Steady advance of stem cell therapies

Stem cell research and related therapies (including regenerative medicine and cellular therapies) is an industry with a strong possibility of having a significant near-term impact on worldwide public health. One reason is the industry’s linkage between policy, science, industry, and patient advocacy, as was clear in the attendance and programming at the 7th annual World Stem Cell Summit held in Pasadena CA, October 3-5. Other science-driven fields such as synthetic biology, nanomedicine, and aging might benefit from cultivating such a multi-disciplinary perspective. Stem cell therapies are useful not only in cell-replacement therapies, but also in disease modeling, drug discovery, and drug toxicity screening.

Disease therapeutics and clinical trial focus
Stem cell therapies are currently being applied to over 50 diseases particularly in the areas of heart, lung, neurodegenerative, and eye disease, and cancer and HIV. Dozens of companies are developing therapeutic solutions which are in different stages of clinical use and clinical trials. Some high-profile therapies include Dendreon’s Provenge for prostate cancer, Geron’s first-ever embryonic stem cell trials for spinal cord injury, Fibrocell’s laViv cellular therapy for wrinkles, and well-established commercial skin substitutes (Organogenesis’s Apligraf and Advanced BioHealing’s Dermagraft).

Policy
Stem cell policy issues under consideration include medical tourism, standards for large-scale stem cell manufacturing, and lingering ethical debates over the use of embryonic stem cells.

Science
Contemporary stem cell science advances include a focus on techniques for the direct reprogramming of cells from one lineage to another without having to return to pluripotency as an intermediary step, improved means of generating and measuring induced pluripotent cells, and progress in approaches to neurodegenerative disease, for example establishing causal factors for early-onset Parkinson’s disease, generating neuronal cells and dopaminergic cells, and neural stem cell lumbar implantation clinical trials.

VC life extension opportunity redux

The VC life extension investment opportunity could happen in at least three areas: first and most importantly, translational medicine, second, health social networks and third, distantly, standardized longevity treatments and delivery systems.

Translational medicine
The biggest and most obvious longevity play for VCs is in translational medicine, shepherding and commercializing science findings from basic research to patient therapies. The vast majority, perhaps even 90% or more, of basic research findings never go beyond the lab or journal publication. A particularly high profile example of an early stage longevity company that rocketed from test tube to IPO to big pharma acquisition is Sirtris, bought by GlaxoSmithKline for $720M in June 2008. Some other early stage translational medicine longevity startups are Elixir Pharmaceuticals, Juvenon and Sierra Sciences. In the plethora of remedies claiming science support, it is critical to tightly link the research evidence to the intervention. For example, in this year’s exploding brain fitness market, there is much claim of scientific support but little published clinical trial evidence.

Health social networks
Another VC play is health social networks (for example, PatientsLikeMe, CureTogether, DailyStrength, HealthChapter, Experience Project and peoplejam). Not only can patients connect and generate group-synthesized, curated and moderated knowledge, but health social networks can also facilitate the development of personalized medicine by being a repository for the quantitative data of genomics, ongoing biomarker measurements and electronic healthcare records. Big pharma can approach patient communities for field studies and clinical trials.

Longevity treatment delivery
Standardized longevity treatments and delivery programs via private clinics are not a traditional VC play, but some interesting 10x business models may be possible. In the several years before longevity treatments are more proven and automatically administered via traditional healthcare channels, these services can be provided by private clinics as they are now.

What would improve the longevity treatment market is standardization; standardization of doctor qualifications, certifications, validations, services and treatments, and treatments supported by scientific research clearly evidenced to consumers. Currently, longevity doctors offer heterogeneous suites of services which is challenging for consumers to parse. Positioned appropriately, there is more than adequate demand despite the current lack of insurance reimbursement. Since longevity treatments are currently outside the purview of traditional medicine, third party certification (for example led by the Methuselah Foundation) could help validate doctors and treatment programs, and contribute to industry standards.