Patients affected by rare and orphan diseases, and their advocates fighting for more research and increased funding to bring effective treatments to the marketplace, just got a big boost from a $ 10 million anonymous gift to the University of Pennsylvania.   Penn has put the funds to good use by establishing the Penn Center for Orphan Disease Research and Therapy focused on discovering novel treatments for rare diseases.   There are close to 7,000 rare diseases, including mesothelioma, AIDS and meningitis, of which less than 200 of them currently have any effective treatments.

J. Larry Jameson, M.D., Ph.D., Executive Vice President for the Health System and Dean of the Perelman School of Medicine said, “The Center will be a natural extension of Penn’s expertise in orphan diseases, and this strategic investment will galvanize support for orphan disease research around the globe.”

Several of Penn’s staff have been leading the way in mesothelioma research, and they have been rewarded for their groundbreaking efforts.  Steven M. Albelda, M.D., vice chief, Pulmonary, Allergy, and Critical Care Division, director of Lung Research and director, Thoracic Oncology Laboratories, received the Wagner Medal for Excellence in Mesothelioma Research award in 2010.  In addition, Sunil Singhal, M.D., Thoracic Surgeon, Assistant Professor, who treats pleural diseases, including mesothelioma, was one of the recipients of the National Lung Cancer Partnership’s Young Investigator Research Grants.

Orphan diseases in the United States are those that affect fewer than 200,000 people.  Currently, an estimated 25 million people in the United States are living with an orphan disease.  Patients with rare diseases often have to travel across the country for treatment at a facility with a team of doctors and surgeons that specialize in their disease.  Specialists are aware of the latest research and they are able to develop a more informed treatment plan than would a physician who does not specialize in the disease.

In a press release announcing the Center, Penn Medicine explains the Center’s function as:

“Working together with other academic institutions, the National Institutes of Health, Pharma, and private philanthropy, the Center aims to reduce dramatically the technological and financial burden of investigators working in isolation by supporting a range of collaborative approaches:

• Robotics for large-scale drug screening,
• Biospecimen repositories to store samples,
• Genotyping and bioinformatics services to identify causative genes,
• Cell-based systems for developing new tests,
• Nano-scale systems for developing new ways to deliver drugs, and
• Small and large animal models for testing potential treatments.”

Although Penn did not specifically mention the International Rare Disease Research Consortium, focused research on rare diseases through this Center, could aid the consortium in meeting their goals of providing diagnostic tools for each of the rare diseases, and to develop 200 new treatments by 2020. 

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We have all heard the word “tumor” and simply think of a mass of tissue. But what is a tumor, really? The definition of tumor implies abnormal cell growth; this results in an abnormal lump or mass of tissue. Tumors (also referred to as a neoplasm) are not always cancerous (malignant); they can also be non-cancerous (benign).

Benign tumors usually grow more slowly than malignant tumors; however their growth can interfere with the ability of healthy tissues to thrive. At times, they may grow large enough to apply pressure to vital body organs resulting in serious health problems. Surgical removal is recommended at times for benign tumors.

Malignant tumors grow at a faster rate, in general, than benign tumors. The concern with malignant tumors is that they may spread to other body tissues and cause damage to them.

Several factors to determine the prognosis for treatment of a tumor include its size, location and type. The patient’s health is also a consideration. At times, there are no symptoms and as a result the tumor is left alone. If a tumor is suspected to be malignant, however, a doctor removes a sample of tissue (called a biopsy) to be sent to a pathologist who studies the tissue under a microscope to confirm if it is malignant or benign.

Tumors are categorized into a system called a tumor grade, which can be determined by the pathologist. The tumor grade is a way to classify cancer cells to see just how abnormal the cells look under a microscope and how quickly they are likely to grow and spread. The specific factors to determine the tumor grade varies by the type of cancer. The tumor grade is combined with the cancer’s stage, as well as the patient’s overall health, to determine treatment by the medical professionals.

The tumor grades signify the various degrees of severity, and are described as Grades 1, 2, 3 or 4. Grade 1 tumors are the least aggressive in behavior. There are a variety of grading systems which differ for each type of cancer. Examples include the Gleason system for prostate cancer, the Bloom-Richardson system for breast cancer and the Fuhrman system for kidney cancer.

There are a large variety of tumor types and your particular type should be investigated to gain further information. Tumors are listed in the information section of KnowCancer.com. At the bottom of the listing you will see the tumor types with descriptions of their signs and symptoms, risk factors, treatment and additional information that may be helpful.

