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duminică, 5 august 2012

Focusing On Strengths Improves Social Skills Of Adolescents With Autism

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Main Category: Autism
Also Included In: Pediatrics / Children's Health
Article Date: 04 Aug 2012 - 0:00 PDT Current ratings for:
Focusing On Strengths Improves Social Skills Of Adolescents With Autism
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The junior high and high school years are emotionally challenging even under the best of circumstances, but for adolescents with autism spectrum disorders (ASD), that time can be particularly painful. Lacking the social skills that enable them to interact successfully with their peers, these students are often ostracized and even bullied by their classmates.

However, a new study conducted by researchers at the Koegel Autism Center at UC Santa Barbara has found that by playing on their strengths - high intelligence and very specific interests - these adolescents are as capable as anyone else of forging strong friendships. In addition, the research findings demonstrate that the area of the brain that controls such social behavior is not as damaged in adolescents with ASD as was previously believed. The findings appear in a recent issue of the Journal of Positive Behavior Interventions.

"The problem is that their restricted interests can dominate their lives and further push away people they'd like to get to know," said Robert Koegel, director of the Koegel Autism Center and the study's lead author. He is also a professor of counseling, clinical, and school psychology and of education in UCSB's Gevirtz Graduate School of Education. "They're so highly focused on that interest, people think they're weird. But by involving themselves in an activity around the interest, they not only make friends but also become valued members of the group. Their specialized skill becomes a strength."

The research team, which also includes Lynn Koegel, the center's clinical director, and Sunny Kim, a graduate student in education at UCSB, took a creative approach to helping three boys with ASD to interact with their peers. Rather than discourage their sometimes-obsessive interests, the researchers helped set up social clubs around them and invited students who do not have ASD to join. The clubs provided a venue for the ASD students to display their special interests and abilities, and helped them engage with their peers in a more meaningful way.

Koegel offered the example of a student with ASD who has a keen interest in computer graphics. The team created a graphic design club in which students would design logos for various companies and businesses. Because most of the students lacked the necessary expertise, they depended on their classmate with ASD to make the venture a success. "When he was able to interact on a topic in which he was interested, he was able to demonstrate more normal social behavior," Koegel said. "He not only made friends with his fellow members, he was elected club president."

According to Koegel, the findings are also significant because they indicate a higher degree of brain functionality than researchers had previously associated with ASD adolescents. "It has been commonly believed that the part of the brain related to social skills is so damaged that adolescents with ASD are incapable of normal social interaction," he said. "We demonstrated that not to be the case. Once you can motivate kids to try things, they make dramatic and rapid improvement, which shows the brain is not as damaged as first thought."

Conducted through the Koegel Center's Eli & Edythe L. Broad Asperger Center, the study sheds important light on a period of growth and development that is presenting new issues as children who were diagnosed with ASD reach adolescence and young adulthood. "This study is so important because it suggests so much optimism," Koegel said. "It shows the brain isn't as damaged as people thought. And it shows that otherwise unhappy individuals can lead more fulfilling lives."

He added that the research team was pleasantly surprised to see that the students with ASD became highly valued members of their groups, and were given a great deal of dignity and respect. They also noted that, without any instructions or encouragement from any of the researchers, many school peers enthusiastically joined in these club activities and had a great deal of enjoyment throughout and beyond the time frame of the study. "In short, this was a lot of fun for everyone," Koegel said.

Article adapted by Medical News Today from original press release. Click 'references' tab above for source.
Visit our autism section for the latest news on this subject. Other researchers involved with the study include John Danial, a doctoral student at UCLA; and Rosy Fredeen and Derek Rubenstein, doctoral students at UCSB at the time the research was conducted.
University of California - Santa Barbara Please use one of the following formats to cite this article in your essay, paper or report:

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n.p. "Focusing On Strengths Improves Social Skills Of Adolescents With Autism." Medical News Today. MediLexicon, Intl., 4 Aug. 2012. Web.
5 Aug. 2012. APA

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'Focusing On Strengths Improves Social Skills Of Adolescents With Autism'

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joi, 15 decembrie 2011

New Drug That Improves Memory And Prevents Brain Damage In Mice May Prevent Alzheimer's Disease Progression

Main Category: Alzheimer's / Dementia
Also Included In: Parkinson's Disease;  Huntingtons Disease;  Stroke
Article Date: 15 Dec 2011 - 7:00 PST

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A new drug candidate may be the first capable of halting the devastating mental decline of Alzheimer's disease, based on the findings of a study published in PLoS one.

