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CathyWilliams.jpgBy Cathy Williams

Cathy Williams is a cancer survivor and former patient at MD Anderson. She and a group of volunteers paint storefront windows with pink ribbons every October to raise awareness and funds for inflammatory breast cancer (IBC).

Williams began painting the ribbons on business windows in exchange for donations to breast cancer research in 2006. Despite her own diagnosis of IBC in April 2008, she has continued her efforts but now focuses solely on highlighting IBC.

I'm an inflammatory breast cancer survivor.

Easter Sunday, I celebrated four years since my diagnosis. Immediately after diagnosis, before the shock wore off, I found myself asking, "Why me?"

I have the answers
I've always been an advocate for breast cancer awareness. My mom was diagnosed with breast cancer at 32, died at 43, and two of her sisters also died of breast cancer. 

At my doctor's insistence, I got my first mammogram at 28, had one every five years until I was 38 and then every year after that.

Why did I have to get breast cancer that doesn't show up on a mammogram? Why did I have to get the most aggressive and deadly form of breast cancer? Why, with my careful breast cancer screening, was I diagnosed with late-stage breast cancer? 

No sooner than I had formulated the questions, I knew the answers.

Neurofibromatosis is worthy of study, say cancer and genetic experts, because the DNA mutations that cause its three types may yield crucial information about how cancer develops -- and new approaches to cancer treatment.

John Slopis, M.D.
, associate professor in the Department of Neuro-Oncology and medical director of the MD Anderson's Neurofibromatosis Program, explains the basics of the disease. (See related story on NF patient Reggie Bibbs.)

What is neurofibromatosis?
Neurofibromatosis is a name given to three different genetic conditions: neurofibromatosis 1, neurofibromatosis 2 and multiple schwannomatosis. These conditions are all called neurofibromatosis or NF because they all cause tumors to grow on nerves.

What is neurofibromatosis 1?
Neurofibromatosis type 1 involves tumors of the nerves from the spine to any extremity and including the skin, tumors of nerves in some organs and tumors of the optic nerves (from the eyes to the brain). NF 1 is also associated with other complications, including bone tumors, spinal scoliosis, high blood pressure, short stature (limited growth in children), brain tumors and learning disabilities.

The genetic mutation is found on chromosome 17 and is inherited from either the mother or the father. In half of the cases, the person with NF 1 will be the first born in the family, who can then pass the gene for NF 1 on to his or her children. People with NF 1 have a great variety of different symptom patterns.

What about NF 2?
Neurofibromatosis type 2 is also genetic, passed down in the same pattern as NF 1, but the genetic mutation is on a different chromosome -- chromosome 22. The disorder is quite different from NF 1.

In NF 2, tumors grow on any nerve from the spine or brainstem. The disorder is diagnosed clinically by loss of hearing or dizziness in the patient due to growth of tumors on the acoustic nerves (these are called 8th nerve schwannomas).

Tumors can grow on other structures in the spinal cord (ependymomas) or on the bone of the skull (meningiomas). Tumors of the spine can be very serious. Most people with NF 2 have very similar patterns of problems. NF 2 does not cause the other complications seen in NF 1.

And the third type of neurofibromatosis?
Called multiple schwannomatosis, it looks a lot like NF 2. But these tumors of the nerves never occur in the brain, brain stem or anywhere above the neck. We know there is one gene mutation that causes it in about a third of cases. This gene mutation is called INI-1. It was only recently discovered. The other two-thirds of cases must be caused by gene mutations, but we have not yet identified these. This condition is best known because it causes patients to experience a great deal of nerve-related pain.

What is the connection with cancer or cancer treatment? 
Most of the tumors of NF are benign tumors that grow slowly and do not become malignant. This means that these tumors are a lot like normal tissue. However, when they grow in the wrong place, the mass of the tumor causes problems. These can cause pressure on the nerves to the ear or eye, spinal cord, brain stem or brain. Sometime the tumors cause pain. Only NF 1 tumors are known to transform into serious malignant cancers called malignant peripheral nerve sheath tumors.

The connection with cancer is that many cancers result from mutations of normal DNA. If we can work out the way a mutation changes the growth of a normal tissue into a tumor or cancer, we can find clues to help cure the cancer.

