NF2 Research and Media News 2011

Congressional Compromise Provides Funding for NF Research in FY12

December 28, 2011 – House and Senate negotiators have agreed to include $12.8 million for NF research in the 2012 Department of Defense budget…

Annette Bakker, Ph.D. Appointed Senior Vice President of Research and Drug Development

December 28, 2011 – The Children’s Tumor Foundation has appointed Annette Bakker, Ph.D., to the newly-created position of Senior Vice President of Research and Drug Development…

IBM Contributes Data to the National Institutes of Health (NIH) to Speed Drug Discovery and Cancer Research Innovation

December 19, 2011 – IBM (NYSE: IBM) today announced it is contributing a massive database of chemical data extracted from millions of patents and scientific literature to the National Institutes of Health. This contribution will allow researchers to more easily visualize important relationships among chemical compounds to aid in drug discovery and support advanced cancer research…

NF Forum on June 8-10, 2012 at Hilton New Orleans Riverside, LA – Save the Date!

December 16, 2011 – The NF Forum allows those living with NF, and their families, to connect, support, and learn from each other while attending seminars on relevant topics pertaining to neurofibromatosis. In 2012, for the first time, the Children’s Tumor Foundation will hold its annual NF Forum adjacent to the NF Conference. This will provide an unprecedented opportunity for the Forum to hold family-targeted sessions, benefiting from the presence of many international physicians, researchers, and clinicians, sharing the latest in medical advancements. Mark your calendar and plan to attend!

ADA Business BRIEF: Communicating with People Who Are Deaf or Hard of Hearing in Hospital Settings

December 16, 2011 – People who are deaf or hard of hearing use a variety of ways to communicate. Some rely on sign language interpreters or assistive listening devices; some rely primarily on written messages. Many can speak even though they cannot hear. The method of communication and the services or aids the hospital must provide will vary depending upon the abilities of the person who is deaf or hard of hearing and on the complexity and nature of the communications that are required. Effective communication is particularly critical in health care settings where miscommunication may lead to misdiagnosis and improper or delayed medical treatment.

Children’s Tumor Foundation Funds Two New Drug Discovery Initiative Awards, and Increases DDI Award Levels for 2012!

December 15, 2011 – … CTF is delighted to announce the recipients of the Drug Discovery Initiative Awards from our fall 2011 round. Dr. Toshifumi Tomoda of the Beckman Research Institute receives a $15,000 in vitro Award to study autophagy-inducing compounds as candidate therapeutics for NF2. Dr. David Little of the University of Sydney receives a $30,000 in vivo Award to study MEK inhibition as a therapeutic approach in mouse models of NF1 related tibial pseudarthrosis… CTF is also pleased to announce that for 2012 we are significantly increasing DDI Award levels! We now offer $25,000 for in vitro DDI Awards, $50,000 for in vivo DDI Awards, and $75,000 for Advanced DDI Awards. There are two DDI Award deadlines in 2012: February 28th and August 31st. No preliminary data is required for DDI Award applications, and cutting-edge ideas are encouraged. If you have any questions about the DDI program, please contact Min Wong at CTF.

Consensus recommendations for current treatments and accelerating clinical trials for patients with neurofibromatosis type 2

December 14, 2011 – Neurofibromatosis type 2 (NF2) is a tumor suppressor syndrome characterized by bilateral vestibular schwannomas (VS) which often result in deafness despite aggressive management. Meningiomas, ependymomas, and other cranial nerve and peripheral schwannomas are also commonly found in NF2 and collectively lead to major neurologic morbidity and mortality. Traditionally, the overall survival rate in patients with NF2 is estimated to be 38% at 20 years from diagnosis. Hence, there is a desperate need for new, effective therapies. Recent progress in understanding the molecular basis of NF2 related tumors has aided in the identification of potential therapeutic targets and emerging clinical therapies. In June 2010, representatives of the international NF2 research and clinical community convened under the leadership of Drs. D. Gareth Evans (University of Manchester) and Marco Giovannini (House Research Institute) to review the state of NF2 treatment and clinical trials. This manuscript summarizes the expert opinions about current treatments for NF2 associated tumors and recommendations for advancing therapies emerging from that meeting. The development of effective therapies for NF2 associated tumors has the potential for significant clinical advancement not only for patients with NF2 but for thousands of neuro-oncology patients afflicted with these tumors…

Patient Information, Neurofibromatosis Type 2 (NF2).

December 14, 2011 – This leaflet is for patients and relatives who attend the neurofibromatosis type 2 (NF2) clinic in Cambridge… Cambridge University Hospitals NHS Foundation Trust has been designated as one of the four specialist centres for the management of NF2 by the National Specialised Commissioning Team (NSCT), the part of the NHS that deals with organizing care for people with rare diseases. This means that people who have been diagnosed with NF2 have access to a team of people with the specialist knowledge and experience necessary to provide the best care…

Neurofibromatosis Working Group at MD Anderson

December 14, 2011 – The Neurofibromatosis Working Group is a collaborative effort between the departments of Neurosurgery, Investigational Cancer Therapeutics and Neuro-Oncology. Our aim is to provide outstanding care for patients with neurofibromatosis and to perform cutting-edge research leading to new treatments for these disorders…

A holiday story about the meaning of perseverance, and its role in success

December 10, 2011 – During the holiday season, we often read inspirational stories that raise our awareness of the plight of the needy and celebrate a spirit of helping the unfortunate who have suffered from hard luck, injury and hurt…

Republican sportswriter to run 50K race as $50,000 fund-raiser for Wilbraham girl with rare form of cancer

December 10, 2011 – Celia had a disease I had never heard of: Neurofibromatosis 2. It was a rare type of cancer, but something about that disease was off the charts…

Watch the NF2 webinar with Scott Plotkin, MD

December 07, 2011 – If you were not able to attend our NF2 webinar with guest presenter Scott Plotkin, MD, you can watch it here! You will need windows media player to view…

Stem Cells and Regeneration of the Auditory Nerve

December 07, 2011 – Overview: Cochlear implants function by electrically stimulating auditory neurons in the absence of hair cells, to enable hearing in severe to profoundly deaf individuals. The efficacy of this device therefore depends on a critical number of surviving auditory neurons. Stem cell transplantation therapy is emerging as a potential strategy for auditory nerve rehabilitation, as differentiated stem cells may provide a source of replacement auditory neurons to the deaf cochlea. The successful engraftment of stem cells into the cochlea will require both the directed growth of new processes and the formation of functional connections with existing structures, and we are investigating these questions using both in vitro and in vivo experimental models. We are particularly interested in whether transplanted stem cells are capable of making functional connections in the brainstem, thereby improving hearing with a cochlear implant.

