Medicine's Holy Grail

 

A few weeks ago, CBS Sunday Morning featured a segment about Alex's Lemonade Stand and advances in treatments for childhood cancer.  It is a truly moving human-interest piece that touches also on the Holy Grail of medical research.  The cure for cancer.  You may be wondering, “what’s the cure for cancer got to do with personalized medicine?”  This is where I tell you – it’s got EVERYTHING to do with it.

For all of the advances in medicine over the past century, one of the biggest mysteries is still cancer.  Medical science is only just beginning to understand and explain why cancer happens, and even then, it’s not the same for every person or every form of cancer.  Going back to the segment on CBS Sunday Morning, one of the things discussed is the discovery that certain types of cancer seem to be linked to a mutation in a gene called anaplastic lymphoma kinase (ALK).  More and more cancer diagnoses are starting to sound like alphabet soup: HER2 positive or negative breast cancer; BRAF mutation in melanoma; chronic myelogenous leukemia (CML) caused by BCR-ABL; colon cancer with or without K-ras mutation.  It can be confusing and it is definitely overwhelming, but here’s what you need to know:  THIS IS GREAT.  Why, you ask? 

Here’s why…One of the biggest shifts in recent years is how cancers are treated and managed.  Therapies and treatments have moved from a generalized “attack every fast growing cell” to a targeted approach.  Having this genetic level of specificity allows modern science to get at the root of what’s causing the cancer cells to grow and thrive.  For example, the ALK mutation that drives certain types of lymphoma seems to be turned off by the drug crizotinib.  In some cases, the treatment has been so successful, that the cancer is quite literally GONE.  Finis. Cured.  Those are some pretty amazing results.  We can get those results because the therapy is acting at the source in a highly specific manner.  Another positive outcome to therapies working at the source is that the side effects are far less than what you see with traditional chemotherapy.  Newer therapies have mild to moderate side effect profiles allowing patients to continue with daily life versus their older chemotherapy counterparts.  Sadly, those cases where cancer disappears completely are still the exception.  Many treatments still fail and cancer progresses.  Treatments that work amazingly well in some patients don’t work at all in others, and scientists don’t always know why.  More research and understanding will be necessary before that miracle cure is found…but there’s promise.

The drug development pipeline is rich with products that are highly specific to a disease type.  Therapies for diseases you’ve likely heard of: fibromyalgia, lupus, multiple sclerosis; and diseases you’ve probably never heard of: Fabry Disease, hereditary angioedema, alpha-1 antitrypsin deficiency.  One of the very exciting things about the drug development pipeline is that ~40% of it is cancer therapies.  Even more exciting is that many of these therapies are oral, not infused.  Unfortunately, the downside to these highly-specific therapies means that they treat only small numbers of patients comparatively.  It doesn’t sound so bad until I tell you that the price goes up when the number of patients treated goes down.  Many of these therapies are at or above $100,000 per year.

In subsequent posts we can dig into the specifics of some of these new therapies.  A lot of these new products entering the market come with companion testing that is required in order to qualify for treatment.  Personalized medicine is here to stay.  We’ll help you become educated.  Ask us your questions, tell us your thoughts.

-A