Rare Disease Day 2014: Join Together for Better Care

Rare Disease Day 2014 There are more than 6,800 rare diseases that affect an estimated 25-30 million Americans. Although these diseases don’t occur often, they can have serious repercussions for the people living with them. Some rare diseases are hereditary, and others are due to environmental factors.

Mesothelioma: A Rare and Incurable Cancer

Mesothelioma is one of the rare diseases that are related to environmental factors. The only known cause of this deadly disease is exposure to asbestos. There are 3,200 people diagnosed with this aggressive and incurable cancer each year in the United States. Many of those diagnosed are not even aware that they were exposed to asbestos at their jobs or in their day-to-day lives. Symptoms of mesothelioma can take decades to appear. So in many cases, by the time they are diagnosed, victims may not have long to live.

Rare Disease Day, February 28, 2014

Today is Rare Disease Day, a worldwide event that was started in 2008 by EURODIS, a non-governmental alliance of patient organizations and individuals who are active in the rare disease field. Over the last six years, this special day has inspired more than 1,000 events — all with the goal of raising the public and policy makers’ awareness of rare diseases.

According to EURODIS, the day has “notably contributed to the advancement of national plans and policies for rare diseases in a number of countries.” This year’s theme, “Join Together for Better Care,” focuses on bringing everyone with, working in the field of, or creating policies for rare diseases out of isolation to work together in hope and solidarity.


How to Participate in Rare Disease Day

More than 80 countries are expected to participate in Rare Disease Day today. If you or a loved one is suffering from mesothelioma or another rare disease, here are 6 ways to participate in Rare Disease Day 2014:

  • Become a friend
  • Raise and join your hands
  • Tell your story
  • Download communications material
  • Join the conversation on social media
  • Organize an awareness-raising activity


  • http://www.rarediseases.org/rare-disease-information/rare-diseases
  • http://rarediseases.info.nih.gov/files/Rare_Diseases_FAQs.pdf
  • http://rarediseaseday.org

Should Lung Cancer Screening Guide Include Asbestos Exposure?

The U.S. Preventive Services Task Force recently recommended that heavy smokers receive annual lung cancer screenings using low-dose CT scans instead of X-rays.

The recommendation is limited to longtime, pack-a-day smokers ages 55 to 79. However, some patient advocacy groups feel the guidance should be expanded to include those who may smoke less but have other risk factors such as exposure to asbestos, a known lung carcinogen, according to a recent posting on Boston.com.

Because of the strength and heat resistance of its fibers, asbestos was used in a wide range of commercial and residential building products, as well as in automobile parts, fabrics, gaskets, and many types of fire-resistant coatings. We now know that exposure to asbestos can result in deadly diseases such as pleural mesothelioma, a rare cancer that occurs in the membranes lining the lungs.

It can take decades for symptoms related to pleural mesothelioma to appear, which makes it critical that those exposed to asbestos-containing materials be tested. Most asbestos exposure occurs on the job but some occupations are more at risk than others. Those who worked in the construction and automobile industries, in shipyards, or spent time on U.S. Navy ships are at higher risk of exposure and should be tested regularly by a medical professional familiar with asbestos-related illnesses.

While testing for cancer comes with its own risk of excess exposure to radiation from CT scans—the preferred testing method—the task force determined that the benefits outweigh the risks for certain patients. A CT or computed tomography scan is better at identifying tumors, according to Boston.com The new recommendation could help doctors spot cancer earlier on, which is key to the treatment of mesothelioma.

If you or a loved one has been exposed to asbestos and received a diagnosis of mesothelioma, you may want to learn about potential legal action. Call Sokolove Law today and a paralegal trained in asbestos litigation can guide you through your options.

Asbestos Lawsuits Require an Autopsy?

A court in Seattle is forcing a man dying of mesothelioma to have an autopsy after his death before his family can sue for asbestos exposure. Seventy-one-year-old James Ross refuses the autopsy required by the King County court on moral grounds. He realizes that this will increase the difficulty in proving that his cancer was caused by asbestos exposure from the defendants, Kaiser Gypsum and T.H. Agriculture and Nutrition. Both of the defendants hold that the suit should be dropped if Ross refuses the autopsy. Under Washington State law, religious grounds permit people to forego an autopsy, but Ross simply claims to object to the marring of his body after death. Due to that, the defendants believe that the lawsuit should be thrown out. Additionally, since Ross’ life has not yet been taken by mesothelioma, the defendants have refused to have the suit move to a trial.