Regeneration of human auditory nerve. In vitro/in video demonstration of neural progenitor cells in adult human and guinea pig spiral ganglion

December 07, 2011 – Abstract: Time lapse video recordings of cultured adult human and guinea pig spiral ganglion (hSG and gpSG) show that mitogen responsive progenitor/stem cells develop in the form of spheres that proliferate and differentiate into mature neurons and glia cells. Neurospheres, cultured with EGF and bFGF showed expression of nestin and incorporation of 5-Bromo-2-deoxyuridine (BrdU). Newly formed BrdU labelled cells were positive for B-tubulin, and also for GFAP demonstrating that neuronal cells were derived from a dividing population of progenitor cells. Dissociated spheres cultured either with glia cell line-derived neurotrophic factor (GDNF) or brain-derived neurotrophic factor (BDNF) and neurotrophin-3 (NT-3), induced differentiation of the progenitor cells. Video microscopy showed that neurons develop from subcultured spheres maintained for up to four weeks. Neurons showed fasciculation and migration with a speed of 10–30 um/h, and some cells had up to 6 mm long neurites coexpressing TrkB and TrkC receptors. Precise dissection suggests that the neurons formed are cochlea-specific. The results suggest that the mammalian auditory nerve has the capability for self-renewal and replacement. Transplantation of progenitor cells together with established means to induce neural differentiation and fiber growth may facilitate strategies for better repair and treatment of auditory neuronal damage.

2012 Young Investigator Awards is now open!

December 06, 2011 – The appl. process for the 2012 Young Investigator Award program is NOW OPEN! Please note the deadlines for the pre-application and full application. Please be sure to carefully read the guidelines of the application process… APPLICATION PROCESS DEADLINES: January 27, 2012: Pre-Application Due February 24, 2012: Invite to submit Full Appl. will sent via email April 2, 2012: Full Application Due

Announcing CTF’s 2011 Young Investigator Award Recipients

December 06, 2011 – The Children’s Tumor Foundation is delighted to announce the funding of SIX Young Investigator Awardees for the 2011 round. The recipients include three postdoctoral awardees and three graduate students; three focused on aspects of NF1 including tumors, bone dysplasia and learning disabilities; and three focused on NF2 or schwannomatosis. Four awardees are US-based and two are international. Details of the Awards, as well as Past Awardees are listed…. The 2011 Young Investigator Awardees for NF2 are:

  1. Miriam Smith, Ph.D., University of Manchester, United Kingdom. Project: Identification of novel genes predisposing to schwannomas and meningiomas by exome.
  2. Jianzhong Yu, Ph.D., Johns Hopkins University. Project: Molecular genetic characterization of the Merlin tumor suppressor protein complex.
  3. Alejandra Petrilli Guinart, University of Central Florida. Project: Investigating a new drug target for NF2.

How to spot bogus research.

December 06, 2011 – The words “clinically proven,” loved and abused by infomercials and health-store potions, no longer carry the same weight they once did…

Project Endeavor Provides High-Speed Internet Service for Deaf and Hard Of Hearing

December 06, 2011 – …Qualified deaf and hard-of-hearing individuals can connect to the Internet through Project Endeavor’s discounted Internet packages, which include discounted service plans and free video communications equipment. Project Endeavor also offers videos that demonstrate the benefits of using the Internet, in both text and American Sign Language (ASL)…

Cochlear implantation (CI) in patients with NF2, and patients with VS in the only hearing ear.

December 04, 2011 – …CONCLUSIONS: Cochlear implantation (CI) in patients with NF2, and patients with VS in the only hearing ear is a reasonable hearing rehabilitation option. Early surgical intervention with preservation of the cochlear nerve should be considered. Auditory benefit can be expected in most patients and in good candidates, excellent benefit with good speech discrimination and telephone use may be achieved. Surgical resection through translabyrinthine approach does not affect speech test outcomes. Radiosurgery has good auditory results. Although not crucial, promontory stimulation for evaluating the integrity of the cochlear nerve is recommended. MRI up to 1.5 Teslas with external head fixation can be done safely in cochlear implanted patients.

Weight Loss & Cachexia…

December 02, 2011 – …Cachexia is a clinical wasting syndrome. Symptoms include weakness and a marked and progressive loss of body weight, fat, and muscle. Anorexia and cachexia frequently occur together; however, cachexia can occur in individuals who are ingesting adequate calories and protein but who are not absorbing nutrients well….

Clinical Trials for regenerating muscles in humans are soon to start…

December 01, 2011 – Scientists have regenerated functional muscle tissue in mice, opening the door for a new clinical therapy for major muscle trauma. The team used a novel protocol to coax human muscle cells into a stem cell-like state and grew the reprogrammed cells on biopolymer microthreads. Surprisingly, the micro…..

The Inhibitory Effect of Honokiol, a Natural Plant Product, on Vestibular Schwannoma Cells.

December 01, 2011 – … CONCLUSIONS: Honokiol, a low molecular weight natural product, inhibits cell proliferation and promotes apoptosis in schwannoma cells by targeting the ERK pathway. Our data suggest that honokiol can be evaluated as a chemotherapeutic agent for VS.

Get up to 75% off on medications you use everyday.

December 01, 2011 – Get your pharmacy card and start saving up to 75% on thousands of prescriptions drugs you use everyday… The Pharmacy Discount Card is a FREE, pre-activated card that offers discounts on prescriptions that range from 0%-75% depending on the drug and location. The use of the discount card, is just like shopping at a discount store. The products are the same, but the pricing is different…

NIH Early Independence Awards – January 30th Deadline!

November 29, 2011 – The National Institutes of Health (NIH) has announced a funding opportunity for the NIH Director’s Early Independence Awards for junior investigators wishing to “skip the post-doc” and immediately begin independent research. Budgets may be up to $250,000 in direct costs per year for up to five years…..The deadline for submitting Early Independence Award applications is January 30, 2012 with Letters of Intent due by December 30, 2011.

NF2 mutation screening (NCG referrals) – clinical pro-forma

November 25, 2011 – Central Manchester and Manchester Children’s University Hospital (in the UK)….. This form should accompany all requests for mutation screening of the NF2 gene. It is not necessary to complete this form for follow on tests where a mutation has already been identified within a family…..

AdvocureNF2 Funds Research

November 16, 2011 – This year (FY11), AdvocureNF2 will be funding two NF2 researchers: Dr. Marco Giovannini at House Research Institute (HRI), who will be starting a Phase 2 clinical trial of RAD001 for NF2. In addition: Dr. Brad Welling at Ohio State University (OSU) is to develop a medical therapy for NF2 VS and meningiomas utilizing two novel small-molecule compounds, AR-12 and AR-42. As well as to screen a library of pure, structurally-defined natural compounds for potent growth inhibitory activity in schwannomas and meningiomas.
3rd State of the Art International NF2 Conference.

November 14, 2011 – The Manchester NF2 Multidisciplinary Team would like to invite you to attend the third State of the Art International NF2 Conference. May 21-22, 2012 in Manchester, UK. The meeting will focus on Epidemiology, Genetics and Natural History of NF2, NF2 surgery and radiosurgery, Auditory Rehabilitation and Animal models and preclinical and clinical trials. …

NF2 Webinar with Scott Plotkin, MD

November 14, 2011 – NF Network is pleased to present an all new NF2 webinar. You don’t want to miss this webinar with guest presenter, and NF2 expert Dr. Scott Plotkin, MD, PhD on Wed, Nov 30, 2011 8:00 PM – 9:00 PM EST. Just a couple of the topics Dr. Plotkin will discuss are advancements in NF2 research and the genetic inheritance of NF2.