When given to mice with Alzheimer's, the drug, known as J147, improved memory and prevented brain damage caused by the disease. The new compound, developed by scientists at the Salk Institute for Biological Studies, could be tested for treatment of the disease in humans in the near future.

"J147 enhances memory in both normal and Alzheimer's mice and also protects the brain from the loss of synaptic connections," says David Schubert, the head of Salk's Cellular Neurobiology Laboratory, whose team developed the new drug. "No drugs on the market for Alzheimer's have both of these properties."

Although it is yet unknown whether the compound will prove safe and effective in humans, the Salk researchers' say their results suggest the drug may hold potential for treatment of people with Alzheimer's.

As many as 5.4 million Americans suffer from Alzheimer's, according to the National Institutes of Health. More than 16 million will have the disease by 2050, according to Alzheimer's Association estimates, resulting in medical costs of over $1 trillion per year.

The disease causes a steady, irreversible decline in brain function, erasing a person's memory and ability to think clearly until they are unable to perform simple tasks such as eating and talking, and it is ultimately fatal. Alzheimer's is linked to aging and typically appears after age 60, although a small percentage of families carry a genetic risk for earlier onset. Among the top ten causes of death, Alzheimer's is the only one without a way to prevent, cure or slow disease progression.

Scientists are unclear what causes Alzheimer's, which appears to emerge from a complex mix of genetics, environment and lifestyle factors. So far, the drugs developed to treat the disease, such as Aricept, Razadyne and Exelon, only produce fleeting memory improvements and do nothing to slow the overall course of the disease.

To find a new type of drug, Schubert and his colleagues bucked the trend within the pharmaceutical industry of focusing exclusively on the biological pathways involved in the formation of amyloid plaques, the dense deposits of protein that characterize the disease. To date, Schubert says, all amyloid-based drugs have failed in clinical trials.

Instead, the Salk team developed methods for using living neurons grown in laboratory dishes to test whether or not new synthetic compounds were effective at protecting the brain cells against several pathologies associated with brain aging. Based on the test results from each chemical iteration of the lead compound, which was originally developed for treatment of stroke and traumatic brain injury, they were able to alter its chemical structure to make a much more potent Alzheimer's drug.

"Alzheimer's is a complex disease, but most drug development in the pharmaceutical world has focused on a single aspect of the disease - the amyloid pathway," says Marguerite Prior, a research associate in Schubert's lab, who led the project along with Qi Chen, a former Salk postdoctoral researcher. "In contrast, by testing these compounds in living cell cultures, we can determine what they do against a range of age-related problems and select the best candidate that addresses multiple aspects of the disease, not just one."

With a promising compound in hand, the researchers shifted to testing J147 as an oral medication in mice. Working with Amanda Roberts, a professor of molecular neurosciences at The Scripps Research Institute, they conducted a range of behavioral tests that showed that the drug improved memory in normal rodents.

The Salk researchers went on to show that it prevented cognitive decline in animals with Alzheimer's and that mice and rats treated with the drug produced more of a protein called brain-derived neurotrophic factor (BDNF), a molecule that protects neurons from toxic insults, helps new neurons grow and connect with other brain cells, and is involved in memory formation.

Because of the broad ability of J147 to protect nerve cells, the researchers believe that it may also be effective for treating other neurological disorders, such as Parkinson's disease, Huntington's disease and amyotrophic lateral sclerosis (ALS), as well as stroke.

