With approximately 350 new cases of rhabdomyosarcoma diagnosed annually in the US, Memorial Sloan-Kettering's pediatric sarcoma team sees approximately 18 newly diagnosed patients with the disease each year. While nearly three-fourths of those 350 patients have the more common embryonal rhabdomyosarcoma, most of the remainder have, like Nate, the less common alveolar variant. Approximately one patient in four with newly diagnosed rhabdomyosarcoma will have Stage IV rhabdomyosarcoma, as Nate did, although almost half of those with Stage IV rhabdomyosarcoma will have alveolar rhabdomyosarcoma.
Our Advantage
Thanks to a long history of treating rhabdomyosarcoma, Dr. Wexler notes that Memorial Sloan-Kettering's pediatric sarcoma team can offer patients a number of advantages. "These begin with our radiologists' ability to accurately interpret imaging studies with often subtle findings," he explains. The advantages extend into the availability of support staff who can quickly accommodate the need to perform imaging studies for patients, often while patients are under anesthesia. The Memorial Sloan-Kettering sarcoma team also includes pathologists, who can accurately diagnose rare tumors like aveolar rhabdomyosarcoma and who can order supporting molecular diagnostic testing -- especially important for such uncommon variants of the disease.
Dr. Wexler goes on to mention that pediatric sarcoma patients treated at Memorial Sloan-Kettering benefit from the expertise of radiation oncologist Suzanne Wolden, pediatric surgeons Michael LaQuaglia and Mark Kayton, and orthopedic surgeons John Healey, Patrick Boland, Edward Athanasian, and Carol Morris. "All members of teams that help me care for my patients," he says. Also playing a crucially important role in Memorial Sloan-Kettering's pediatric sarcoma team's success are the nurse practitioners, nurses, child-life staff, and volunteers. "Each of these team members is uniquely able to care for and cheer children with rare cancers while the children undergo highly toxic treatments," Dr. Wexler explains.
Investigational Approaches
"In Nate's specific case," says Dr. Wexler, "we were able to offer him treatment through a pilot clinical trial for children with advanced-stage, 'high-risk' rhabdomyosarcoma, incorporating seven -- rather than the standard three -- chemotherapy drugs, all with known activity against rhabdomyosarcoma, given in unique combinations, scheduling, and dosing." The trial, IRB 03-099, has generated sufficient preliminary information on the safety and feasibility of the strategy that the larger nation-wide cooperative group has adopted several of its key features for their current and future clinical trials.
When asked to describe Nate's response to treatment, Dr. Wexler is matter-of-fact: "His reaction was, for better and for worse, typical. Most children will respond promptly and dramatically to this therapy. Most children will, unfortunately, suffer terrible side effects with this therapy. Most children, thankfully, are resilient and live so much 'in the moment' that as soon as they are feeling better, they have 'moved on' and want merely to return to 'normal.'"
The Goldsteins are part of a large and extended family that was able to offer support not just to Nate but to his parents and siblings. "They belong to a community that seemed to have an infinite supply of volunteers to help with tasks ranging from the mundane -- grocery shopping -- to the heroic -- accompanying him to his seemingly never-ending daily radiation treatments," Dr. Wexler says. "Nate himself is a very sweet and very determined -- and very bright -- little boy. He understood that he was being made sick so that he could ultimately get better."
According to Dr. Wexler, at the time of his diagnosis, Nate's chances of responding to treatment were excellent. "Most children, even with advanced-stage rhabdomyosarcoma, will respond to the initial treatment, their cancers often entering into complete remission," notes Dr. Wexler. "Most children, however, will ultimately relapse." Half of these relapses occur within 15 to 18 months of the initial diagnosis, and three-quarters occur within two years of the initial diagnosis. Most children who remain relapse-free by three years from diagnosis will never have their cancers recur. "Now that he is more than 30 months from diagnosis and still in remission," Dr. Wexler says, "Nate's chances of being cured are significantly greater than his chance of recurring."
More than a Patient
But Dr. Wexler does not look at Nate as only a patient to be treated. "My personal feelings for Nate are profound," he says. "He is a beautiful, delightful, and charming little boy, whom I have come to love deeply. I love when he comes into my office and greets me with a hug and a kiss. I love when I see his bashful smile. I miss seeing him in clinic on a regular basis -- but I am thrilled that he's feeling well enough and is doing well enough to no longer need to be seen so often. I would not have missed, for all the money in the world, the opportunity to see him break a board with his "diseased" left leg at his martial arts dojo this summer! I am looking forward, God willing, to joining his family at his Bar Mitzvah in five years and two weeks -- actually on November 5, 2011, to be precise. Yes, it's already in my calendar. And I hope that I am still around and well enough to dance at his wedding. If he waits, as I did, until he's in his mid-30s, I plan to be there but I don't know if I'll be dancing very much!"
Dr. Wexler takes no personal credit for saving Nate's life, saying "I was one member of a team. While I will never understand, in theological terms, why Nate got sick, I believe nonetheless that I am merely a messenger who was permitted to bring about his healing. In this case, I was someone who was fortunate enough to work at a singular institution at a particular moment in time when I was able to offer a family and a little boy with a very serious case of cancer a unique option for treatment that was not available elsewhere."
"Although I am thrilled beyond words that Nate is doing so well," Dr. Wexler says in conclusion, "my satisfaction will not come until we either find a way to prevent these terrible cancers from developing in the first place, or until we find a far gentler and less toxic way of curing them."
