Music Is Medicine

No, the title is not a cliche.  It's the name of a fabulous organization founded by the daughters of one of my colleagues here at Johns Hopkins.  Leora and Ariella are musicians themselves, and they firmly believe that music can truly help in the healing process.  Founded when the girls were still teenagers, Music is Medicine is now an organization with national reach.  Quite a feat for two sisters from Baltimore.

Last year, I wrote about a visit one of my patients had from Drew Seeley, her favorite singer.  He wrote her a song, and I'll never forget the look on her face when he showed up in her hospital room to sing it for her.

Yesterday, lightning struck twice, when my patient, Bo, received a visit from Savannah Outen, who flew to Baltimore to serenade him with a song she wrote just for him.

 Bo is a musician himself.  In fact, I've seen him play at a fundraiser to help raise money to cover some of his medical costs.

So after Savannah played, Bo picked up the guitar, and in a truly priceless moment, played for her as she sang his requests.  Her version of Landslide (performed here by Fleetwood Mac and here by Smashing Pumpkins) was absolutely beautiful.  Moments like this, unscripted and real, remind me of all that is good in people.

I was there to hear the music, but Bo and his family had a surprise for me, too.  A framed picture of me and Bo... in one of my... uh... better moments in clinic.  Yes, that's me, hard at work curing cancer!

The song Savannah wrote and sang for Bo, "Brave and True," will be available soon on iTunes.  If you can't wait that long, check out this song to hear what a beautiful voice she has.

Related Posts:
Music Can Heal
Fishing, and the Kindness of Strangers
A Musical Wish

The Power of Social Media

In the past, I've discussed the financial challenges facing those of us who conduct research on rare diseases and the important role private foundations play in facilitating our work.  What I haven't mentioned before is the role social media can play in linking researchers with foundations.

As some of you may know, I'm on Twitter.  Well... sort of.  I've been on Twitter recently about as much as I've been posting on my blog.

But through Twitter, I found the Rally Foundation.  Honestly, I'm not sure I would have found them otherwise.  I'm sure glad I did, though.  They're a fabulous organization.  Founded by Dean Crowe, they have raised a ton of money to support childhood cancer research, and they have supported projects at 35 cancer centers across the country.  I'm honored to be on their medical advisory board.

And today I was thrilled to learn that they are going to fund a new project in my lab.

We have been studying a gene called WT1, which is turned on in a large number of sarcomas, including many cases of Ewing sarcoma, osteosarcoma, and rhabdomyosarcoma.  The role of WT1 in childhood cancer is unclear.  We have a theory -- that WT1 helps tumors to coax blood vessels into supporting their growth.  Money from Rally Foundation will allow us to test this theory, and to begin the process of identifying drugs that will block the activity of WT1.  Our hope is that, since WT1 is turned off in almost all noncancer cells in growing children, a drug that targets WT1 will be an effective treatment for childhood cancers with very few side effects.

We're obviously a long way from developing such a drug, but with the help of Rally Foundation, we're a step closer today than we were yesterday.

And every journey begins with a single step.

Related Posts:

The Importance of Research Foundations
The Stomp Out Cancer Project
The Sarcoma Program Goes 21st Century

Welcome Back!

It's a warm, sunny day today.  Spring is finally here, from the looks of things.  That means it's time for a new beginning.

On my way home from work last night, I stopped for a drink with an old friend of mine.  When I say old... I've known this friend since we were kids.   I can't remember a time when I didn't know him.  We were joined by two of his other friends, who I was meeting for the first time.  When I first meet people, and they ask what I do, my answer evokes either a blank look, a look of pity, or a lot of questions.

Thankfully, this time I got questions.

The two most common questions I'm asked are how I ended up doing what I do, and what is the most rewarding part of my job.  I'd like to share my answers here, as a part of my new beginning.

How did I end up as a pediatric oncologist?  I wasn't one of those kids who knew at age 5 he wanted to be a doctor.  In fact, I went to college hoping to be a research scientist.  It was only after my sophomore year that my father suggested to me that if I wanted to do medical research, a medical degree might be helpful.  So I started medical school in the Medical Scientist Training Program, on a track to get both an MD and a PhD.  Thankfully, just seven years later, I graduated with both degrees, ready to take the next steps.