Article adapted by Medical News Today from original press release. Click 'references' tab above for source.
Visit our alzheimer's / dementia section for the latest news on this subject. The research was funded by the Fritz B. Burns Foundation, the National Institutes of Health, the Bundy Foundation and the Alzheimer's Association.
Salk Institute Please use one of the following formats to cite this article in your essay, paper or report:

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Sleep Apnea - CPAP Face Mask Improves Overall Cardiovascular Health

Editor's Choice
Academic Journal
Main Category: Sleep / Sleep Disorders / Insomnia
Article Date: 15 Dec 2011 - 8:00 PST

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Patients with obstructive sleep apnea who use a face mask during their slumber hours were found to have significantly improved blood pressure, levels of stomach fat (visceral fat), and cholesterol and blood sugar levels - all factors closely related to metabolic syndrome and heart health, researchers reported in NEJM (New England Journal of Medicine).

As background information, the authors explain that approximately 18 million people in the USA live with obstructive sleep apnea. Many sufferers are unaware of their condition, because they are asleep when the breathing is halted - whoever sleeps with them is generally the first to notice.

Obstructive sleep apnea (British spelling: apnoea), also known as OSA is a disorder of sleep in which the sufferer stops breathing for at least ten seconds while asleep. The throat muscles (soft tissue in the back of the throat) collapse and close, resulting in blocked airways. Each episode of halted breathing is called an apnea. Apnea means without breath.

Patients with sleep apnea may wake up during an apnea, but are rarely aware of their problem - they do not know why they woke up.

Obstruction ventilation apnée sommeil
During an "apnea" the airway becomes blocked

Standard treatment involves a CPAP machine - this includes a mask that is attached to continuous airway pressure. A significant proportion of CPAP machine users stop using them within a year because they are cumbersome devices.

Lead author, Surendra Sharma, from the All India Institute of Medical Sciences in New Delhi, said:

"These patients need to be properly counseled for regular use of CPAP machines. In a real- life situation, the machine will be used for a longer period and more benefits will be observed."

Pharmaceutical giant, Pfizer Inc. funded the trial. Clinical trials government number NCT00694616. The company does not make or sell sleep apnea devices.

CPAPmask
A CPAP mask (Source: Philips)

Obstructive sleep apnea patients were randomly selected to have therapeutic CPAP followed by placebo CPAP (sham CPAP). This was followed by a 1-month washout period, after which the groups swapped (the placebo ones used the mask for three months, while the other group used the placebo).

At the beginning and end of each three-month intervention, the researchers took measurements of the participants' blood pressure, insulin resistance, fasting blood sugar (glucose) levels, fasting blood lipid profile, visceral fat, carotid intima-media thickness, and glycated hemoglobin levels.

The authors reported the following results on 86 patients who completed the trial; 87% of whom had metabolic syndrome: Those on the CPAP treatment had considerable improvements in the following readings, compared to those on sham CPAP in the following:

- Systolic blood pressure
- Diastolic blood pressure
- Serum total cholesterol
- Non-high-density lipoprotein cholesterol
- Low-density lipoprotein cholesterol
- Triglycerides

Metabolic syndrome reversal was found in 11 of 86 patients on CPAP therapy, compared to 1 of 86 on sham CPAP therapyThe authors concluded in an Abstract in the journal:
"In patients with moderate-to-severe obstructive sleep apnea syndrome, 3 months of CPAP therapy lowers blood pressure and partially reverses metabolic abnormalities."

Written by Christian Nordqvist
Copyright: Medical News Today
Not to be reproduced without permission of Medical News Today

Visit our sleep / sleep disorders / insomnia section for the latest news on this subject. "CPAP for the Metabolic Syndrome in Patients with Obstructive Sleep Apnea"
Surendra K. Sharma, M.D., Ph.D., Swastik Agrawal, M.D., Deepak Damodaran, M.D., Vishnubhatla Sreenivas, Ph.D., Tamilarasu Kadhiravan, M.D., Ramakrishnan Lakshmy, Ph.D., Priya Jagia, M.D., and Atin Kumar, M.D.
N Engl J Med 2011; 365:2277-2286December 15, 2011 Please use one of the following formats to cite this article in your essay, paper or report:

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Christian Nordqvist. "Sleep Apnea - CPAP Face Mask Improves Overall Cardiovascular Health." Medical News Today. MediLexicon, Intl., 15 Dec. 2011. Web.
15 Dec. 2011. APA

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marți, 13 decembrie 2011

Therapy Improves Stem Cell Engraftment In Umbilical Cord Blood Transplant Recipients

Main Category: Stem Cell Research
Also Included In: Transplants / Organ Donations
Article Date: 13 Dec 2011 - 1:00 PST

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A therapy involving a natural compound may improve the ability of stem cells from umbilical cord blood to engraft in patients receiving a stem cell transplant for cancer or other diseases, a phase I clinical trial led by Dana-Farber Cancer Institute scientists indicates.