What types of research are you conducting in the Neurofibromatosis Clinic at MD Anderson?
We're evaluating the use of positive emission tomography/computed tomography imaging (PT/CT), both of which provide three-dimensional images of functional processes in the body, to identify tumors in the early stages of malignant transformation.

We're working on whole-gene sequencing of the NF 1 gene to identify specific mutations that predict the development of malignancy in a given patient with NF.

And we're attempting to characterize the tumor genetics and signaling mechanisms of NF1 and NF 2. We hope to discover more targeted systemic therapies, which home in on the condition's distinct biological pathways.

Related story:
Go Ahead Ask: Neurofibromatosis Patient Invites Questions, Makes Friends
A Houston-based, internationally known activist, Reggie Bibbs has neurofibromatosis type 1, a genetic disorder that ranges in severity from almost imperceptible to major.Read more about Reggie

MD Anderson's Institute for Cancer Care Excellence recently published an analysis of anticipated health care reform legislation in the noted journal CANCER and, along the way, illustrated why its research is vital to the future of MD Anderson and the cancer care community.

The review article, which appeared online Nov. 8 in CANCER, outlined the many implications of the 2010 Patient Protection and Affordable Care Act and the Health Care and Reconciliation Act on Cancer Care Delivery. Signed into law last March by President Obama, the two health care reform acts open insurance coverage and ensure access to health care services to millions more Americans.

While the authors write that the true impact of these reforms will not be realized for several years, the cancer care community should anticipate changes in the short-term related to quality reporting and measurement, health care delivery systems and payment structures. They also outline what reforms mean for cancer-specific interests such as hospital re-admissions, prevention services, molecular diagnostics, clinical trials and professional education and call on cancer care providers to  engage in ongoing discussions about quality reporting and care delivery.   

Read the full article in CANCER.

MD Anderson's Institute for Cancer Care Excellence, created in 2009, has been at the forefront of cancer patient care, exploring outcomes, delivery and cost of multidisciplinary care but there is much work to do. Led by Thomas W. Burke, M.D., executive vice president and physician-in-chief; Thomas W. Feeley, M.D., vice president for medical operations and head of the Division of Anesthesiology and Critical Care, and Ron S. Walters, M.D., associate vice president for medical operations and informatics, the Institute for Cancer Care Excellence will play a crucial role at MD Anderson in developing the next generation of cancer care delivery systems, keeping patients the focus of care, enhancing safety and delivering care at a reasonable cost.

The WIN Consortium is a new kind of organization - a network of research and medical institutes, universities and industry partners - coming together around personalized cancer therapy. It is a response to the need to make faster progress in fighting cancer, and the realization that we can only achieve this by working together.

On July 6, the partnering organizations (visit for a full list) gathered at the Palais de Congres in Paris and formally launched the WIN Consortium. MD Anderson President John Mendelsohn, M.D., was unanimously elected to chair the WIN Directorate, the executive group in the organization. 

Drs. Thomas Tursz and Vladimir Lazar from Institut Gustave Roussy were elected vice-chair and chief operating officer, respectively (see photo), with additional officers to follow. The newly elected chairs of the Scientific Advisory Board were this year's meeting organizers: Dr. Leroy Hood, head of the Systems Biology Institute, and Dr. Richard Schilsky, past president of ASCO.

GAP.WIN2010.jpgWIN's main goal is to address scientific questions about cancer in different populations across the globe. Founders participate with equal rights and duties, with the final shared aim of significantly improving the outcome for cancer patients.

The WIN objectives fall into the following areas:

1. To validate, harmonize and standardize tools allowing early diagnosis and individualized approaches for cancer treatment.

2. To initiate and conduct new types of clinical trials, based on assigning treatments that target the genetic and molecular abnormalities identified in an individual patient's cancer.

3. To generate shared tumor specimens and databases including all available clinical, imaging and biological characteristics of patients enrolled in such trials.

4. To assist investigators in raising the necessary funding to conduct such trials, through either grant applications in various countries or direct collaborations with pharmaceutical companies.