Why oncology?  I was attracted to the science.  Understanding how cells work fascinated me, and when I realized that cancer is a perversion of the normal processes of cell biology, I knew what I wanted to study.

Why pediatrics?  That's even easier.  I love kids.  I love their undying optimism, their boundless enthusiasm for life, they neverending need to explore the world around them, and their seemingly limitless ability to love those around them.

The choice to become a pediatric oncologist turned out to be pretty easy for me.

Source

What is the most rewarding part of my job?  That's pretty easy, too.  Earlier this week, I received an email from an old patient.  He wrote to tell me that it was the 15th anniversary of the day we met and I told him he had cancer.  He has now lived more time post-diagnosis than pre, and felt moved to tell me how much he loved his life and how happy he was to have met me.

I received a similar message two years ago.  An old patient wrote to me on her birthday.  She told me she had recently been researching her diagnosis... and realized that someone with her type of leukemia, who had the chromosome mutations that her leukemia had, ought to have died.  Not only was she celebrating another birthday, but she was preparing for her wedding, and she wrote to thank me for taking such good care of her.

How could I ever have considered any other career?

It's spring.  Time for new beginnings.  Time to renew my efforts in the lab, so that even more kids will be able to look back on the day they were diagnosed and be thankful for the modern treatments, born of an intense research effort, that have erased the day when a childhood cancer diagnosis was a death sentence.

Related Posts:

Who am I, and why am I here?

Childhood Cancer Awareness Month

September is Childhood Cancer Awareness Month.  If you are a regular reader of my blog (even if I'm not a regular writer), you are already aware of childhood cancer.  But perhaps you didn't know that cancer is the Number 1 disease killer of children.  The only more frequent cause of death in childhood is trauma.  Or perhaps you didn't know that 10,400 children will be diagnosed with cancer this year in the US, but that many women will be diagnosed with breast cancer every 2 weeks.  This might be why a paltry 4% of the National Cancer Institute's annual budget goes to pediatric cancer research.

So now you're aware.

But here's a more important thing to be aware of:  pediatric cancer is weak; although it can kill the body, it can't kill the spirit of childhood.  Don't believe me?  Check out the spirit of the kids I take care of every day.

This is not a drill!

 Today was the day.


I recently wrote about chemotherapy shortages.  They've been in the news more and more over the past 12 months.  But until today, at least in my practice, they were worries.

But now the drug in short supply is methotrexate.

Acute lymphoblastic leukemia (ALL) is the most common cancer in children.  Cancer is the most common cause of death (other than trauma) in children.  Upwards of 80% of children with ALL are cured with modern treatment regimens.

But I don't know how to cure ALL without methotrexate.

Or osteosarcoma.

Or lymphoma.

There are several companies that supply methotrexate in the US, but all are experiencing production or distribution delays or suspensions.  The net result?  We had a meeting today to figure out if we have enough methotrexate to treat our current patients.  And, if we have a shortfall, to figure out who gets treated... and who doesn't.

The good news is we found a few vials we didn't know about yesterday, and for at least the next 2 weeks, all current patients can get treated on schedule.  But, if we don't get another shipment in 2 weeks, or if an adult-sized patient is diagnosed with Burkitt's lymphoma presents to the hospital tomorrow?  We won't have enough drug. 

And someone won't get treated.

I have already discussed the many reasons for drug shortages.  The list of drugs in short supply is mind-bogglingly long.  But we can work around a lack of Zofran.  We can find alternatives for Gentamicin.

But I can't cure ALL without methotrexate.

Managing Pain

Pain.

Little scares a new oncology patient as much as the idea of pain.  Unfortunately, pain permeates my practice.  Often, pain is the initial symptom that leads to a new cancer diagnosis.  Cancer patients undergo frequent painful procedures -- biopsies, bone marrow aspirates, surgeries ... even a simple blood draw involves a small amount of pain.  Because of this, managing pain is something I have some experience doing.

So I was surprised when I read this article in The Washington Post this morning and discovered that "some [pain doctors] began decrying the increasingly widespread use of opioids and questioned whether the drugs worked."  Really?  There are pain doctors who question whether opiates (morphine, for example) work?

