New cancer drug for children safe, effective

A first-of-its-kind drug targeting a fused gene found in many types of cancer has been found to be effective in 93 per cent of paediatric patients tested, scientists say.
Most cancer drugs are targeted to specific organs or locations in the body.
 
Larotrectinib is the first cancer drug to receive government approval,breakthrough therapy designation for patients with a specific fusion of two genes in the cancer cell, no matter what cancer type.
"In some cancers, a part of the TRK gene has become attached to another gene, which is called a fusion," said an assistant professor.

"When this occurs, it leads to the TRK gene being turned on when it's not supposed to be and that causes the cells to grow uncontrollably.
"What is unique about the drug is it is very selective; it only blocks TRK receptors," said the lead author of the research.
Larotrectinib, targets TRK fusions, which can occur in many types of cancer.
While the TRK fusions occur in only a small percentage of common adult cancers, they occur frequently in some rare paediatric cancers, such as infantile fibrosarcoma, cellular congenital mesoblastic nephroma, and papillary thyroid cancer.
"Every patient with a TRK fusion-positive solid tumour treated on this study had their tumour shrink. The nearly universal response rate seen with larotrectinib is unprecedented," the author said.

TRK, short for tropomyosin receptor kinase, is a gene that plays a key role in brain and nervous system development and has a limited role in nervous system functions such as regulating pain in later life.

Larotrectinib belongs to a class of molecules known as kinase inhibitors, which work by cutting back on the enzymatic activity of a key cellular reaction, researchers said.

The selectivity of the drug means it does not cause the severe side effects associated with many traditional cancer treatments, and none of the patients with TRK fusions had to quit the study because of a drug-induced side effect, they said. 

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Precision cancer therapy effective in both children and adults

Three quarters of patients, both adults and children, with a variety of advanced cancers occurring in different sites of the body responded to larotrectinib, a novel therapy that targets a specific genetic mutation. Results of this multisite phase 1/2 trial have been published in the journal on February 22, 2018. Unlike most cancer therapies, this oral treatment is based on the genetic traits of the tumor and not the organ where the cancer originated. 
 
An acquired genetic defect, TRK fusions accelerate cancer cell growth. Larotrectinib is highly selective for inhibiting this process. Fifty-five patients, ranging from 4 months to 76 years of age, with 17 unique tumor types, were treated with larotrectinib. Three quarters of patients enrolled responded to therapy and 86% of responding patients remain on study or have undergone curative surgery. No patients discontinued treatment due to drug-related side effects.

Several pediatric patients that enrolled in the study had infantile fibrosarcoma, a type of cancer that harbors a TRK fusion and is difficult to treat since it responds poorly to chemotherapy. Radiation therapy is also not a good option since it has devastating long-term effects for young patients.

"This is truly a magic bullet for our patients with TRK-positive cancer," said a researcher, who helped design the pediatric part of the study. "In some cases, this cancer can be treated surgically - often requiring amputation or another disfiguring surgical procedure. After treating our patient with infantile fibrosarcoma with larotrectinib, the cancer shrunk sufficiently and we were able to surgically remove the tumor while preserving the patient's leg."

This study is part of a noteworthy drug development program. Typically, testing of new therapies in a pediatric population is done after the drug is licensed for adults, if at all. However, larotrectinib was simultaneously studied in children and adults. A special liquid formulation was developed for administering appropriate doses to very young patients. This early pediatric focus helped to accelerate clinical development by aiding in the rapid accrual of appropriate patients.

The government granted larotrectinib breakthrough therapy designation that resulted in an expedited review. Drugs may qualify as breakthrough therapies when preliminary clinical data indicate that the new treatment offers substantial advantages over existing options for serious or life-threatening diseases. 

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