The International Society for Cellular Therapy (ISCT), the global society of clinicians, researchers, regulators, technologists, and industry partners dedicated to the translation of cellular therapy into safe and effective therapies to improve patients’ lives, announced the launch of its publication and reference guide on the use of unproven cellular therapies.

The documentation has been authored by the ISCT Presidential Task Force, comprising 23 leading figures in the field of cell therapy. The publication, constructed over two and a half years, is the most recent and comprehensive initiative by ISCT to combat and raise awareness about unethical and unproven cellular therapies and medical tourism.

The publication is being launched at the ISCT South and Central America 2015 Regional Meeting, in Santiago, Chile at the University of Los Andes, October 14 to 16.

The web-based open access shared knowledge document is intended to be used by the entire cellular therapy community, from patients and patient associations to medical practitioners, bioethicists, scientific organisations, regulators and all those involved with cellular interventions across the world. This will enable all concerned to make more informed decisions about treatments in this growing field of medical care.

The publication is part of a long-term program by ISCT to deal with unproven cellular therapies. This ISCT programme involves establishing a multilateral task force comprising patient organisations, professional societies, and regulatory agencies as well as implementing a long-term program of regulatory harmonization. This publication is the first deliverable from the Presidential Task Force. The programme aims to continuously provide updated guidance to patients and practitioners to support dialogue and informed decisions on the access to cellular therapies.

The document, entitled ‘On Unproven Cellular Therapies 2015. Talking About Unproven Cell-Based Interventions’ is available on www.celltherapysociety.org and will be published in the ISCT’s scientific journal Cytotherapy in January 2016. The various sections of this manuscript 1) define unproven cellular therapy, 2) deal with industry issues including cell manufacturing, 3) investigate globally diverse and contradictory regulations, 4) demonstrate how unproven treatments negatively influence genuine cell-based products, 5) propose action to improve communication among international monitoring organizations, and 6) outline the next stage of the ISCT initiative to promote ‘proven cellular therapy’.

“It has been essential for ISCT to take the lead on dealing with unproven cellular therapies. This will be a vast and complex but critical task, involving all parties in the cell therapy field. There is no magic wand to wave, and as the cell therapy field continues to evolve, it will be critical to provide guidance to the entire cell therapy community to make sure that the potential of cell therapy is realized. Unproven cellular therapy has the possibility of tarnishing the reputation, business case for, and thus potential of proper cellular therapy treatments in the minds of patients and investors alike,” said Daniel J. Weiss, ISCT chief scientific officer.

Cellular therapy is a new field of medical treatment involving the administration of living cells, mainly various types of stem cells and immune cells, into patients as therapies for multiple disorders that range from chronic, controlled conditions, such as arthritis, Crohn’s disease or wrinkles, to untreatable, debilitating, life-threatening, or incurable diseases like cancer, ALS and spinal cord injury. As it is at the forefront of medical treatment, there are only currently up to 30 approved cellular therapies that are available to patients, depending on their circumstances. According to clinicaltrials.gov, there are currently over 30,000 studies in cellular therapy in development and progressing through the clinical trial process. Though the failure rate is high through the approval process, many cellular therapy treatments, once approved, have the potential to treat a range of conditions with no current therapies.

The lack of currently effective treatments for some conditions has led a number of patients to seek alternative, unregulated treatments. The ISCT report estimates the market for unproven cellular therapies to be worth as much as USD 2.4 billion (EUR 2.13 billion, GBP 1.57 billion). It is estimated that currently approximately 60,000 patients every year globally are treated with unproven cellular therapies. ISCT estimated that charges for individual treatments can be as much as USD 40,000 (EUR 35,500, GBP 26,000) per treatment. This also does not account for the considerable psychological and potential physical harm to patients and families through raising and dashing hopes of a ‘cure’ that has little or no real benefit.

As a result, ISCT, as the leading authority in the cellular therapy field with 23 years experience and comprised of over 1,300 international members, was ideally placed to compile a guide for practitioners, patients and those associations concerned with cell therapy. ISCT drew from a range of experts in the field and longstanding relationships with global regulatory agencies.

The ISCT guide summarizes a range of challenges, and provides education for patients and those dealing with the cellular therapy industry. The guide highlights that regulation varies widely according to country or product type and confusion is increased where regulation for conventional drugs is applied to cell or gene therapies. Many unproven cellular therapies are offered in countries where treatments are less regulated. However, the guide highlights there has been an increase of unproven cell therapy in countries with strict regulation but untightened loopholes.

Due to the complexities of new cellular and gene therapies, regulatory approval is often a lengthy and complicated process, and in some cases, too long for effective intervention for some patients. In addition, countries with poor regulatory oversight could encourage and provide a harbor for cellular treatments without a full understanding of the implications and risks involved in these treatments. ISCT has included in the guide an overview of regulations for treatments per country, and inter-agency and country collaborations.

In addition, providers of unproven cellular therapy can produce poor-quality articles in journals that lack robust scientific peer-review and persuade some research ethics boards to provide approval for unsuitable research. Consequently, this can allow some clinics to register studies in proper clinical trial databases. These factors make it very difficult for patients and the medical community to choose legitimate cellular therapies.

Due to the critical nature of the issue of the unethical use of unproven cellular therapy, ISCT will invite a range of other societies, regulatory departments and international government agencies to contribute additional supporting materials. ISCT views future and ongoing contributions to the guide of key importance, to ensure the document be kept up to date and remain representative of all involved as the cell therapy sector and therapies develop.

“Enhancing the power of cells to introduce new treatments to the market is moving from a pioneering phase to a more mature status. This has tremendous implications, especially for those conditions with few current approved effective treatments. However, this has led some clinics to offer treatments with, at best, questionable data, and at worst, terrible consequences for patients,” said Massimo Dominici, ISCT president and chair of the Presidential Task Force.

“This is why ISCT considered it critical to lead in compiling this comprehensive report. This will also give patients the information they need to make the decision that is right for them and promote greater cooperation amongst the entire cellular therapy sector to tackle the unproven cellular therapy issue. ISCT openly invites other societies and other relevant parties to contribute to the reference guide. This will make sure that ISCT is able to bring a united voice from the whole cell therapy community to inform patients of the dangers of unproven cell therapy.”