Children’s Tumor Foundation Raises $1.5 Million at recent Benefit Dinner

November 14, 2011 – The Children’s Tumor Foundation’s 2011 Annual Benefit Dinner was our largest and most successful event ever — raising $1.5 million and still counting for neurofibromatosis research (NF). …

Psychosocial Aspects – Recurrent brain tumour: the impact of illness on patient’s life

November 14, 2011 – Despite advances in therapies that offer improved survival rates, clinical course of brain tumours leads to a progressive functional deterioration in patients with modifications in their psychological reaction to the disease. Patients with brain tumours are rarely assessed for quality of life and psychological variables, and even fewer studies have assessed patients who have experienced a recurrence of brain tumours. …

2011 Halloween Bash raises $100,000 for children’s tumor research!!

November 1, 2011 – The 2011 Halloween Bash has come and gone and it was a huge success. On Saturday, October 22nd, 300+ people packed the Redux Lounge in downtown Walnut Creek to attend the 7th annual Bash, a benefit through Advocure NF2 to fund children’s tumor research. Over $40,000 was raised that night and with contributions from Varsity Painting and the Thoms family, the total amount raised for research is $100,000…..

SOME NF2 Research Labs to be Supported

October 30, 2011 – There are several worldclass research labs studying NF2.

SOME NF Clinics with an excellent and progressive NF2 component

October 30, 2011 – SOME NF Clinical Centers with an excellent and progressive NF2 component, that are currently participating in progressive NF2 care, NF2 drug trials, and/or NF2 studies.

In Reversing Motor Nerve Damage, Time Is of the Essence: ‘Wait and See’ in Injuries Like Carpal Tunnel Syndrome May Miss a Window for Recovery

October 29, 2011 – When a motor nerve is severely damaged, people rarely recover full muscle strength and function. Neuroscientists from Children’s Hospital Boston, combining patient data with observations in a mouse model, now show why…..(would this have revelance to correcting NF2 facial nerve damage?)

Call for Entries to the 2012 Neuro Film Festival

October 23, 2011 – The American Academy of Neurology Foundation is calling on all neurology patients, caregivers and others to submit a short video to its 2012 Neuro Film Festival, telling their story about why more research is needed into finding cures for brain disorders. Winners could win up to $1,000 and a trip to New Orleans. The deadline to enter is January 31, 2012. A good way to get NF2 awareness out there in a big way. Deadline is January 31, 2012.

University Hospitals Neurofibromatosis 2 Center

October 22, 2011 – UH Neurofibromatosis 2 Center in Cleveland, OH is comprised of a multidisciplinary group of surgeons and specialists in neurosurgery, neurology, oncology, otolaryngology and genetics…. They offer standard and innovative treatment options including management through medication, radiation or surgery. Hearing rehabilitation using auditory brainstem implants (ABI) or cochlear implantation (when appropriate) are available as well.

2012 NF Forum Topics Survey

October 22, 2011 – In preparation for the 2012 NF Forum: CTF is conducting a survey to gauge interest in what topics everyone wants at the next NF Forum. The proposed NF2 topic selections are limited. However in the next step, it allows you to free form as many NF2 topics as you want. Tell them what you want! Save the Date: NF Forum 2012, Hilton New Orleans, June 8-10, 2012.

Living with NF2

October 20, 2011 – Losing all hearing in his right ear at 16 and in his left ear at 22 are only two of the ordeals Dan Knodel has experienced in his ongoing battle with NF2.

Student Copes with NF2

October 20, 2011 – As his hearing gradually diminishes, Mark Pyeatt finds ways to compensate that allow his college and career goals to remain unchanged.

Crucial Funding For NF Research

October 13, 2011 – …..Increasing pressures on the defense budget are resulting in cuts to various programs, and we need your help in sending the message to Congress that federal funding for NF research should continue at a robust level. This is more important now than ever before. The FY2012 Defense Appropriations Bill passed by the House of Representatives earlier this year included $12.8 million for NF research. The Senate did not provide this level of funding in its version of the bill. We need your help in requesting that Congress retain the House-passed funding level for the NF CDMRP when House and Senate leaders meet to negotiate the differences in their two bills. Time is of the essence – please write your Member of Congress today! A template letter of support is included on this page to help make this easy……
Click here to view and download template letter
Click here to search for your Representative
Click here to search for your Senator

Facial Reanimation of Patients with Neurofibromatosis Type 2

October 9, 2011 – ….. Patients were treated between 2006 and 2009. 3 patients were men and 2 were women. The age of presentation of debilitating facial paralysis ranged from 13 to 50 years old. All patients were treated with a single stage free gracilis muscle transfer for smile reanimation. Each obturator nerve of the gracilis was coapted to the masseteric branch of the trigeminal nerve. Measurement of oral commissure excursions at rest and with smile, preoperatively and postoperatively, revealed improved and near symmetric smile in all cases…..

NF2 Workshop held in LV last year, to be published

October 4, 2011 – The Children’s Tumor Foundation is delighted to announce that a Foundation-spearheaded paper outlining progress in NF2 clinical trials and strategies for further accelerating these trials has been accepted for publication in the American Journal of Medical Genetics (AJMG). This paper, “Consensus Recommendations for Current Treatments and Accelerating Clinical Trials for Patients with Neurofibromatosis Type 2” was authored by 17 international NF2 clinical and research experts….. The new AJMG publication should appear in the near future.

Sorafenib in a NF2 study, in the UK

September 30, 2011 – Adults who have Neurofibromatosis 2 (NF2) and at least two skin tumours (nerve tumours under the skin also referred to as ‘schwannomas’) are invited to participate in a research study being undertaken in Plymouth and Manchester; Professor C. Oliver Hanemann, a consultant neurologist at the Peninsula College of Medicine and Dentistry in Plymouth, is the Chief Investigator. Professor Gareth Evans is the Principal Investigator in Manchester. …

Medical Drug Shortages

September 26, 2011 – Doctors and officials from Health and Human Services and the Food and Drug Administration testified on the effects of drug shortages on cancer patients.

Key Molecule That Blocks Abnormal Blood Vessel Growth in Tumors Identified

September 25, 2011 – A new and better understanding of blood vessel growth and vascular development (angiogenesis) in cancer has been made possible by research carried out by a team of scientists from Moffitt Cancer Center, the University of Florida, Harvard University, Yale University and the Children’s Hospital of Los Angeles.

NIH Offers Director’s Transformative Research Awards of up to $25M/Year for up to Five Years

September 23, 2011 – Announcing a terrific funding opportunity at NIH: The Common Fund’s NIH Director’s Transformative Research Award initiative, formerly known as the Transformative Research Project (TR01), is created specifically to support exceptionally innovative and/or unconventional research projects that have the potential to create or overturn fundamental paradigms. These projects tend to be inherently risky and may not fare well in conventional NIH review. As compared to the other NIH Director’s Awards – the Pioneer, New Innovator Award, and Early Independence Awards – the primary emphasis of the Transformative Research Awards initiative is to support research on bold, paradigm-shifting, but untested ideas, rather than to support exceptionally creative individuals who wish to pursue new, potentially high impact research directions.

Results of world-first viral therapy trial in Ottawa cancer patients published in Nature

September 19, 2011 – News Release – Researchers from the Ottawa Hospital Research Institute (OHRI), the University of Ottawa (uOttawa), Jennerex Inc. and several other institutions today reported promising results of a world-first cancer therapy trial in renowned journal Nature. The trial is the first to show that an intravenously-delivered viral therapy can consistently infect and spread within tumours without harming normal tissues in humans. It is also the first to show tumour-selective expression of a foreign gene after intravenous delivery…….

