Pay It Forward

I got a wonderful email the other day from the mother of a patient of mine, Kelly. She wrote to tell me not only how well her daughter is doing now, but that she is planning a benefit concert to raise money for some of the doctors at Hopkins who helped her daughter. In her words, “since I can never actually "repay" any of you, it is my deepest hope that I can somehow pay it forward.”

With her permission, I want to tell you Kelly’s story. My sincere hope is that someone with a child who is very sick will read this story, see the pictures, and feel some hope that their child, too, can come through it and do well.

Kelly came to Johns Hopkins when she was 11 for treatment of an unclear disorder of her immune system that had not responded to numerous therapies over the previous 2 years. The immunologist who saw her at Hopkins thought she might benefit from a novel treatment we have developed using high doses of a chemotherapy drug called cyclophosphamide (Cytoxan) to essentially “reboot” the immune system in patients who have severe autoimmune diseases (an autoimmune disease is a condition where the patient’s immune system attacks the patient’s own body as if it were an infection).

Twenty-one days after being admitted to the hospital for evaluation, Kelly was treated with high dose Cytoxan. This treatment initially made a very sick child even worse. She had blood infections. She needed transfusions. She couldn’t eat and needed a tube for feeding.


But then she got better.

It was slow at first, and when she was discharged from the hospital after 48 long days, Kelly was still being fed through a tube. But she was able to stop antibiotics, slowly decrease her pain medications, and eventually eat again. Two weeks after discharge from the hospital, Kelly was well enough to return home to California. She has continued to improve dramatically, and I’ve had the pleasure of watching her progress in photos her mother has sent me… first, wearing a bikini on the beach in Malibu, and most recently playing with the animals at the fair.



Kelly now eats a regular diet, her feeding tube is gone, she goes to school with her friends, and you could never tell by looking at her that she was ever so sick. Her story continues to inspire me. Children like Kelly, who endure so much, and emerge looking as if nothing had ever happened, are the reason I do what I do. And if her mother can “Pay it forward” and make this concert happen, I’ll be there dancing in the aisles with her.

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The Story of K

Is Cancer Contagious?

This question actually comes up a lot in my practice.

When a family is first coming to terms with a cancer diagnosis, so many questions pass through their minds. Does it run in families? Do my other kids need to be checked? Is it contagious?

In humans, the answer is “No,” although now that we know cervical cancer is usually caused by Human Papilloma Virus (HPV; a sexually transmitted infection) this answer is a bit fuzzy. Although viruses like HPV that can cause cancer are contagious, cancer itself is not.

But is that true for all animals? Apparently not. Recently I came across this fascinating article about one of my favorite animals from childhood: The Tasmanian Devil.



When not chasing Bugs Bunny, Tasmanian Devils live in, well… Tasmania. They are marsupials, carrying their young in pouches like a kangaroo or opossum. They are the largest carnivorous marsupial to escape extinction.

Sadly, though, over the last decade, the population has crashed. In some areas by as much as 90%. The cause? Cancer. A cancer that is contagious!

How does that happen? The cancer, known as Devil Facial Tumor Disease, causes a tumor on the face of the Tasmanian Devil, and when an animal with such a tumor bites another Devil (which isn’t a rare event, as you might imagine), the cancer cells are transmitted to the victim and grow into a tumor. The tumor makes it hard for the animal to feed, so it starves.

Why doesn’t the animal’s immune system protect it against the cancer? In most other species, if you inject cells from one animal into another, the recipient’s immune system destroys them. That’s why organ transplants don’t work without strong immune suppressive medications. This immune defense is based on differences in a set of genes called MHC genes that are so variable that (for the most part) only identical twins share the exact same gene sequences. This holds for humans, dogs, cats, mice, monkeys, kangaroos… almost every animal.

Except, apparently, the Tasmanian Devil. Tasmanian Devil MHC genes are not very diverse, and this allows the cancer cells to evade the immune system and grow.

But the mystery does not end there. There are other animals with very little MHC diversity, like cheetahs and beavers, but they don’t have contagious cancer. Also, Devil Facial Tumor Disease is new, first spotted in 1996. This suggests that the situation is more complex than it would seem on the surface, and raises the possibility that the cancer cells have evolved in ways that make them more transmissible.

How? No one knows. It’s just one more of the many unsolved mysteries surrounding cancer and the immune system.

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Kaposi's Sarcoma and the Virus/Cancer Connection (Part 2)

Kaposi's Sarcoma and the Virus/Cancer Connection (Part 3)

HPV, STIs, and Teenaged Girls

Oh, by the way...

These four words, just at the end of an appointment, usually are prelude to something of tremendous importance that the patient has spent most of the preceding hours trying to decide how to talk about.

Today I saw a 23 year old young man with relapsed metastatic osteosarcoma. He has already been treated with all five of the chemotherapy drugs that we know typically work against osteosarcoma. We looked hard, but there are no open Phase II clinical trials for which he is eligible. After much discussion and many emails, we decided on a low dose chemotherapy regimen in combination with another drug that we hope will help the chemo work better.

