Archive for the ‘Orphan Cancers’ Category

Drug Development for Rare Cancers To Speed Up With New Strategy

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Posted 19 Sep 2011 — by James Street
Category Drug Testing, Drugs, ependymoma, Orphan Cancers, Osteosarcoma
by Tanya Thomas on  September 18, 2011 at 5:59 PM (Cancer News)

Researchers have identified promising new therapies for ependymoma, a rare tumor with few treatment options. St. Jude Children’s Research Hospital investigators led the effort, which used a new, faster drug development system that combines the latest drug screening technology with the first accurate animal model of the tumor.

Investigators identified several dozen new and existing drugs as possible ependymoma treatment candidates. The drugs were found by screening 5,303 existing medicines, natural products and other compounds for activity against the tumor, which develops in the brain and spine of children and adults. The work is published in the current edition of the scientific journal Cancer Cell.

The list of candidate drugs included 5-fluorouracil (5-FU). 5-FU has been widely used to treat a variety of adult cancers but has not been formally tested against ependymoma. Based on study results, St. Jude is planning a clinical trial of 5-FU in young ependymoma patients, said senior author Richard Gilbertson, M.D., Ph.D., director of the St. Jude Comprehensive Cancer Center. Gilbertson credited the method used in this study with highlighting 5-FU’s potential.

Researchers hope to use the same system to expand chemotherapy options for patients with other cancers. “This approach should significantly advance the efficiency and speed with which we discover and develop new treatments for rare cancers and cancer subtypes,” the investigators noted. Jennifer Atkinson, Ph.D., a former St. Jude postdoctoral fellow, is the first author. R. Kiplin Guy, Ph.D., chair of the St. Jude Department of Chemical Biology and Therapeutics, and Gilbertson are corresponding authors.

New study will use dual approach to find mutations in osteosarcoma

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Posted 13 Jun 2011 — by James Street
Category Bioinformatics, Circulating Tumor Cells, Etiology and cause of osteosarcoma, genetic research, genetic research, Orphan Cancers, Osteosarcoma Outcomes

A new United Kingdom-based osteosarcoma study will use advances in genomic research and analysis to identify genes that give rise to the condition — as well as create personalized blood tests for children and young adults. Results of the study, according to the Wellcome Trust Sanger Institute press release, may assist physicians in improving osteosarcoma treatment and the creation of improved methods in the measurement of disease regression.

“We hope that this research project will improve the way patients with cancer are monitored and will guide the best drug treatment for the cancer in each patient,” Adrienne Flanagan, MD, PhD, medical director of the Royal National Orthopaedic Hospital, stated in the release.

The new study will will look for tumor-specific DNA in the bloodstream of patients. The complete genome of 50 osteosarcoma patients will be sequenced, and researchers will look through the patients’ plasma before and after chemotherapy treatment to find patient-specific rearrangements in the DNA that leaks from the osteosarcoma into the bloodstream.

Developing a picture of the unique profile of mutations of each patient’s cancer and then using these mutations to monitor the amount of cancer-derived DNA circulating in the blood is what the clinicians hope will enable them to deliver treatments to patients in a personalized fashion. Researchers are also looking for novel genes giving rise to osteosarcoma.

Patients in the study are being treated at the Royal National Orthopaedic Hospital, as well as the University College London Hospital. Research is being performed in collaboration with the University College London Cancer Institute, as well as the Wellcome Trust Sanger Institute.

Reference:

A comprehensive candidate gene approach identifies genetic variation associated with osteosarcoma

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Posted 29 May 2011 — by James Street
Category Epigenetics, genetic research, genetic research, Orphan Cancers, Osteosarcoma, SNP variations

Osteosarcoma (OS) is a bone malignancy which occurs primarily in adolescents. Since it occurs during a period of rapid growth, genes important in bone formation and growth are plausible modifiers of risk.

Genes involved in DNA repair and ribosomal function may contribute to OS pathogenesis, because they maintain the integrity of critical cellular processes. We evaluated these hypotheses in an OS association study of genes from growth/hormone, bone formation, DNA repair, and ribosomal pathways.