Over the years, I have seen a variety of pain management styles. From doctors who prescribe intramuscular injections of pain medications to small children recovering from surgery, through sophisticated regimens involving patient-controlled analgesia and the use of non-drug techniques designed to specifically combat different types of pain. Pain management skills vary widely, and careful use of appropriate therapies can make all the difference to a suffering child.

Unfortunately, because the drugs which are the mainstay of pain treatment, opiates, are highly addictive, their use is politicized.  The article in today's Washington Post, for example, was focused on a patient advocacy group, the American Pain Foundation, which gets the lion's share of its funding from the pharmaceutical industry.  Unfortunately, this creates the appearance of a conflict of interest when the group strongly advocates for the use of specific narcotic pain medications (such as OxyContin) to control chronic pain.  And the appearance of a conflict of interest, whether or not the conflict exists, is sufficient to cast doubt on everything the Foundation has to say, even when what they say is spot on.

The use of narcotic pain medications is clearly expanding, and as a result, overdoses are an increasingly common cause of death in this country.  That doesn't mean these drugs should not be used.  They are highly effective at controlling acute, and even chronic, pain.  But like all medications, they need to be used appropriately, under medical supervision, prescribed by doctors who are experienced in their use, know how they work, know what kinds of pain they help, and know the risks and limitations of their use.

The diseases I treat cause pain.  One of the most common fears among cancer patients is the fear of dying in pain.  The treatments I use cause pain.  Some of the procedures we perform to monitor the progress of my patients cause pain.  Without highly effective drugs to treat pain, I could not do my job.  Rather than politicizing these drugs, we should be advocating for increased education about their proper use, about choosing the right drug for the right type of pain, and increasing research into the mechanisms of pain so that newer, more effective, safer drugs can be developed.

Photo Credit

Related Posts:
What Rufus Can Teach Us About Pain
Fentanyl Revisited
Narcotics for pain control:  When is enough too much?

Special Kids

All of the kids I take care of are special.  Is that cliche?  Maybe so, but it's true.  I truly have learned something from each and every one of them -- though not always what I thought I would learn.

Some kids stand out.

My first "real" patient, who was 13 at the time, and is now, at the advanced age of 27, joining the military.

The patient who got a transplant for her horrible leukemia... who was so sick going into her transplant that I said to her during her "consent conference"... "Well, if your kidneys fail during the transplant, at least your donor can give you one of his, too, and since your immune system will be his, you won't have to worry about rejection."  Not only did her kidneys not fail, but she is alive, well, in remission, and sometimes takes her hormones.

Marta also stands out.  Marta was a teen mother before I met her, though she was finishing her freshman year of college.  That, alone, impressed me.  But as I got to know Marta, first during her initial treatment, and then while we were unsuccessfully treating her relapse, I got to know a warm, caring, wonderful mother.  A young woman who faced adversity with grace, never complaining about her fate.  A young woman who made mature decisions, including continuing college through all of her treatment.  A young woman who accepted hospice care when she needed it, but continued to do what she could to extend the time she would have with her child.

From some kids, I learn about a disease.  From some kids, I learn compassion.  From some kids, I learn grace.  From Marta I learned how to face life, no matter what life has in store.

I'll miss her.

Another Kind of Tears

 Photo Credit

Tears are a fact of life in my business.  Parents cry when I tell them their child has cancer.  Children cry when they undergo painful procedures.  We all cry when a child dies.

But sometimes the tears are tears of joy.

Jade came to my clinic for another opinion.  She has a benign tumor, but it's in a bad place.  As I have shared before, sometimes it isn't better to have a "benign" tumor.  In order to remove Jade's tumor, she would need disfiguring surgery.  She and her father were told there was no choice.


Thankfully, they were told wrong.

Even though Jade's tumor is benign, it can be treated with chemotherapy.  It's a small tumor, and it's not causing her any symptoms right now.  Even if we can't make it go away, if we can keep it from growing, she will be fine.

After discussing her options, I asked Jade and her father if they had any questions.  Her father started crying, and then Jade did, too.  They were so relieved to have non-surgical options!

Photo Credit

How nice to make someone cry tears of joy for a change.

Related Posts:
When Benign Isn't Better and Malignant Is Preferred
A Long Year for Mike
"I Don't Know How You Do Your Job"

You're Kidding, Right?

