4-year-old donates blood cells to fight neuroblastoma

Neuroblastoma accounts for six percent of all cancers in children, and it's deadly in kids under 5.

Many times, the cancer has spread to the lymph nodes before parents even notice a lump.

Meet Cashel, a boy who fought the disease for two years and is now helping others.

“He had chemo. He had stem cell transplant too, which is high-dose chemo too. They rescue it with their own stem cells,” explains Cashel's mom, Alita Conoley-Wurzbach.

Plus 12 rounds of radiation and immunotherapy. The cancer was first discovered as a lump on his neck, but the primary tumor was a grapefruit-sized one on his stomach.

“He had his primary tumor removed. That was a 14-hour surgery here at Texas Children’s,” says Conoley-Wurzbach.

This trip, Cashel donated his own blood cells to boost the research.

Dr. Heczey is looking at genetically engineering natural killer T cells, which help fight neuroblastoma.

“What if we genetically engineer T cells to specifically attack the tumor cells, as well,” Heczey suggests.

The clinical trial is evaluating a therapy called CMD-501, in which the patient’s natural killer T cells are genetically modified in the lab to better attach to tumors. But for now, Cashel and his mom have a feeling of:

“Empowerment, because there is so little control you have over as a parent with a sick child,” Conoley-Wurzbach says.

Cashel is a little guy who is now healthy and helping others beat this tough disease.

The study uses natural killer T cells to fight neuroblastoma, and Texas Children’s Hospital in Houston is still recruiting participants.

MEDICAL BREAKTHROUGHS
RESEARCH SUMMARY

TOPIC: 4 YR OLD CASHEL DONATES BLOOD CELLS TO FIGHT NEUROBLASTOMA
REPORT: MB #4550

BACKGROUND: Neuroblastoma is a form of cancer that develops from immature nerve cells found in several areas of the body. Most commonly it is found in and around the adrenal glands, which sit atop the kidneys. It can also develop in other areas of the abdomen, neck and near the spine, and in the chest. Most commonly it affects children age 5 or younger, though it may rarely be found in older children. Signs and symptoms vary depending on which part of the body is affected. The most common form, in the abdomen, may cause symptoms such as abdominal pain, a mass under the skin that is hard to the touch, and changes in bowel habits (constipation or diarrhea). In the chest there may be pain, wheezing, or changes to the eyelids and eyes. Other signs and symptoms that may indicate Neuroblastoma can be lumps of tissue under the skin, dark circles around the eyes, fever, unexplained weight loss, back pain, proptosis or eyeballs that seem to protrude form the sockets, and bone pain.
(Source: https://www.mayoclinic.org/diseases-conditions/neuroblastoma/symptoms-causes/syc-20351017)

TREATMENT: The treatment of Neuroblastoma depends on a number of factors, including the risk group of the cancer, the child’s age, etc. It may include more than one type of treatment. Some options include surgery, chemotherapy, and radiation therapy. Doctors may use regular imaging tests to be sure the tumor is going away or does not get bigger. Children at low risk usually don’t need very intensive treatment; in fact, some might not need treatment at all because some of these neuroblastoma will mature or go away on their own. If the tumor can be easily removed, even if some is left behind, the child can be watched carefully without further treatment because the remaining tumor will often mature and go away on its own. Children on the high- risk end of the scale require more aggressive treatment; they may require a stem cell transplant, immunotherapy, or retinoid therapy.
(Source: https://www.cancer.org/cancer/neuroblastoma/treating/by-risk-group.html)

NEW TECHNOLOGY: Researchers are developing a new strategy to treat patients with neuroblastoma. The Geno Kit 2 study is focusing on natural killer T cells or NKT cells for short. These are a small subset of cells in the blood, which carry some anti tumor properties. Researchers discovered they can traffic specifically to Neuroblastoma sites, attacking the white blood cells inside the tumor cell called tumor associated macrophages or TAMs. These TAMs support the growth of the neuroblastoma, creating a more nourishing environment for it to thrive. Therefore, NKT cells indirectly can alter the neuroblastoma ability to grow. The team is working to genetically engineer NKT cells to specifically attack the tumor cells.
(Source: Andras Hecczy)