After reviewing the treatment plan and all of the potential side effects, the patient signed informed consent and we made plans for follow-up, including blood tests to monitor for side effects.

“Oh, by the way…”

This is when the patient told me he was hoping to hook his boat up to the back of his van and drive out west with a friend.

My initial reaction was… fear. I had just laid out a treatment plan with a long list of side effects. Without prompt medical attention, some of these side effects could be fatal. How was I going to tell this young man that he can’t go camping in the middle of nowhere while taking these medications? What if he went anyway, and something horrible happened to him? Something that could have been prevented had he stayed locally?

Should I let him go, or try to talk him out of it?

It didn’t take me long to decide he should go on his trip. His prognosis is poor, even if this treatment helps. At the moment, he is in good condition and will really enjoy his trip. As long as he fully understands the consequences of his decision, he should live as full a life as he can for as long as he can.

I just hope he has a safe trip and a great time.

Thank Goodness for Ethics Committees

(Blogger’s Note: Due to the sensitive nature of this case, even more details than usual have been changed.)

The patient was coming to me to sign consent to donate bone marrow. Just one problem. She’s pregnant.

Does that matter?

What if I told you that the bone marrow donor is 23 years old and is donating for her sister who has leukemia?

What if I told you that the donor is 19 and is donating for an unrelated child with severe combined immune deficiency syndrome (SCIDS)?

What if I told you that the donor is 17 and is donating for her father, who has lymphoma?

There are so many variables that play into the answer to the first question I posed: Does it matter? Certainly if she is pregnant and undergoes general anesthesia, she exposes her unborn child to a small but measurable risk. How about regional anesthesia? How about the blood loss associated with marrow donation? Bone marrow transplant for a child or young adult with certain types of leukemia can be considered standard of care. What if the transplant is experimental, rather than standard?

Although these questions may seem abstract, they are not. In fact, one of the situations above happened to me recently. It was the kind of situation that made me happy that our hospital has an ethics committee.

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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!

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Related Link

The Orwellian Language of Pediatric Oncology

George Orwell is most famous for his novel, 1984, which described a future world run by Big Brother, who controls all aspects of society. One of Big Brother’s methods of control is the manipulation of language, referred to by Orwell as Newspeak – the use of words to mean essentially their opposite.

I was struck this past weekend by how we doctors use words in ways not originally intended. An oncologist might say the following about her patient, “Frank failed cisplatin/doxorubicin, so we are going to use ifosfamide and etoposide.” What the doctor meant was that Frank was treated with cisplatin and doxorubicin, but these drugs did not control his disease. The drugs failed Frank, even though the doctor said that Frank failed the drugs.

In my most recent blog post, I discussed how it can be good to be wrong. But is that what I really meant? Of course not. It’s never good for me to be wrong. What is good is that I told the family there were two possible explanations for their child’s CT scan results, one more likely and one unlikely – and the unlikely (but better for the patient) result turned out to be correct.

On Sunday, the mother of my patient updated her CarePage. Her son, she said, is “getting bored with cancer.” Reading this reminded me of something I often tell patients when they are in the hospital and not much appears to be going on: “It’s better to be bored. Bored is good.” That sounds like something from 1984. Or from the Oliver Stone movie Wall Street (“Greed is good.”). But in this case, it’s true. Being bored with your cancer can only be a good thing. It means things are going well, there are no acute crises, and (to quote my patient’s parent’s blog again) “adrenaline [isn’t] flowing like a volcano.”

Sometimes Newspeak DOES reflect the truth.

It’s Good to be Wrong

Sometimes my job is tough, especially when I have to give someone bad news. In September, I described telling a child and his family about a new nodule on his CT scan. Ordinarily, such a nodule means metastatic disease. Metastatic disease is an ominous sign.

In that post, I talked about how to deliver bad news. What I didn’t mention was our discussion of alternative explanations for the CT scan findings. Although I told the family that this nodule probably represented a return of the cancer, I also told them it could also be caused by inflammation, infection, or some other benign process.

Was this false hope? Was I just trying to soften the blow? Or could it really be that the nodule we saw was caused by something else?

We decided not to act immediately, but to wait a brief period and repeat the scan. If the nodule was cancer, it should still be present, and it should be larger. If it was inflammatory, it could be gone.

Three days ago we repeated the scan. The radiologist’s report reads, “Interval resolution of previously visualized nonspecific 2 mm nodule.” The nodule is gone!

I had told the family I thought the cancer was back. I also told them I hoped I was wrong. Sometimes, it’s good to be wrong.

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Back to the Future: Another Meeting in Denver

Last week I attended the annual meeting of the Children’s Oncology Group. Like last year, the meeting was held in Denver, Colorado. The Children’s Oncology Group is the organization that coordinates the majority of clinical research into childhood cancer in North America. We conduct clinical trials ranging from early Phase I studies of brand new drugs through Phase III trials designed to optimize the treatment of children with a wide variety of cancers.