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Mesothelioma is an aggressive, incurable cancer caused by asbestos exposure that often leaves patients with few treatment options and a prognosis of less than one year after diagnosis.  Asbestos was widely used in building materials in homes, schools, factories and other industrial settings for much of the 20th century. Its use was largely restricted in the late 1970s because of its toxicity. The heavy use of asbestos left workers and their families who breathed asbestos dust decades ago vulnerable to contracting the asbestos cancer, which typically takes 30 years or more to present symptoms.

Asbestos is proven to cause not only mesothelioma, but lung cancer, asbestosis and other respiratory diseases, with close to 3,000 new cases of mesothelioma being diagnosed each year in the United States.  With the known health hazards the International Agency for Research on Cancer (IARC), the National Toxicology Program (NTP), and the US Environmental Protection Agency (EPA) all recognize asbestos as a human carcinogen – meaning asbestos causes cancer or helps cancer grow.

The 12th Report on Carcinogens, a science-based, public health document mandated by Congress, also recognizes the toxicity of asbestos in all forms.  The report, released in June by the U.S. Department of Health and Human Services, identifies substances, chemicals, metals, pesticides, drugs and compounds that are known or reasonably expected to cause cancer in humans. The report lists 54 substances as known carcinogens and 186 substances that are suspected of causing cancer in humans.

The report states that cancer studies in humans “have shown that exposure to asbestos causes respiratory-tract cancer, mesothelioma of the lung and abdominal cavity (pleural and peritoneal mesothelioma), and cancer at other tissue sites.”

Substances such as asbestos are listed as known carcinogens when there is a convincing body of evidence from peer-reviewed scientific studies involving humans to show a cause-and-effect relationship between exposure and development of cancer. Asbestos has been listed as a known carcinogen since publication of the 1st Report on Carcinogens in 1980. Scientific studies have found that occupational exposure to all forms of asbestos increases the risk of cancer, according to the U.S. Department of Health and Human Services.

Glass wool is among the eight newly listed suspected human carcinogens in the 12th Report on Carcinogens. A synthetic fiber, glass wool is used in building insulation and in special use applications as high-efficiency air filters and in aircraft and spacecraft insulation. Scientific studies involving animals show that glass wool causes cancerous tumors in animals’ lungs. The ability of glass wool fibers to cause cancer in animals varied depending on the types of fibers.

Much like asbestos fibers, glass wool fibers that are biopersistent and remain in the lungs are most associated with causing cancer. Asbestos was used to make insulation and insulation wrap. Older homes and buildings may contain vermiculite insulation containing asbestos.

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It was a day not unlike other days my wife and I have spent at our home in North Georgia. It was a bit warm, sunny and otherwise reasonably comfortable. The day itself was a bit special, because we were there to take a quiet weekend interlude to celebrate our birthdays. We were glad to have some much needed time together to get away from all the tumult of our everyday lives and basically just relax, to do some of the things that we wanted to do.

We decided to take one of our favorite walks, down a gravel county road, much of it by a river that is near our home. In order to extend the walk to make it five miles when we have the time–like we did today–we took an extra “loop” which goes to a small wooden church tucked deep in the woods.

The sign outside the church says it was founded in the latter part of the 19th century, but some of the gravestones are dated even before then. They still have services there every Sunday.

When we got to the church, we were drawn to a freshly dug grave in the cemetery.  On the top of the dirt was a beautiful bouquet of several dozen yellow roses. Like the turned dirt, these flowers were fresh and just opening, an indication that whoever rested there was only recently buried.

My wife and I started to wonder out loud: Who was this person? What happened to them? Apparently a woman, who loved her so much to have placed those beautiful flowers on her final resting place?

Many questions, and no answers. No name plate, no date of birth or death. Just the flowers on the grave.

For reasons that I will never understand, my wife and I had a few tears in our eyes. We had a prayer together that she didn’t suffer in her death, that she had others to pray for her, that although she was missed, she hopefully will be remembered.

And then it struck me that so much of what we do as part of our every day lives is to care for those we rarely meet, many whom we never know.

I thought at that moment of the many colleagues, friends, and volunteers I work with every day. I thought of their commitment to our cause and our effort to reduce the burden and suffering from cancer for others. I thought about how we try to improve the quality of life of so many in their time of need. I thought about how many we touch every day who we will never know personally, and serve them in ways we can never measure. I thought about how much we hope we can bring with just a little bit of good into an otherwise difficult day.

Many of us run through our days without thinking too much about who we are and why we exist. In that moment, in that cemetery by that wooden church in the middle of nowhere, the answer to that question became just a bit clearer for my wife and me.

