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Wednesday, August 6, 2008

Does Cell Therapy Demand a Different Investigatory-to-Commercial Paradigm?

I've just finished reading a great post by blogger Sonya Fatah. It's a very balanced and thorough post (click here to read it) on the stem cell medical tourism phenomenon complete with ample anecdotal patient testimonials - including some longer-term follow-up stories - as well as content from interviews of Indian embryonic stem cell transplant [pioneer / capitalist / fraud / savior - depending who you ask] Dr. Geeta Shroff (pictured here). Sonya's post also outlines the reaction of Dr. Stephen Minger et al (including colleagues in India) who tow the more western medicine party line that all this treating patients commercially prior to the rigor of clinical trials, peer review, and regulatory compliance/approval is...well, just wrong.

Word is that ISSCR is busy preparing a public policy statement regarding the stem cell medical tourism industry. It will be curious to see how they balance the various perspectives here given that they are an 'international' society with a clearly 'western' bent. They apparently had a very lively session at their latest conference on this topic.

I'm a firm advocate of regulatory frameworks to protect people from the predators, fraudsters, or even just the sloppy. I'm also an ardent believer in the benefits of peer review, transparency, and accountability. As a product of western inculcation (education) I also believe in the benefits of the clinical trial system and long-term follow-up as a means of vetting therapies before we let them loose on a 'suspeptible' public, regardless how well-meaning or even beneficial the efforts to make such therapies available earlier might be.

Having said all that, even in the West we make certain - albeit regulated - exceptions for no-option patients, etc. And patients - upset they cannot access what are perceived to be relatively safe, low-risk, potentially life -saving/altering stem cell treatments in the US - are voting with their feet and checkbooks traveling to Central America, the Caribbean, India, Asia, and anywhere else they can pay for the privilege to access unproven treatments. It's hard to argue with patients suffering from crippling, painful, and/or terminal ailments that they should be denied the right to choose their treatment.

Would it be better if these treatment providers - most of them companies charging amply for their treatments - would publish their science, conduct clinical trials even whilst they charge patients for treatment, be more transparent with their products/protocols, and/or conduct and publish the results of long-term follow-up? Sure. But this is not as much a part of the culture of eastern medicine. Clinical trials, regulatory approval, peer review...are all hallmarks of western science. Furthermore, different attitudes toward IP protection means transparency and publication are more easily exchanged for trade secret protection.

Has this therapeutics globalization triggered more a clash of cultures than one science and medicine? Could it be said that cell therapies - not unlike herbal remedies, acupuncture, chiropractic regimes or other forms of relatively harmless treatment modalities - do not require as strict a regime of clinical trial and regulatory oversight prior to commercial access? Are they inherently safe enough to allow for a different investigatory pathway or have we just not yet discovered the potentially harmful - if not deadly - side effects presented by predecessor pharmaceuticals, biotechnologies, and even the sister gene therapy which mandate the kind of process we've invented? Would our efforts be better spent on patient education rather than opposing the development and practices of such clinics?

Perhaps only time will tell. I definitely don't have the answers but I can most certainly understand those who - when faced with unbearable alternatives and a perception of very little downside - decide to travel and pay for treatments that cannot be accessed in North America. I am also sure that we in the West must be more than careful in judging or attempting to impose our values or worse - our systems - on those who think differently.

It is easy to accuse companies charging amply for these treatment of being fraudulent or, at the very least, of being motived solely by profit to sell something unproven to those with no options. This is tricky position to take while sitting in a country noted for its free enterprise with its predatory lending practices, legalized gambling, credit card companies charging double-digit interest rates, capitalist institutions more than eager to take advantage of the purchaser's freedom of choice to buy beyond their means, and a health care system with some of the highest costs in the world while allowing a majority of its citizens to go without coverage.

In this world of increasingly globalized medicine there will be medical jurisdiction shopping just like there are havens for taxes, drugs, weapons, etc. We must find ways to work together to a better, harmonized end. The FDA - at least at CBER - is open to accepting data produced outside the US in trials that follow basic strictures. Innovative organizations, academic institutions, and companies should seek ways to collaborate with the companies providing these treatments to attempt to "validate" them to western standards while opening them up to peer review.

Let's keep an open mind and discourse as we seek global solutions to balancing patient protection, choice, education, and access.

We must do everything we can to encourage all involved to conduct clinical trials, perform long-term follow-up, publish their science, and be open to peer review. We should try to better understand what is happening in these jurisdictions rather then simply expecting or demanding they comply with our systems. We should be cautious in our criticism and open in our consideration of whether new treatment modalities open the door for different regulatory pathways that provide safe choices for patients as soon as reasonably possible.

4 comments:

Lee Buckler said...

It came to mind this morning that the Dendreon Provenge case is another example of US patients showing increasing frustration with the FDA saving people from their own power of choice. The agency is worried at this point, as I understand it, about safety - only efficacy. I understand the need for a standard which new therapeutics must meet to be approved for sale but we're also denying patients access to a therapy that is relatively safe and the evidence to-date shows work well for some patients. In the instance of Provenge, these patients are not taking this lying down. There must be a model that better balances the interests in certain circumstances.

--Lee

Lee Buckler said...

This week's (4 Aug 2008) GEN podcast is on a related topic: CLINICAL TRIAL ETHICS IN THE DEVELOPING WORLD

http://www.genengnews.com/gencasts.aspx

Jon Rowley said...

Lee,

Another great post. Medical Tourism is a strange phenomenon, and I am still uncertain if I am for or against. Some of the questions you pose remind me of a few posts by Ogan Gurel at Life Science Chronicle. Ogan thinks Safety should be the main driving force behind FDA decisions; see (http://blog.aesisgroup.com/2007/07/02/fda-tortoise-hare-or-something-else.aspx) & (http://blog.aesisgroup.com/2007/07/06/fda-reform-redux-on-business-models-regulatory-reform-and-safety.aspx) and that the market should be left to decide efficacy. He argues that as long as therapies do no harm, then the ones that work will eventually win out. It is an interesting way to think of FDA reform, and would have some interesting impacts on cell therapy. Would love to hear your thoughts on these concepts..... JON

Anonymous said...

I dont understand the concept of "medical tourism", either something works or it doesnt and thats the bottom line.

The problem is that we get so caught up in thinking about political arguements (ie patients have a right to decide vs everyone offshore is a charlatan) that we forget the basic reasons why people do go offshore

take autologous therapy...autologous fat stem cells have been known for almost a decade to be useful in stimulation of angiogenesis...places such as www.cellmedicine.com do autologous adipose with some interesting data...they have evenpublished some of their other approaches...but this is an example of something based on good science...you cant compare autologosu adipose or autologous bone marrow "medical tourism", with crazy embryonic stem cell injections into people