“Only four (4) U.S. Food & Drug Administration-approved therapies in the last 30 years”

That’s quote from the National Brain Tumor Society website posting regarding “Clinical Trial Endpoints”: http://braintumor.org/advance-research/integrated-initiatives/clinical-trial-endpoints/

 

Does four (4) seem low to you? It seems low to me.

The NBTS posting puts that in perspective: “With nearly 700,000 Americans living with a brain tumor, and only four (4) U.S. Food & Drug Administration (FDA)-approved therapies in the last 30 years, and no cure – the needs of the brain tumor community are clear. Brain tumor patients need new therapies that will either eradicate their brain tumor or better manage the tumor and its manifestations; as many brain tumor patients also experience adverse changes in their physical, cognitive, and psycho-social well-being which significantly impacts their ability to maintain ‘normal’ lives while receiving treatment.”

A couple of numbers popped out of this paragraph and uncomfortably whacked my eyeballs: “four (4)”, “30 years” and “700,000”.

We continue to spend significant $ (and $$$) on research and have only got four (4) new approved therapies in the last 30 years? What’s going on?

While I’m not smart enough or insightful enough to diagnosis this issue, I think part of it traces to Clifton Leaf’s observation that “the public’s immense investment in research has been badly misspent”. He notes that scientists seldom collaborate and share their data, why new drugs are so, so expensive and why young scientists are “…now abandoning the search for a cure.”

If you want to know more I highly recommend that you read his well-researched and documented book, “The Truth in Small Doses”: http://www.amazon.com/The-Truth-Small-Doses-Cancer/dp/1476739994

At the same time, I am more than glad that the NBTS is attempting to bring all the disparate parties together to, well, work together.

It’s about time.

John

PS - Sorry, I just don't have the energy today to write the right amount of outrage or incredulity into this post. Feel free to add your own.

Precision Medicine Conference (Highlights)

I just read the “Precision Medicine Conference” highlights posted on the National Brain Tumor Society website: http://blog.braintumor.org/precision-medicine-conference-highlights/  and found myself hopeful, if confused.

I’m hopeful because the highlights sound surprisingly upbeat, positive and, well, optimistic. Why? Because (as best I can tell) we are starting to really leverage our understanding of the human genome as it relates to more effectively treating brain tumors.

Dr. Jennifer Helfer, PhD, explains this much more clearly in her conference recap by stating that  “Precision medicine, also referred to as personalized or individualized medicine, is defined on Wikipedia as ‘a medical model that proposes the customization of healthcare—with medical decisions, practices, and/or products being tailored to the individual patient.’"

Part of this introductory statement is still confusing to me – why is the idea of “medical decisions, practices and/or products being tailored to the individual patient” a radical/newsworthy idea? Haven’t we always been doing that? If not, why not?

As best I can tell, some of this stems from the importance of an individual  victim's genetics in addition to the tumor's classification, e.g. glioblastoma. Being able to research this linkage, according to Dr. Jeffrey Flier, Dean of the Harvard Medical School, is being led - somewhat strangely - by patients  who are “collecting and sharing their own medical information via sites like 23andMe" - https://www.23andme.com/

This information appears to give medical professionals better direction on how to treat patients’ individual and unique brain tumors. The challenge, it appears, is that more doctors need to become more familiar with this information in order to administer and interpret this genetic information.

If you do nothing else, watch this video entitled “Discovering the PD-1 Checkpoint: Winners of the 2014 William B. Coley Award for Tumor Immunology” - https://www.youtube.com/watch?v=B532URzuJOU