Navigation Links
IU researchers report first effective treatment of tumors arising from common genetic disease NF1
Date:11/1/2012

Physician-researchers at Indiana University School of Medicine have reported the first effective therapy for a class of previously untreatable and potentially life-threatening tumors often found in children.

Announcing their findings in the online first edition of Lancet Oncology, the researchers said the drug imatinib mesylate, marketed as Gleevec as a treatment for chronic myeloid leukemia, provided relief to a significant number of patients with plexiform neurofibromas, tumors caused by neurofibromatosis type 1, or NF1.

"Although this was a small study, the results were significant, particularly given that such patients have had few treatment options for what can be a very debilitating disease," said first author Kent Robertson, M.D., Ph.D., associate professor of pediatrics. "We believe these findings warrant larger trials of both imatinib mesylate as well as other similar compounds that would appear promising in laboratory tests."

Affecting about 1 in every 3,000 children born, NF1 is the most common neurological disorder caused by mutations in a single gene. Including a much rarer related type, NF2, neurofibromatosis is a genetic disorder that is more prevalent than cystic fibrosis, Duchenne muscular dystrophy and Huntington's disease combined, according to the Children's Tumor Foundation.

The mutation produces a variety of symptoms, from mild to severe. Patients can develop caf au lait spots and disfiguring tumors on or just under the skin. Internally, tumors can develop along nerve tissue and cause problems if they begin to press against vital organs or the windpipe. Some patients suffer from chronic pain.

The tumors have been nearly impossible to treat effectively. Because they are relatively slow growing, they are not affected by radiation treatments or cancer chemotherapy drugs. They also are often not good candidates for surgery because they are dangerously close to vital organs.

In the study reported in Lancet Oncology, of 23 patients who received the drug for at least six months, six experienced a 20 percent or more decrease in the volume of one or more plexiform neurofibromas, and 30 percent of patients had improvements in symptoms.

Primary investigator D. Wade Clapp, M.D., Richard L. Schreiner Professor and chairman of the Department of Pediatrics, noted that even relatively small reductions in tumor size can result in significant relief of symptoms for patients, such as improved breathing and restoration of bladder control.

In earlier laboratory research, the researchers determined that Gleevec was effective in tissue culture and mouse models of NF1 tumors after discovering that a cellular signaling mechanism that Gleevec targets in chronic myeloid leukemia also played an important role in development of NF1 tumors. Reporting that finding in the journal Cell in 2008, the research team also reported that in a compassionate use protocol, they had treated a 3-year-old girl with a life-threatening tumor compressing her airway. The girl's tumor shrank by half within three months of treatment.

Although Gleevec has been widely used as a treatment for chronic myeloid leukemia and has been prescribed in some cases for long periods without serious side effects, 13 of the initial 36 patients enrolled in the IU study dropped out before their results could be analyzed at six months of treatment. Nine of the 13 left due to problems taking the drug or side effects. The authors noted that they used the previously established maximum tolerated dose for Gleevec for this study to ensure that any drug activity against any NF1 tumors would be observed. This approach was taken among patients who had been living with slow-growing tumors for long periods of time and therefore were less likely to accept drug side effects than patients with malignant tumors, the authors suggested. Dosages have been modified in subsequent studies.


'/>"/>

Contact: Eric Schoch
eschoch@iu.edu
317-274-7722
Indiana University
Source:Eurekalert  

Related medicine news :

1. OHSU researchers discover potential way to repair brain damage in multiple sclerosis
2. Penn researchers find error reporting improves perceptions of safety and may reduce incidents
3. Common Antidepressants Too Risky During Pregnancy, Researchers Say
4. Mechanism found for destruction of key allergy-inducing complexes, Stanford researchers say
5. Feinstein Institute researchers discover that bean used in Chinese food could protect against sepsis
6. Mayo Clinic researchers develop new tools to better treat ADHD patients in early stages
7. Moffitt Researchers find genetic predictors of fatigue for some prostate cancer patients
8. Penn Medicine researchers map strategy for choosing wisely on low-value health care services
9. Researchers ID potential patient population who may benefit from novel anti-platelet treatment
10. Moffitt researchers identify unique immune gene signature across thousands of patients solid tumors
11. Mount Sinai researchers identify predictors for inpatient pain
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
IU researchers report first effective treatment of tumors arising from common genetic disease NF1
(Date:2/11/2016)... ... February 11, 2016 , ... From March 4 through 6, ... Annual Meeting at the Walter E. Washington Convention Center in Washington, D.C. , ... condition of hyperhidrosis (excessive sweating) and its treatment options. Specifically, the company will ...
(Date:2/11/2016)... ... February 11, 2016 , ... Husted Kicking has completed its ... on February 6th & 7th, 2016 according to kicking coach Michael Husted. , “This ... to the NFL’s combine in Indianapolis,” says Husted. “The NFL uses a third party ...
(Date:2/11/2016)... , ... February 11, 2016 , ... The book, “Computers ... IT services, what questions to ask your IT consultant before signing a contract and ... your computer network. , “With companies relying heavily on e-mail and technology, it’s more ...
(Date:2/11/2016)... AZ (PRWEB) , ... February 11, 2016 , ... As ... with support of the Million Dollar Round Table Foundation (MDRTF), has gifted $10,000 to ... according to Colleen’s Dream Foundation President Billy Cundiff. , “We are honored to support ...
(Date:2/10/2016)... ... February 10, 2016 , ... The 9th annual meeting ... Congress (WMIC), will be held in New York City, NY on September 7 ... The congress will highlight and emphasize how imaging reveals a greater understanding of ...
Breaking Medicine News(10 mins):
(Date:2/11/2016)... The primary goal of this research is ... the usage of liquid biopsy. Key information the survey ... Timeframe of liquid biopsy adoption amidst future users - ... type - Sample inflow to conduct liquid biopsy tests ... and so on. - Correlation analysis of sample type ...
(Date:2/11/2016)... Calif., Feb. 11, 2016 PRO-DEX, INC. (NasdaqCM: PDEX) ... ended December 31, 2015. The Company also filed its Quarterly ... year 2016 with the Securities and Exchange Commission today. ... 31, 2015 --> --> ... increased $2.6 million, or 95%, to $5.4 million from $2.8 ...
(Date:2/11/2016)...  Kindred Biosciences, Inc. (NASDAQ: KIN ), a ... of pets, today announced the submission to FDA of ... Application (NADA) for Zimeta™ (dipyrone injection, KIND-012).  Positive topline ... for the control of pyrexia (fever) in horses were ... --> The Chemistry, Manufacturing, and Controls ...
Breaking Medicine Technology: