Future of Medicine should not be Elysium

To have a long and healthy life was and is a dream of humankind since the inception of human civilization. Based on current pace, within 20-50 years medical science will achieve level where many difficult to manage diseases, like cancers and some infectious diseases become easily treatable.

Most recent example is hepatitis C virus story. Just 5 years ago, it seemed that there was no hope for people affected by Hep C. There was no approved vaccine or approved drugs capable of stopping this virus that causes extensive liver fibrosis. However now, if completely unexpectedly, we have anti-viral medicine that show up to 99% effectiveness rate (in some combination) and with less side effect (double benefit). No one could imagine this.

What about recent success in cancer therapy? Treatments with patient’s own tumor-specific re-engineered T cells or with antibodies to awaken the body’s own immune system had achieved an unprecedented level of success reaching up to 50% effectiveness rate.

These are all amazing news for anyone and especially for individuals with these medical conditions. However, even though the future seems bright, there is something unsettling in all this. It is the price tag.

Costs are so prohibitively high that almost 95% of world population would not able to afford it on their own (for example, tumor immuno-therapy with the patient’s own immune cells can cost more than 500,000$ , and Hep C treatment can cost between 50,000 – 120,000$ per treatment ).

More worrisome is the fact that unlike small molecules used in conventional pharmacology that can be easily manufactured in mass scale with minimal investment as generics, many advanced new treatments now days are based on large, complex molecules, called biologicals like humanized antibodies, or even more complex procedures like harvesting and re-engineering patient’s own T cells. These are completely different categories of medicine with no real way for mass scale production.

How can we afford this costly medicine to live longer and healthier?

Movie, Elysium, shows one such scenario. In this movie, society are divided into super rich and everyone else. Super rich live on a giant orbital station and are completely free of any disease thanks to medical technologies. These medical technologies are inaccessible to regular earthly people.

In my opinion, with the few rare exceptions, we do not control our medical health trajectory. Hence, every human should have an inherent right to have an access to the most advanced medical technologies. However, no federal or state budget can afford medical cost so high.

So what is the solution? how to make cutting edge medical technologies affordable to average person? The solution can lie in technology but in different kind of technology that is in infancy right now. It is gene therapy (gene silencing, cutting, replacing, modifying). Gene based medical technologies has two major advances over other type of biologicals: (a) gene therapy will be much cheaper (everyone who worked with PCR or RT-PCR can verify that costs for primer nucleotides are way cheap compared to antibodies), (b) gene therapy will treat the cause (genes) rather than outcome (proteins) of medical conditions.

Current medicine is based on repeated engagement with body’s proteins (receptors, cytokines, transcription factors). Gene therapy will be based on single or a few engagements with body’s DNA or RNA for long-term effect (for example, using CRISPR-Cas9 system). Such intervention can produce permanent fixes. An Overall cost will be low and majority will be able to afford it.

posted by David Usharauli


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