Unfortunately, this year I was only able to attend part of the meeting. I did get to spend a full day attending various meetings related to ongoing and upcoming bone cancer-related clinical trials. It was an exciting day because we are entering the era I spoke about as “the future” when I was interviewing for medical schools back in the 1980’s – the era of “molecular medicine.” All of the upcoming studies under discussion involved the use of at least one drug that works differently from traditional chemotherapy. Drugs that target specific biological differences between normal cells and cancer cells. These drugs have tremendous potential to improve our treatment of children (and adults) with cancer by being more effective and having fewer side effects. As George Allen, the coach of my favorite football team when I was a kid, used to say, “The future is now.”

Like last year, I also took some time to relax while I was out west. Last year, I wandered around Denver, but this year I rented a car and explored the mountains. South of Denver, west of Colorado Springs, lies Pikes Peak, which already had snow at the summit.

Nearby were the amazing rock formations of Garden of the Gods.

Needless to say, the scenery was stunning, and my little camera doesn’t begin to do it justice.

I can’t wait to get back there for a vacation, when I can really spend time in the Rockies and enjoy it all.

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Bravo New Jersey!

Photo Credit

It’s that time of year again. The days are getting shorter, Halloween decorations are appearing, and that can only mean one thing. It’s time to get your flu shot.

Every year, the flu shot changes, because every year the strain of influenza virus that circulates changes. Last year’s flu shot does not protect you this year. So every year, people need a new shot.

It also seems that every year, the official recommendation for who needs a flu shot changes as well. This year is no exception. This year, the Centers for Disease Control, the agency that makes the official recommendations, has said that all children over the age of 6 months should be vaccinated against influenza.

The State of New Jersey has taken this recommendation to heart, and this year is requiring influenza vaccination for children to be allowed to attend preschools and day care centers. This law sparked a protest outside of the New Jersey Statehouse on October 16 by parents who oppose this requirement.

Personally, I think this was a good decision, based on sound science. States already require immunization against a large and growing number of infections for all children who attend school. So the idea of requiring a flu shot to attend school (even if it’s preschool) is not out of line with current policies.

In fact, influenza vaccine is probably more important than some of the others that are routinely given. For example, children are required to receive a diphtheria immunization (that’s the D in DTP and DTaP). Prior to universal immunization, diphtheria was a feared disease, and there were 13,000-15,000 deaths in the US every year from diphtheria. Between 1998 and 2004 there were 7 cases in total reported to the CDC. In contrast, every year on average 200,000 Americans are hospitalized because of influenza, and 36,000 of them die. Children are at risk as well. Every year an average of 20,000 children under the age of 5 are hospitalized for complications of influenza. Getting influenza is also a risk factor for potentially serious bacterial infections in children.

Another important factor is that children easily spread influenza to their parents and other household contacts. My colleagues at the University of Maryland demonstrated that a school-based influenza immunization program was able to decrease transmission of influenza to adults who come into close contact with vaccinated children. So by requiring that more children receive this important immunization, New Jersey is likely to see a decrease in influenza-related illness not only in children, but in adults as well. And since so many people die from influenza every year, this is probably a good thing.

Cough Medicine for Kids: Update 2008

Photo Credit

A year ago, I wrote about an effort by the Baltimore Commissioner of Health, Dr. Joshua Sharfstein, to convince the US Food and Drug Administration (FDA) to ban the use of over the counter (OTC) cough medicines for young children. Last October, an FDA advisory panel voted 13 to 9 to recommend that these medicines not be used for children under 6. Unfortunately, it has taken a year for anything substantive to happen.
Better late than never, the Consumer Healthcare Products Association, a trade organization representing OTC drug manufacturers, announced that “the leading manufacturers of these medicines are voluntarily transitioning the labeling on oral OTC pediatric cough and cold medicines to state ‘do not use’ in children under four years of age.” This is a big step forward, because most bottles still say to “consult your pediatrician” before treating kids that age – implying that it is safe, as long as a pediatrician is guiding the dosing.

The American Academy of Pediatrics has been saying for years that these drugs do not work and are unsafe for young children. In fact, the AAP advocates a label saying they should not be given to anyone under the age of 6 as well as a ban on marketing them for that age range. Over 7,000 children a year are seen in emergency rooms for problems related to taking OTC cough and cold medications. The vast majority of them are under the age of 3, so even if pharmaceutical companies do not change their labels to match what pediatricians want, a label suggesting these drugs not be used for children under 4 will help a lot.

Why is this topic so important? Because it shows that just because a medicine works for adults doesn’t mean it works for kids. Too many drugs are marketed to kids without being tested on children, based on the assumption that kids are just small adults. Thankfully, Andrew von Eschenbach, the Commissioner of the FDA, in his own blog, recognizes that this just isn’t so. Hopefully, the future will be full of drugs that are safe to give to children because they are appropriately tested on children, with the same rigor that they are tested on adults. We owe this to our children.