ddn Online

The Blog of Drug Discovery News

Research efforts can help fragile young lives

Whether we are researchers or journalists, as we explore the landscape of the drug development world we are reminded just how fragile human life can be.

This week, I came across a story that a team of pediatric cancer researchers have identified variations in a gene as important contributors to neuroblastoma, the most common solid cancer of early childhood.

The study team, led by researchers at The Children’s Hospital of Philadelphia, found that common variants in the LMO1 gene increase the risk of developing an aggressive form of neuroblastoma, and also mark the gene for continuing to drive the cancer’s progression once it forms.

The team’s work appears online in Nature. According to their report, a cancer of the sympathetic nervous system that usually occurs as a solid tumor in the abdomen, neuroblastoma accounts for 10 percent of childhood cancer deaths.

According to its website, The Cancer Center at The Children’s Hospital of Philadelphia cares for more children with cancer than any other pediatric hospital in the United States. It is ranked second in children’s cancer care in the U.S. by U.S.News & World Report.

It is so sobering to think that the youngest and most vulnerable—our children—sometimes are engaged in a fight for their lives as they battle myriad issues that come with adult-sized problems.

The researchers found a significant association between neuroblastoma and the LMO1 gene, located on chromosome 11, detecting the strongest signal among patients with the most aggressive form of the disease. This portion of the study identified SNPs, changes in a single letter within the DNA sequence, which predispose a child to developing neuroblastoma.

The research team utilized genetic tools to decrease LMO1’s activity, and showed that this inhibited the growth of neuroblastoma cells in culture. Increasing LMO1 gene expression had the opposite effect, causing tumor cells to proliferate.

Because other genes in the LMO family are known to be active in acute leukemias, other researchers have been investigating potential anti-leukemia drugs to target portions of the LMO pathway.

This is just one example of all of the great work going on in labs in hospitals, academic institutions and other facilities in the United States and around the world.

The research offers great hope for children battling cancer. It also is further proof that the work being done in research labs around the world are yielding results that can take steps to eradicate the diseases that take a great toll on us emotionally and physically, regardless of sex, age or race.

Further, the study by researchers at The Children’s Hospital of Philadelphia shows how we can expand our knowledge base for translating genetic discovery to clinical issues through integrative genomics, combining SNP discovery arrays with gene expression arrays and other functional approaches.

With the great results coming from this work, perhaps the suffering of some of the small and meek can be eased.

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December 7, 2010 - Posted by | Academia & Non-Profit, Announcements and Events, Labwork & Science, Uncategorized

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