In-depth biotechnology features: The ins and outs of the biotech industry

Desperate patients are ready to do everything to access to new treatments and Google searches can help them find medicines in the early stages of development rather than those with proper authorization. They latch on to this false hope and travel to search for treatments, sometimes with deadly consequences. When you are chatting about health tourism, you are probably thinking about medical tourists travel to the jungle to obtain some plant used in traditional ethnomedicine. 21st-century patients who have reached a therapeutic “dead end” might also consider biotech solutions still under development, not yet on the market or even outright forbidden in their locality. We can't blame them, given the inescapable media hype surrounding these novel therapies! Is it surprising that when patients read “Researchers Bring Gene Editing to Patients with Deadly Diseases” (San Jose Mercury News) they think the cures are already here?“ R. Alta Charo, J.D. (N Engl J Med 2016) The biotech market is definitely more challenging and heterogeneous than its traditional molecular counterpart, due to regulatory, marketing and technology issues. As a result, there is quantitative and qualitative inconstancy in products availability worldwide. Innovative products such as stem cells are a special victim of this phenomenon such that the European Committee for Advanced Therapy (CAT) has raised concern about it. The CAT is concerned about a phenomenon known as stem-cell tourism in which severely ill patients travel to clinics around the world where unauthorised stem-cell-based treatments are offered in the absence of rigorous scientific and ethical requirements.” European Committee for Advanced Therapy on the use of unregulated stem-cell based medicinal products You might expect that variable- or low-quality product issues are limited to developing countries, but guess what? There is also variability within Europe! It may sound like the regulatory standards for ATMPs (Advanced Therapies Medicinal Products) are tough to meet, since only 6 cell therapies have been authorized and only 3 are on the market (Zalmoxis; Glybera; Holoclar). However, many products have been authorized via regulatory pathways specific to individual nations, like the UK's EAMS for drugs targeting life-threatening conditions, that are not up to continental standards. Though the ATMP legislation aims to unify quality requirements, there is still significant variability from country to country. Here's the catch: the ATMP cannot ban these products, but they are limited to the country where they were approved. As a result, patients travel internationally but within the EU to obtain specific therapies. Behind the stories, what are the facts? There are plenty of articles covering horror stories in cell therapy tourism to countries in Asia and South America that are often more flexible from a regulatory perspective than Europe. But adverse events are not limited to the far reaches of the world: they can happen close to home, too. In Germany, a clinic called X-Cell Center raised concern in 2010 when a 10-year old child from Azerbaijan suffered internal bleeding after bone marrow-derived stem cells were injected into her brain. Around the same time, an 18-month old girl from Italy died from complications resulting from a similar procedure (eurostemcell.org). More recently in 2014, five Americans tested seropositive for Coxiella burnetii, the agent of Q fever. Suspiciously, these patients traveled to Germany to receive live cell therapy a treatment in May 2014. (center for disease control and prevention). But even if establishments like X-Cell Centre are closed, they can move to another country where regulation is less strict. Sure enough, X-Cell's founder opened stem cell clinics called Cells4health in Lebanon and India after the German outpost was shuttered. In another infamous example, patients with neurodegenerative diseases were given an Italian treatment called Stamina®, which is based on mesenchymal stem cells. After the Italian Parliament raised €3M for a trial in 2013, it was billed as a compassionate treatment and administered to a broad range of patients. However, a leaked protocol of Stamina administration revealed many gaps, most troubling regarding GMP (Good Manufacturing Practice). Further, a week after the leaks, the health ministry revealed that the condition of 36 patients treated with Stamina’s therapy had not improved, contrary to clinic publication. Treatment was halted a few months later. English, German, French and other patients could start a sort of medical tourism and ask Stamina to provide them with such unproven therapies” Prof Michele De Luca, Centre for Regenerative Medicine at the University of Modena, IT And what next? Authorities are aware of the phenomenon of unregulated and egregiously sloppy procedures. Accordingly, they are working to clamp down on these and control the market in order to discourage patients from traveling to undergo risky procedures. Even if there is seemingly scant evidence of adverse effects, patients must be wary of products authorized at the national level in Europe and reconsider traveling to obtain procedures that have not yet withstood the scrutiny of the continental authority. Featured Image: Tourism (CC2.0, Gregory Bodnar/Flickr) Figure 1: European Commission (P0/Pixabay) Figure 2: Neural Stem Cell (CC2.0, Joseph Elsbernd/Flickr) Figure 3: Mouse embryonic stem cell growing on mouse embryonic feeder cells (CC2.0, Joseph Elsbernd/Flickr)

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