Invest In Research, But Don’t Forget The Miracles

It’s time to talk cancer science and discovery.


I am currently in Chicago, attending the annual meeting of the American Society of Clinical Oncology (ASCO).  This is what most of us consider the premier international cancer meeting, and is attended by over 30,000 physicians and cancer scientists, hearing information on over 5000 studies, some of which inevitably will set the standards of care in cancer treatment for years to come.


As I write this, I am listening to a series of talks from leaders in cancer medicine.


One presentation that has just concluded was given by Dr. John Niederhuber, the director of the National Cancer Institute.


What he reminded us that despite our advances in cancer treatment, and while we are struggling to get sufficient funds for cancer research, we can never forget that miracles do occur.


Dr. Niederhuber reviewed many of the issues facing cancer research today, including the lack of new funding sources, and our need to extend our capabilities to provide state-of-the-art care for patients in every part of this country, no matter their economic circumstances, the color of their skin, or the language they speak.


He spoke of the advances that we are making in cancer research, especially our ability to analyze the genetic makeup of cancers, and the implications of that research in determining how each patient’s cancer can best be treated.


Dr. Niederhuber pointed out what many of us know: as we learn more about what makes a cancer cell a cancer cell, we continue to uncover more and more ways to interfere with that cancer cell.  We suspect that in the future each patient’s cancer will require a unique combination of specifically selected targeted therapies based on genetic analysis of that patient’s tumor.


But, as Dr. Niederhuber pointed out, who is going to pay for the development of all of these potential life-saving targeted agents?   As one gets into more detail of an individual’s cancer, we will inevitably face the fact that the “market” for a specific targeted therapy may be very small.


That means that for-profit companies won’t be willing to make the substantial investments (now well over $1 billion for each successful cancer drug) necessary to bring “small market” targeted therapies to clinical trials and cancer treatment. 


Dr. Niederhuber suggested that there may be a role for government in collaboration with pharmaceutical companies to address this problem, so that we can take advantage of the overwhelming amount of scientific information that becomes available daily.  He suggested that it may be the role of NCI in partnership with other entities to provide the financial resources that for-profit companies may not be willing to invest.


But we have heard much of this before from many sources.  What humanized and differentiated this discussion was a specific case that Dr. Niederhuber presented as he concluded his talk. 


His story reminded all of us that we can never give up hope, even in the most dire of circumstances. He took me back 36 years ago when I was a young, new clinical associate at NCI.  It made me recall why I went into clinical cancer research.  It refreshed the memory of the hopes that my colleagues and I had that we would be able to make the significant breakthroughs in cancer treatment that would eventually cure this horrible disease.


Dr. Niederhuber’s story was that of a young lady, a mother of two, who was in her late twenties and had an extensive, uncommon cancer called cutaneous T cell lymphoma.


She came to the Clinical Center at the National Cancer Institute’s main campus in Bethesda, Maryland near the end of her life.  Weak, unable to move, wrapped in blankets to preserve her body heat because her skin was so cracked it retain its own heat.


The young woman received several investigational therapies, but none were successful.  She was placed in hospice care.


Dr. Niederhuber then spoke about what anyone would call a miracle.


The NCI has a program to accelerate the clinical investigation of drugs that appear promising for the treatment of cancer.  They may be drugs that are “orphaned,” in the sense that they may only help a small number of patients with uncommon cancers.  Or, as was the case here, they may be drugs which had been tried unsuccessfully before, but for one reason or another have been “rediscovered” based on new research or new, valid theoretical considerations.


NCI had such a drug that had been previously tried and failed as a chemopreventive agent.  But a researcher made the case that this drug may be effective in T cell lymphomas.


The lady received the drug.  Her response was miraculous, and as Dr. Niederhuber reported, she is cancer free today, several years after she was the first human with cutaneous T cell lymphoma to receive the drug.


Those are the types of moments we lived for when we were young physicians in training at the NCI back in the early 1970’s.  And, those are the moments all of us—physicians, patients and families alike—live for today: the hope that even when there is no hope, there will be a miracle.


Miracles however are just that: miracles.  They are very rare, and don’t happen often.  As an oncologist, I can share with you that these moments are very, very uncommon.


I imagine the real message here is that we must always continue to have hope.  But we also need to work to make today’s miracles tomorrow’s realities.


The federal government, through NCI, is by far the largest supporter of cancer research in this country.  But for the past several years, the funding for NCI has been flat.


In what economists call “real dollars”, based on the impact of inflation and the inexorable rise in basic costs, that means there has actually been a decrease in cancer research funding.


With all of the truly exciting developments in cancer research, and the reality that our investments of the past four decades are leading to incredible and previously undreamed-of results, this is clearly not the time to stop funding this effort.


We talk of miracles, but we must always be investing in and building the infrastructure.  We must never stop providing the resources for our scientists—especially the new investigators with bright, bold new ideas—to continue their research.


The research infrastructure story is clearly not as heart-warming as a good miracle, but our success in treating cancer is going to be more based on incremental step-wise advances rather than sudden miracles.  It is slow, plodding work that will produce the miracles of tomorrow.  If we fail to invest today, we will not have successes in the future.


As Dr. Niederhuber pointed out, we need to take our discoveries from the bench to the bedside, and then throughout the country and the world.  We must find the ways to get our discoveries and our miracles into every corner of this country, for every person with cancer who can benefit from them.


To me, that is going to be the true miracle.  And, as Dr. Niederhuber’s story reminds us, we must never give up hope that this miracle will one day come true.

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