(Another of David's rants about insurance companies)

I've written many times about the absurdities I've encountered dealing with insurance companies.  I've had another experience that I'd like to share.

The patient came into clinic for chemotherapy.  The PA examined him and heard an irregular heartbeat.  Neither of us know the patient well, so we looked through his chart to see if anyone has ever noticed an irregular heartbeat before.  No one had.  We ordered an EKG, and it showed an abnormal rhythm.  The patient has had several EKGs in the past, and this rhythm was a new finding.  One possible explanation for the new abnormal rhythm would be if the patient's central line had moved a bit and was irritating the right ventricle of his heart.  The best way to figure out if that could be happening is to get a chest x-ray to see where the catheter tip is.

We ordered a chest x-ray, only to learn that the patient's insurance company doesn't cover radiology procedures at Johns Hopkins.  Since an abnormal heart rhythm can cause sudden death, diagnosing a cause is an emergency, so we called the insurance company for "permission" to order the x-ray.

You may think this is a "no brainer."  After all, a chest x-ray is cheap, and we could discover the cause of the patient's abnormal heart rhythm and fix it relatively easily.  The insurance company, however, needed to authorize the x-ray.  So I was told the nurse would review the case and get back to us.


Once again, I find my judgment about a case under review by someone with a financial stake in the decision.  In this case, a nurse, sitting in an office in another state, not able to see my patient, was going to decide if my decision to order a chest x-ray was justified.

No.  I'm not kidding.

But if you ask, insurance companies don't make medical decisions, they make coverage decisions.

Related Posts:
Why David Hates Insurance Companies
Not Medically Necessary

How Much Time Do I Have?

I think that is the hardest question I have ever been asked. The emotional weight that goes along with asking how many days are left in a child’s life is staggering. I have been asked this question many times during my career, and I still don’t know how to answer it.


Some doctors answer this question based on disease-specific statistics. For example, since most patients diagnosed with a brainstem glioma die within a year of diagnosis, some will tell the family of a patient who receives this diagnosis that the child has less than a year to live.

Photo Credit

Some doctors answer this question based on personal experience. It is personal experience that has led me to dread this question the most. I would like to share two of these experiences to illustrate why.

It was a Monday night. I was home having dinner with my family when I was paged by the resident covering our inpatient unit. My patient had just completed an MRI, ordered to help us determine why he had lapsed into a coma. The MRI had just been read, and the radiologist had called the resident with the result: not only had my patient’s tumor grown substantially, but it was causing an “uncal herniation” (this is when a part of the brain is compressed, often by blood or a tumor, cutting off the blood supply to vital structures and often rapidly leading to death). Since the family had already decided not to pursue further treatment of their child’s cancer, the progression was not a surprise, but I went back the hospital immediately to talk to them about the herniation, since that was a sign that their child could die as soon as that very night. The previous week, when asked how long they had left, I had estimated several weeks, so this change would come as quite a shock.

Picture Posted By Permission

When I got to the child’s room, we spoke at length about his prognosis. His parents asked me how long they would have with him. I answered honestly that I thought he would die within days, and that he could possibly die within hours. The next morning, when he was still alive, we started to arrange a discharge with hospice care. It was Friday when he was finally able to go home.

That was 52 days ago, as I type this. Since discharge, my patient has regained consciousness, resumed eating, and is considering an exercise program to regain some strength. He is on his second hospice company. My estimates of his lifespan were both way off base.

Picture Posted By Permission

Two weeks after my Monday night conversation, another patient of mine was admitted to the hospital for end of life care. After a couple of days in the hospital, his mother asked me how long he had left. I told her that I had no idea, and shared with her the story of the patient with the herniation, who I thought would die within days and who was still alive almost 3 weeks later (at that point). I told this mother that, based on my experience, the best I could do was to guess how long she had with her son. She really wanted an answer, though, so I guessed for her. I told her that her son’s lifespan could be measured in weeks. Probably not days, certainly not months or years, but weeks. He died three weeks later.

I suppose sometimes I do guess correctly.

Related Posts:
 
I went in to say "Good bye"
When My Patients Die
Oh, by the way...

Another Ethical Dilemma

The ethics of modern medicine has always fascinated me, and Pediatric Oncology has provided me with more than my fair share of ethical issues to contemplate. I want to share today’s, and see what people think about this particular, emotionally charged, situation.