If you can find a place in your heart for someone you never met and never knew, then perhaps our humanity for each other hasn’t been dissolved by all that is going on around us. If we can be touched by a stranger, then perhaps we are still able to touch those who look to us for help and guidance in their times of need. I know thousands of people-along with millions of volunteers–who do that every day.

Sandra and I spent our minutes by the church, then continued our walk. We didn’t have much to say to each other for a while, but sensed that the moment in the cemetery told us a lot about who we are and who we aspire to be.

The comforting thought is that we are very grateful we are not alone.

By Nancy Meredith
Michele Carbone, MD, PhD, one of the world’s premiere authorities on mesothelioma and director of the University of Hawaii Cancer Center, has once again concluded a groundbreaking study that could have far-reaching implications of the environmental risks on the health of some Americans.  Carbone and his research team have concluded that exposure to high levels of the mineral erionite, used in road gravel in several US states, can lead to the development of mesothelioma.  He added that “there is reason for concern for increased risk [of mesothelioma] in North Dakota in the future.”

Airborne Erionite Becomes Lodged in Lungs – Similar to Asbestos
According to a study published in the Proceedings of the National Academy of Science, North Dakota and 11 western states have erionite-containing rock deposits.  The researchers focused on Dunn, ND where rocks containing erionite have been used to produce gravel for the past 30 years.  They found that the hazards of exposure to the mineral have increased recently with expanded oil exploration and increased traffic on the over 300 miles of road covered with erionite-based gravel.  The urbanization of the area has led to erionite dust becoming airborne where it is then inhaled and lodges in people’s lungs.

Up until this study, the only known cause of mesothelioma was through exposure to asbestos.  Asbestos is a known carcinogen and has been linked to mesothelioma and lung cancer, as well as to asbestosis and other respiratory diseases.   Mesothelioma has a 30 to 60 year latency period, and most often diagnoses are not made until symptoms appear and the disease has progressed to an advanced stage leaving the patient with life-threatening complications.

Turkish Villages Used for Comparison
Carbone began his study years ago in Cappadocia, a region of Central Anatolia in Turkey, where an epidemic of malignant mesothelioma had been reported.  His team found then that mesothelioma was associated to exposure to erionite, that has some of the same physical properties as asbestos.  However, the researchers found that erionite is significantly more potent than asbestos.  They found that “erionite was 500–800 times more tumorigenic than chrysotile asbestos and 200 times more tumorigenic than crocidolite asbestos.”

To determine the potential health implications to Americans, Carbone and the researchers compared erionite from the Turkish villages to that from Dunn, North Dakota.  The erionite concentrations found in the air measured in North Dakota “along roadsides, indoors, and inside vehicles, including school buses, equaled or exceeded concentrations in Boyali, [Turkey] where 6.25% of all deaths are caused by malignant mesothelioma.”

With their findings, the researchers concluded that erionite exposure for North Dakota school bus drivers and children, road workers, and others regularly using the erionite roads, may exceed the often used benchmark for risk management actions of an estimated risk probability of one additional cancer per 10,000 people. 

If the latency period for erionite plays out the same as for asbestos exposure, the number of cases of malignant mesothelioma could soon be on the rise.  In addition, adults as young as their 30’s could contract mesothelioma from exposure in their childhood on the school buses and the local playgrounds.

The researchers recommend “novel preventive and early detection programs in ND and other erionite-rich areas of the United States.”  Hopefully, some of the medical research that is still in the lab will prove useful for early detection of mesothelioma in the upcoming years to offer these Americans an effective treatment and cure for the disease if contracted from erionite exposure.

The study was supported in part by the National Cancer Institute, the American Association for Cancer Research Landon Innovator Award, and by grants from the Butitta Mesothelioma Foundation, and the Mesothelioma Applied Research Foundation.

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Malignant mesothelioma clinical trials continue to be one of the best options for patients struggling to find a new, effective treatment.  Offered through hospitals and medical research institutions, the trials test new methods of screening, prevention, diagnosis, or treatment of the disease.  They also provide researchers with critical information needed to allow them to move their findings from the lab to the patients.

Mesothelioma, an asbestos-related cancer of the lining of the lungs, abdomen or heart, is a disease being fought internationally, and clinical trials are offered throughout the world.  According to clinicaltrials.gov, there are currently 30 open studies for malignant mesothelioma being conducted worldwide.  Trial sites include the United States, Canada, Belgium, France, Germany and Italy.

Mesothelioma is highly aggressive and is resistant to many cancer treatments.  The prognosis for mesothelioma patients is usually grim: the average survival time varies from 4 – 18 months after diagnosis.  For the close to 3,000 Americans diagnosed with the disease each year, clinical trials may offer them a breakthrough treatment not yet available through their physician.

