Neuroblastoma Awareness: Hero: Baby Aerabella

Hi, My name is Danica. My daughter, Aerabella was diagnosed on Sept 22nd
2011 at 9 months old with stage 2A Neuroblastoma. She has a large tumor in
her chest that was found due to an odd cough. Her family doctor insisted a
chest x-ray be done at the Akron Children's Hosptial. We thought she may
have eaten something and had it lodged. Imagine our shock when we were shown
an x-ray with a mass approx 5cm*3cm*2cm in her chest. She was admitted to
the oncology floor later that night. We spent 13 long days and nights at the
hospital while numerous tests and procedures were completed. Bella had her
port placed Sept 23rd and started Chemo that night. She has three more
cycles of Chemo to go. Then hopefully surgery to remove the remaining tumor.
We were very lucky to have caught this early and her prognosis is very good.
She has been doing quite well, the chemo seems to have given her more energy
and made her hyper. She is at the stage where she loves getting
into everything and exploring new things.She has her own facebook page and
has added neuroblastoma awareness in hopes to make her friends all aware of
this horrible cancer. Her address is facebook.com/babyaerabella

Neuroblastoma Awareness: ALK gene discovered by St. Jude scientists associated with FDA approved adult cancer drug

A drug recently approved by the U.S. Food and Drug Administration for treatment of an adult cancer targets a malfunctioning gene discovered more than a decade earlier at St. Jude Children's Research Hospital. The story highlights how scientific findings from St. Jude can be translated into therapies and tests that in addition to helping children, also help adults.

The drug is Xalkori (crizotinib). The FDA approved Xalkori in August as the first targeted therapy for patients with ALK-positive non-small cell lung cancer (NSCLC) that is locally advanced or metastatic. Xalkori is manufactured by the pharmaceutical company Pfizer.

The ALK gene was discovered by St. Jude scientists searching for genes affected by a chromosomal change common in the cancer cells of patients with anaplastic large cell lymphoma (ALCL).The blood cancer accounts for 10 to 30 percent of pediatric non-Hodgkin lymphoma. In 1994 Stephan Morris, M.D., then a St. Jude junior faculty member; Thomas Look, M.D., then chair of a St. Jude department; and their colleagues, published the first of several reports detailing the discovery of ALK and the gene's pivotal role in driving the cancer. ALK is short for anaplastic lymphoma kinase, the name investigators gave the protein whose assembly instructions the gene carried.

ALK is now widely recognized as a potent promoter of several adult and childhood cancers, including ALCL and neuroblastoma, a childhood tumor of certain nerve cells. The work done by Morris, Look and their colleagues in a fifth-floor laboratory of the Danny Thomas Research Center eventually helped to launch a new targeted cancer treatment.

Hiroyuki Mano, M.D., of the University of Tokyo, led the 2007 research into the molecular drivers of NSCLC. The study showed some NSCLC tumors were driven by an ALK rearrangement. Following this discovery, Pfizer expanded a Phase I clinical trial of Xalkori to include patients with ALK-positive advanced NSCLC. Xalkori blocks the cancer-causing activity of the ALK protein. Additional clinical trials with the drug are now underway in other cancers, including neuroblastoma and ALCL, the lymphoma that launched the search.

The ALK discovery and related later research led to five U.S. patents for St. Jude. The patented work includes methods for detecting the chromosomal rearrangements that unleash the cancer-causing ability of the ALK gene as well as tools to identify and characterize drugs for cancers caused by ALK deregulation. Morris, Look and their colleagues also worked with another pharmaceutical company to design a diagnostic assay to identify patients with the ALK mutation. The test, a fluorescence in situ hybridization (FISH) assay, has been marketed for more than a decade. In August, it won FDA approval as a diagnostic test for use with Xalkori.