I received an email today from a colleague in another state. He trained under me, and I guess he thinks I did a good job, since he emails me for advice from time to time. He met a new patient today – a 23 year old woman with a new diagnosis of osteosarcoma. Unfortunately, she is 20 weeks pregnant.

One of the mainstays of osteosarcoma treatment is high dose methotrexate. Methotrexate is a very effective drug for terminating pregnancies, and this is where the ethical dilemma begins. The patient has a choice to continue her pregnancy or not. Except that the state in which she lives does not allow medical assistance funds to be used to terminate a pregnancy, and she has Medicaid as her sole source of health insurance. She cannot afford to pay for an abortion herself. If she chooses to continue the pregnancy, either out of necessity or out of desire to do so, giving her methotrexate will be fatal to the baby inside of her.

If the patient chooses to remain pregnant, my friend has some very difficult decisions to make: should he wait to treat her until the baby is born? This would give the tumor as much as 4 months to spread before treatment, a huge risk to the patient. Should he begin therapy early, maybe once the third trimester begins, and just not use methotrexate? This would allow the baby to develop to full term prior to delivery, and only delay beginning chemotherapy a few weeks, but would provide less than optimal care to his patient. Could an obstetrician ethically deliver the baby early, say at 30 weeks, a time when the child’s development is likely to be normal (but, of course, may not be) despite being premature? This would allow him to use methotrexate almost 3 months earlier than if the baby is delivered at full term.

I told him what I think I would do, but I’m glad that for me this is just an ethical puzzle, instead of a real situation where I have to make real decisions.

Related Posts:

The Irony of Patient Autonomy
Thank Goodness for Ethics Committees

How Small a Chance Is Too Small?

I’ve written about difficult cases before, but this case is really eating at me. It touches on issues of patient autonomy and how much influence we, as physicians, have over our patients. But the real issue is one of futility. How small a chance at survival is too small to offer a very toxic treatment?

The patient is a young boy with leukemia. We have been treating him for over a year, and his leukemia just won’t go into remission. For the past 3 months he has been in the hospital, first to receive chemotherapy and then to manage the side effects we caused. His bone marrow is nearly empty, but almost 80% of what few cells are present are leukemia cells. His only potential curative therapy is a bone marrow transplant.

Therein lies the problem. Not only does he have refractory leukemia, but his lungs are significantly injured from his previous chemotherapy, functioning at just about 55% of their predicted ability. For my patient, this is a major problem, because sick lungs make a bone marrow transplant very risky. Also, he doesn’t have a matched donor, so we would have to do a highly experimental type of transplant called a “reduced intensity haploidentical transplant.” This means we would give less than the usual amount of chemotherapy (to try to decrease the toxicity), and would use a donor that is only half matched.

I presented this case to the adult BMT group last week. Not one of them thought going through with the transplant was a good idea. They are convinced the patient will do badly, that he will end up dying in the ICU on a ventilator, and would never be cured with this approach anyway. Better to send him home on hospice care.

I polled a number of pediatric colleagues by email. They all said the same thing – better to do hospice care than a transplant that will most likely make the patient sicker before he dies anyway.

Our group has discussed the case extensively. We all agree that the chance of cure for this child is less than 5%. There is a 95% chance he will die faster and in more pain if we go ahead with the transplant than if we send him home on hospice care. So is the more humane choice to not offer the family a transplant, knowing the odds are overwhelmingly against success, knowing that the transplant will most likely make an already tragic situation worse?

Who gets to decide if the 5% chance of a cure is worth the risk? Is this chance of success so small as to qualify as futile?

If I know the father the way I think I do, if I hold out ANY chance of cure, he’ll take it, no matter the cost. That means, if I offer the family the transplant, he will go for it. But is that fair of me? He doesn’t know what it’s like to watch a child die in the ICU. I don’t think I can fully explain to him how awful a death his son may have, especially compared with what it would be like for him to die peacefully at home. Given that, can he truly give informed consent?

On the other hand, if I don’t offer the transplant, I take away even that slim chance of survival. Can I ethically do that? Or is that a decision the parents get to make?