In a report published by the Intercultural Cancer Council less than 5% of the over 10 million adults with cancer in the United States participate in clinical trials.  They found that two of the primary reasons include lack of awareness of studies and mistrust of the research.  Education of the clinical trial process is one way to allay their fears and expand participation in the trial process.

US clinical trials are closely monitored and are run according to strict guidelines.  The National Institutes of Health (NIH) explains that federally funded clinical research has safeguards in place to protect the participants, including explaining to the participants:

• What will happen during a study including eligibility criteria, evaluations and all procedures to be performed;
• The possible risks, benefits, alternatives, and responsibilities of the clinical trial are explained thoroughly to patients before they agree to participate;
• That a clinical study is reviewed by an independent review board prior to the start to ensure that potential risks are clearly stated, and that investigators take sufficient and appropriate measures to address risks before the study proceeds;
• The study will be closely monitored once it is underway by an independent committee for safety and conformation to the study documents;
• That their privacy will be protected; and
• The study will be ethical and will follow international and federal “good clinical practices” guidelines.

If someone is suffering from mesothelioma, they should check with their physician to determine if any current studies exist for their particular case.  The NIH adds that for “study participants with complex illnesses, ‘protected’ does not mean ‘risk-free’—all studies carry some risk.”  When determining if you should participate in a trial it is important to work with your medical team to determine if the benefits outweigh any risks.

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New York City mesothelioma lawyer Joseph W. Belluck, who is experienced in asbestos exposure issues, said he was disturbed to learn that a Brooklyn hospital has been cited for negligent failure to protect workers from exposure to the well-known carcinogen during demolition and renovation work.  He added that OSHA fines against Interfaith Medical Center reflect the seriousness of neglecting workers’ welfare and well-known rules and regulations about asbestos in the workplace.

The U.S. Department of Labor’s Occupational Safety and Health Administration has proposed fines of up to $ 48,000 against Interfaith Medical Center of Brooklyn for 14 violations of workplace health and safety standards during work to remove asbestos at the facility.

Among other infractions, the hospital failed to train staff, warn contractors of the presence of asbestos, properly label asbestos-containing materials and dispose of asbestos properly, according to an OSHA news release, which detailed the citations and the proposed penalties against Interfaith Medical Center.

“The connection of asbestos and asbestos removal to cancer, and the safety measures required are longstanding and well known,” said Belluck of the New York City personal injury law firm, Belluck & Fox, LLP. “It’s shocking that Interfaith Medical Center would ignore them.”

Workplace asbestos exposure can result in asbestosis, mesothelioma and lung cancer.

“It is particularly distressing to realize that workers at Interfaith possibly may have been exposed to asbestos for years as their employers ignored this potentially deadly risk,” Belluck said. “OSHA makes clear information available and will provide training about the hazards of asbestos exposure and the appropriate safety measures for dealing with it. There’s no excuse for any facility to simply disregard long-known hazards and long-defined rules and regulations.”

Belluck is a nationally recognized attorney whose practice focuses on asbestos-exposure/mesothelioma litigation, as well as consumer, environmental and product injury cases. Belluck has won several major cases involving injuries from asbestos.

“People who have been diagnosed with mesothelioma and lung cancer should contact an attorney to help them protect their legal rights,” the New York lawyer said.

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As long as there is the possibility for newer and better drugs to fight cancer, there remains hope. A few of the recent breakthroughs in cancer drugs are listed below:

Breast cancer that has metastasized in patients who have already been treated with chemotherapy at least twice can be treated with a new drug called eribulin mesylate. The FDA approved use of this drug on November 15, 2010. Trademarked under the name Halaven, it is manufactured by a company called Eisai, Ltd., and has been shown to improve overall survival in patients by 2.5 months on average.

Eribulin is a laboratory-made form of halichondrin B, which is a substance derived from a sea sponge. It is a targeted therapy drug, meaning it is made to affect a specific type of cell; in this case it targets the protein called tubulin in cells to interfere with cancer cell division and growth.

Cancer patients with bone metastases from solid tumors are approved to use a drug called denosumab, trademarked under the names Xgeva and Prolia, and is manufactured by Amgen, Inc. Denosumab helps to prevent skeletal-related events and was approved by the FDA on November 18, 2010. These skeletal-related events include pathological fractures, radiation therapy to bone, surgery to bone or spinal cord compression due to cancer.

Advanced prostate cancer has been shown to have an improved survival rate using the drug cabazitaxel over those receiving standard chemotherapy. The FDA approved this drug on June 17, 2010 and can be considered as an alternative for men where standard chemotherapy has failed. It is registered under the name Jevtana, an injection made by the company Sanofi-Aventis. It is effective in patients previously treated with a drug called docataxel, and can provide alternative options for survival.

