Medhealth Review

Drug Combination Could Be Effective for Rhabdomyosarcoma. But are Clinical Trials Possible for Humans?

A new study by NCI in mice could be positive for kids suffering from Rhabdomyosarcoma. The term refers to particular cancer affecting the soft tissues and muscles. 

The trial in mice involved two different drugs, which, when used in combination, could reduce the growth of cancer cells. The two drugs are ganitumab, an experimental drug, and trametinib, the targeted drug.

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The positive outcome has compelled researchers to try the same combination in humans. However, there was an unexpected delay. One of the drugs, ganitumab, was discontinued after it failed to be effective in a massive study on patients suffering from Ewing Sarcoma. However, the researchers highlight that the drug was stopped due to failure in adults and not in children.

Cancer in children is different as compared to adult cancers. So, drugs that may be less effective for adults may positively impact children, mainly when combined with other medications. 

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“Every year, a couple of drugs are put on the shelf that we would have been interested in continuing development of, but because the adult study failed, there’s no path to move forward. If you’re a drug company, developing a drug for a pediatric indication may not make financial sense because pediatric cancers are so rare. And that’s the harsh reality,” Douglas Hawkins, M.D, Group Chair, NCI-funded Children’s Oncology Group.

The remaining stock is expired. The researchers are left with no options but to look for alternative options. The other studies involving the drug have been halted as well. 

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