Details of the trial (abstract 653), which involved 12 patients who underwent reduced-intensity chemotherapy and then received a transplant of cord blood stem cells treated with the compound FT1050, will be presented at the American Society of Hematology's 2011 annual meeting on Monday, Dec. 12, at 2:45 p.m. PST.

FT1050-treated blood-forming stem cells are being tested as a possible solution to one of the major shortcomings of transplants involving stem cells from umbilical cord blood: the relatively small number of stem cells infused in such procedures often take longer to engraft or take root in patients than do the more numerous stem cells involved in transplants from adult donors. The delay can leave patients susceptible to dangerous infections and other complications.

"There is a significant need to improve the speed and quality of engraftment of cord-derived stem cells," says trial leader Corey Cutler, MD, MPH, of Dana-Farber and Brigham and Women's Hospital. "FT1050 has shown the ability in preclinical research to activate hematopoetic [blood-forming] stem cells so they engraft more quickly and with a higher degree of success."

Umbilical cord stem cell transplants are an option for patients who do not have a closely-matched adult donor. Because the current pool of potential donors is smaller for non-Caucasians than for Caucasians, members of ethnic minorities tend to receive transplants from cord blood at a higher rate than Caucasians do.

The goal of the phase I trial was to assess the safety of FT1050-treated cord blood cells in adult patients receiving umbilical cord blood stem cell transplants, and determine if the treated cells accelerate engraftment. In the 12 patients who participated in the trial, engraftment occurred approximately three to four days faster than happens with standard cord blood cells. Levels of white blood cells known as neutrophils returned to normal in the patients after a median of 17.5 days, similar to the rate in standard stem cell transplants. Side effects of the FT1050-treated cord blood cells were minimal. In none of the study patients did the stem cells fail to engraft.

The phase I trial was sponsored by Fate Therapeutics, Inc., of San Diego, Calif., which is developing ProHema, a biologic product consisting of hematopoietic stem cells treated with FT1050 for patients undergoing stem cell transplantation. FT1050 was identified by Leonard Zon, MD, a hematologist and director of the Stem Cell Program at Children's Hospital Boston, using chemical screens conducted in zebrafish, and is the first potential therapeutic derived from a zebrafish model to make into clinical trials.

"We're encouraged by the results of this study for patients receiving umbilical cord stem cell transplants after reduced-intensity chemotherapy treatment," Cutler says. "Further studies are planned to test FT1050-treated hematopoietic stem cells in a larger group of these patients."

Article adapted by Medical News Today from original press release. Source: Dana-Farber Cancer Institute
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13 Dec. 2011. APA

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"Twinning" U.S.-Based And Rwandan Physicians Improves Lymphoma Outcomes In Children

Main Category: Lymphoma / Leukemia / Myeloma
Article Date: 13 Dec 2011 - 1:00 PST

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In an African county lacking any specialists in children's cancers, a team approach that "twins" Rwandan physicians with Boston-based pediatric oncologists has shown it can deliver expert, curative care to young patients stricken with lymphoma.

The first-of-its-kind strategy is credited for curing at least 5 of 10 children at a rural Rwandan hospital; two others are in remission while receiving chemotherapy, and three children have died. The long-distance team approach was designed by Sara Stulac, MD, MPH, director of pediatrics for Partners In Health, and during the last year further developed and formalized through a partnership with pediatric oncologist Leslie Lehmann, MD, Kathleen Houlahan, RN, BA, MHA, pediatric oncology nurse and nurse director of the Jimmy Fund Clinic at Dana-Farber/Children's Cancer Center, and Larry Shulman, MD, medical oncologist and chief medical officer of Dana-Farber Cancer Institute.

