SEP 12, 2019 1:57 PM PDT

Infant with leukemia saved by liver cancer drug

A young child just months old recently successfully received cancer treatment for an uncommon type of leukemia. The drug used in the treatment was not a targeted leukemia drug but instead sorafenib, a drug typically used for treating adults with inoperable liver cancer and advanced kidney cancer.

The new research was published in the journal Leukemia and led by senior author Elliot Stieglitz, MD, a physician-scientist in the UCSF Division of Pediatric Hematology/Oncology and the Helen Diller Family Comprehensive Cancer Center.

The young infant, who is now a healthy toddler, had juvenile myelomonocytic leukemia (JMML), but did not respond to the typical standard of care of chemotherapy, so could not receive a stem cell transplant. In an attempt to look for alternative treatment options, Stieglitz’s team started to consider precision medicine based on genetic profiling. This move parallels a change in the way we are beginning to treat cancer on a greater level, with doctors considering therapy options based on the genes behind cancers not the kind of cancer itself.

In conducting a molecular profile of the infant’s cancer cells, the researchers found a mutation called an FLT3 fusion, which signifies the fusion of two genes together. This was a surprise, as it had yet to be discovered in pediatric cancers before – however, it opened up a new avenue for treatment. Dr. Mignon Loh, co-author of the paper and Chair in Pediatric Molecular Oncology explained:

"We know that fusions are more likely to respond to targeted therapies than other types of mutations. Sorafenib, which was developed at UCSF, is a type of targeted therapy known as a kinase inhibitor that works by blocking the action of an abnormal protein that signals cancer cells to multiply."

An infant with JMML was saved by a drug used for adults with liver cancer. Photo: Pixabay

And in fact, sorafenib was just the treatment that the child needed in order to gain enough strength to receive a stem cell transplant. The significance of this case, say the authors, can be extrapolated to other cases as well.

"The patient's history reveals that the one-size-fits-all treatment approach does not work well for all children with JMML," said Stieglitz. JMML affects approximately 1.2 million children annually. The authors hope their research will create a ray of hope for these youngsters and their families.

Sources: Science Daily, University of California - San Francisco

About the Author
  • Kathryn is a curious world-traveller interested in the intersection between nature, culture, history, and people. She has worked for environmental education non-profits and is a Spanish/English interpreter.
You May Also Like
DEC 10, 2019
Cancer
DEC 10, 2019
Using anthrax to fight cancer?
Surprising research published recently in the International Journal of Cancer says anthrax may act as a potential bladder cancer treatment. Yes, you read c...
JAN 15, 2020
Clinical & Molecular DX
JAN 15, 2020
Laser microchip picks up cancer markers in urine
A future where patients no longer need to endure expensive, painful and complicated cancer tests could soon become a reality. Researchers have developed a...
JAN 11, 2020
Cancer
JAN 11, 2020
Should we be concerned about talc powder and ovarian cancer?
After the outcry against baby powder and concerns regarding its link to ovarian cancer, still, no investigations have clearly linked the product to the dis...
JAN 16, 2020
Cancer
JAN 16, 2020
FLASH proton therapy: faster and more effective
A new technique called FLASH proposes a new type of radiation therapy. The technique is composed of an ultra-high dose rate of radiotherapy and uses electr...
JAN 21, 2020
Cell & Molecular Biology
JAN 21, 2020
Repurposing Existing Drugs to Treat Cancer
Drugs have to be rigorously tested before they can be offered to patients, so it can be much easier to find more than one application for a medication....
FEB 23, 2020
Cancer
FEB 23, 2020
Celular aging reexamined
New research published in Genes and Development has identified one of the mysteries of aging. According to scientists at Sanford Burnham Prebys Medical Dis...
Loading Comments...