina foundation and their position precedes the saga of the Stamina case. The reasons for these misleading attacks are not apparent. But Ricordi’s position of and that of The Cure Alliance have been clearly and previously outlined (www.TheCureAlliance.org and www.CellR4.org).

There are many scientists and leaders in various fields, who support the need to review the guidelines and existing regulations for cellular therapies. Several countries including the UK and Japan are considering revisions of current regulations for stem cell/cellular therapies, always in respect of patient safety, but without imposing unsustainable costs and time frames, which today greatly limit testing of novel cell based potentially therapeutic approaches. Imposing to cellular therapies the same regulations as for drug development is considered by many not only scientifically unfunded, but also severely limiting innovation and the development of new therapeutic strategies, considering that now over $1 Billion and 7 to over 9 year time frames could be required for the development and approval of a new molecular entity.

Among the many who support this need ‘review (never deregulate) there are also former leaders and deputy commissioners of the FDA and industry leaders, not only pharmaceutical, such as Andrew Grove, the former CEO of Intel.

Ricordi’s recent editorial on CellR4 has also been misquoted, but is visible to all (http://www.cellr4.org/article/110), together with his opening address at the 12^th Congress of CTS where starting from minute 21:10 his position is clearly outlined.

The editorial was an attempt to mediate between conflicting positions in the field of cell therapy, reporting extracts and topics for discussion from some of the most important debates that emerged from recent articles, editorials and online commentaries. The purpose was to solicit papers/commentaries on topics that are at the center of current controversies, to be published in the next issue of CellR4 (deadline for submissions August 15^th). The objective was to encourage a dialogue and a constructive debate in an effort to understand each other positions and perhaps even start working collaboratively towards the definition of possible solutions in the interest of patients, who some of us still consider central to our work and to our professional mission.

Camillo Ricordi directs the Cell Transplant Center and the Diabetes Research Institute (DRI) at the University of Miami, where one of the most successful initiatives has been the “Fast Track Center for Testing”, a center for hands-on due diligence and verification of scientific claims on potential breakthroughs towards the development of a cure. This program has allowed for the efficient and rapid verification of ideas, discoveries, alleged novel technologies and/or innovative treatments. In recent years this center has been equipped with state-of-the- art platform technologies and shared resource infrastructure as well as skilled personnel to assist in the practical verification of cell products and cell-based therapies. The central objective was to contribute to the evaluation of ideas with real potential toward a cure and at the same time help to unmask technologies or proposed strategies which in good or bad faith are presented as promising or potentially breakthrough. This has been considered an important service not only for patients and the general public, but also for foundations, corporate entities requiring hands-on due diligence on selected claims and for the scientific community in general.

Camillo Ricordi and The Cure Alliance’s concern is to verify in the fastest and most efficient way possible putative breakthroughs and avoid the speculations that often surround possible cures or centers where unproven treatments with stem cells are proposed for everything. On the other side, it should also be recognized that the stem cells are becoming more than a promise for several clinical applications and that over 300 clinical trials around the world will help to verify safety and efficacy for each indication.

These clinical applications and trials need to be performed with the maximum transparency, appropriate IRB and informed consents, quality controls and safety precautions. In addition, these clinical trials cannot continue indefinitely in the absence of mandatory reporting and rigorous scientific verification of the results obtained, including monitoring of possible side effects and defined stopping rules.

In this direction, Camillo Ricordi and The Cure Alliance have never been pro- or against Stamina, but are pro-patients and pro-verification of the claims for treatments for disease conditions now afflicting humankind that impose unsustainable suffering and health costs, while certainly also representing an attractive market opportunity for commercial interests.

Regarding the specific trial in Italy, an over three hour meeting was held on Sunday July 7, between members of The Cure Alliance and the University of Miami Cell Transplant Center, including Camillo Ricordi, with Davide Vannoni and Gianfranco Merizzi from Stamina foundation and Medestea. The meeting has allowed the group to view and discuss a large volume of data, testimonials, expert opinions, tests and quality controls performed on cell products. The huge volume of material received is still under evaluation and confirms the position of Camillo Ricordi in favor of a scientifically rigorous and objective verification of the protocol and results that will be obtained in the approved clinical trails.

While The Cure Alliance doesn’t have an official position on the subject, as it includes individuals with different opinions, Camillo Ricordi is in favor of the scientific verification of the clinical results and claims of Stamina, which some experts have found significant, while others have discredited. The question for Ricordi is not if Stamina has performed enough animal studies or has enough scientific publications, is not if and why patents have not been allowed (incidentally, rejection of a patent has nothing to do with the possible effectiveness of a treatment proposed in the patent itself), is not whether it is right or wrong to spend € 3 million for clinical trials when other excellent scientists are struggling and must compete for increasingly scarce funding sources, to carry out research a much, or possibly even more important. Ricordi’s opinion is that such stage and level of discussion is obsolete, as hundreds of patients have been treated, the Italian Judiciary system has overturned decisions by regulatory agencies and the opinions of scientists have been overruled by the reactions of the public and the virtually unanimous vote of the Italian Parliament in favor of the clinical testing of the controversial cellular therapies offered by Stamina. It is now critically important for everyone to move forward and shed light on this whole saga.

At the end of this verification process the possibilities are at least 3:

1. The the proposed cell therapy works for the selected indications and will eventually improve the conditions of hundreds of millions of patients worldwide.

2. A partial therapeutic effect is observed which is indicative of a biological effect but the observed improvements are not sufficient to justify the continuation of the clinical trials and the costs associated with them.

3. The proposed method does not work, no clinically beneficial effect is observed in the treated subjects and the possibility’ of fraud and speculation leveraging on the expectations and desperation of patients and their families will be investigated at the appropriate levels.

In summary, Camillo Ricordi and The Cure Alliance are not for, or against anyone, but they are in favor of a verification process that is in the interest of all and that will put an end to behaviors, defamatory coalitions and polarizing misleading publications which are as false as non-constructive.

As recently introduced in Italy in the process of evaluation of grants and proposals for funding that now often requires (or welcomes) the contribution of foreign “impartial” experts, we hope that also in this case the appropriate authorities will consider the service of foreign experts who could be of assistance to perform an impartial assessment of the data and results obtained.

The Cure Alliance will continue to focus on promoting collaborative research efforts towards the definition of therapeutic strategies and will continue to work towards resolution of current impediments, challenges and obstacles on the path to develop new cures.