New strategy likely to speed drug development for rare cancers (ependymomas)

September 16, 2011 – 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 Sept. 13 edition of the scientific journal Cancer Cell…….

Cancer Growth In Head And Neck Suppressed By Turmeric

September 16, 2011 – According to investigators at UCLA’s Jonsson Comprehensive Cancer Center a pilot study using human saliva revealed that curcumin, the primary component in the spice turmeric, suppresses a cell signaling pathway that powers the growth of cancer in the head and neck. The investigation is presented Sept. 15 in Clinical Cancer Research, a peer-reviewed journal of the American Association of Cancer Research…….

Malay Folk Medicine points to Cancer Cure Tree

September 15, 2011 – A drug from a tree that belongs to the pomegranate family found here in Sarawak has shown promise in the fight against certain types of cancer. Known for hundreds of years to the Malay folk community, the plant grows in the highlands of central Borneo……

An Immune System Trained to Kill Cancer

September 13, 2011 – “…Dr. June said his team hopes to use T-cells against solid tumors, including some that are very hard to treat, like mesothelioma and ovarian and pancreatic cancer. But possible adverse reactions are a real concern, he said, noting that one of the protein targets on the tumor cells is also found on membranes that line the chest and abdomen. T-cell attacks could cause serious inflammation in those membranes and mimic lupus, a serious autoimmune disease…..”

Combining Curcumin (Diferuloylmethane) and Heat Shock Protein Inhibition for Neurofibromatosis 2 Treatment: Analysis of Response and Resistance Pathways.

September 12, 2011 – The NF2 gene product, merlin, is activated (dephosphorylated) by contact inhibition, and promotes growth suppression. We investigated the effect of curcumin (diferuloylmethane), a molecule with anti-inflammatory and anti-tumorigenic properties, on human schwannoma cell growth and the regulation of merlin by curcumin in both NF2 cells and neuroblastoma (non-NF2) cells. Curcumin inhibited the growth of HEI-193 schwannoma cells in vitro, and down-regulated the phosphorylation of Akt and ERK 1/2….. The combination of curcumin and a heat shock protein inhibitor synergistically suppressed schwannoma cell growth. Our results provide a rationale for combining curcumin and KNK437 in the treatment of NF2.

Following A Path To A Potential Therapy For NF2, A Rare Tumor Disorder

September 4, 2011 – The proteins that provide cells with a sense of personal space could lead to a therapeutic target for Neurofibromatosis Type 2 (NF2), an inherited cancer disorder, according to researchers at The Wistar Institute. Their findings, which appear in the April 12 issue of the journal Cancer Cell, could have profound implications for NF2….

Definitive NF2 article by Dr. Gareth Evans

September 1, 2011 – In this GeneReview: Summary • Diagnosis • Clinical Description • Differential Diagnosis • Management • Genetic Counseling • Molecular Genetics • Resources • References • Chapter Notes. Last Update: August 18, 2011.

A benefit through Advocure NF2 Inc to fund children’s tumor research.

August 31, 2011 – “Saturday, October 22nd, 7:30PM, Vice Niteclub, 1251 Arroyo Way, Walnut Creek, CA. …This year will be the 7th annual Halloween Bash and our goal is to raise $100,000. All donations will be matched by the Thoms family so we could conceivably raise a total of $200,000. Of this money, $0 will go to administrative causes. All of it will go to research being performed by doctors and scientists working on a cure/ therapy to control tumor growth. While we are not close to eliminating NF2, we are extremely close to containing it and preserving the quality of life for Camille and thousands of others affected…..”

NIH Bench-to-Bedside Program

August 26 – Established in 1999, the Bench to Bedside (B2B) Program funds research teams seeking to translate basic scientific findings into therapeutic interventions for patients and to increase understanding of important disease processes (including in rare diseases, such as NF)……A B2B award provides up to $135,000 a year for two years……Letters of Intent are now being accepted for the 2012 cycle and are due on September 28, 2011. More information about the program and the application process can be found at:, or submit any questions by email to:

Various NF video interviews at CTF

August 21 – According to CTF: “all videos ARE captioned”. Dr. Scott Plotkin – Progress of NF2 clinical trials, Gareth Evans – Link Between NF2 and Schwannomatosis, Dr. Jaishri Blakely – Schwannomatosis or NF2, Dr. Gareth Evans – Background – NF2 & Schwannomatosis, Dr. Gareth Evans – Visual Traits to NF2?, Matthias Karajannis – What’s happening with NF2 Clinical Trials, etc.

Five New Neurofibromatosis Drug Discovery Initiative Awards

August 20 – The Children’s Tumor Foundation (CTF) is delighted to announce the funding of five new Drug Discovery Initiative (DDI) Awards totaling $125,000, from applications received for our spring 2011 deadline.

2011 NF Conference Abstract Now Available!

August 20 – The NF Conference is the largest annual gathering of researchers and clinicians focused on neurofibromatosis and has been convened by the Children’s Tumor Foundation (CTF) for more than 20 years.

Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2

August 19 – This trial studies whether Everolimus is efficacious in treating Neurofibromatosis type 2. Identifier: NCT01419639.

Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

August 15 – The purpose of the study is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients. Secondary objectives include determining if RAD001 treatment will improve hearing ability in NF2 patients. Identifier: NCT01345136.

A benefit through Advocure NF2 Inc to fund children’s tumor research.

August 15, 2011 – “…This year will be the 7th annual Halloween Bash and our goal is to raise $100,000. All donations will be matched by the Thoms family so we could conceivably raise a total of $200,000. Of this money, $0 will go to administrative causes. All of it will go to research being performed by doctors and scientists working on a cure/ therapy to control tumor growth. While we are not close to eliminating NF2, we are extremely close to containing it and preserving the quality of life for Camille and thousands of others affected…..”

New clues to fighting genetic disease.

August 12, 2011 – “Scientists have hit on a genetic trick that opens up fresh avenues for the treatment of devastating diseases, such as cystic fibrosis, muscular dystrophy and certain forms of cancer,” reported The Guardian…..

Gene Therapy Cures Adult Leukemia

August 11, 2011 – Two of three patients dying of chronic lymphocytic leukemia (CLL) appear cured and a third is in partial remission after infusions of genetically engineered T cells…..(now it’s NF2 and solid tumors’ turn)….

NFRP Funding Opportunities

August 9, 2011 – The Neurofibromatosis Research Program (NFRP) has announced the funding mechanisms for the FY11 appropriation of $16M…..

CTF Funds SIX Neurofibromatosis Young Investigator Awards

August 8, 2011 – The Children’s Tumor Foundation (CTF) is delighted to announce the funding of SIX Young Investigator Awardees from application received for our 2011 program. The recipients include three postdoctoral awardees and three graduate students; three focused on aspects of NF1 including tumors, bone dysplasia and learning disabilities; and three focused on NF2 or schwannomatosis…..

NF2 research Progress Report at OSU – August 2011

August 1, 2011 – Our research at OSU (partially funded by AdvocureNF2) focuses on the generation of mouse models for NF2-associated schwannoma and meningioma and the identification of novel therapeutics for these tumors. We have made the following progress in the past year…..

Young NF2er with awesome message at a NJ beach near Ocean City!
July 25, 2011 – Kim Underwood Gillet’s son Devon, at a beach in Ocean City, NJ! It was his first time at a boardwalk. He had a BLAST! He came all the way from Canada to come to the Ride4NF fundraiser, hosted by Jaime L. Cirillo on July 23:) Awesome message and photo, thank you!

