Neurofibromatosis Times

HDAC6 Inhibitors – Commercial Collaboration Opportunity for Neurofibromatosis Researchers

Kim Hunter-Schaedle — Wed, 10 Mar 2010 15:27:30 +0100

Acetylon Pharmaceuticals, a new biotech company developing technology from Harvard and the Dana-Farber Cancer Institute, is seeking neurofibromatosis researchers to assess its selective small molecule, orally administered inhibitors of HDAC6 in preclinical models of neurofibromatosis. While Acetylon’s initial clinical priority is multiple myeloma, there is potential benefit of non-isoform specific HDAC iRead More...

2010 NF Conference: Announcing Our First Sponsors

Kim Hunter-Schaedle — Mon, 08 Mar 2010 17:07:49 +0100

We are delighted to announce the first four sponsors for the Children’s Tumor Foundation 2010 NF Conference: AstraZeneca, the Biotechnology Industry Organization, Nexgenix Pharmaceuticals, and NF Ireland. All four entities have in unique ways been long-time supporters and partners in the Children’s Tumor Foundation’s mission to end neurofibromatosis. We are delighted to have them on bRead More...

Interersting Series in NY Times on Cancer Research

John Risner — Sun, 07 Mar 2010 03:17:26 +0100

The NY Times published a three part series titled "Target Cancer" last week that is very informative. While the focus is on melanoma, many of the issues the patients, researchers and clinicians faced during the trial are universal and have specific relevance to NF research.

Story link: http://nyti.ms/aV4iov

Foundation Funded Research Shows Promise for Lovastatin in Healing NF1 Bone Fractures

Kim Hunter-Schaedle — Fri, 05 Mar 2010 13:28:32 +0100

Statin drugs, widely prescribed for cholesterol management, are well known in the neurofibromatosis community for the ongoing Phase II clinical trials of the drug Lovastatin for the treatment of NF1-related learning disabilities. However Lovastatin may also have promise for the treatment of NF1-related bone fractures. Pseudarthrosis or ‘false joint’ can occur in children with NF1 when a long bone such as in the leg fractures but fails to heal because new bone is unable to beRead More...

Foundation Grants Available to Fund Small Neurofibromatosis Clinical Trials

Kim Hunter-Schaedle — Tue, 02 Mar 2010 21:32:13 +0100

The Children’s Tumor Foundation is now offering Clinical Trial Awards up to $125,000 to fund pilot-scale clinical testing of potential candidate drugs for the treatment of tumors and other manifestations of NF1, NF2 or schwannomatosis. Two CTF-funded neurofibromatosis trials are currently underway and recruiting patients: a Phase Zero trial of lapatinib for treatment of NF2 vestibular aschwannomas (Dr. Jaishri Blakeley, Johns Hopkins University) and a Phase I trial of sorafenib for treaRead More...

Some Clarity on NF2-Related Cataracts?

Kim Hunter-Schaedle — Fri, 26 Feb 2010 14:50:57 +0100

Over half of individuals with NF2 will develop cataracts in the lens of the eye called PSCs. A new report* (links to abstract) presents some ideas for the biological basis of these cataracts. The NF2 gene was inactivated in a genetic mouse model only in the developing cells that will give rise to the lens, called fiber cells. Unlike normal fiber cells, the NF2 inactivated fiber cells were unable to stop dividing at the correct time in development, and also continued to express geneRead More...

2010 CDMRP Neurofibromatosis Grant Program OPEN!

Kim Hunter-Schaedle — Wed, 24 Feb 2010 15:22:06 +0100

The Department of Defense Congressionally Directed Neurofibromatosis Research Program (CDMRP NFRP) is now open for applications for Clinical Trial Awards, Investigator Initiated Awards and Investigator Initiated Focused Research Awards. For all programs, pre-applications are due April 29, 2010 and full applications May 20, 2010. A total of $13.75M is available in 2010. For more information and application forms: http://cdmrp.army.mil/funding/nfrp.htm

Genetic Mouse Model Lights Up Neurofibromatosis Tumors

Kim Hunter-Schaedle — Wed, 24 Feb 2010 14:39:26 +0100

A new report* (links to abstract) from Jessica Hawes and Karlyne Reilly at the National Cancer Institute describes a new genetically engineered mouse model of NF1 in which the tumors literally become illuminated. This is thanks to a luciferase reporter gene that ‘lights up’ a cell whenever and wherever tumor cells are growing. ‘Glowing’ cells can be detected through bioluminescence imaging of the living aRead More...

Foundation Funded Breakthrough NF2 Research Published in Top Journal ‘Cell’

Kim Hunter-Schaedle — Thu, 18 Feb 2010 23:04:41 +0100

As we have reported, some major advances have been made recently to unravel the functioning of merlin, the protein made by the NF2 gene. Merlin is a tumor suppressor protein and it does not operate properly in patients with NF2, which leads to the formation of schwannomas and meningioma tumors. However its not obvious how merlin actually ‘moves around’ and operates inside the cell; the traditional view has held that merlin protein acts at the cell membrane. Why is thisRead More...

NF2 Clinical Trials Update

Kim Hunter-Schaedle — Wed, 17 Feb 2010 19:58:40 +0100

Last July the drug bevacizumab (Avastin) was reported to show promise in shrinking NF2 vestibular schwannomas, with some improved hearing seen in patients. That Massachusetts General Hospital study, published by Dr. Scott Plotkin and colleagues in the New England Journal of Medicine, has been followed up by a publication* (links to abstract) from Dr. Victor Mautner in Germany reporting 2 cases in which treatment with bevacizumab - for 3 months in one case and 6Read More...

