Why David Hates Health Insurance Companies

A Rant

Photo Credit

I am involved in a case right now that epitomizes all that is wrong with commercial health insurance.  All in one case.  How convenient.

My patient is a young adult with a sarcoma diagnosed in her liver.  There is one large mass and several smaller ones.  The tumor is not resectable right now, so she will need chemotherapy.

Two issues have arisen this week:  one related to diagnostic imaging (radiology) and the other related to quality of life.  With both issues I have faced significant roadblocks, placed by the patient's insurance company, that impede my ability to provide the care this young woman needs.

I'll start with the imaging issue.  The patient's sarcoma is not one that typically arises in the liver.  Also, the presence of multiple masses is more consistent with spread TO the liver, rather than the tumor arising FROM the liver.  That means, if we hope to cure this young woman, we need to find the primary tumor.  A CT scan of her chest, abdomen, and pelvis showed nothing.  An MRI of her pelvis, to better evaluate her uterus, a place that this tumor could arise, showed nothing.  Because a tumor like she has can come from anywhere in the body, I ordered a PET scan.  The insurance company denied coverage.

Why?

Because, the physician reviewer told me, there is no evidence that a PET scan is useful in this disease.

Of course, the physician reviewer is not an oncologist, and therefore not a sarcoma specialist, so I'm sure he does not keep up with the latest literature about PET scans and sarcomas.  But I do.  A quick search of PubMed using the terms "sarcoma" and "PET" revealed 471 articles.  I faxed him 4 of them yesterday.  I hope that is sufficient evidence to allow me to get the test I, the treating physician, believe my patient needs in order to maximize my chance of curing her.  I'll find out later today or tomorrow.

The other roadblock involves her quality of life.  My patient will need a chemotherapy drug called ifosfamide to treat her tumor.  This drug has a significant risk of infertility associated with it.  After consultation with a reproductive endocrinologist, we decided that the best was to try to protect her fertility would be to use a drug called Lupron.  Unfortunately, Lupron is expensive, so it requires prior authorization from the insurance company.  I just received an email from our clinic coordinator that read, in part, "It won’t be covered if it’s for fertility reason (per her case manager)."

So... I have some choices to make.  Do I lie and say the drug is being prescribed for another indication?  Do I tell the truth and risk the family having to pay $750 per dose out of their own pockets?  Or do I choose a different drug, one that will not work as well, and know that I am not providing optimal care for this young woman, and am increasing her risk of infertility?

All because her health insurance company wants to save a few bucks.  At least they are "not for profit."  Imagine the difficulties we face when the insurance company is trying to provide dividends for their investors, instead of health care for their customers.

Related Links:
Not Medically Necessary
Cancer Treatment and Fertility, Part 2:  What Can Be Done?
Cancer and Fertility:  How Can Treatment Impact Fertility? (Part 1)

It's Wednesday... The Doctor is Playing Golf

What a horrid, 50's-era cliche!

But today, it was true. This afternoon I played golf in a fundraising tournament for the Heather Brooke Foundation. This is a foundation named in honor of a patient with Ewing's Sarcoma that I once helped care for. When Heather passed away, her mother channeled her grief into helping others. The Heather Brooke Foundation exists to help conquer childhood cancer and to help and educate the families of children with debilitating illnesses.

Today was a beautiful day for golf... if you like playing in gale force winds! Of course, if you're as terrible as I am (other than Putt Putt, today is the 3rd time I've played golf in my entire life), the wind really doesn't matter so much.

But for my buddy Dean, who really plays quite well, the wind was a problem.


We played at The Timbers at Troy, a beautiful local course. It was a fabulous day, and everyone seemed to have a great time.

Last year's tournament raised enough money to buy a PCR machine for my lab. With the help of this machine, we generated the preliminary data that convinced the National Comprehensive Cancer Network to fund our clinical trial for patients with recurrent and refractory sarcomas. You should have heard the applause when I made that announcement at the post-tournament lunch!

Yes, it was a beautiful golfing day.

Related Posts:
When Translational Research Really Translates
Johns Hopkins Team Sarcoma 2008

When Translational Research Really Translates

All of us who work in a lab and see patients say the same thing: “I do this because I hope one day to be able to discover something in the lab that will really help patients.” This is the essence of what is called “Translational Research.”