Jessica Stone update

July 25, 2011 – …It’s been three years since Jessica made the decision to lose her hearing in order to save her life. A tumor caused by her neurofibromatosis II (NF2) was growing on her brain stem removing it would mean silencing her world, a choice she willingly made, but one she knew wouldn’t cure her…..

Beet juice: It does a body very good

July 20, 2011 – Beets offer more than just nitrite. Beet juice is packed with Vitamin A, Vitamin B-6, iron and calcium. They have antioxidant and anti-inflammatory benefits. Beet fibre is good for digestive health.
An Italian study linked the carotenoids found in beets — lutein and zeaxanthin — as unique support to eye health.
In some lab studies, the betanin pigments found in beets have also been shown to reduce tumor cell growth.

Proposed Trial for NF2 in the UK

July 13, 2011 – In a human cell culture model, Professor Oliver Hanemann showed that this new target can be effectively inhibited with a drug that leads to the reduction of the tumour’s growth. Many NF2 sufferers have multiple tumours, including schwannomas, at nerve endings in the skin. These ‘nerve tumours in the skin’ allow an innovative clinical trial design in which one can directly test if the drug gets into the tumour and affects the tumour.

Nilotinib alone or in combination with Selumetinib is a drug candidate for Neurofibromatosis type 2.

July 13, 2011 – Loss of the tumor suppressor merlin is a cause of frequent tumors of the nervous system, such as schwannomas, meningiomas, and ependymomas, which occur spontaneously or as part of neurofibromatosis type 2 (NF2). Because there is medical need for drug therapies for these tumors, our aim is to find therapeutic targets. We have studied the pathobiology of schwannomas, because they are the most common merlin-deficient tumors and are a model for all merlin-deficient tumors. With use of a human schwannoma in vitro model, we previously described strong overexpression/activation of platelet-derived growth factor receptor-ß (PDGFR-ß) leading to strong, long-lasting activation of extracellular-signal-regulated kinase (ERK1/2) and AKT and increased schwannoma growth, which we successfully inhibited using the PDGFR/Raf inhibitor sorafenib.

The Neurofibromatosis Conference: Tumor Targets, Drug Trials and a Schwannomatosis Database

June 15, 2011 – “The 2011 NF Conference kicked off this weekend with some interesting and exciting presentations. On Saturday, Dr. Filippo Giancotti (MSKCC) provided an update on his novel research first reported in 2010 that described for the first time a role for NF2 merlin protein in the cell’s nucleus. Dr. Giancotti has continued to unravel the signaling activities of merlin in the nucleus, and interestingly also showed that in sections of human meningioma tissue, merlin protein is not present in the nucleus. This would provide support for Dr. Giancotti’s unique idea that merlin controls normal cell division and that lack of its nuclear function might be a factor in promoting NF2 tumor growth….”

Advocure honors Dr. Marco Giovannini, director of HRI’s Center for Neural Tumor Research for his work on NF2.
From left to right: Jane Tran Ly of CNTR, Sheila Heal of Advo, Dr. Marco Giovannini – Director of CNTR, Roland Thoms of Advo, Nicolas Xavier Bonne of CNTR, and Jérémie Vitte of CNTR
May 27, 2011 – In January 2008, Dr. Giovannini founded the Center for Neural Tumor Research at HRI, with a mission to translate research on the cellular and molecular basis of neurofibromatosis type 2 (NF2) related tumorigenesis into tools relevant for the treatment of the disease.
Transcript of interview with Dr. Giovannini to follow. Please check back soon. Thank you.

Proposed Phase II Trial of Rapamycin on NF2 Tumors in FY11

May 26, 2011 – In this newly funded NF2 clinical trial, RAD001 (rapamycin) is assessed as a candidate drug therapy for NF2 by its effects on the growth and/or shrinkage of the characteristic NF2 tumors: vestibular schwannomas, meningioma and ependymoma. RAD001 has been widely tested in patients and is known to be safe: indeed it was one of the first drugs to enter large scale clinical trials for NF1 plexiform tumors. If there are promising results, this trial will pave the way for future combination therapy drug trials, since animal studies have suggested that RAD001 might be additive if combined with other drugs such as Bevacizumab or Lapatinib, also being tested as candidate NF2 drug therapies. The Principle Investigator of this proposed study is Dr. Matthias Karajannis, New York University Langone Medical Center.

Five New Neurofibromatosis Drug Discovery Initiative Awards

May 26, 2011 – The Children’s Tumor Foundation (CTF) is delighted to announce the funding of five new Drug Discovery Initiative (DDI) Awards totaling $125,000, from applications received for our spring 2011 deadline. DDI Awards fund studies to test candidate neurofibromatosis (NF) drugs for NF1, NF2 or schwannomatosis, offering $15,000 for in vitro cell-basedscreens, up to $30,000 for in vivo animal model screens and up to $50,000 to continue outstanding in vivo research projects. Launched in 2006, it is one of CTF’s most successful programs.

South Jersey’s Ride4NF Bikerun

May 20, 2011 – As many of you already know, this is the second year that I will be hosting South Jersey’s Ride4NF Bikerun and Benefit to raise money for NF Network to help fund research so that we may someday soon find a cure.
This year, I will be 35 years old, one year away from the age my Mom was when NF2 took her life away. I will fight this disorder my whole entire life if I have to, so that I can live to see my kids grow up, and so that no other children have to lose a parent, or no parent has to lose a child to NF2. ……more.

2011 NF2 Fundraising Plan – a nationwide Halloween Bash!!!!

May 17, 2011 – The Plan: Throw a Halloween Bash party/event in your city to raise money to fund children’s tumor research, on behalf of Advocure NF2.
The Backstory: For the last 7 years, Roland and Nicole Thoms have held a Halloween Bash on the Friday (or Saturday) night before Halloween. Through entrance fees, a silent auction, sponsorships and other donations, the event raises about $30,000 each year.
Why we are doing this: Last year, we provided $20,000 to Dr. Welling of Ohio State University for his research on developing drugs and testing natural compounds against NF2 associated schwannomas and meningiomas. This year, our goal is to raise $100,000 to not only continue funding Dr. Welling but to also identify other research partners to control and eventually cure NF2. 100% of funds raised go to our goals. Checks to be made out to Advocure NF2.
Today: Contact Roland Thoms and join forces on the 2011 nationwide Halloween Bash. 925-575-1593 (California) or ……more.

Surgery Helps a Young Girl Take Back Her Smile

May 17, 2011 – In 2008, Cowen suffered partial paralysis after undergoing surgery to remove a tumor from the right side of her brain stem. Cowen was bound to a wheelchair for months as she worked to regain strength and overcome paralysis on the left side of her body, paralysis that made it impossible for Cowen to move the muscles that would allow her to smile.
But a year later, Cowen underwent an innovative surgery at the Massachusetts Eye and Ear Infirmary in Boston — called free gracilis transfer — to restore muscle movement to her face. ……more.

Metabolic Modulation of Glioblastoma with Dichloroacetate (DCA)

May 15, 2011 – Medical Researchers at the University of Alberta reported today evidence that the orphan generic drug Dichloroacetate (DCA) may hold promise as potential therapy for solid tumors and perhaps the deadliest of all human cancers: a form of brain cancer called glioblastoma.

