Last year, Dr. James Watson, the noble prize winner, part of a team responsible for the discovery of the structure of DNA declared that beating cancer was possible by 2020. His argument in the NY Times was that if we really focused money into basic science cancer research rather than clinical cancer treatment centers, this recommendation turned out to be politically unacceptable and he was kicked out of the cancer advisory center.

What seems to really connect with Dr. Watson's statement is that our most cutting edge cancer therapeutics are often based on ancient hypothesises or assumptions.

Angiogenesis

Take Angiogenesis as an example(the need for tumors to create their own blood supply to sustain themselves), this mechanism of action was proposed by Dr. Judah Folkman back in the early 70's and after much resistance in the scientific community accepted decades later. As a result of Dr. Folkman's work and the the development of drugs that choked off the creation of new blood supplies to cancer cells our latest generation of Cancer therapeutics have emerged (Erbitux, Herceptin, Velcade and Tarceva). One seemingly important point here should be the glacial pace of Cancer development over the last couple of decades and our need to innovate out of this deadly lethargic pace of innovation.

Understanding how to differentiate between cancers

The good news is that biotech's and pharma companies are heavily investing in new experimental therapies (arguably the number 1 target for therapeutic development) through the use of newer genomic technologies and an increased understanding of how to really separate out different variations of cancer. Herceptin (Trastuzumab) only works well for certain types of breast cancer, typically on those breast cancers that over express HER2 but when it's appropriately used it can have a substantial effect, further reducing relapse risk in some women by 50%.

Future Cancer treatments

Increasingly, there's a consensus that not only do cancers have to be specifically targeted based on their specific genomic mutations (which can be a daunting task based on the latest findings of 23,000 mutations in a sequenced lung cancer cell)  but it seems likely that cancers will also have to be treated with a combination of therapeutics to reduce the risk of resistance developing (a similar concept to treating bacterial infections). As a result many current studies are ongoing to identify effective combination therapies and over time will likely be a combination of better and better biologic therapies (protein, RNAi and cellular) as well as through the use of targeted radiotherapy and surgery. 

One of the biggest challenges that all of us have within the Garage Biotech space is financing a start up venture, we face somewhat similar challenges to entrepreneurs in the tech space but bootstrapped Biotech start ups have the additional problem of requiring potentially more capital intensive equipment and usually working with more “buggy” biology (cells, DNA/RNA and molecules are very sensitive to environmental changes and hence even minor changes can ruin weeks/months worth of work).

One key entrepreneur and venture investor that I’ve followed over the last decade or so has been Guy Kawasaki, if for no other reason that he provides salient words of wisdom but in a very entertaining format to budding entrepreneurs and has been doing since the early days of Apple computers.

In one of his articles, Guy talked through some of the finer points of “The Art of the Start” and I highly recommend reading his original article. I’ve simmered down some of his key points and connected it to some of the lessons I’ve learned and observed in the biotech industry.

1. Early Cash flow is king – Not Profits! : This is a tough one for us in the land of biotech start ups as it’s often touted that it takes 7-12 years to develop a therapeutic and anywhere between $800M-$1BN USD to get a therapeutic drug approved by the FDA. Statistically, this may be true (and I wouldn’t want to doubt Tuft University’s numbers in aggregate) but the reality of it is that a really solid platform technology with proof of concept data (even in vitro) could be enough to raise, angel, venture or even grant funding and I have known of Biotech entrepreneurs in the past that have been able to raise substantial sums (in the millions) of even non-diluting grant funding from the government and non-profits. Other interesting models are starting a service business (even if only temporarily) or in the medical device/tools space it may be possible to start selling/partnering your technologies sooner to jump start a positive cash flow.
2. Bottom up forecasting is the way to go!: market segmentation is a class that’s taught in business schools globally and can also lead to some pretty confused thinking for first time entrepreneurs if you listen too much to your B-School Prof theories on assessing essentially established markets and apply that rational to tiny or non-existent  but rapidly developing markets. It doesn’t really matter if the market for your particular drug/device or service is in the billion dollar range, your market size might be limited to the 10 researchers that would be willing to try out your device in the first year in their lab or how much you can get for your first licensing deal in year 1. My advice is to see past the excel worksheet and really think about how you personally will sell your biotech product!
3. Truly understand that you’re building a scrappy start up (under staff and chose your battles): this is perhaps my favourite of Guy’s points and links in with cash being king, for a bootstrapped start up you have a precarious lack of both time and money, this means on the one hand you should buy as much off the shelf (rather than bespoke) equipment as possible but on the other hand realize that you will likely have to under staff your start up initially to gauge the true level of resource need but be careful not to fall foul of any existing rules or regulations in our highly regulated industry (OSHA being just one example)!

I’ve highlighted a few points that I felt are particularly important to bear in mind as you start up your projects/garage biotech. The catch in our industry of course is that there is a vast difference in how you’d structure your project/biotech dependent on your individual focus, be it tools, devices, diagnostics or therapeutics.