Our laboratory studies cancer stem cells. I have blogged about these cells before. Cancer stem cells are thought to be a small population of cells within a tumor that are resistant to chemotherapy and are capable of regenerating a new tumor. Thus, these cells are thought to be responsible for local relapses and for metastatic disease. Because they are resistant to chemotherapy, our usual treatments don’t get rid of them, so finding ways to kill these cells is critical to the further advancement of cancer treatment.

There are many theories to explain the resistance of cancer stem cells to chemotherapy. One of these is that there are important metabolic differences between cancer stem cells and most other cancer cells. One such difference might involve a signaling pathway called mTOR. mTOR stands for “mammalian target of rapamycin.” Rapamycin is a drug that is used primarily to prevent the rejection of transplanted organs. It turns out that rapamycin works by interfering with the function of a specific enzyme that was given the name mTOR.

For a variety of reasons, inhibiting mTOR activity has been predicted to make cells more sensitive to chemotherapy. In collaboration with another researcher at Johns Hopkins, Jonathan Powell, our laboratory has done some experiments that seem to show that inhibiting mTOR increases the sensitivity of cancer stem cells to chemotherapy.

Last winter, we responded to a call from the National Comprehensive Cancer Network for research proposals utilizing an mTOR inhibitor for the treatment of cancer. Based on our laboratory data, we proposed a clinical trial that would treat patients with a combination of a chemotherapy drug (liposomal doxorubicin, or Doxil) and an mTOR inhibitor (in this case temsirolimus, or Torisel).

In March we learned that our proposal would be funded.

On Monday, I signed the contract that will allow our clinical trial, now approved by the Institutional Review Board, the Food and Drug Administration, and the Johns Hopkins Clinical Research Committee, to begin to enroll patients. We hope to treat our first patients in late August.

I can’t wait to see whether we are able to help the patients willing to enroll in this trial! How gratifying it would be to know that work in our lab led to a new way to treat cancer patients. That, after all, is why we do this.

Related Posts:

The Stem Cell Thing

Stem Cells, or "A Rose By Any Other Name..."

Cancer Stem Cells and Familial Cancer Risk for Breast Cancer

Team Sarcoma 2009

This weekend is the conclusion of International Sarcoma Awareness Week. We at Hopkins kicked off the week with our 2nd annual All Wheels Welcome ride down the Baltimore and Annapolis Trail. It was a marvelous event, with over 150 participants of all ages – including current and former patients, as well as their friends, families, and supporters. I think we were all amazed by Team Luca, the family and friends of a current patient of mine, who all showed up in bright red shirts and were an inspiring presence.

I want to extend a personal Thank You to everyone who helped organize the event and to everyone who participated. We raised almost $13,000 in just one day. This year’s event was larger than last year, and we hope next year will be even bigger!



The highlight of the week every year is the Team Sarcoma Core Bike Ride. This year, my home state of Maryland had the honor of hosting the Core Team, who rode from Cumberland to Washington, DC, spreading awareness and forming bonds of friendship.

I had the honor of meeting the group on Wednesday night for dinner in Shepherdstown, WV. After a fabulous meal, I spoke briefly about some of the work we have done in the lab that has led to a new clinical trial that was just approved this week by our IRB. We expect to be open for enrollment in mid-August!




After my presentation, the true fun began, as I was able to speak with the dedicated men and women who make up the Core Team. It was truly an honor, and a whole lot of fun!

The week culminated with a reception in Washington, DC, attended by representatives of several local medical centers, including our own, as well as dignitaries from each of the countries represented on the Core Team.

None of this would be possible without the leadership of Bruce and Bev Shriver. They are truly remarkable individuals, and all of us in the Sarcoma World owe them a great debt.

I don’t know about you, but I can’t wait for next year!

Related Posts:
Sarcoma Video
Johns Hopkins Team Sarcoma 2008
The Importance of Research Foundations

Another Trip, Another Conference

I recently had the pleasure of attending the 14th annual meeting of the Connective Tissue Oncology Society in London. This was the kind of medical conference I like the most. It was small, with ample opportunity to meet colleagues from around the world. More importantly, the presentations were first rate, and I learned a lot from my time there.