Dr. William E. Evans, St. Jude director and chief executive officer, said the ALK story captures an important aspect of the hospital's commitment. "Our focus is on finding cures for pediatric diseases, but our discoveries often provide insights that can be building blocks for advances in other diseases, including adult cancers. We are committed to facilitating this so that the most good can come from our discoveries," he said.

Working through the St. Jude Office of Technology Licensing, Pfizer obtained licenses to the hospital's patented research tools. Several other companies have executed licenses with St. Jude to use these patent rights.

This year about 210,000 new cases of lung cancer will be diagnosed in the U.S. Current estimates are that approximately 3 to 5 percent, or 6,500 to 11,000 patients with non-small cell lung cancer, carry the ALK rearrangement and may be candidates for treatment with Xalkori.

Today, Morris is a member of the St. Jude Pathology and Oncology departments. Look is a professor of pediatrics at Harvard Medical School and the Dana-Farber Cancer Center in Boston. Morris is still asking questions about ALK, including the protein's normal functions. He said he is thrilled that his work offers new hope for thousands of lung cancer patients. "We knew in 1994 when we initially discovered ALK that it was an outstanding drug-development target," Morris said. "It is heartening to now see patients benefiting from our research."

Source St. Jude Children's Research Hospital

Source: http://www.news-medical.net/news/20110930/ALK-gene-discovered-by-St-Jude-scientists-associated-with-FDA-approved-adult-cancer-drug.aspx

Progesterone could fight against neuroblastoma

High doses of the hormone progesterone can kill neuroblastoma cells while leaving healthy cells unscathed, scientists at Emory University School of Medicine have found in laboratory research.

The results, published in the journal Molecular Medicine, suggest that progesterone could be used to fight neuroblastoma, the most common form of cancer affecting small children.

More research is necessary to determine the optimal dose, how long progesterone treatment should last and if it should be used alone or in combination with radiation or chemotherapy. Emory scientists are also exploring whether it can stop the growth of other brain cancer types such as glioblastoma and astrocytoma. Progesterone has also been reported to slow growth of several other types of cancers in the laboratory, but has not been used clinically against neuroblastoma.

The first author in the team of researchers is Fahim Atif, PhD, instructor in emergency medicine, with senior author Donald G. Stein, PhD, Asa G. Candler professor of emergency medicine and director of Emory's Department of Emergency Medicine Brain Research Laboratory. Daniel Brat, MD, PhD, professor of pathology and laboratory medicine in Emory School of Medicine was a collaborator on the research team.

The discovery grew out of studies of progesterone's protective effects in brain injury. Based on Stein's pioneering work, medical centers across the country are now testing progesterone in the setting of acute traumatic brain injury in a phase III clinical trial. While investigating how to enhance progesterone's effectiveness, Atif and his colleagues observed that it could protect healthy neurons from stress but caused cells from a tumor cell line to die.

In a mouse model, progesterone treatment cut tumor growth in half over eight days, while no drug toxicity was seen with healthy neurons or in live animals. The researchers showed that progesterone can decrease the levels of proteins produced by tumor cells that attract new blood vessel growth and help tumor cells invade other tissues.

"This fits with what we know about one of progesterone's roles during pregnancy, which is to regulate the growth of placenta," Atif says. "Placental cells behave in a way that resembles tumor cells, invading the uterine wall and tapping into the mother's blood vessels."

In studies performed elsewhere, doses of progesterone that were lower than the most effective dose in the Emory study actually accelerated cancer growth. Based on their results, the Emory researchers propose that for fighting certain types of cancer, high doses of progesterone may be better than low doses.

Progesterone's effects on cancer are known to be complex. There may be differences between progesterone, the natural hormone, and synthetic progestins. The National Institutes of Health's Women's Health Initiative study showed that women who received hormone replacement therapy with combined estrogen and progestins had an increased risk of heart disease and breast cancer, although some studies have identified a potential "safe period" if hormone replacement therapy lasts less than two years.