NF Forum: NF and Health Insurance

Garrett Gleeson — Wed, 17 Feb 2010 19:51:06 +0100

This is the second in a series of articles outlining the agenda for the 2nd Annual NF Forum in Las Vegas from April 9-11. The NF Forum is a patient and family medical symposium designed to educate individuals (of all ages) and their families about NF, management options, clinical trials, insurance concerns, social challenges and many other expressions of NF1 and NF2.

Attaining health insurance and receiving the care promised to those who have health insurance are ongoing Read More...

Humanizing a Mouse Learning 'Maze' for NF1 Clinical Trials

Kim Hunter-Schaedle — Thu, 11 Feb 2010 19:55:34 +0100

The 2005 report by Alcino Silva’s group that mice with genetically-based NF1 related learning disabilities showed improved learning and memory abilities following treatment with statin drugs quickly advanced to patient testing, and these drugs are now in Phase II NF1 clinical trials. However a challenge has been adapting the tests used to monitor mouse learning to one that can be used in humans, to see if the drug is working in patients. Now researchers at Children’s HospRead More...

CTF Brings Neurofibromatosis to the BIO CEO & Investor Conference

Kim Hunter-Schaedle — Tue, 09 Feb 2010 19:42:20 +0100

The past two days have been a whirlwind at the 12th Annual Biotechnology Industry Organization CEO and Investor Conference at the Waldorf Astoria in New York City. Traditionally a forum for biotech and pharma companies and investors to partner, the Children's Tumor Foundation was among a small number of medical research foundations invited to participate. The meeting kicked off yesterday for CTF at 9am with a platform presentation on neurofibromatosis and our research programs, whicRead More...

WHO DAT!?

John Risner — Mon, 08 Feb 2010 19:37:16 +0100

“Dat” is Superbowl Champion Jon Stinchcomb holding THE Lombardi Trophy. Jon is from Lilburn Georgia and cousin to the Leathers family. He and his brother Matt (played for the Raiders in Superbowl XXXVII) have been amazing members of the Children's Tumor Foundation family, holding events like the Countdown to Kickoff and even Read More...

Rep. John Murtha

Endurance Admin — Mon, 08 Feb 2010 19:25:25 +0100

We mourn the loss of Congressman John Murtha, who passed away on Monday, February 8th, after complications from gall bladder surgery.

A former Marine Corps officer, Rep. Murtha was the first veteran of the Vietnam war elected to Congress in 1974, and focused on military affairs during his long and successful career in Congress. As Chairman of the defense subcommittee of the House appropriations committee, he was a tireless advocate for our soldiers and their families.

AlonRead More...

Young Investigator Unravels New NF1 Drug Target

Kim Hunter-Schaedle — Thu, 04 Feb 2010 19:02:47 +0100

Current Children’s Tumor Foundation Young Investigator Awardee Dr. Sutapa Banerjee (Washington University School of Medicine) is first author of a new publication* (links to abstract) in Cancer Research that identifies a new candidate drug target for treatment of NF1 tumors. Senior author Dr. David Gutmann is Dr. Banerjee’s mentor and also heads the optic pathway glioma site of the Children’s Tumor Foundation NF Preclinical Consortium.

GlaxoSmithKline Enters Realm of Rare Diseases

Kim Hunter-Schaedle — Thu, 04 Feb 2010 18:10:21 +0100

Following a path blazed by Genzyme and now followed by a number of other biotechs and pharmas, Glaxo Smith Kline has announced they will be opening a division dedicated to finding medical treatments for rare diseases.

GSK has generously provided drug for the ongoing Children's Tumor Foundation-funded Phase Zero trial to assess lapatinib as a candidate treatment for NF2 vestibular schwannoma. The new GSK rare disease program comes in the wake of other program cRead More...

Racing4Research Pictures

Garrett Gleeson — Wed, 03 Feb 2010 16:37:39 +0100

BIO Patient Advocacy Video Highlights Children's Tumor Foundation

Kim Hunter-Schaedle — Tue, 02 Feb 2010 13:56:34 +0100

Patient advocacy groups like the Children's Tumor Foundation play many roles from funding medical research, providing support, resources and a community 'voice' for patients. At the Biotechnology Industry Organization 2009 Annual Conference, a forum for pharmaceutical and biotechnology companies, and more recently for patient advocacy groups to interact and partner with these entities, CTF's Chief Scientific Officer Dr. Kim Hunter-Schaedle featured in a short video highlighting thRead More...

BIO Podcast Previews CTF Investor Conference Presentation

Kim Hunter-Schaedle — Mon, 01 Feb 2010 20:43:01 +0100

Children's Tumor Foundation Chief Scientific Officer Dr. Kim Hunter-Schaedle has been invited by the Biotechnology Industry Organization (BIO) to present the Foundation's translational neurofibromatosis programs in preclinical drug screening and clinical trials at the 12th Annual BIO CEO and Investors Conference (www.ceo.bio.org) at the Waldorf-Astoria Hotel in New York on February 9th at 9am. BIOtech Now’s Tracy Krughoff talked to Dr. Hunter-Schaedle to Read More...