ASK Karl

Question: What’s new in cancer research? 

Answer: What's new: a way of making treatment research more effective! A proposed tissue bank will allow sharing of material from biopsies and associated data, for the first time giving scientists access to tissue samples and associated clinical and research data. It is called the National Biospecimen Network – NBN.  

Here’s an example that I hope will explain the urgent need for NBN: 

Suppose you are comparing how well different laundry detergents clean stained clothing. In order to compare the results you must account for the type of cloth and the stain. If you use a new detergent on the entire batch (the sample), maybe it works better than the rest on 20% of the items. ...

... But if you don't know what type of cloth and stain it cleaned best you haven't learned anything useful.  And when it's time to clean your next shirt you're back to trial and error, and the chance you'll get it wrong 80% of time. 

I think the above describes the problem with clinical cancer research today pretty well. But when you get it wrong on patients – test a new treatment that does not help many – the costs are significant: 

  • the majority of patients suffer toxicity for no benefit; the expensive failure gives pause for drug sponsors to try again; 

  • a potentially useful new drug (for some patients) is not approved; 

  • the wariness of patients to participate in this kind of trial-and-error research increases costs and delays evaluations.... the list goes on. 

Here's where NBN comes in. To know what the differences are in the tumors (the stains) and the patients (the cloths), you have to first collect the tumor, blood, urine and other biospecimens in a very controlled way - and at each participating center. 

Biospecimens are fragile and can change based on how long you take to remove them, the temperature at which they’re stored, what methods you use to freeze them, etc. 

Once you have the tissue, you can use standardized advanced tests to discover what the defects are in the cells - how they are different from normal cells and other tumor cells of the same cell type. This is called molecular profiling

The goal of profiling is to characterize the patient and the tumor - to identify the unique aspects of the tumor and the patient that relate (correlate) to response to treatment with different agents, or how these markers predict prognosis (the way your cancer is likely to affect your survival). This is what is sometimes called discovery and validation of biomarkers

NBN will not only bank biospecimens. It will also store clinical data associated with the tissue (the patient’s diagnosis, health status, and treatment history) but with identifying information stripped from the records and replaced with a unique code in order to protect the privacy of the participants. 

NBN will provide open access to the tissue and clinical data, as well as the research findings that are done using the tissue. Access to tissue will be based on the merits of the science, but all scientists can “mine” the findings to discover important patterns (correlations) between clinical outcomes with patient and tumor characteristics, dramatically accelerating productive research. 

Notably, NBN will require researches to publish all research finding based on NBN-acquired tissue (including failures) so that resources can be applied to the most promising directions and not wasted on less promising research paths. 

Importantly, NBN will make results of different studies comparable and statistically powered*, and help identify sub-populations that can benefit from targeted drugs, making personalized medicine possible. 

*Statistically powered findings require large numbers of tissue to ensure that the findings are not due to chance. For example, you may need 1,000 tissue samples of a certain type of cancer to see that an association of a marker to an outcome is not due to chance - just as you might need to flip a coin 100 times to see the true outcome odds are 50:50. Only ten coin flips can mislead.

Standards is a mundane word, but it's key to progress and the cornerstone of NBN. Medical research is severely restrained today despite having lots of tissue samples, because we do not yet have standards for collecting, assessing, or describing it. It’s is a tower of Babel. 

For example, if I measure your height with my hand, you can’t compare it with your height measured by other hands. Consider how important standards are for more complex activities such as commercial passenger jet travel. If each airport and pilot used different signals and procedures we would not often arrive safely to our destinations! 

NBN will provide the foundation for accelerated clinical research, built on common language for describing the tissue and associated data (informatics software), and having sufficient numbers of comparable tissue samples stored in standardized biorepositories (or biobanks). 

Is there a need for greater efficiency in clinical research on cancer? 

Yes, and the need is urgent! I believe that the NBN infrastructure is as important to driving cancer research as our electric power system is to our economy. Consider how backward our economy would be if each company had to produce it’s own power? 

Despite the promise of innovation based on new insights and technologies, "the number of new drug and biological applications submitted to FDA has declined significantly"1, and only one in ten new cancer drugs win marketing approval. 

Unfortunately, one in three women will get cancer in their lifetime, and one in two men.2 Therefore we should all be concerned about the evidence of stagnation, especially given the unprecedented opportunity to innovate and make progress against cancers. 

1. FDA – The "Innovation/Stagnation: Challenge and Opportunity on the Critical Path to New Medical Products." - 

2. SEER Cancer Incidence Statistics 2002

Finally, the success of NBN will depend on the participation of patients – a factor that will depend on public understanding of it’s importance, and will also depend on trust in the NBN system - which will provide stewardship over our tissue and clinical records. Note that tissue contains DNA, which is the recipe for cell behavior, but also for who we are as individuals, families, and sometimes ethnic groups. 

Given the nature of the information, strong consent procedures and privacy safeguards will be required, as well as informed patient representation and oversight within NBN. And given the concerns, and in the interest of establishing public trust and funding, I am very pleased to report that patient perspectives are being sought early in the design phase of NBN.

~ Karl Schwartz, BA, MFA
President, Patients Against Lymphoma

Caregiver and Patient Advocate
Patient Consultant to the FDA/Oncologic Drug Advisory Committee
Participant in the NCI Progress Review Group for Blood Cancers

Lymphoma Foundation of America
Main Office
1100 N. Main Street
Ann Arbor, MI 48104
Tel: (734) 222-1100 | Fax: (734) 222-0044

Patient Hotline: 1-800-385-1060

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