Lehmann will present the results (abstract 4222) at the American Society of Hematology's 2011 annual meeting, Session 901, Monday, Dec. 12, at 6 p.m. PST in Hall GH of the San Diego Convention Center.

"We show that we can safely deliver care using this model an American-trained pediatrician supervising a Rwandan-trained generalist who are together supervised through phone calls from a U.S-based pediatric oncologist," says Lehmann, who is clinical director of the pediatric stem cell transplant program at Dana-Farber/Children's Hospital Cancer Center.

While nations in the developing world have traditionally devoted their limited public health resources to epidemic infectious diseases such as malaria and diarrhea, life-threatening non-communicable conditions like cancer and heart disease are of growing concern.

"There are not enough pediatric oncologists in the world" to provide specialized cancer care for children in developing countries, Lehmann says. "And there's not a single trained pediatric oncologist in Rwanda," a country of more than 11 million people.

In the Western world, 80 percent of children can be cured of lymphoma, but this success rate requires definitive diagnosis, expert administration of chemotherapy, and experienced follow-up care. The "twinning" team approach leverages in-country medical and nursing resources by adding the long-distance supervision and treatment-planning of Dana-Farber/Children's pediatric specialists in blood cancers.

The children described in Lehmann's report were treated over the past four years at the Rwinkwavu government hospital in rural Rwanda. The hospital is supported by Paul Farmer's Partners In Health, which works with governments in impoverished areas of the world to improve health care.

About five years ago, Stulac, a pediatrician then based in Rwanda as the clinical director for Partners In Health's project, set up the care model based at Rwinkwavu, along with Sara Chaffee, MD, of Dartmouth-Hitchcock Medical Center. Rounding out the group are Alain Uwumugambi, MD, a general physician trained in Rwanda; Merab Nyishime, RN, head pediatric nurse at Rwinkwavu Hospital, and a Rwandan nurse coordinator, Jean Bosco Bigirimana, RN. All are authors on the paper.

Lehmann says that accurate diagnosis of lymphoma is essential and sometimes difficult. "You don't want mistakes," she emphasizes. Children underwent biopsies and staging X-rays in Rwanda, and the results were sent to Brigham and Women's Hospital in Boston for all diagnoses.

Next, the pediatric oncologist (Lehmann) oversaw the development of a treatment plan. Patients received surgery at the national referral hospital, as needed. When a patient required chemotherapy, the drugs were prescribed by one of the U.S.-trained pediatricians, and administered by Rwandan nurses under the supervision of the local general physician and supported by the American-trained pediatrician, Lehmann explained.

For radiation therapy, children were taken to facilities in bordering Uganda. Throughout treatment, Lehmann consulted with the team in weekly conference calls or more often, if needed.

The 10 patients covered in the ASH report ranged from 3 to 15 years in age. All were diagnosed with lymphoma, which is more common in children in Rwanda than in the U.S. Five patients completed therapy four receiving CHOP or ABVD chemotherapy, and the fifth had the disease removed surgically and has not needed chemotherapy. Those five children have no evidence of disease in follow-up ranging from four months to four years and are considered cured, Lehmann said.

Two patients are currently on chemotherapy and their cancer is in remission. Two children died from treatment-related complications, and a third died when the lymphoma progressed during treatment.

"This is the beginning of a new model," says Lehmann. "In the past, doctors weren't comfortable having complicated oncology care delivered without a local oncology specialist available. Having a specialist on-site would be ideal but as global health moves into oncology, there are not enough oncologists to provide that kind of care so alternative approaches must be developed and carefully assessed."

The study was funded by Partners In Health.

Article adapted by Medical News Today from original press release. Source: Dana-Farber Cancer Institute
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13 Dec. 2011. APA

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duminică, 11 decembrie 2011

Breast Cancer Survival In Younger Breast Cancer Patients Improves With Bone Drug

Editor's Choice
Main Category: Breast Cancer
Also Included In: Bones / Orthopedics;  Cancer / Oncology
Article Date: 10 Dec 2011 - 18:00 PST

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Zometa (zoledronic acid), used to protect bone health in pre-menopausal ER-Positive breast cancer patients, has been found to improve survival considerably. In fact, it had as beneficial an effect on survival as chemotherapy, researchers from the University of Vienna, Austria, reported in the 2011 CTRC-AACR San Antonio Breast Cancer Symposium this week.

