A Legacy of Hope for Young Leukemia Patients

Oncology Live®Vol. 19/No. 17
Volume 19
Issue 17

Joseph V. Simone, MD, is best known for leading the research efforts that resulted in the first curative combination treatment for certain pediatric patients with acute lymphoid leukemia. Under his leadership, the St. Jude Children’s Research Hospital in Memphis, Tennessee, was designated the first and only National Cancer Institute cancer center dedicated to children. He was honored in the Pediatric Oncology category with a 2017 Giants of Cancer Care® award.

Joseph V. Simone, MD

In the 1960s, programs for treating leukemia were few and far between—especially for children. The disease was just beginning to be understood, and rules and regulations for taking care of both adult and pediatric patients were underdeveloped and inefficient, as the first treatment for systemic cancers had been developed only a decade earlier.

A key player who emerged during those early years of pediatric cancer care was Joseph V. Simone, MD. Most notably, he chaired the Department of Hematology and served as associate director for clinical research at St. Jude Children’s Research Hospital before becoming the director and CEO of the hospital from 1983 to 1992.

The Chicago native began his career in medicine as a resident at Presbyterian-St. Luke’s Hospital. Initially studying internal medicine, Simone soon discovered his interest in hematologic diseases. That led him to the Pediatric Department at the University of Illinois, which had a big commitment to blood disorders and was conveniently located down the block.

After years of training in hematology and oncology, Simone left Chicago to take a job at the St. Jude Children’s Research Hospital in Memphis, Tennessee—a position suggested by a friend who had passed on the opportunity.

Until this point, almost all children with leukemia died of their disease. But by cultivating a communication model to better reach the patients, Simone began to see the difference in treating children versus treating adults.

The level of complexity of the treatment changes when speaking with a child rather than to an adult, so “we use words and concepts that the kids will understand. It’s tricky, though, because you can scare them, the parents are scared—and sometimes I am scared. But I think [having] that group—parents, doctor, our superb nurses, and the patients involved—is something [for which] we do not provide enough training for people,” Simone admits.

St. Jude’s prominence grew with Simone’s reputation. After one year, he had reorganized the clinic and laid the foundation for the reputation of efficient, personalized, patient-based care that St. Jude is revered for today.

Patients are what drove Simone to make St. Jude a home away from home. Situated on the Mississippi River that serves as the state border, many of his patients lived in Arkansas and had to travel by bus or ask for a ride from neighbors just to get the treatment they needed. To alleviate this emotional and economic pressure, Simone and his colleagues developed a program where the hospital would pay for the families to stay in nearby hotels. This philosophy is demonstrated in the roots of St. Jude’s current financial assistance policy for which it is highly praised. The hospital vows to never turn away a patient because of the inability to pay.

One of these patients who lived “across the river” in Arkansas was one of Simone’s first patients who was cured. At 10 years old, the boy appeared to be cured, but Simone and his colleagues were careful to warn the mother that this may not be permanent. Simone continued to follow the patient’s progress on a regular basis, and the boy continued to move along, developing as a normal child would.

A Mentor

This case inspired Simone, motivating him to find cures for more children. “When you get one like that, you get very, very anxious to get more. That pushed us on,” he says.When asked about the great strides made in the 1960s and 1970s for childhood leukemia, Simone credits his mentor, Donald Pinkel, MD. Pinkel was the first director of St. Jude and has been cited as one of the preeminent physicians in the fight against childhood cancers.

“He was there before the building was built—he was already putting in the structure for taking care of patients,” Simone says of Pinkel. “He helped all of us youngsters learn how to conduct clinical trials so that we could find out what kind of treatment was better for that particular leukemia.”

Simone’s philosophies, medical and otherwise, have been formed by watching others. He cites working with seasoned physicians, listening to parents of children with cancer, and treating patients as the best lessons learned throughout his career.

One of the medical philosophies he followed came from Pinkel, which Simone said was best described as an attitude. Pinkel had a certain attitude regarding how physicians should handle and treat patients and their families, stating that both hope and truth are essential components of pediatric care.

Simone went on to serve as the associate director for clinical research. Together with his colleagues, Simone made great strides in the research field. Most notably, he developed the first curative combination treatment for select children with acute lymphoid leukemia, which led to the first patient with the disease to be successfully taken off therapy.

