Many classes of therapy can be robustly manufactured with little risk of issues and do not need the full cost in time, effort, and funds of Good Manufacturing Practice (GMP) specified by the FDA in order for any given batch of the drug to be demonstrated to be safe. The most frequently used AAV vector serotypes, for example, are relatively safe in this way. One can manufacture a batch of an AAV drug with any one of the very experienced manufacturers in the same way one would for research in animals, and then run all of the quality assays needed to demonstrate that the batch is safe. This costs a lot less than full GMP but should produce essentially equivalent outcomes in safety.
Medical tourism allows companies to bring drugs to the clinic in a responsible way without having to spend vast sums conforming to what the major regulators of the world, the FDA and EMA, believe is sufficient. Here I’ll point out an example of a company doing this for VEGF gene therapy, a potential way to upregulate angiogenesis in order to reverse the age-related loss of capillary density that impairs function in tissues throughout the body. VEGF gene therapy in mice has been demonstrated to modestly extend life span. Forms of VEGF gene therapy have been trialed successfully in humans, and as noted here, have been approved in Russian for some years.
Keeping up with the longevity gene therapies
The recent revelation that Khloé Kardashian has received a gene therapy purported to promote tissue rejuvenation came as something of a surprise to many. The celebrity influencer received the capillary-boosting VEGF treatment from regenerative medicine specialist in Mexico. The therapy, which is claimed to help combat the age-related loss of vasculature in tissues and organs, was provided by Unlimited Bio, which operates out of Próspera, an autonomous special economic zone in Honduras designed to foster rapid biomedical innovation. The company says it is on a mission to conduct 100 clinical trials of genetic preventive therapies within 10 years, using the regulatory structures of Próspera to enable faster approvals and streamlined processes.
The founding mission of Unlimited Bio is ambitious: run 100 clinical trials within 10 years, building multiple gene therapies under one umbrella and combining them to combat the effects of aging. The first therapy to be offered by Unlimited Bio is a gene therapy delivered via plasmid, which delivers the genetic instructions for vascular endothelial growth factor (VEGF) into cells to stimulate the growth of new blood vessels (angiogenesis). “We wanted to start with a simple, well-established gene therapy. This therapy was first approved in Russia and Ukraine in 2011 for lower limb ischemia, and more than 10,000 patients have received it safely over 15 years. Of course, not everything that works for a disease will have benefits in healthy people, but VEGF is a unique case. Capillaries are essential for oxygen and nutrient delivery. With age, capillary density declines, which may contribute to sarcopenia and other age-related conditions. By enhancing capillarization, VEGF essentially upgrades our body’s ‘delivery system’ – adding more roads for nutrients and oxygen to reach cells. That’s why we believe it has strong longevity potential.”
Unlimited Bio licensed the VEGF therapy for use in Prospera in preventive indications, and within six months of incorporation, had its first product on the market. While only a small number of people have received the treatment to date, the Kardashian effect is already being seen. “Since several well-known influencers received the therapy, interest has grown rapidly. I can say it is the most affordable preventive gene therapy worldwide – comparable to stem cell treatments.”