The scientists reported that not only did zoledronic acid reduce death risk by 36%, but also that the risk of breast cancer recurrence dropped 28%. Patients were administered zoledronic acid along with adjuvant endocrine treatment, including ovarian function suppression.

The researchers presented data after 48, 62 and 84 months of follow-up. At 7 years there were "drastically" fewer breast cancer recurrences and better survival rates - and no reports of toxic side effects.

Professor of Surgery, at the University of Vienna's Medical School, Michael Gnant, M.D.,said:

"We have confirmed what this trial showed initially, which was both exciting and surprising. The continued success of this treatment means we can intervene early and still observe persistence of the benefit of treatment."

Dr. Gnant is also President of the ABCSG (Austrian Breast- and Colorectal Cancer Study Group).

The trial involved 1,803 women, all of them premenopausal with early-stage ER (estrogen receptor)-positive breast cancer. They were randomly selected to one of the four arms of the study. They were administered the following drugs or combinations for 36 months: AnastrazoleTamoxifenZoledronic acid plus anastrazoleZoledronic acid plus tamoxifenIn 2008, an initial report showed that there had been a significant improvement in disease-free survival in the females receiving zoledronic acid.

According to the latest data at 84 months post-treatment, the patients on zoledronic acid had a 36% lower risk of death and a 29% smaller chance of the cancer coming back (recurrence). There were no reports of any patient developing renal failure or osteonecrosis (bone death) of the jaw. Gnant explained that this means the treatment is not only effective after seven years, but also safe.

The participants aged over 40 with presumed complete ovarian blockade were 34% less likely to experience cancer recurrence, and also had a 44% lower risk of death. For patients under 40 there appeared to be no significant survival benefits.

The team concluded that zoledronic acid, added to adjuvant endocrine therapy (including ovarian function suppression), should be a considered treatment option for females with ER-positive early breast cancer who have not yet reached the menopause.

Zoledronic acid, also known as zoledronate, molecular formula C5H10N2O7P2, is a bisphosphonate, which is made and marketed by the Swiss company Novartis under the brand names Aclasta, Reclast, Zomera and Zometa. Zometa is prescribed to prevent bone fractures in patients with several types of cancers. Zoledronic acid is also used for osteoporosis treatment, and hypercalcemia of malignancy.

Written by Christian Nordqvist


Copyright: Medical News Today
Not to be reproduced without permission of Medical News Today Visit our breast cancer section for the latest news on this subject. Please use one of the following formats to cite this article in your essay, paper or report:

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Christian Nordqvist. "Breast Cancer Survival In Younger Breast Cancer Patients Improves With Bone Drug." Medical News Today. MediLexicon, Intl., 10 Dec. 2011. Web.
11 Dec. 2011. APA

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joi, 8 decembrie 2011

Combination Of Everolimus And Exemestane Improves Progression-Free Survival For Women With Metastatic Breast Cancer

Main Category: Breast Cancer
Article Date: 08 Dec 2011 - 2:00 PST

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In an international Phase III randomized study, everolimus, when combined with the hormonal therapy exemestane, has been shown to dramatically improve progression-free survival, according to research from The University of Texas MD Anderson Cancer Center.

The study, known as Breast Cancer Trials of Oral Everolimus (BOLERO-2), was presented at the 2011 CTRC-AACR San Antonio Breast Cancer Symposium by Gabriel Hortobagyi, M.D., professor and chair of MD Anderson's Department of Breast Medical Oncology. Earlier findings were simultaneously reported in the New England Journal of Medicine.

Everolimus, an immunosuppressant agent first used to prevent rejection of organ transplants, also has anti-angiogenic properties. It inhibits the mammalian target of rapamycin (mTOR) protein, a central regulator of tumor cell division and blood vessel growth in cancer cells; the mTOR pathway is activated in hormone-resistant breast cancer, explained Hortobagyi. Currently, the oral agent is approved for both the treatment of kidney cancer and pancreatic neuro-endocrine tumors, with MD Anderson's research leading the way for the latter's usage approval by the FDA.