Prognostic factors for the incidence and recovery of delayed facial nerve palsy after vestibular schwannoma resection

May 11, 2011 – Preservation of facial nerve function in vestibular schwannoma (VS) resections remains a significant operative challenge. Delayed facial palsy (DFP) is one specific challenge yet to be fully elucidated. The aim of this study was to evaluate DFP among VS resection cases to identify significant prognostic factors associated with its incidence and clinical recovery……..more.

Neurofibromatosis awareness month

May 7, 2011 – Sarah Gorden is 26-years-old and lives in Westbank with her husband Hans and their dog and two cats.
She was diagnosed with NF2 at the age of 13 years old. She was having trouble walking and Doctors discovered her brain and spine were loaded with benign tumours, and she was diagnosed with NF2.
After surgery to remove several tumours from her spine she regained my ability to walk.
But the battle was far from over……..more.

Health Fair at the 2011 NF Forum for NF2 & Schwannomatosis Patients

May 5, 2011 – The Children’s Tumor Foundation is partnering with Mayo Clinic to offer a “Health Fair” for individuals with NF2 or Schwannomatosis on July 29, 2011 as part of the NF Forum. …….more.

Paving the Way to Future Treatments

May 4, 2011 – Marco Giovannini, M.D., Ph.D. leads the Laboratory of Neural Tumor Research in the Wallis Annenberg Research Center at the Institute. His primary area of study is Neurofibromatosis Type 2 (NF2), a genetic disorder that is characterized by multiple tumors on the cranial and spinal nerves, and by other lesions of the brain and spinal cord. Tumors affecting both of the auditory nerves are the hallmark and hearing loss beginning in the teens or early twenties is generally the first symptom…….more.

Back to the Future: Proceedings From the 2010 NF Conference

May 4, 2011 – The full 2010 NF Conference report is now available for FREE download.

Bristol-Myers Squibb Offers Funding Grants for Clinical & Discovery Research

May 4, 2011 – “The Collaborative Science Center of Excellence (CSCoE) welcomes you to the Bristol-Myers Squibb Investigator Sponsored Research (ISR) application website. ISR is also referred to as Investigator Initiated Research, Investigator Sponsored Trials, and Investigator Initiated Trials. This website provides information to investigators interested in submitting an ISR proposal.”
Bristol-Myers Squibb has updated its process to receive web applications for investigator-sponsored research (ISR). Specific time periods for submissions are now offered. Bristol-Myers Squibb will be accepting research proposals/applications beginning April 15, 2011 through midnight June 30, 2011. It is imperative that your application is received on or before June 30, 2011, to ensure that it is considered during the established review cycle. An internal Scientific Research committee will be assessing all submitted applications with the intent of conveying the committee’s decision by August 15, 2011.
Open to both U.S. and non-U.S. Clinical Research Investigators……more.

Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

May 3, 2011 – The purpose of the study is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients. Secondary objectives include determining if RAD001 treatment will improve hearing ability in NF2 patients.
RAD001 is an oral drug that is approved by Food and Drug Administration (FDA) for other types of tumors, it is not approved by the FDA for treatment of NF2 related tumors……more.

House Ear Institute Changes Name to House Research Institute

May 3, 2011 – House Ear Institute Board of Trustees announced last week that the nonprofit has changed its name to House Research Institute. The change to Research in the name more accurately reflects the institute’s expanded research mission.
“Although our focus is still the ear, we are finding more and more that hearing dysfunction often goes beyond the ear,” said James Boswell, chief executive officer of House Research Institute. “While we remain at the cutting-edge with regard to development of hearing prosthetics like the cochlear implant and the auditory brainstem implant, some of our most innovative science is in the areas of genetics, regeneration, prescription drug-induced hearing loss, neural tumors, and autism.”…..more.

House ‘Dear Colleague’ letter NOW CIRCULATING, deadline May 10th

May 2, 2011 – Attached please find a draft letter for your use in contacting your members of Congress about the ‘Dear Colleague’ letter that is circulating. The deadline to sign the letters is Tuesday May 10th.
Congressman Gutierrez’s office sent out a ‘Dear Colleague’ letter today asking for member of Congress to sign onto two letters supporting NF research. The first is supporting $16 million for the Army’s NF Research Program in the Fiscal Year 2012 Department of Defense Appropriations bill and the other is asking for NF specific report language to be included in the Fiscal Year 2012 Labor, Health and Human Services, Education Appropriations bill.

Radiographic regression of cranial meningioma in a NF2 patient treated by bevacizumab

April 26, 2011 – Neurofibromatosis type 2 (NF2) is a rare genetic disorder characterized by the development of multiple tumors of the central nervous system including bilateral vestibular schwannomas (VSs) and multiple meningiomas. Treatment options in NF2 tumors include surgery and radiotherapy. Bevacizumab, a humanized mAb against vascular endothelial growth factor (VEGF), has recently been proposed to NF2 patients with inoperable VS, leading to hearing improvement and some decrease in VS size [ 1, 2]. Interestingly, regression under bevacizumab treatment has also been reported in a sporadic case of non-NF2 anaplastic meningiomas [ 3].
This case describes a 51-year-old female with NF2. A right 40-mm VS responsible for deafness was removed at the age … more

Scientists Create Stable, Self-Renewing Neural Stem Cells

April 26, 2011 – In a paper published in the April 25 early online edition of the Proceedings of the National Academy of Sciences, researchers at the University of California, San Diego School of Medicine, the Gladstone Institutes in San Francisco and colleagues report a game-changing advance in stem cell science: the creation of long-term, self-renewing, primitive neural precursor cells from human embryonic stem cells (hESCs) that can be directed to become many types of neuron without increased risk of tumor formation….. more

Transcript of Dr. Brad Welling’s interview with Advocure on March 30, 2011

April 24, 2011 – I conducted a phone interview with Dr. Brad Welling of Ohio State University (OSU) on Wednesday, March 30th. I was introduced to the work of Dr. Welling by Marie Drew, chairperson of Advocure NF2. Based on his commitment to the NF2 community and his promising work with drug developments and naturals to find an effective treatment on tumors, I directed funds generated through 2010 fundraising, through Advocure, to his research efforts….. more

Major NF2 funding opportunity from the Children’s Tumor Foundation in FY11

April 22, 2011 – In Fall 2010 CTF announced a two-year expansion of NFPC (2011-2013) and issued a Request for Applications. CTF is now issuing a SECOND Request for Applications specifically seeking applications from candidate NF2 Centers. Up to THREE NF2 Centers will be funded – each will be eligible to receive up to $150,000 per year for two years as well as up to 3 x $25,000 milestone-driven incentive awards. This is a tremendous opportunity for outstanding NF2 centers to collaborate with industry and with other expert preclinical centers, and to access guidance from a CTF-appointed expert industry advisory board and project manager…. more

‘The Gift’

April 19, 2011 – Paco Rodriguez died tragically on November 22, 2009. His “gift” was a vital liver that allows NF-Mid-Atlantic’s Meaghan Kingsley to live on while facing her battles with NF2. Don’t miss the documentary “The Gift”, on E-60 airing on ESPN, 4/19/11 at 7:00 p.m. (EST) and throughout the week. Check your local listings for ESPN’s E-60.

