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Unproven cell therapies: How can access be provided to patients?
Cytotherapy, 2016; 18: 695
LETTER TO THE EDITOR
Unproven cell therapies: How can access be provided to patients? IAN MCNIECE MD Anderson Cancer Center, Houston,TX, United States To the Editor: In the January 2016 issue of Cytotherapy (volume 18 number 1), the International Society for Cell Therapy (ISCT) Presidential Task Force on Unproven Cellular Therapies presented a series of articles addressing issues around the use of unproven cellular therapies. Although the topic is covered extensively, input from patients and patient advocacy groups is lacking. I strongly agree that in the long term, it would be ideal to have clinical trials performed under rigorous regulatory oversight with state-of-the-art manufacturing. However, for many patients with debilitating diseases, this does not currently provide an immediate solution for treatment or an avenue to cure. This unmet need drives patients to seek treatment offshore or from providers working outside the regulatory framework. So, in addition to defining the issues around unproven cellular therapies, there remains a need to provide access to stem cell and other cellular therapies for today’s patients. The most advanced cellular products are being evaluated in early stage clinical trials with the time to market at least 5 years in the future.These studies typically have rigorous eligibility criteria and attempt to evaluate cell therapy in a well-defined and homogenous patient group. However, many patients looking for treatment with cellular therapies have comorbidities, making them ineligible for such clinical trials. It seems regrettable that the ISCT Task Force provides no solution for these patients. The message that for safety reasons it will be necessary to wait until these therapies are proven does not resonate with patients and their families. I have a grandson with developmental problems and am aware of the issues parents face in trying to get treatment for their children. Also, a good friend has a child with developmental problems who received stem cell treatment in Peru. Before the treatment, the child had regular seizures that disappeared after treatment. I was opposed to the treatment, but I cannot refute the fact that, while not curative, the treatment had a major beneficial impact on this child
(whether or not it was a placebo effect). In a clinical trial with a high bar for response and cure, this benefit might have been defined as not significant and lost, barring the opportunity of a second treatment and further potential for improvement. The need for access to stem cell therapies for existing patients is a significant challenge. However, I believe it is possible today to safely apply unproven cell therapies within an acceptable regulatory framework on a compassionate basis. For example, given the safety of MSCs in clinical trials and the potential to manufacture allogeneic MSCs for banking, there could be a treatment offered in the United States under an umbrella Investigational New Drug that would make this option available to patients with diverse conditions. There could be a central manufacturing facility providing donor MSCs; cost would be relatively low (about $3,000 per product, and clinical service could be provided by research centers involved in the specific disease. The ISCT could lobby the National Institutes of Science to obtain funding for such a trial, offering a compassionate option for such patients. Because the therapy is of proven safety, the worst outcome envisaged would be lack of efficacy. Given the breadth of expertise on the ISCT Presidential Task Force on Unproven Cellular Therapies, I think other cell-based options could also be developed. In the absence of effective treatment alternatives, there will continue to be a need for unproven cell therapies for many conditions, and patients will continue to travel oversees to “clinics” that are less than optimal in many cases. Here is an opportunity for the ISCT to help deliver safe but unproven cell-based treatment for patients and be a leader in this field. More regulations will not change this need but rather make it more difficult for patients to receive therapies locally, leading to an increase in the tide of medical tourism. I therefore propose that any discussion of unproven cell therapies should include consideration for immediate solutions for today’s patients, as well as defining more rigorous long-term goals.
Correspondence: Ian McNiece, PhD, MD Anderson Cancer Center, Houston, TX, United States. E-mail: [email protected]
ISSN 1465-3249 Copyright © 2016 International Society for Cellular Therapy. Published by Elsevier Inc. All rights reserved. http://dx.doi.org/10.1016/j.jcyt.2016.02.007
LETTER TO THE EDITOR
Unproven cell therapies: How can access be provided to patients? IAN MCNIECE MD Anderson Cancer Center, Houston,TX, United States To the Editor: In the January 2016 issue of Cytotherapy (volume 18 number 1), the International Society for Cell Therapy (ISCT) Presidential Task Force on Unproven Cellular Therapies presented a series of articles addressing issues around the use of unproven cellular therapies. Although the topic is covered extensively, input from patients and patient advocacy groups is lacking. I strongly agree that in the long term, it would be ideal to have clinical trials performed under rigorous regulatory oversight with state-of-the-art manufacturing. However, for many patients with debilitating diseases, this does not currently provide an immediate solution for treatment or an avenue to cure. This unmet need drives patients to seek treatment offshore or from providers working outside the regulatory framework. So, in addition to defining the issues around unproven cellular therapies, there remains a need to provide access to stem cell and other cellular therapies for today’s patients. The most advanced cellular products are being evaluated in early stage clinical trials with the time to market at least 5 years in the future.These studies typically have rigorous eligibility criteria and attempt to evaluate cell therapy in a well-defined and homogenous patient group. However, many patients looking for treatment with cellular therapies have comorbidities, making them ineligible for such clinical trials. It seems regrettable that the ISCT Task Force provides no solution for these patients. The message that for safety reasons it will be necessary to wait until these therapies are proven does not resonate with patients and their families. I have a grandson with developmental problems and am aware of the issues parents face in trying to get treatment for their children. Also, a good friend has a child with developmental problems who received stem cell treatment in Peru. Before the treatment, the child had regular seizures that disappeared after treatment. I was opposed to the treatment, but I cannot refute the fact that, while not curative, the treatment had a major beneficial impact on this child
(whether or not it was a placebo effect). In a clinical trial with a high bar for response and cure, this benefit might have been defined as not significant and lost, barring the opportunity of a second treatment and further potential for improvement. The need for access to stem cell therapies for existing patients is a significant challenge. However, I believe it is possible today to safely apply unproven cell therapies within an acceptable regulatory framework on a compassionate basis. For example, given the safety of MSCs in clinical trials and the potential to manufacture allogeneic MSCs for banking, there could be a treatment offered in the United States under an umbrella Investigational New Drug that would make this option available to patients with diverse conditions. There could be a central manufacturing facility providing donor MSCs; cost would be relatively low (about $3,000 per product, and clinical service could be provided by research centers involved in the specific disease. The ISCT could lobby the National Institutes of Science to obtain funding for such a trial, offering a compassionate option for such patients. Because the therapy is of proven safety, the worst outcome envisaged would be lack of efficacy. Given the breadth of expertise on the ISCT Presidential Task Force on Unproven Cellular Therapies, I think other cell-based options could also be developed. In the absence of effective treatment alternatives, there will continue to be a need for unproven cell therapies for many conditions, and patients will continue to travel oversees to “clinics” that are less than optimal in many cases. Here is an opportunity for the ISCT to help deliver safe but unproven cell-based treatment for patients and be a leader in this field. More regulations will not change this need but rather make it more difficult for patients to receive therapies locally, leading to an increase in the tide of medical tourism. I therefore propose that any discussion of unproven cell therapies should include consideration for immediate solutions for today’s patients, as well as defining more rigorous long-term goals.
Correspondence: Ian McNiece, PhD, MD Anderson Cancer Center, Houston, TX, United States. E-mail: [email protected]
ISSN 1465-3249 Copyright © 2016 International Society for Cellular Therapy. Published by Elsevier Inc. All rights reserved. http://dx.doi.org/10.1016/j.jcyt.2016.02.007