I’m always interested in finding out more about how innovative Garage biotech’s have surmounted these challenges so please feel free to get in touch or leave a comment.

The first step with tracking progress with garage biotech projects is to ensure that there is an accurate record of all new work that is performed. It's not quite as fun as jumping straight with a new bit of lab equipment but is vital to ensure you get the most of your project. Anyone who has spent any considerable amount of time in the lab knows that there is a substantial amount of trial and error involved in any experiment and the important thing to ensure is that all of the individual adjustments to particular procedure is tracked for repeatability later.

Most lab scientist currently still use notebooks, strategically placed by their lab benches to track their progress but you could argue the need to manually insert each procedure would be too time consuming for a part time scientist. One method of stream lining this necessary process of tracking experimental progress by garage biotech enthusiasts is through the use of  webcam videos that can (if needed) be saved and then later modified to ensure that your work can be easily reviewed at a later date and time and also potentially be easily distributed. An excellent illustration of this technique was the Video Diaries created by Will Smith’s fictional character in the movie “I am legend” which showed the power and simplicity of tracking experiments in a video format (these webcam software programs are already widely commercially available).

I’ve included one example of a video that shows and explains how to extract DNA, pretty neat!

Many of us in the biotech world know that the idea of a lone biotech entrepreneur creating a successful biotech company in his garage is a laughable idea to most of the biotech/pharma industry but it is none the less a very alluring idea to those of us with an entrepreneurial affinity. Recently, I've come across a string of scientists who have and run their own labs in their garages (many former academics and commercial scientists) and it made me revisit the idea of Garage Biotech.

It's very difficult to get any sort of statistics on the number of Garage Biotech's being started or run currently by scientists or small entrepreneurs as many are hesitant to disclose their projects until they're close to completion (not unlike the Garage entrepreneurs of Silicon Valley) but anecdotally there seems to have been a rise at least in regards to resources available to this types of biotech entrepreneurs with dramatically dropping assays, used lab equipment sold on ebay and increasingly sophisticated open source bioinformatics tools.

There have also already been signs of success for some small biotech/bioinformatics start ups:

Eric Engelhard - is bioengineering a honeybee in his garage and hoping to use this technology to develop non-venom producing honey bees.

James R. Graham - was inspired while making electronic music on a half-dozen computers in his bedroom and used the algorithms that he developed to create Diction, a tool that converts the genome into a series of waveforms and locates crucial regions that are actively producing proteins utilizing the human genome which is publically available at the NIH website.

Agribiotics - an agricultural biotech start up was aquired by Nitragin for $24M and was developed by a garage biotech entrepreneur.

Spaltudaq - Johnny Stine a lone entrepreneur in Seattle and a former scientist at Icos founded a small therapeutic biotech called Spaltudaq which has been successful in setting up three partnerships to make rabbit-based antibody drugs for biotech and pharma companies. These partnerships could generate as much as $200 million, if he manages hit all of his targeted milestones (which as we know in the biotech world can be difficult).

So it seem that even though hard statistics may be hard to find in this growing niche, there's definitely growing promise!

During the last couple of years there has been a silent revolution in the understanding and use of "off label" cognition enhancing prescription drugs, it began with students competing to improve their test scores and has expanded out to busy professors and executives in search of that professional edge. To some it's an extremely worrying trend and to others it's mearly a natural progression of medical technology...

Cosmetic Neurology?

Last year Nature, the scientific journal published the results of an informal online poll asking whether readers attempted to sharpen "their focus, concentration, or memory" by taking cognition enhancing drugs. One in five respondents said they did. The majority of the 1,400 readers who responded said that healthy adults should be permitted to take brain boosters for non-medical reasons which at the time was an incredibly brave finding for Nature to publish due to the resistance in mainstream society against prescription drug use in healthy people.

The discussion within the Nature article focused on the two main cognition enhancing drugs that are currently being used "off label" by some individuals in the hope of enhancing their performance and they are likely very familiar therapeutics to the majority of you, as they are used for the treatment of ADHD and Insomnia:

• Adderall
• Provigil

At the moment those embracing Nootropics, i.e. new chemicals or therapies to enhance their own cognition, are beginning to develop an interest in many of the experimental therapies that are being developed to treat cognitive decline in Alzheimer's and Dementia which include Ampakines and Cholinesterase inhibitors that have shown some promise within the clinic. We can all connect at least partially with the idea of striving to be better, faster, stronger and the idea of enhancing our own cognitive abilities can be seductive. However, it’s important to remember that our brains are finely balanced biochemical machines and by altering the concentration of certain chemicals and neurotransmitters within a healthy mind we run the risk of unexpected consequences, not least of which could result in classical mental illnesses or new dysfunctions.

For those of you on the bleeding edge of this fascinating Nootropic trend, I wish you the best of luck! Please make sure to tread carefully and let us know your thoughts in the comments section, as Forest Gump once said “Life is like a box of chocolates, you never know what you’re going to get!” Corny, I know!