CDMRP Research Funding for 2011 (UNCLASSIFIED)

News Release: April 18, 2011 – Comprehensive Program Announcements are expected to be released during spring/summer of 2011. The announcements will include detailed descriptions of funding mechanisms, evaluation criteria, submission requirements, and deadlines. Each Program Announcement may be downloaded from the website (, the CDMRP website (, and the CDMRP eReceipt website ( upon its release…. more

Path to Potential Therapy for NF2, a Rare Tumor Disorder

April 18, 2011 – The proteins that provide cells with a sense of personal space could lead to a therapeutic target for neurofibromatosis type 2 (NF2), an inherited cancer disorder, according to researchers at The Wistar Institute. Their findings, which appear in the April 12 issue of the journal Cancer Cell, could have profound implications for NF2 and related cancers, such as mesothelioma….. more

$16M Appropriated for NF Research in FY11

Friday, April 15, 2011 – I am pleased to announce that Congress has appropriated $16 million for NF research for fiscal year 2011. This research, conducted through the Department of Defense, is critical both to those living with the challenges of NF and the brave men and women serving our country. As many of you know, research into treatments and cures for NF and its various manifestations has implications for those beyond the NF community. Advancing our understanding of mast cells to stop tumor growth helps us understand how we may also speed wound healing for our war fighters. Understanding the mechanisms of pain in NF can allow us to understand the signaling pathways that can block the pain our soldiers may face. Imaging technologies advanced to understand NF tumor growth are proving essential in MRI volumetric imaging to the broader community, particularly our armed forces. We are grateful that those serving in Congress recognize the importance of NF research for those serving our country, and those living with NF. We know that the importance of NF research validates that recognition…… more

Hearing with Our Brain

Friday, April 15, 2011 – The House Ear Institute (HEI) in Los Angeles was established in 1946 as a non-profit organization by Howard P. House, M.D. for the exploration of the auditory system from the ear canal to the cortex of the brain. Institute scientists investigate hearing loss and ear disease at the cellular and molecular levels, as well as the complex neurological interactions between the auditory system and brain…… more

RegeneRx Reports New Statistically Significant Data Confirming Repair Of Corneal Damage In Dry Eye Model

Friday, April 15, 2011 – RegeneRx Biopharmaceuticals, Inc. (OTC Bulletin Board: RGRX) (“the Company” or “RegeneRx”) today announced positive new data related to RGN-259, its preservative-free ophthalmic drug candidate. In a second “dry eye” study conducted by Ora, Inc. using their Preclinical CAE™ Murine (mouse) Model, four active concentrations of RGN-259 were compared to three control groups, consisting of a negative control (vehicle) and two positive controls (doxycycline and Restasis™)…… more

Proteostasis Regulators

Thursday, April 14, 2011 – Advances in our ability to characterize and pharmacologically control the Proteostasis Network create new opportunities to ameliorate a wide range of diseases. It is now possible to use orally available small molecules, or Proteostasis Regulators, to restore the natural balance of the Proteostasis Network. It has been demonstrated that Proteostasis Regulators can fold mutated proteins, potentially offering a significant benefit to patients suffering from genetic diseases associated with loss of protein function. Scientists have also shown that Proteostasis Regulators can control other biological pathways within the Proteostasis Network to ameliorate degenerative diseases associated with protein aggregation, such as Alzheimer’s and Huntington’s diseases…… more

Advocure’s new brochure is ready

Friday, April 08, 2011 – Advocure’s new brochure is ready. We would like to thank both Jennette Braaten and Bill Reese for donating their time and expertise in designing our brochure. Thank you.

NF2 Webinar: “Ask the Doctor”

Tuesday, April 05, 2011 – At 8:00 PM – 9:30 PM EDT today, NF Inc. Network invites you to come and bring your NF2 questions to the “Ask the Doctor” Webinar. Dr. Scott R. Plotkin, from the NF Clinic at MGH in Boston, will be the presenter. Learn about the latest NF2 research while having the opportunity to address your specific NF2 questions to a leading specialist about the general care and management of NF2. This meeting is for anyone who is facing a diagnosis of NF2, living with NF2 or providing care for someone affected with this disorder. Thank you.

Cancer Treatment Breakthrough, UK

Tuesday, April 05, 2011 – Scientists from the School of Pharmacy at Queen’s University Belfast and Almac Discovery Ltd, announce the first publication describing a potential new anti-angiogenic therapy based on a natural protein first discovered at QUB. Anti-angiogenics prevent the growth of new blood vessels in tumours and starve them of nutrients, leading to inhibition of tumour growth. [applicable to NF2?]….. more

Treating Neurofibromatosis Type II (NF2)

Tuesday, April 05, 2011 – Washington University physicians at the Siteman Cancer Center are forming a comprehensive center for the treatment of neurofibromatosis type II (NF2). NF2 is a hereditary disease that often results in multiple brain and spinal tumors. In this Cancer Connection podcast [see website], Siteman neurosurgeon Michael Chicoine, MD, discusses the symptoms of NF2 , treatment and the new NF2 center. According to Chicoine, there are only two other comprehensive NF2 centers in the country, one in Los Angeles and one in Boston…… more


Sunday, March 27, 2011 – ….NF2 is caused primarily by dysfunction of the NF2 gene product called merlin which inhibits directly PAK1, a Rac/ CDC42-dependent Ser/Thr kinase. In collaboration with Dr. Hiroshi Maruta at the Hamburg University Hospital (UKE) in Germany, we are screening a number of pharmacological inhibitors of PAK1 in a murine xenograft model for NF2. Tumors are formed from subcutaneous implantation of a schwannoma cell line into the flanks of immunodeficient mice. It is our objective to find a PAK1 inhibitor which reduces the growth of schwannomas in our NF2 mouse model. This drug(s) will have tremendous clinical implications for the future, particularly if it could be used in place of invasive surgery, which often leaves patients debilitated…… more

Noninvasive Brain Stimulation May Improve Swallowing After Stroke

Sunday, March 27, 2011 – Stroke patients who received electrical brain stimulation coupled with swallowing exercises showed greater improvement in swallowing ability than patients who did not receive this stimulation, according to a pilot study reported in Stroke: Journal of the American Heart Association…… more

CTF Young Investigator Uncovers New Functions of NF2 Merlin Protein

Friday, March 25, 2011 – Timmy Mani, a Children’s Tumor Foundation 2008-2010 Young Investigator Award recipient, has published a paper in Molecular Cell Biology describing new findings that help understand how the NF2 protein merlin regulates cell growth. These findings were made during Timmy’s time as a CTF YIA in the lab of Dr. Wallace Ip at the University of Cincinnati. It is known that merlin function requires it to bind to the interior of the cell membrane. Timmy’s research shows that a key player in making this binding happen is the phosphoinositide PIP2. When merlin protein is modified so that it can no longer bind PIP2, the cells grow uncontrollably. This finding suggests an important role for PIP2 in controlling cell growth and that PIP2 function will be an interesting area to explore in looking for new drug targets for NF2 therapies…… more

Trailor of upcoming NF2 movie, ‘Perseverance Factor’

Thursday, March 24, 2011 – Movie trailer of Rebecca Durek’s upcoming May 2011 NF2 documentary release, ‘Perseverance Factor’.