Methods: We evaluated 4836 tag-SNPs across 255 candidate genes in 96 OS cases and 1426 controls.

Logistic regression models were used to estimate the odds ratios (OR) and 95% confidence intervals (CI).

Results: Twelve SNPs in growth or DNA repair genes were significantly associated with OS after Bonferroni correction. Four SNPs in the DNA repair gene FANCM (ORs 1.9-2.0, P=0.003-0.004) and 2 SNPs downstream of the growth hormone gene GH1 (OR 1.6, P=0.002; OR 0.5, P=0.0009) were significantly associated with OS.

One SNP in the region of each of the following genes was significant: MDM2, MPG, FGF2, FGFR3, GNRH2, and IGF1.

Conclusions: Our results suggest that several SNPs in biologically plausible pathways are associated with OS. Larger studies are required to confirm our findings.

Author: Lisa MirabelloKai YuSonja BerndtLaurie BurdettZhaoming WangSalma ChowdhuryKedest TeshomeArinze UzokaAmy HutchinsonTom GrotmolChester DouglassRichard HayesRobert HooverSharon Savagethe National Osteosarcoma Etiology Study Group
Credits/Source: B

More Wilms Tumor Cases in Saint Clair County

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Posted 13 May 2011 — by James Street
Category Big Pharma, Clinical Trials, Ethics of Science, Finance and Politics of cancer research and treatment, Orphan Cancers

Updated: Thursday, 12 May 2011, 7:14 PM EDT
Published : Thursday, 12 May 2011, 7:10 PM EDT

By ROBIN SCHWARTZ
WJBK | myFOXDetroit.com

MARINE CITY, Mich. (WJBK) – The cancer survivors are young, beautiful, innocent children all born healthy, but suddenly thrust into a battle for their lives.

Their mothers recently spoke out a Marine City Commission meeting demanding answers.  In the last four years, five children, all from Marine City, have been diagnosed with the rare kidney cancer Wilms Tumor. The youngest, six-month-old Ireland Kulman, was just diagnosed in March.

Each year, only about 500 children in the U.S. get this cancer.  The odds of this happening in a city of 5,000 people are astronomical.

“The odds are just about one in 500-trillion, which to put that into perspective, that would be like buying the winning Mega Millions ticket two weeks in a row,” said math professor Scott Anderson.

Now, the Saint Clair County Health Department has said it’s not just Marine City.  They’ve confirmed three new Wilms Tumor cases, two in the Port Huron area, and one more in Richmond on the border of Saint Clair and Macomb counties.

FOX 2 also got a call from Carole Phillips, who lives in New Baltimore just 15 minutes away.  Her daughter is also a Wilms Tumor survivor diagnosed in 2005.  It’s not yet clear if her case is related.

“I firmly believe that there is a link.  The likelihood of it being a coincidence, I think, is non-existent,” she said.

“It breaks my heart and it blows my mind that there (are) so many cases,” said Kris Tranchemontagne.

She is one of the Marine City moms.  Her daughter was diagnosed in October 2008.  She said officials recently tested the area drinking water, but nothing unusual was found.  The troubling mystery remains.

So, where does the problem lie?  In the air?  In the water?  Somewhere else in the environment?  No one knows for sure, but the families immediately look with suspicion just a few miles north to Sarnia, Ontario, also known as “chemical valley.”

“They make 40-percent of the petrochemicals for all of Canada,” said St. Clair Channel Keeper Doug Martz.

“They’re definitely dumping chemicals.  There (are) spills constantly,” said Danielle Williams.

Her ten-year-old daughter is another Wilms Tumor survivor.  She brings an enlarged picture of “chemical valley” with her to every meeting.  It’s a place the Problem Solvers have highlighted before back in 2003 after the big blackout.  We reported on a massive spill of the cancer causing chemical vinyl chloride that went undetected for days.

The families are calling on the federal government to join the investigation.

The total number of local Wilms Tumor cases could continue to grow as more families come forward.

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Dr. Jeffrey Taub, director of Hematology and Oncology at Children’s Hospital, joined Huel Perkins to talk about this story.  Click on the second video in the player above to watch their conversation.