Another type of cancer being fought using new drugs is cutaneous T-cell lymphoma, which is any of a group of T-cell non-Hodgkin lymphomas; the most common types are mycosis fungoides and Sezary syndrome. The drug called romidepsin registered under the name Istodax (made by Gloucester Pharmaceuticals Inc.) has been approved by the FDA on November 5, 2009 to treat cutaneous T-cell lymphoma in patients who have received at least one prior systemic therapy.

A common theme evident in these examples of recent breakthroughs in cancer drugs: no matter what stage of cancer or what previous treatments existed, there remains the hope that a new drug may perhaps be an alternative solution for increased survivability.

Phil Mickelson, the famous left-handed golf champion, has a number of impressive titles: winner of four major golf championships and the winner of 39 PGA events. But he also has another more impressive title: a cancer supporter.

Rewind the clock a couple years when Phil’s wife, Amy, was diagnosed with the shocking news of her breast cancer in May, 2009. More shocking: only six weeks later, Phil’s mom, Mary, was also diagnosed with breast cancer. While prayers and support were being sent out in abundance for Amy and Mary, it became evident that Phil’s world was rocked, too. To support his wife, Phil had serious decisions to make. How should he use his time? What would be best for the children? What type of support did Amy need? His decision to then withdraw from two major golf events shortly after his wife’s diagnosis displayed Phil’s character, wisdom and fortitude.

Phil also took a brief leave of absence from the PGA Tour after hearing of his mother’s diagnosis. What an example of solid priorities. Phil’s absence resulted in a “Pink Out” event created and hosted at the Crowne Plaza International shortly after the PGA Tour in 2009. His fellow golfers all wore pink in support of the Mickelson family. Phil, like the rest of us, sometimes needed to depend on the strength of the people around him so he could stay focused where it was needed most. A true champion.

Mickelson won the 2010 Masters Tournament on April 11, 2010. In a poignant finish, Amy and the children joined Phil to celebrate the win. The embrace was recorded for the entire world to see. It’s moments like these that make golf history.

Phil and Amy show themselves to be true champions in spirit as they continue to thrive through the health challenges of cancer. Earlier this year, Amy was back to walking the course to follow Phil’s game after being forced to stay away during her recovery last year. In the meantime, Phil continues to play an amazing golf game, which makes him different from most of us. It’s hard to list, let alone remember, all of the prizes he has won in his career. But his biggest prize: maintaining his role as a supportive family man when faced with cancer in his world.

It is a role that we can each aspire to imitate!

In a session at the Biotechnology Industry Organization (BIO) meeting in Washington, DC this month, the International Rare Disease Research Consortium (IRDiRC) leaders agreed that encouraging more countries and pharmaceutical companies to join the consortium is critical for meeting their lofty goals laid out earlier this year. Their intention is to provide diagnostic tools for each of the close to 7,000 rare diseases, such as mesothelioma, AIDS and celiac disease, and to develop 200 new treatments by 2020. 

A rare or “orphan” disease status is assigned to a disease or disorder if it affects fewer than 200,000 Americans at any given time.  Although the number of people with mesothelioma and some other diseases may be low, the loss of life and quality of life places an undue burden on the patients and their families. Between 85 and 90 percent of orphan diseases are serious or life-threatening, yet only about 200 of them currently have any effective treatments.

The initial funding for the consortium is managed through the founding agencies, U.S. National Institutes of Health (NIH) and the European Commission.  As additional countries commit to the effort, they will each pay $ 10 million.   “We hope that when it starts rolling other countries come on board to create diagnostics for rare diseases and speed up the development and approval of treatments for rare diseases,” Indridi Benediktsson of the European Commission’s Health Directorate, told delegates at BIO.

Stephen Groft, Director of the Office of Rare Diseases at NIH, added that rare diseases are a “global issue and the needs are the same regardless of country.”  He said to meet the goals it calls for a “tremendous, coordinated effort.”

The consortium leaders acknowledge that there will be many barriers to overcome, such as intellectual property rights, but they all agree that rare diseases need to be an international priority.  It can take 8-12 years to develop a new drug, and they note that there needs to be methods and agreement for testing and re-purposing existing drug compounds to meet their goals.

Industry watchdogs have their eyes on the consortium’s meeting scheduled for October, in Montreal, Canada, where the consortium will finalize their research agenda. The agenda will be used to build interest within the pharmaceutical industry and attract investigators, clinicians and countries into the rare disease effort.

BIO Session on Rare Disease Consortium

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