Progesterone has a long history as a treatment designed to prevent preterm birth. If progesterone is to be used with small children, any potential effects on development must be weighed against the risks of standard treatments.

Source: Emory University

Source:http://www.news-medical.net/news/20110714/Progesterone-could-fight-against-neuroblastoma.aspx?page=2

News:MYCN amplification can serve as a favorable prognostic indicator for neuroblastoma

source:http://www.news-medical.net/news/20110812/MYCN-amplification-can-serve-as-a-favorable-prognostic-indicNeuroblastoma, a malignant tumor that primarily affects infants and young children, is a leading cause of death for children with cancer. When amplification of the MYCN oncogene is found in the tumor, it usually indicates an aggressive tumor with rapid progression of the disease and a poor outcome. Thus MYCN amplification has come to be used as a prognostic indicator.

However, sometimes a favorable outcome can be achieved even if MYCN amplification is present. A study reported in the current issue of the journal Pediatric and Developmental Pathology focuses on four such cases.

Four patients were diagnosed with neuroblastoma between the ages of 6 and 13 months and were treated with high-dose therapy and autologous stem cell rescue. Three of the patients are alive and well, having survived from 19 months to 7 years following treatment. One patient, with stage 4 disease, died eight months after being diagnosed.

Although MYCN amplification was confirmed in all four patients, it was not expressed in some of the common ways. Fluorescence in situ hybridization was used to identify the gene amplification in these cases. MYCN protein expression was not detected through immunohistochemistry.

Tumors with MYCN amplification typically have an undifferentiated or poorly differentiated subtype with a high mitosis-karyorrhexis index. Large cell type and presence of prominent nucleoli also characterize MYCN amplification and indicate aggressive behavior of the tumor. In the four cases examined in this study, the tumors all showed a poorly differentiated subtype and a low mitosis-karyorrhexis index. They also did not qualify as a large cell type and lacked prominent nucleoli.

These cases display a unique combination of unfavorable and favorable prognostic indicators for neuroblastoma. The authors speculate that a lack of excess MYCN protein expression despite gene amplification could cause this rare genotype-phenotype discordance. The findings could indicate that MYCN amplification does not automatically mean a poor prognosis.ator-for-neuroblastoma.aspx

Hero:Linas Modestas Zibas

our 4 years son Linas Modestas Zibas still fighting with high risk Neuroblastoma stage 4

Angel: Sierra Rayn Chamblee

Sierra Rayn Chamblee. Diagnosed with stage IV neuroblastoma at 21 months old, battled for 11 months, lost the fight at 2 1/2 years old.

May God bless this family in this time of sorrow.

Hero: Noahs Story

Our world turned upside down on December 18, 2007. Noah was having spuratic side pain so we took him to the pediatrician and they said he had intestinal flu and if the pain continues bring him back, well the pain continued and he was then diagnosed with intussusception. He underwent a barium enama only to find a 4" tumor sitting above his right kidney. This tumor turned out to be Neuroblastoma cancer, which sprouted out of the right adrenal gland. His was diagnosed at stage 2b.
He underwent four rounds of low-dose chemo and had the tumor removed on April 7, 2008. It wasn't 100% removed and our home Oncologist reassured us that with Noah's histology and biology it wouldn't come back.
Noah had reoccurance on October 5, 2008. We are now in treatment with Memorial Sloan Kettering Hospital in New York. He had 3 high-dose rounds of chemo, radiation & surgery. Noah was NED (no evidence of disease) for 15 months and then relapsed in April of 2010. He endured 3 rounds of chemo and after scans in June 2010 he is again NED and is now getting 3f8 Monoclonal Antibodies every 8 weeks. Please continue to pray for our little boy. Read the updates for the current information on Noahs journey. Our greatest praise goes to God our Father who gives us the comfort, peace and strength through Noah's healing. We also have such a tremedous support group and wonderful prayer warriors who have gotten us through these tough times.