The blog post title above is also the title of a report by Sigma-Aldrich (published with partners AAAS and Science). The report is based on 608 survey respondents, 45% of which were in the US; 23% of respondents were graduate students. It’s not so long, as reports go, and you might easily read it on the bus or while eating lunch. I’ll give you a few nuggets from the report here to whet your appetite.

When asked about their barriers to progressing their research, 62% of respondents named insufficient funding, with inappropriate duration of grants and insufficient scope of grants coming next, both with 18%. Twenty-two percent of responders said their funding for translational research decreased over the past 12 months (17% said it increased). Also, the majority of respondents (62%) said that collaboration with their business school would benefit their research groups, but only 13% are actually collaborating.

That last bit of information Pat Sullivan found shocking. He’s the former liaison for academic partnerships at Pfizer and now works for Sigma-Aldrich. He thinks there is an opportunity for pharma to help educate universities on how to best fill the voids in research left by pharma cutbacks.

I asked him for further thoughts on the report, and he supplied this:

Through aggressive downsizing, Pharma has reduced its capacity to identify new commercially viable therapeutic disease targets and new drug candidates. These reduced capabilities have driven Large Pharma to seek out new partnerships with academic researchers working in the translational research fields. However, in order to take full advantage of this new opportunity, academic researchers and institutions will need to conduct their research programs in a new way. In fact, they will have to model and conduct their translational research programs after the processes and workflows currently being used in Large Pharma. This will require the translational researcher to work on interdisciplinary scientific teams and with non-scientific departments like their Business and Law schools. Institutions that set up their programs to promote collaborations across departments will learn how to leverage their scientific discoveries into commercial opportunities. They will also learn how to choose external partners to help them to generate quality data and obtain key reagents needed to attract Pharma collaborations and funding. Institutions and Funding agencies will also have to support Principal Investigators seeking to work in the translational research field by recognizing contributions beyond just publishing.  

Eventually, institutions that conduct translational research will learn to focus their programs on the drug development process that will be the most efficient for them to execute and add the most value to Large Pharma. In most cases, they will focus their programs on the early drug development phases involving new disease target validation and lead chemistry discovery. If the translational research program focuses on an orphan disease, it will be required to work with several external partners to conduct preclinical and clinical activities and obtain manufacturing capabilities.  

Thoughts? You can access the report at the link below.

Enjoy your lunch,

Brady Huggett

Translational Research report

The author, at the Great Wall of China.

On the 8^th of December 2011, I was invited to represent Kuwait at the Human Variome Project (HVP) meeting, being held for the first time in Beijing, China. The meeting was a result of many months of negotiation and the finalization of a historic partnership agreement between China and the Project.

HVP is an international consortium of researchers and clinicians, and later UNESCO representatives from more than 30 countries.  It formally inaugurated in 2006 in Melbourne, Australia, with the major goal of documenting (and placing in a database) all polymorphisms and mutations in human sequences, as well as associate these with human health and diseases. This ensures that global information on genetic variation is collected, curated, interpreted and shared freely and openly.

I want to stress the words “openly” and “freely,” because for a scientist this is the best way to share data. For an entrepreneur, though, this is a waste of effort, growth, and potential income, if not secured first by patenting.

Let me explain the problem. There is no doubt that the HVP will uncover thousands of DNA changes associated with disease predisposition and outcome, and they will be useful for disease diagnosis, prognosis and theranostics.  For example, yesterday I found a base substitution in the MLH1 gene of an Arab patient with hereditary non-polyposis colorectal cancer (HNPCC). The genetic change was non-synonymous (meaning it changed the amino acid composition of the MLH1 protein from proline to serine). The DNA change may have altered the protein function, predisposing the individual to cancer. However, one really cannot tell for sure because it can be a polymorphism found in the normal population.  One of the major aims of the HVP is to stratify these DNA changes into polymorphisms or disease causing mutations.

This is achieved for HNPCC by the Insight group, one of many databases curated for the HVP.  Now that federal appeals court ruled that genes (or more accurately, DNA sequences) can be patented, overturning a lower court decision by Judge Sweet, biotechnology companies are rushing to gain from genome wide association (GWAS) and linkage studies.

On the 18^th of January 2012, we learned that Myriad Genetics has acquired exclusive license to intellectual property covering the analysis of the RAD51C gene for risk of hereditary breast and ovarian cancer. The six heterozygous mutations found in the RAD51C gene by a German group confer increased susceptibility to breast and ovarian cancers (for more info, click here).

My dilemma, and the reason behind my post, is to ask how will the patenting industry cope with thousands or more disease-associated DNA sequences coming out from the HVP? Moreover, after further validation, do I submit my unique sequence I found in the MLH1 gene to the Insight database, where it may help clinicians and patients, or do I patent it first?

By the way, I know that I and my colleagues in the Middle East and beyond have hundreds of these risk-associated sequences. We do want to do something with them that reflects the true human spirit!

Fahd Al-Mulla

I always say to my colleagues “Success has no formula, but failure does.” Often, biotech spin-offs yield more in experience than they do history. But I would like to share the story of a colleague of mine who went from a humble academic job in the microbiology department in a national university to a clinical success story. His lab in Tokyo is now providing cell-based immunotherapy via 6,000 transfusions a year, from all over Japan and from neighboring countries.

Hiroshi Terunuma started his career as a medical microbiologist in the Yamanashi University School of Medicine in the late ’90s, when he worked on HIV carriers in whom RNA transcriptome analysis was essential to understand the nature of the immune system. He had no other way to study the RNA transcriptome than to try a reproducible expansion of human immune cells, including T cells and NK cells, in the lab. He did years of basic work without even glimpsing the light at the end of the tunnel. But then he had a two-year tenure in Zambia for studying the immune systems of HIV patients, and even though he came back to Japan with additional expertise, still he and his colleagues had to burn the midnight oil to achieve a successful lab-expansion of the human immune cells. Finally, at the end of a decade of hard work, he got a breakthrough and patented it, though he found his invention was going to be more useful in treating cancer patients!

By then, the Lymphokine Activated Killer cells and Dentritic Cell based treatments had started as clinical applications in Japan, and his invention of being able to grow the Natural Killer Cells without feeder layers meant he had become an immunotherapist for cancer patients. Around this time, he was introduced to Tsutomu Kaneko, whose business expertise combined with the clinical and research expertise of Terunuma to produce a win-win for all involved. With this type of customized and autologous cell-based procedures being considered as clinical procedures as per the Japanese law, the two men produced the Autologous Immune Enhancement Therapy (AIET) for cancer using the patient’s own Natural Killer cells and with other immune cells such as T cells. Today, AIET has treated more than 10,000 patients and several Asian countries have started this treatment as part of the Terunuma team’s outreach via technology transfer. The patents are swelling; the company he started with Kaneko is debt free and earning a profit. Above all, though, the product is a worthy contribution to cancer patients.

I would summarize by saying the key elements for success were:

• • The years of hard work in the bench to find the appropriate culture methodologies.
• • Repositioning the invention to an application that was essential and in demand
• • The availability of clinical data on autologous immune cell therapies for cancer
• • A synergy-based partnership helped the scientists and clinicians contribute in areas alien to them.
• • The “clinical procedure” recognition of the autologous immune cell treatment in Japan

Samuel JK Abraham