Signals Blog

With contributions from James Smith, a recent Oxford University graduate and current SENS Research Foundation Summer Scholar working at the Harvard Stem Cell Institute.

There were two momentous events on the night of August 23, 2014. First, the inaugural Rejuvenation Biotechnology conference drew to a close. Second, the largest earthquake in nearly 25 years hit the San Francisco Bay Area.

Perhaps a result of several obligatory refreshments at the end of the day, James’ sleep was not disturbed by the significant shaking. In fact, he was quite surprised to hear of it in the morning, much to the amazement of several colleagues.

Conversely, David – for better or worse always at the heart of the action (as per a previous post) – was in a highly flexible, wood and tin cottage two miles from the epicentre in Napa. Fortunately, we both survived, remedied a major deficiency in British education, previously unaware of ‘earthquake protocol,’ and report key take homes of a stellar ‘final days play’ in San Jose.

Here are some thoughts from the three day event.

Basic science is required

A key and repeated message is the continued need for basic scientific research. As mentioned in the blog post from the first day of this conference, the discovery of the CRISPR system exemplifies this. Another example, green fluorescent protein – or GFP – was provided by Brock Reeve of the Harvard Stem Cell Institute. The protein is now used widely as a tool for cell biology visualization. Its discovery was not driven by application, but purely by the desire to understand why jellyfish luminesce. There is also a need for more directed basic scientific research in understanding various diseases and the processes of aging in general. Alzheimer’s disease was highlighted as an area for such research; even now, relatively little is known about the causes of this disease and thus developing successful treatments has had limited success.

One size does not fit all when it comes to funding

Funding basic research is problematic; it is here, in open-ended research with no obvious commercial output, that government will need to play a significant role. Of course, once basic breakthroughs are made, translational science is paramount. Organizations such as Breakout Labs, which supports early-stage companies moving from the lab to the economy, may play an important role in getting companies to later stages that can attract the attention of angel investors and venture capital firms.

We need to change our attitude towards regulation

As we discussed in our second post on the conference, the general attitude towards regulation seems to be fundamentally changing. Despite this, a stark reminder of the general opinion of regulation came when a speaker raised the idea that treatments could be administered (and therefore paid for), once safety has been established, with no evidence of any efficacy. This was met by applause from many in the audience, and dissent from far fewer. It seems surprising that such an ethically questionable policy would be supported, in which desperate patients could be given hope from treatments that ultimately have no proven benefit. There must be awareness that regulation is necessary and inevitably beneficial for all. Even in industry, efficacy and safety should supplant profit at the top spot.

Regulatory reform could be beneficial

Having said this, fundamental change in the way new treatments are regulated could be beneficial. As an example, Japan’s new approach to stem cell therapy approval appears to have attracted significant support. In Japan’s new, fast-track approval path for stem cell therapies, phase 3 clinical trials are not required and efficacy need only be demonstrated in small patient samples. Such an intermediate approach to regulation might be beneficial elsewhere, as long as patients are aware of the evidence, or lack thereof, for a particular product.

Collaboration above all else

The need for collaboration was a resonating message. We think there are two ‘directions’ in which collaboration can occur: discovery and translation. In discovery, collaboration amongst scientists from different areas is fundamental to the discovery and identification of new treatments, new targets for treatment and greater understanding of disease. In translation, collaboration between the scientists involved in discovery with patients, clinicians, industrial representatives and regulators is crucial to seeing advances in science lead to advances in patient health.

So long as the interests of all parties remain aligned in providing the greatest patient benefit in combination with sustainable business, there is hope. Unlike our ignorance to seismic activity, we shall be keeping a keen eye on how the rest of this potentially ground-breaking chapter in biotech is written.

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David Brindley
David is an international thought-leader in the translation of life-science innovations into commercially viable products and services. His expertise spans the ‘Valley of Death,’ encompassing regulation, basic science, process engineering and finance. This distinctive skill set positions David at the forefront of socially responsible investments – in particular initiatives that make impactful contributions to global health. David currently holds a joint appointment between the University of Oxford and the Harvard Stem Cell Institute and is an active Fellow of the Royal Institution of Great Britain and the Royal Society for the Advancement of Arts and Manufacturing. In addition to being an Editorial Board member of a range of international academic and industrial journals, David is also a founder of Translation Ventures, a boutique consultancy that is actively engaged in maximizing the financial and societal value realized from cutting edge scientific innovations. Disclosure: David A Brindley has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in any postings apart from those disclosed. D.A.B. is subject to the CFA Institute’s Codes, Standards, and Guidelines, and as such, the author must stress that his contributions to this site are provided for academic interest only and must not be construed in any way as an investment recommendation.