Success did not come without its challenges. Reflecting on his work, Simone recalls finding curative treatments. He shared his findings during a national meeting and was met with resistance from the community. This, he says, was because the subject of leukemia was taboo in the community; suggesting that there was a cure for these patients was unheard of. However, this did not stop Simone and the team at St. Jude.

“We pushed on, and we got some terrific results over the next several years, and people started calling us, saying that they wanted to come to St. Jude to see how we did this,” Simone says. “We had a whole trail of people from Germany, Poland, Japan—all over the world— who wanted to see how we could possibly get that result. That is really how it exploded.”

One of the biggest challenges of a pediatric oncologist, Simone says, is working with the patients and their families to understand the illness and outcome. “You must adapt,” he says. It is the doctor’s responsibility to help the patient understand that he or she is trying to help them, not hurt them. Additionally, Simone says, a large part of treating a child is counseling the parents and cultivating the skill in which to do that effectively. He has seen this very challenge drive people away from the field, but it is this relationship that he feels is integral to truly treating a child with cancer.

In 1983, under Simone’s leadership, the hospital was designated as the first and only National Cancer Institute cancer center dedicated entirely to children. The following year, St. Jude established what is now the world’s largest long-term follow-up clinic geared specifically toward pediatric cancer survivors. Other successes during his directorship included researchers’ finding the first 2 specific translocations known to cause pediatric acute lymphoblastic leukemia and developing individualized chemotherapy regimens based on patient tolerance.

By the end of Simone’s time at St. Jude, the once-dismal survival rate for acute lymphoblastic leukemia had reached 73%. Currently the survival rate for children with acute lymphoblastic leukemia sits in the 90% range.

Simone’s success in building St. Jude as a cancer center had institutions knocking at his door to help them do the same. After St. Jude, Simone was recruited to be the physician- in-chief of Memorial Sloan Kettering Cancer Center. From there his career path took him to the Huntsman Cancer Institute at the University of Utah, where he was the first senior clinical director, and then moved on to the Shands Cancer Center at the University of Florida. During these appointments, he also served as an external adviser for cancer centers such as Moffitt Cancer Center, University of Southern California Norris Comprehensive Cancer Center, Cleveland Clinic, and University of California San Diego Moores Cancer Center.

Away from the Clinic

Simone reflects on his time building these cancer centers with a chuckle, stating, “I assume I did a good job because they paid me.Away from the clinic, Simone has also shaped practice within the larger oncology community. He led the development of the American Society of Clinical Oncology’s Quality Oncology Practice Initiative, which is an oncologist-led, practice-based program. Additionally, he served on the editorial board for Journal of Clinical Oncology from 1982 to 1983, before serving as an associate editor from 1984 to 2001.

Other notable positions he has held over his career were medical director and chair of the National Comprehensive Cancer Network, president of the Association of American Cancer Institutes, and vice chair of the Pediatric Oncology Group. He has also served on the board of directors for the American Society of Clinical Oncology.

Although mostly retired, Simone currently serves as the president of Simone Consulting, which provides services including strategic planning, program structure, quality assessment, recruitment, and physical facilities. He continues to write his own column, “Simone’s OncOpinion,” about his observations of the current oncology space, with advice for physicians in the field.

Simone has undoubtedly left a mark on the pediatric cancer world during his career. From his time as a resident at the University of Illinois and director of St. Jude Children’s Research Hospital to his directorship of the Shands Cancer Center, Simone has parlayed his expertise in the laboratory and the clinic into leading the charge on building up cancer centers around the United States.

With over 50 years in oncology, Simone has had a professional career that has filled his garage with awards but he says nothing compares with interacting with the patients he has treated. Being able to watch his former patients go on to high school, college, and marriage is worth more than any medal, trophy, or ribbon.

A day has not passed when Simone has regretted his decision to leave internal medicine to treat children with cancer. “There is no question in my mind that my biggest impact in my career was developing treatments for childhood leukemia. Everything is dwarfed against that,” he says. “I think that having become a pediatric oncologist was a godsend for me.”

Although he says that his entry to pediatric oncology was a “quirk,” he knew that he could make the biggest impact on children dealt the unluckiest of hands.

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