5. To promote research and education in personalized cancer therapy, primarily through an annual meeting in Paris.

The second annual meeting was held July 7-10 and featured a long list of prominent speakers from many of the WIN member organizations, covering a large spectrum of issues surrounding personalized cancer therapy. The conference closed with a follow-up business meeting, as the WIN Consortium got down to managing the first details of forming and starting to work on the big challenges ahead.

A trail-blazing lung cancer clinical trial that matched drugs to patients based on a molecular analysis of their tumors took center stage today at the opening session of the American Association for Cancer Research 101st Annual Meeting 2010

The Phase II clinical trial known as BATTLE demonstrates the potential of using drugs that target specific genetic networks or other molecular defects to tailor personal therapy for patients. That's not done now in lung cancer, as it can be to the benefit of some breast and colon cancer patients, because there are no established molecular signatures to predict who would benefit from each drug, says clinical trial leader Edward Kim, M.D., associate professor in M. D. Anderson's Department of Thoracic/Head and Neck Medical Oncology.

BATTLE -- short for Biomarker-integrated Approaches of Targeted Therapy for Lung Cancer Elimination -- evaluated four drugs in 255 stage IV non-small cell lung cancer patients who had received between one and nine previous treatments.

"We know that new targeted therapies help a fraction of lung cancer patients, but we haven't been able to identify who those people are," Kim says. "BATTLE demonstrates the feasibility of a more personalized approach by taking a new biopsy of each patient's tumor, swiftly evaluating that tumor tissue for specific biomarkers, then assigning drugs to patients based on that analysis." 

For example, the researchers found one of the four drugs in the trial, sorafenib, didn't work at all against tumors with one common, more easily treated mutation. However, sorafenib had good effect controlling tumors that featured a KRAS mutation, which has no proven treatment.

By using an innovative statistical approach to identify patients in Phase II clinical trials who are most likely to benefit from a targeted drug, BATTLE points the way toward focused Phase III trials that will require fewer patients, take less time and will be more likely to succeed.

Lung cancer research, Kim says, is littered with large Phase III trials that enrolled hundreds or thousands of patients, then showed no or only minor effects. Many failed to enroll enough patients to finish.

The BATTLE abstract at AACR can be viewed here.

Read the news release from M. D. Anderson - BATTLE Links Potential Biomarkers to Drugs for Lung Cancer

By Wendy Gottsegen, Director, External Communications

We also don't recommend that you don't drink the water, either. Confused? Well there seems to be buzz on the Internet about a type of water that a Dallas-based company is marketing and this water's relationship to M. D. Anderson.   

So we thought we'd un-muddy the water for our patients, employees and supporters. M. D. Anderson doesn't recommend the water. 

Dont drink the waterWhat we did do was test the water (this time the pun is not intended) for the company, who compensated us to do so. The tests were very specific, not comprehensive and the results were turned over to the company without interpretation by M. D. Anderson experts.

Because of the advanced equipment at our institution, we get requests from industry from time to time to use the equipment for commercial endeavors. We evaluate these requests, and if it's not disruptive to patient care and research, we may accommodate for a fee. This is what happened in the case of the water from Dallas.

M. D. Anderson is focused on patient care, research, education and prevention of cancer. You'd be hard-pressed to get us to stray from that mission, so when you see us appear to recommend something not necessarily related to cancer, please ask questions. In fact, please call us at 877-MDA-6789 and let us know.

Our lawyers have crafted some nice language fully explaining this situation. Please share it with anybody who might ask you about M. D. Anderson and some new-fangled "nutraceutical" water from Dallas.

M. D. Anderson statement:
Recently, you may have heard or read about a company that sells Evolv, a "nutraceutical beverage," which is being promoted in part based upon testing done at The University of Texas M. D. Anderson Cancer Center, but also is being mistakenly viewed as endorsed by M. D. Anderson. M. D. Anderson conducted limited chemical analysis of the product to evaluate its anti-inflammatory activity for a fee at the request of the manufacturer. No efficacy or toxicity data were generated at M. D. Anderson nor was the product tested on humans. Moreover, M. D. Anderson does not have any involvement with the company, the product is not produced by M. D. Anderson, and M. D. Anderson does not endorse the product or recommend its use.


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