 Photo Credit

The Irony of Patient Autonomy

One of the cornerstones of medical ethics is the concept of patient autonomy. MedicineNet.com provides a succinct definition: “The right of patients to make decisions about their medical care without their health care provider trying to influence the decision. Patient autonomy does allow for health care providers to educate the patient but does not allow the health care provider to make the decision for the patient.”

At lunch the other day, I was talking about a particularly challenging case when this concept came up. After thinking for a few minutes, I was struck by a sense of irony that I wanted to share in case others have thoughts about this.

The patient is a teenager with leukemia. When she was diagnosed with leukemia, we didn’t really give her parents any choices about treatment. We told them their daughter has leukemia, she needs chemotherapy, and we think she should get this particular regimen. Sure we received informed consent to treat the patient (whether this is truly informed consent is a topic for a future post), but it’s not like we gave the parents a choice of regimens or the option to forego chemotherapy. And because leukemia in children is curable, even if the parents had refused chemotherapy, we would have gone to court to force her to be treated.

Did we respect the patient’s autonomy here? Based on the definition above, I would say we did not. We certainly influenced the decision – by not giving the parents any choices.

Fast forward to last week, when a bone marrow examination revealed residual leukemia. The next course of action at this point is not clear: should she go straight to a bone marrow transplant? If so, should it be a standard transplant, or a more experimental approach? How about more chemotherapy? Is so, which regimen? The right choice is not clear. My “lunch date” is not a physician. So he asked me, “How will the family decide?” Full of respect for the patient’s autonomy, I answered, “Well, I will lay out the options, list some pros and cons, and the family will make a decision.”

This is when Marco asked the question that sparked this posting: “How will they make that decision? They don’t have the expertise to make that call, do they? Isn’t that what you are trained for?”

And that’s when it struck me. This family has had two key decision points: the day the girl was diagnosed, and the day her marrow showed persistent leukemia. At the first point, my expertise was unimportant… any oncologist would have said the same thing (that she needed chemotherapy), and the patient’s autonomy was only a secondary consideration. At the second point, when the optimal choice is NOT clear, when the decision should be MOST informed by someone experienced, that’s when I was explicit about my desire to lay out choices and let the family decide.

I still don’t think I should tell the family what decision to make… I just think it’s ironic that when the decisions are more complex, and my expertise matters more, the patient’s autonomy is even more important.

Maybe that’s how it should be. What do you think?

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)

A Long Year for Mike

A year ago this week, Mike's sinuses were acting up again.  It wasn't uncommon for Mike to have sinus problems during winter, but last December things were worse than usual.  A trip to his pediatrician led to a CT scan.  The scan found a mass, not a sinus infection, and that's how Mike and I met.

Mike spent last Christmas worrying about the mass in his sinuses, and whether it was cancer.  After a year of surgery, chemotherapy, and radiation, Mike celebrated a very important day earlier this month:  End of Chemo Day.

We celebrated with Mike in clinic.  His family brought trays of sandwiches and desserts to share with the staff.  Mike plays guitar, so he had a special cake.

The food was awesome, the cake was divine.

But the best part of the day?  The smiles associated with the news that the MRI and the PET scan couldn't find any cancer in Mike anymore!

I'm pretty sure this year Christmas will be a whole lot more merry in Mike's house!

Related Post:

A Musical Wish

Not Medically Necessary

After a courageous 5-year battle, R lost his fight with cancer. He lived a good life while he was being treated, finishing high school, starting college, and checking items off his bucket list. This holiday season is hard for his family. His absence is palpable … and painful. What could make this time any worse?


The letter they got from their insurance company.

Apparently I ordered a CT scan back in September. The insurance company sent the family a letter telling them that they have deemed the scan “not medically necessary,” and they are refusing to cover it.

I’m used to the things health insurance companies do to try to avoid “medical losses” (their term for payments made to health care providers). I often have to fight with them when they don’t want to cover treatment that is part of a clinical trial (even when the trial is standard of care), or when they don’t approve of my choice of antibiotic, or don’t want to pay for a bone marrow transplant. Those denials, however, are usually couched in terms like “this is not a covered benefit.” This allows the company to claim that they are not making medical decisions : they are making a determination of what is covered – it is up to the physician and patient to decide what treatment is necessary, they are only deciding whether they will pay for it based on the terms of the policy.