The Emergence of Personalized Medicine

Thursday, March 24, 2011 – The 2011 Mass Bio Annual Meeting, from Monday, March 21 – Tuesday, March 22, will focus on Personalized Medicine: Every Patient Tells a Story. Leading experts will discuss successes and failures, road blocks, business models, regulatory issues and other challenges around Personalized Medicine, while also sharing solutions and best practices…….. “we are seeing the emergence of personalized medicine. This is based in understanding an individual’s genetic makeup, and then identifying why a person develops cancer or heart disease or any other malady. This information helps doctors to determine which patients are likely to benefit from specific molecularly-targeted medicines. Though this seemed like science fiction just a few years ago, the first results of personalized medicine are already emerging specifically from cancer trials. The result we hope will be faster and more meaningful clinical trials and a faster path of getting drugs to the market.In response to this, drug companies are realizing that if they are developing a new drug they need to be thinking about developing a ‘companion diagnostic’ – e.g. a blood test so they can identify, prior to prescribing the drug, the patients that will most benefit from the treatment. This is revolutionizing the way drug companies are planning their drug development strategies and excitingly encouraging partnering between drug and diagnostic companies early on to ensure that no promising drug is left behind due to lack of a diagnostic” (CTF blog)…… more

Tiny LNA-based compounds inhibit entire disease-associated microRNA families

Monday, March 21, 2011 – A study published online in this week’s Nature Genetics demonstrates that tiny Locked Nucleic Acid (LNA)-based compounds developed by Santaris Pharma A/S can inhibit entire disease-associated microRNA families. This provides a potential new approach for treating a variety of diseases including cancer, viral infections, cardiovascular and muscle diseases…… more

FDA Approves Gadavist For Central Nervous System Scans

Thursday, March 17, 2011 – The U.S. Food and Drug Administration approved Gadavist (gadobutrol), a gadolinium-based contrast agent, for use in patients undergoing magnetic resonance imaging (MRI) of the central nervous system…… more

Clinical trial for dry mouth

Thursday, March 17, 2011 – A clinical trial using an all-natural lozenge to treat dry mouth, a condition that impacts 40 percent of American adults, is under way at Georgia Health Sciences University College of Dental Medicine…..more

Helping To Identify Small Tumors Early

Thursday, March 17, 2011 – … research is focused primarily on the electrophysiological assessment of hearing through evoked responses and neural activity of the auditory nerve for patients with hearing loss or acoustic neuromas (benign tumors growing on the balance nerve that can affect the auditory nerve and hearing)…… more

Gene therapy reverses symptoms of Parkinson’s disease

Thursday, March 17, 2011 – A gene therapy called NLX-P101 dramatically reduces movement impairment in Parkinson’s patients, according to results of a Phase 2 study published today in the journal Lancet Neurology. The approach introduces a gene into the brain to normalize chemical signaling. The study is the first successful randomized, double-blind clinical trial of a gene therapy for Parkinson’s OR ANY NEUROLOGICAL DISORDER….. more

Multifocal Extracranial Meningioma Metastases

Tuesday, March 15, 2011 – Subcutaneous nodular scalp and pulmonary lesion histologic symptoms (Figure 2) revealed atypical meningioma (WHO grade II). The neurofibromatosis type 2….. more

Researchers locate brain’s loudness map

Tuesday, March 15, 2011 – ABC Online – Scientists in the UK have for the first time identified a region in the brain that processes changes in sound loudness, which could lead to improvements in brain implant technology….. more

Interview with Dr. Brad Welling – scheduled for March 30th

Friday, March 11, 2011 – Dr. Welling with Ohio State University is conducting research, partially funded by Advocure NF2 Inc, testing drugs and natural compounds against NF2-associated tumors. Please Reply with your questions to Thank you…… more

Advocure’s newsletter: ‘NF2 Compass’ – Spring 2011 issue is released

Mar 10, 2011 – Contains recent NF2 News and articles of NF2 importance, including a patient’s perspective of the NIH’s ongoing ‘NF2 Natural History Study’, and a list of current NF2 clinical trials & studies.

‘A Struggle for Hope’

Monday, March 07, 2011 – Featuring Courtney Gale, and the manifestations of NF2. This film has been entered in the 2011 Neuro Film Festival from the American Academy of Neurology at Let’s put our brains together to support brain research. Directed by Debbie Wolfe. Reported by Donna Winchester. For further information or press inquiries, contact Debbie Wolfe at: Please vote for us! Here’s how:
1. Visit this URL:
2. Next, click on the “Vote Now” button.
3. Then scroll down, as ‘A Struggle for Hope’ is near the bottom. Thank you.

Request for Information (RFI) for the NIH Director’s Early Independence Award Program

Sunday, March 06, 2011 – A major concern among young researchers is the gloomy prospect that when they are done with their Ph.D or MD training, they won’t be able to get funding to set up their own lab. This might include Children’s Tumor Foundation Young Investigator Award recipients ready to become independent neurofibromatosis researchers. It is a worrying situation as young researchers are the ‘seed corn’ of science, ensuring future continued research progress. Faced with the current financial situation, though, many young scientists leave scientific research for other career paths. To address this, and especially to ensure support for the brightest young stars, the National Institutes of Health is planning an Early Independence Award (EIA) that would allow researchers to move directly from completing their Ph.D or MD, and on to running an independent lab. At this time the NIH is asking for YOUR feedback and input on this proposed program. Deadline for feedback: March 18th. For more information and to offer your comments…… more

CDMRP’s FY10 Annual Report

Wednesday, March 02, 2011 – CDMRP’s vision is to find and fund the best research to eradicate diseases and support the war-fighter for the benefit of the American public. Consumer involvement with CDMRP has exceeded 500 organizations since it began in 1992. CDMRP continues to value the experiences of our consumer advocates…… more

PTC Therapeutics Announces Achievement of Milestone in BMI1 Collaboration with the Wellcome Trust – PTC Receives $2.2 Million Payment

Wednesday, March 02, 2011 – PTC Therapeutics, Inc. (PTC) today announced that it has identified a chemical series of molecules that penetrates the blood-brain barrier in animal models and reduces levels of BMI1, a protein linked to drug-resistant cancers. The research marks a milestone in PTC’s collaboration with the Wellcome Trust and triggers drawdown of a $2.2 million tranche of their $5.4 million Seeding Drug Discovery award…… more

Meningioma Webinar:

Mar 1, 2011- Treatment and Research Updates, at 10am PST.

Feb 28, 2011
International Rare Disease Day.

Feb 21, 2011
Meningioma Mommas is a 501(c)(3) not-for-profit organization dedicated to raising meningioma awareness and funding for meningioma specific research.

Feb 17, 2011
Back to the future: Proceedings from the 2010 NF Conference.
Full report.

Feb 12, 2011
Opening options for neurofibromatosis 2. The Galloways’ generous gift made the phase II Avastin trial for NF2 at John Hopkins, MGH, and NCI possible.

Feb 11, 2011
New Study Suggests New Therapies for NF2 Patients.

Feb 9, 2011
Movie trailer and highlights to upcoming debut film entitled, “Perseverance Factor”. Story of Rebecca Dufek’s (NF2er) recent attempt to summit Mount Kilimanjaro, the highest peak in Africa on Oct 10, 2010. Her bio and story can be found here.

Jan 17, 2011
FDA Approves Avastin; Serious Side Effects

Jan 6, 2011
Two Acceleration Methods Lead To Major Advance In MRI, Allowing Much Faster Brain Scans.

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