Emily faced one harrowing decision after another. Her 4-year-old son, James, had been diagnosed with stage 4 high-risk neuroblastoma, a rare cancer of nerve cells. James immediately began chemotherapy at a hospital in the Washington, D.C., area, where they lived.
Within a few days of diagnosis, however, Emily connected with well-known pediatric cancer advocates, who urged her to seek treatment at Memorial Sloan Kettering Cancer Center (MSK).
“I was thinking, ‘You have to be kidding me — I’m in the nation’s capital, why do I have to go to New York? Why do I have to leave my young daughter?’ ” Emily recalls. “But they kept saying, ‘MSK is where you need to be for neuroblastoma treatment.’ ”
The advocates recommended MSK pediatric oncologist Shakeel Modak, MD. When Emily saw that his team was spearheading the latest neuroblastoma research and clinical trials, she quickly agreed.
“Dr. Modak treated James’ case — as he does all his cases — with urgency,” she says. “He did a Zoom session with us from India, where he happened to be at that time, and walked us through our options,” she says. “He’s the most dedicated man alive.”
Dr. Modak said James could finish his initial chemotherapy in Washington but should come to MSK for surgery — which would be extensive — and continue his frontline treatment there. After meeting the surgical team, including pediatric surgeon J. Ted Gerstle, MD, Emily and her husband, Blake, say they had no doubt that MSK was their best shot at removing James’s complex, high-risk tumor.
Emily and Blake relocated to the New York area in 2021, at the height of the COVID pandemic, leaving their 18-month-old daughter in the care of her grandparents so James could be treated by the pediatric specialists at MSK Kids.
The disruption turned out to be worth it. At MSK, James had access to new therapies that have improved the prognosis for neuroblastoma, including a monoclonal antibody drug called naxitamab and a cancer vaccine being tested in a clinical trial.
Surgeries To Remove Neuroblastoma
Dr. Gerstle successfully performed two 12-hour surgeries to remove the primary tumor and all other soft tissue disease. Then James underwent multiple rounds of chemotherapy, including an additional high-dose round to clear the remaining neuroblastoma cells.
But it didn’t eliminate all the disease. Despite the aggressive treatment, James still had 20 spots of neuroblastoma throughout his bones and bone marrow.
At most hospitals, the preferred option was more chemotherapy — a devastating prospect for a young child who had already undergone so much. But Dr. Modak had another plan. Even though neuroblastoma is rare, MSK doctors had seen many cases, allowing them to use treatment data to understand the significance of subtle changes in the cancer’s behavior. In James’s disease, the chemotherapy appeared to have altered the neuroblastoma to a more mature form called ganglioneuroma.
“Dr. Modak told us, ‘We have the data, and chemotherapy isn’t going to do anything for James’s disease,’ ” Emily says. “He was able to make a risk assessment, including the potential toxicity of staying on that path. When you’re at a cancer center like MSK, having this level of data and analysis really helps you make better decisions.”
Access to a New Neuroblastoma Immunotherapy Treatment Using Humanized 3F8
Dr. Modak switched James to treatment with humanized 3F8, a monoclonal antibody drug called naxitamab (Danyelza®). The antibody works by stimulating the patient’s immune system to destroy residual neuroblastoma cells. Humanized 3F8, conceived and developed by MSK physician-scientist Nai-Kong Cheung, MD, PhD, had been tested in phase 1 and 2 trials at MSK and had received approval from the U.S. Food and Drug Administration (FDA) just a few months earlier.
The results for James were dramatic. After several rounds of naxitamab, the ganglioneuroma lesions disappeared, and James was declared to have no evidence of disease (NED) — the best news a cancer patient can receive.
Using a Cancer Vaccine To Prevent Neuroblastoma From Recurring
Even though naxitamab appeared to have erased the cancer, Dr. Modak wanted to do everything possible to prevent the high-risk neuroblastoma from returning. He proposed that James receive a pioneering treatment: a cancer vaccine that would train his immune system to continuously be on guard to spot and fight malignant cells. He recommended that James consider a clinical trial led by MSK pediatric hematologist-oncologist Brian H. Kushner, MD, which was testing the vaccine in high-risk neuroblastoma patients.
Because James seemed well, it was not easy for his parents to contemplate putting their son through another medical intervention. But neuroblastoma has a high relapse rate, and they were afraid the disease could come back. Dr. Modak said the vaccine was the best insurance to prevent that.
“With any novel treatment, there are always risks, and it’s hard being a pioneering mother giving a child a cancer vaccine when there is uncertainty,” Emily says. “No matter how hard you research things as a parent, at a certain point, your ability to assess things is going to hit a dead end, and you have to trust your oncologist. Dr. Modak never oversold anything to us about the antibody treatment or the vaccine. When he was optimistic, we believed it.”