CTOS, as the group is called, has grown dramatically in the past few years, both in numbers and in the quality of the research. My first CTOS meeting, two years ago in Venice, was smaller and many of the papers presented were good… but not great. This year, there were 30% more attendees, and the quality of the work presented has improved dramatically as well.

CTOS is truly an international and interdisciplinary organization, embodying what I believe is truly necessary for the advancement of care for patients with sarcomas. The organization has members from Europe, Asia, Australia, North America, and the Middle East. Sitting in the same conference room listening to the same presentations were radiologists, surgeons, orthopedists, radiation oncologists, medical oncologists, pediatric oncologists, pathologists, nurses, and patient advocates. These interactions, across international boundries and across the boundries of medical disciplines, are vital for progress in caring for patients with rare diseases.

My favorite session captured that spirit perfectly. Entitled “Bone Sarcomas 2 - Surgery and Molecular Biology,” this session included talks on surgical techniques for rebuilding limbs after resection of large tumors as well as two talks on the application of cutting edge molecular biological techniques to understanding the biology of these tumors. I can honestly say I’ve never experienced anything quite like that.

All work and no play makes Jack a dull boy, so I managed to spend some time sightseeing. It was typical London weather – overcast, misty, and cool, but I enjoyed it anyway.

Next year? Miami!

Related Posts:

Back to the Future: Another Meeting in Denver

Osteosarcoma Symposium in Houston

Children's Oncology Group: A Meeting of the Minds in Denver, CO

Related Link

The Stomp Out Cancer Project

Stomp Out Cancer! What a great idea.

This is a terrific organization started by the friends and family of Steven Mackin, a young man who died of Ewing’s sarcoma. Ewing’s sarcoma is the second most common form of bone cancer, mostly striking children and young adults. This will be the second year that a group of musicians is putting together a compilation CD of indie music to raise funds for Ewing’s sarcoma research.

Last year I posted some “guest doctor” posts on the Stomp Out Cancer blog. This year, I am their official Medical Advisor. It’s been an honor to be associated with this group, and I am very excited about this ongoing project! Please take a look at our video announcement above and spread the word.

If you are in a band, or if you know someone who is in a band, and you want to submit music for consideration for inclusion on Stomp Out Cancer’s second CD, follow this link.

What’s New in Cancer Research?

Why do there seem to be so many

cows at the conferences I go to?

I just got back a couple of days ago from Chicago, where I attended the annual meeting of the American Society of Clinical Oncology (ASCO). This is an enormous meeting, attracting over 30,000 participants from across the country and around the world. All of the specialties involved in cancer care are represented – surgeons, medical oncologists, pediatric oncologists, radiation oncologists, social workers, psychiatrists, orthopedists, and others.

The annual meeting is an opportunity for researchers from around the world to present their findings to each other. I thought it might be interesting to discuss some of the interesting presentations about sarcomas that I saw:

Better Treatment for Ewing’s Sarcoma
One of the most exciting talks at the meeting (at least, for me) was Dr. Womer’s presentation of the results of the last Children’s Oncology Group clinical trial for patients with localized Ewing’s sarcoma. In this study, patients were randomly assigned to receive the same chemotherapy (cycles of vincristine/doxorubicin/cyclophosphamide alternating with cycles of ifosfamide/etoposide) every 3 weeks or every 2 weeks. The question was whether chemotherapy works better given closer together (time intensive), or whether there would be too many side effects. Well, the results are in, and time intensive chemotherapy works! The patients who received the chemotherapy every 2 weeks had a 78% 4-year event-free survival rate, compared with 70% for the patients treated at the standard 3 week interval. Although this improvement was limited to patients younger than 18 years old, this is probably because there were too few older patients to evaluate.

Photo Credit

The Genetics of Rhabdomyosarcoma
Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma of children and adolescents. There are several different types of RMS based on microscopic appearance, but the two most common types are called “alveolar” and “embryonal.” These types refer to the microscopic appearance of the tumors (alveolar because the tumor resembles the alveoli, or airspaces, of the lungs; embryonal because the tumor looks like very immature muscle cells). It has long been known that alveolar RMS is characterized by chromosomal translocations (where a piece of one chromosome is “swapped” for a piece of another one), but embryonal RMS is not. Dr. Barr presented data showing that embryonal RMS are characterized by specific changes in chromosome 11 instead.