This letter is different. It is denying coverage because someone decided the scan was “not medically necessary.” That person has made a medical decision.

Who is the person that made this decision? There is no way for me to find out, but it is likely that the person making that decision is not a pediatric oncologist with experience taking care of kids with multiply relapsed, refractory osteosarcoma. The person who made this determination may not be a physician at all. I am certain of one thing, however. This person was not in the room examining my patient when I decided he needed a CT scan. This person was not responsible for the health of my patient. This person was not trying to decide if a treatment was working, and if not, what we should do next. This person was not helping to treat my patient in any way.

What kind of penalty would I face if I were making medical decisions for patients who I did not see, talk to, or examine … whose cases I was not familiar with? Apparently, there is no penalty for someone making medical decisions from the comfort of their corporate office building, looking at nothing more than the interval of time between two scans.

Yes, I will appeal the denial. Hopefully the insurance company will agree to cover the bill. But only Ebeneezer Scrooge would think it reasonable, during the holiday season, to mail such a letter to the parents of a child who just passed away. Only the Grinch, whose heart was two sizes too small, would think it reasonable for someone in an office building to look over my shoulder and decide that a CT scan I ordered to evaluate the progress of my dying patient’s cancer was “not medically necessary.”

 
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I went in to say "Good bye"

We knew this day was coming. Over the summer her cancer came back even though she was getting chemotherapy. We switched gears, giving radiation and chemotherapy aimed at controlling pain, no longer at curing disease. But that doesn’t make this day any easier.

Overnight, last night, it became harder to breathe and her pain worsened. A chest x-ray showed almost no air getting to her left lung. Hoping there was fluid that could be removed, she had a CT scan today; but there was no fluid, only tumor. Tumor that hadn’t been there 10 days ago.

I went in to the hospital today, to see her one last time before she went home. We watched some of the football game together. We talked about her kindergarten teacher, a brave woman who was a tremendous support before she died of breast cancer in August. She told me about the tombstone she wants – a softball diamond with a girl sliding into home plate, with a caption that reads, “Safe at home!”

But rather than complaining, or asking “why me?” the young woman and her family had different plans to discuss. Their community had raised a large sum of money to help cover medical expenses, and there is going to be a lot left over. As her father said, “The community has done a lot for us. We need to give something back.” So, on the day she was going home, my patient was deciding how she was going to help her community.

They decided to give some money to a fund established in memory of her kindergarten teacher. They decided to give some money to a neighbor who, because of sudden illness, had fallen months behind on his mortgage payments. And they talked about how they could still contribute to Ewing sarcoma research. “Just because this is happening to me, doesn’t mean I don’t want to keep trying to help,” she said.

Photo Credit
I hope that when I am in her situation, I can show half as much grace as she did this afternoon.

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A Famous Parent

In the past, I have blogged about patients of mine who have made the news. This past week, while I was reading the newspaper before going to work, my eye was caught by an article in the Sports section about a man with a familiar name.

(Note: All personal information in this post is also seen in the article in the Washington Post, so there is no violation of confidentiality. Also, some links will require a subscription to washingtonpost.com to see.)

The article was about the head football coach at the University of Richmond, Michael London. The article spoke about Mr. London in glowing terms, talking about how he grew up in Richmond, played defensive back for the University of Richmond, and eventually became a detective in the Richmond Police Department’s street crimes unit.

See the video here.

Eventually Mr. London left the police department and became a football coach. I met Michael London when he was the linebackers coach for the University of Virginia’s football team. At this point his daughter had been diagnosed with Fanconi Anemia and needed a bone marrow transplant. The London family investigated several cancer centers, and settled on Johns Hopkins.

The Washington Post article related the story of Ticynn’s transplant, and how Mike felt about being the donor. Reading about a transplant I had directed, but from the father’s perspective, was very moving. We doctors rarely truly know how our patients perceive what they are going through. We usually only know what they tell us. This time, I got to read how a patient’s family dealt with a transplant without the filter that accompanies talking with a doctor.

I remember Mike, and I remember Ticynn. It was an honor to take care of the London family, and I’m thrilled Ticynn did so well (and continues to do well). Reading an article like this, and seeing how our medical encounter fits into the arc of Mike London’s life was a fabulous experience. I only wish I could see all of my patients’ lives in this way.

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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.

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