How Cancer Vaccines Work
Cancer vaccines are based on the powerful idea that the immune system can be trained to seek and destroy cancer. They hold the greatest potential for helping patients like James, who may have lingering cancer cells after their tumors have been removed. A cancer vaccine works by exposing the immune system to specific substances produced by a patient’s tumor, called antigens. Once the immune system sees the antigens, it will attack them and keep the cancer at bay.
The momentum for cancer vaccines has picked up speed in recent years as the science has evolved, and MSK is playing a leading role in the field. In 2024, a generous gift led to establishment of The Olayan Center for Cancer Vaccines (OCCV) at MSK. MSK surgeon-scientist Vinod P. Balachandran, MD, Director of the OCCV, has played a key role in the development and testing of one of the most hopeful examples of this type of therapy: a vaccine for treating pancreatic cancer based on mRNA.
The Advantages of Cancer Vaccines
One advantage of a cancer vaccine compared with other treatments is that it can be given over a brief period and then not needed again, because the immune system learns to fight on its own. In addition, the side effects of cancer vaccines are less than for chemotherapy, surgery, and radiation.
These benefits helped persuade Emily and Blake to enroll James in the vaccine trial. “We thought the vaccine could provide a long-term benefit without piling on more side effects, which would give James a chance to thrive after finally finishing all the treatments,” Emily says.
How the Cancer Vaccine Is Given to Patients
The clinical trial was testing a “bivalent” vaccine, which works by triggering an immune response against two unique antigens on neuroblastoma cells — sugar-lipid molecules called glycolipids. The cancer vaccine was given with an oral yeast-derived agent called beta-glucan that researchers believe enhances the immune response. Preclinical testing on beta-glucan had been carried out by Drs. Modak and Cheung at MSK several years earlier.
James joined the trial in 2022 and got 10 shots over the next three years. Every time he got a shot, he consumed beta-glucan once a day for two weeks. The main side effects from the cancer vaccine were temporary pain and inflammation at the injection site, which were much easier to tolerate than James’s earlier treatments. He received the final shot in April 2025.
James Is Thriving After Neuroblastoma Treatment
Today James is about to turn 9 and is thriving. He loves being outside and is very active — playing lacrosse and skiing, camping, and fishing. He recently joined an elite flag football league in the Washington area called Flagstar.
“I’m really proud of him, remembering how he used to get weak after just a few minutes, and now he’s a monster on the field,” Emily says. “It was a huge milestone for his healing. And a big part of that was MSK’s emphasis on avoiding toxicity. I want him to feel like he has his childhood back and that he can chase after dreams just like any other kid.”
Benefiting From Donations to Rare Cancer Research
Emily credits her son’s successful outcome to a group of parents who formed a unique organization to partner with MSK’s neuroblastoma team almost 20 years ago. The group, called the Band of Parents, funded the majority of the humanized 3F8 research and continues to be a top supporter of the neuroblastoma team at MSK. Band of Parents members across the country also participate annually in Cycle for Survival (MSK’s fundraising program for rare cancers) and in many cities build some of the leading Cycle for Survival teams. The Band of Parents in now the lead funder of James’s vaccine trial, and their Cycle for Survival teams direct their fundraising dollars to the vaccine.
“The parents that came before us, and the people who donated, ultimately saved James’s life,” Emily says.
After understanding the critical role Band of Parents played in funding humanized 3F8, Emily joined the board of the organization to continue its essential work in propelling cutting-edge research and clinical trials for neuroblastoma.
With the encouragement of Emily’s friend, Lynn, whose father was treated at MSK, the family started a Cycle for Survival team under the name Team SuperheroJames and has raised more than $600,000 since they began riding in 2021. Team SuperheroJames has coordinated with Band of Parents’ Cycle for Survival teams to donate all funds they raise to the neuroblastoma vaccine trial and related research. One hundred percent of funds raised through Cycle for Survival goes to cancer research at MSK.
A Mother’s Message for Families With a Pediatric Cancer Diagnosis
Emily wants others facing a cancer diagnosis to be aware that many organizations can be found easily on the internet to help with fundraising for treatment costs, coordinating care, and providing other assistance. “People should consider these organizations before they decide not to pursue treatment somewhere simply because it’s out of state,” she says.
Most of all, Emily is grateful for the dedication of the MSK pediatric neuroblastoma experts.
“A lot of people have said to me, ‘It’s a miracle James is OK now,’ ” she says. “I would say the miracle is that there are people like Dr. Modak, Dr. Cheung, Dr. Gerstle, Dr. Kushner, and the others who have made this their life’s work. They are not willing to stop at outcomes that are just OK but are relentless in pursuing what is best. That’s why James is here.”
Cancer Vaccine Could Prevent Neuroblastoma From Returning After Antibody Treatment: Key Takeaways
- Neuroblastoma is a rare cancer of the nerve tissue that usually arises in the abdomen. It mainly affects young children.
- A drug developed by researchers at MSK Kids called humanized 3F8 was approved by the FDA in 2021. It is effective at treating high-risk neuroblastoma.
- MSK is now conducting a clinical trial testing a vaccine that could reduce the risk of neuroblastoma returning after treatment.