Interestingly, not all alveolar RMS has these chromosomal translocations, and 63% of those that do not, instead have the same chromosome 11 changes seen in embryonal RMS. A detailed genetic analysis also showed that by this test, so-called “translocation negative” alveolar RMS appears to be more similar to embryonal RMS than to “translocation positive” tumors. This raises the possibility that, in the future, we may do away with tumor descriptions based on microscopic appearance in exchange for genetic descriptions that probably more closely reflect the underlying causes and behavior of the tumors.

Alveolar RhabdomyosarcomaPhoto Credit

Synovial Sarcoma
One of the more rare types of sarcoma we treat is called synovial sarcoma. Doctors from the Rizzoli Institute in Italy looked back over the records of 250 synovial sarcoma patients treated there since the 1970’s to try to learn more about this rare disease. They presented some very important findings: 1) radiation therapy improves survival in patients with localized disease, but chemotherapy did not; 2) some patients relapse very late (more than 5 years from the end of treatment), suggesting that we need to follow these patients for a very long time; 3) patients who have a local relapse are often cured with further treatment, and it did not seem to matter whether this was a rapid relapse or a late relapse. This is an important study because it is the largest report on a single series of synovial sarcoma patients treated at a single institution.

Samarium to treat Osteosarcoma
Finally, I had the honor of presenting the results of my Phase I study of samarium-153 for the treatment of patients with osteosarcoma metastatic to bones. Samarium-153 is a radioactive agent that targets bone lesions. Because it was a Phase I study, the goal was to identify a dose of the drug we could give that would allow patients to recover safely and quickly from the side effects. Not only did we find what that dose is, but we also showed that this drug could be given to patients who had a LOT of previous treatment, and they had no more side effects than patients who had not been treated at all. Our next step will be to figure out how best to integrate this drug into treatment regimens based on chemotherapy, and hopefully one day this will be an important addition to our arsenal of therapies for osteosarcoma patients.

Unfortunately, my schedule did not allow me to make it to the presentation of the data regarding vitamin D and breast cancer, but you can read more about that study here.

Related Posts:
Vitamin D and Breast Cancer
Osteosarcoma Symposium in Houston
Children's Oncology Group Meeting
Medicine from the Sea

Osteosarcoma Symposium in Houston

Earlier this month, I had the distinct honor of participating in an international osteosarcoma symposium organized by one of the pioneers of our field, Dr. Norman Jaffe. Entitled “Progress from the Past, Prospects for the Future,” the meeting took place at MD Anderson Cancer Center in Houston, TX. Participants included surgeons, oncologists, radiologists, radiation oncologists, pathologists and laboratory scientists from around the world, as well as patients, parents, and patient advocates.



Dr. Jaffe asked me to speak about the possible role of immunotherapy in osteosarcoma treatment. It was an honor to have been invited, and it was a pleasure to be given the opportunity to discuss my thoughts on what the future of osteosarcoma therapy might include.

So what happened? Dr. Bruland, from Norway, gave an excellent presentation on his work demonstrating that small deposits of osteosarcoma cells might lie dormant in patients’ bone marrow, and that these cells might play a key role in disease relapse. Osteosarcoma is not thought of as a disease that typically involves bone marrow, so if true, this could change the way we go about hunting for sites of disease in our patients as well as changing our understanding of how cancer cells survive therapy to cause relapse.

Dr. Gorlick, from Montefiore Hospital in the Bronx, provided a comprehensive overview of the basic biology of osteosarcoma, providing a framework for future laboratory research that might one day yield new targeted therapies. Dr. Hughes, from our host institution, MD Anderson, discussed his work on one particular biological pathway (called the Notch signaling pathway) that might be important for the development of metastatic disease. There are already drugs available that target this pathway, so the prospect of intervening in this process is tantalizingly close.

Another highlight of the trip for me was the opportunity to see relatives of some of my patients and their families in a context outside of the Pediatric Oncology Clinic.