Preliminary BOLERO-2 data were first presented at ESMO and showed a significant progression-free survival benefit; these updated findings represent six additional months of patient follow-up, Hortobagyi explained.

"This study is based on the concept that we now know more about the resistance mechanisms to endocrine therapy and the experimental arm of BOLERO-2 uses a dual-attack on treatment refractory hormone receptor-positive breast cancer: it simultaneously inhibits the estrogen-signaling pathway with the aromatase inhibitor, exemestane, and the PI3-kinase/AKT/mTOR pathway with the use of everolimus," Hortobagyi said. "For the first time in a large Phase III trial, we have demonstrated that this dual-attack is more effective than a single endocrine treatment for patients who have received prior endocrine therapy."

The international Phase III trial enrolled 724 metastatic breast cancer patients, all of whom were post-menopausal, had hormone receptor-positive disease and evidence of progressive disease. In a two-to-one ratio, 485 women were randomized to receive the combination of everolimus (10 mg daily), and exemestane, an aromatase inhibitor, and compared to 239 women who received exemestane and placebo. All were previously treated with and progressed on letrozole and anastrozole, with the majority of women extensively treated with prior therapies. The median age of the women enrolled was 62 years; the study's primary endpoint was progression-free survival.

In patients receiving the everolimus combination, researchers found a progression-free survival of 7.4 months, compared to 3.2 months in those who took exemestane alone, a finding Hortobagyi describes as "highly significant." The clinical benefit rate complete responses, partial responses and stability exceeding six months was 50.5 percent in those in the combination arm, compared to 25.5 percent in those who received the hormonal therapy alone. Adverse effects, such as shortness of breath, hyperglycemia, mouth sores and fatigue, were all higher in the combination group, but were manageable and did not disrupt patients' quality of life, the researchers found. Survival data are still being analyzed.

Because aromatase inhibitors are associated with bone loss and fractures, as a safety measure, Hortobagyi and the researchers assessed patients for bone-turnover markers. The addition of everolimus was found to significantly reduce the level of such markers an interesting area for future study, noted Hortobagyi.

"Over the years, our treatment approach for such women with metastatic breast cancer has been sequential use of as many hormone therapies as possible, keeping metastatic disease under control for as long as possible. These findings may allow us to change our approach. In this group of heavily pre-treated patients, all of whom progressed on prior endocrine therapy, the addition of this mTOR inhibitor resulted in significant prolongation of progression-free survival and an improved response rate, with only a modest addition of toxicity," said Hortobagyi.

Hortobagyi believes that these findings may be practice-changing for women who meet the criteria for BOLERO-2. Additional studies are planned with everolimus in breast cancer, including in combination with an aromatase inhibitor in the adjuvant setting, as well as additional clinical trials in the metastatic setting.

In addition to Hortobagyi, authors on the international study include: Jorge Baselga, M.D., Massachusetts General Hospital Cancer Center and Harvard Medical School, and the NEJM study's corresponding author; Martine Piccart, M.D., Jules Bordet Institute; Hope S. Rugo, M.D., University of California, San Francisco, Helen Diller Family Comprehensive Cancer Center; Howard Burris, M.D., Sarah Cannon Research Institute; Mario Campone, M.D., Institut de Cancérologie de l'Ouest; Shinzaburo Noguchi, M.D., Osaka University; Michael Gnant, M.D., Comprehensive Cancer Center, Medical University of Vienna; Kathleen Pritchard, M.D., Sunnybrook Odette Cancer Centre; Luc Vittori, Zhiying Xu, Ph.D., Pabak Mukhopadhyay, Ph.D., Tarek Sahmoud, M.D., Ph.D. and David Lebwohl, M.D., the last five of Novartis Pharmaceuticals.

The trial was funded, in part, by Novartis. Hortobagyi reports receiving research funds from, as well as having served as a consultant for, Novartis.

Article adapted by Medical News Today from original press release. Source: University of Texas M. D. Anderson Cancer Center
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