Research paves the way for safer, more effective treatments for children with aggressive blood cancers

Australian nanomedicine researchers have devised a new approach to solving the decades-old clinical problem of targeting therapeutic drugs to target cancer cells in the body.Published in Impact Journal this week scientific translational medicineThe study paves the way for safer and more effective treatment options for children who may develop other types of cancer, not just aggressive blood cancers.

Chemotherapy is the mainstay of treatment for leukemia, the most common blood cancer in children. However, while chemotherapy is very effective against certain types of leukemia, it is less effective against other types known as “high-risk” leukemia. Treatment of high-risk leukemia typically involves administering large doses of toxic drugs into the body, which indiscriminately affect both cancer and healthy cells. This often causes serious side effects and lifelong health problems in survivors.

Finding ways to make therapeutic agents more selective to cancer cells is key to improving treatment success rates while reducing toxicity in children treated for high-risk leukemia. Targeting leukemic cells specifically could not only make the treatment more effective, but also make it safer for use in children. “

Professor Maria Cavalaris AM, Director of Translational Cancer Nanomedicine, Children’s Cancer Institute

In a newly published study, scientists from the Children’s Cancer Institute and collaborating institutions used a special formulation of the anticancer drug doxorubicin (Caelyx). The drug is encapsulated in tiny particles called liposomes. To this formulation they added a “bispecific antibody”. This is an antibody that can recognize a drug on one end and a cancer cell on the other end and bind to it, effectively acting as a bridge between the two. Known as a “targeted drug delivery system”, it functions to deliver a drug to its target, in this case a leukemic cell, where the drug can do its job and kill the cell.

“What makes this new approach particularly useful is its flexibility,” explains lead author Dr. Ernest Molds, a research fellow at the Children’s Cancer Institute. “We can use this system to target any leukemia, including the high-risk subtypes that kill Australian children each year, without having to design a completely new treatment each time. Instead, we just change the cross-linking of the antibody, targeting the same drug against blood cancers in every child.”

“Furthermore, this approach has the potential to counter drug resistance in individual patients. , can make it recognizable.” Altered cancer cells. You won’t be able to escape easily. “

This new approach was shown to work well not only in laboratory-grown leukemia cells, but also in live models of the disease. In these models, targeted drug delivery systems not only reduced leukemia burden, but also significantly increased survival (up to 4-fold in some cases).

Researchers believe that this same approach could be used to improve the selectivity of not just chemotherapy drugs, but any new generation of therapeutics, offering far safer alternative treatment options than those currently offered. I think it will open the way to provide for children. They are also excited about its potential contribution in the early days of precision medicine.

“In the future, each child diagnosed with leukemia may receive targeted treatment for a specific subtype based on the analysis of blood samples,” said Kavalaris. .

“We believe the controlled targeting of nanotherapeutics is a true milestone in the treatment of childhood cancers, and we are very optimistic about what this will bring.”

Matt Weston and his son Jacob, who is currently undergoing treatment for acute lymphoblastic leukemia, are well aware of the importance of this research. “I’ve been following this research project closely, and the work of Maria, Ernest, and the Children’s Cancer Institute team really surprised me,” said Professor Matt.

“Having experienced first-hand how toxic chemotherapy can be through my son’s journey, I can tell you some scary stories of side effects, even with a standard risk diagnosis.” , the treatment is not only much more toxic, but they are also much less likely to survive.

“I am always thinking of those children and their families. It gives me great hope.”

Jacob has already undergone several cycles of chemotherapy in the past eight months and is experiencing a variety of debilitating side effects. But we’re not done yet. He will need another 16 months of maintenance therapy, after which it will take another three years to fully recover. “There’s a long way to go,” says Matt.You can read more about Jacob’s story here.

The research includes several institutions including Children’s Cancer Institute, University of Queensland, University of New South Wales Sydney, University of Western Australia, Curtin University, Prince of Wales Hospital, St Vincent’s Hospital, Perth Children’s Hospital, Telethon Kids Institute and others. agencies cooperated. Key funding bodies include the National Health and Medical Research Council, Cancer Council NSW, Tour de Cure and others.