(everything is bigger in texas!)

Of course, no trip is all work and no play. I’ve never been to Houston, so I tried to squeeze in time to explore the city. I got to visit the (very avant-garde) Contemporary Arts Museum, as well as the sculpture garden across from the Fine Arts Museum (thankfully on a bright sunny day).

(a piece from the sculpture garden)

And the sushi at Azuma was incredible. If you’re ever in Houston, I highly recommend it!

The proceedings of this symposium will be published by Springer in the very near future for those of you who are interested in learning more.

(psychedelic cow guarding the Texas Children's Hospital)

Sarcoma Video

Bruce Shriver, co-director of the Liddy Shriver Sarcoma Initiative, posted an informative video on YouTube recently, called "A Forgotten Cancer". The Liddy Shriver Sarcoma Initiative is a major sponsor of sarcoma research across the country, including in my own laboratory. Check it out below and consider participating in Team Sarcoma 2008.



Another powerful site is The Faces of Sarcoma, which features pictures of sarcoma patients from around the world. It's amazing how diverse the group is, including patients of all ages and races, and from every corner of the globe.

The Stem Cell Thing

One of the best parts of my job is that not only do I have the pleasure of taking care of children and young adults with sarcomas, but I also run a laboratory where we conduct research that we all hope will eventually lead to new treatments for my patients. I am going to talk a little about my laboratory research today.

One of the hottest new ideas in the world of cancer research is the concept of the “cancer stem cell.” As many of you who have been involved with Ewing’s sarcoma patients know, the correlation between response to therapy and cure is not very strong. In other words, although patients with widely metastatic disease can respond well to chemotherapy and to radiation therapy, relapse is frequent and cure rates remain less than 25%.

Why is this?

One possible explanation is the existence of a cancer stem cell. Cancer researchers used to believe that all cells within a tumor were the same, and that any one of them is capable of unlimited growth and of spreading to distant sites. We now know that this is not the case. Only a small fraction of the cells in a tumor are capable of unlimited growth. The rest will only divide a few times before dying. This recognition led to the development of the Cancer Stem Cell Model.

The Cancer Stem Cell Model predicts that the small number of cells capable of unlimited self-renewal would have properties similar to other stem cells, such as bone marrow stem cells and the embryonic stem cells that we read about in the papers all the time. These cells would be responsible for maintaining the primary tumor population and should be relatively resistant to chemotherapy. This would make them the primary cells causing relapse because of their ability to survive the treatments that kill the majority of the other cancer cells. Cancer stem cells have been identified in several types of cancer, including leukemia, breast cancer, brain tumors, and others.

So…. Are there sarcoma stem cells? That remains an open question, but one that we are in the process of studying in my lab.

We have some very exciting preliminary data that makes me think that we may have identified Ewing’s sarcoma stem cells. This, of course, is just the first step. After we are able to prove that these cells exist, the real fun will begin! We have a plan in place to learn as much as we can as quickly as we can about the basic biology of these cells.

Hopefully our research will have a big impact on many aspects of the care of sarcoma patients:

1. We can isolate sarcoma stem cells and try to find drugs that can kill them.

2. We can devise a rapid test for sarcoma stem cells that might allow us to better predict who will relapse and who will not.

3. We can devise strategies to identify these cells in biopsy specimens to be able to determine if treatments have killed stem cells in the patient.

4. We can devise new treatment strategies specifically targeting stem cells that hopefully will result in higher cure rates than currently used chemotherapy with fewer side effects.

It is an exciting time in cancer research…. This new theory goes a long way toward explaining why chemotherapy can make a tumor go away but might not cure the patient. Identifying the cells responsible for relapse will allow us to make big strides in treatment and hopefully dramatically improve our treatments.

I’m lucky to be able to work in both worlds (the lab and the hospital) and be in a position to guide research findings from the laboratory into the hospital and witness first hand this revolution in cancer treatment – away from toxic chemotherapy and toward targeted therapy* more precisely aimed at the cancer.

*An article I wrote for ESUN, the newsletter for the Liddy Shriver Sarcoma Initiative, on targeted therapies for sarcomas