As a result, James Ross and the defendants are now on a death watch until his case can move forward. During his career as a railroad brakeman and conductor with the Burlington Northern Railroad, James Ross was regularly exposed to asbestos. He also came in contact with the deadly substance while he was remodeling a home in the 1960s. Exposure to asbestos is the only known cause of mesothelioma, a deadly cancer of the lungs that usually kills within months of diagnosis. The plaintiff argued on October 31, 2008, that a protective order to prohibit an unauthorized autopsy should be filed. According to court documents, the court cannot force a person to have an autopsy even if his refusal is not on religious grounds. The decision to protect James Ross from an unauthorized autopsy was pending at the time of this writing.

Mesothelioma Drug ONCONASE Status to be Discussed by the FDA

ONCONASE, a drug to treat mesothelioma and other cancers, is scheduled at the end of January 2009 to be the subject of a pre-NDA (New Drug Application) meeting of the Food and Drug Administration (FDA). This is a critical step to be taken before the drug’s manufacturer, Alfacell, can complete its New Drug Application. The intention of the pre-NDA meeting will be for the FDA to give ideas to Alfacell to finish its application. Whether or not the drug will be approved cannot be contested until after the finalized NDA is submitted. During the Phase IIIb trials for ONCONASE, only those patients whose mesothelioma was not successfully treated by other chemotherapies saw a statistical improvement in their condition with ONCONASE.

The lack of an impact by Alfacell’s drug on patients who were treated by other chemotherapy drugs might lead the FDA to not approve ONCONASE. Alfacell is looking to ask the FDA to allow for it to finish its rolling NDA within 48 hours of the pre-NDA meeting. In doing so, the company could get pointers from the FDA to better tailor its application, which will improve the chances of the drug being approved. Shortly after the completion of the NDA, the FDA will view the results of the clinical trials of the drug. If ONCONASE is approved, it will open the way in the near future for offering this drug to unresectable malignant mesothelioma (UMM) patients who have failed to be treated with other chemotherapies. ONCONASE has already been given fast track status to lessen the amount of time required in the FDA approval process. It also is classified as an orphan drug in the United States and Europe. This status is given to drugs that treat patients with rare diseases that affect fewer than 200,000 people.

Mesothelioma Drug Study Shows Promise While Another Drug Company Finds Its Future Foggy

A drug developed by the Proteolix, Inc., pharmaceutical company, carfilzomib, has shown promising results in its first round of clinical trials. Carfilzomib acts as a proteasome inhibitor, which targets specific aspects of the cancer without doing excessive harm to noncancerous cells. Fourteen patients in the first set of trial with solid tumors whose condition had previously failed to be improved by at least two other drugs, including at least one mesothelioma patient, were treated safely and some showed a stabilization of their cancer. Carfilzomib appears to be a safe and well-tolerated drug following its first round of testing. The most prevalent side effects of the drugs included fatigue, nausea, headache, diarrhea, and constipation.

For most patients, the side effects were either temporary, or they were successfully reversed. The most disturbing side effect of carfilzomib — peripheral neuropathy, or slight tingling and numbness in the extremities — was not severe enough in any of the patients to warrant a change or stoppage of their dosage of carfilzomib. Following the first set of clinical trials, carfilzomib began Phase II trials in May 2008, targeting solid tumors in non-small cell lung, ovarian, small cell lung, and renal cell cancers. It is also being studied in a second phase as a combination therapy with other chemotherapies. More information about these continuing clinical trials with carfilzomib can be seen at http://www.clinicaltrials.gov. Alfacell, the maker of ONCONASE, a successfully tested mesothelioma drug, included an audit option in its Annual Report filed on October 14, 2008. As a Nasdaq listed company, Alfacell was required to announce its audit option per Nasdaq Marketplace Rule 4350 (b)(1)(B). Any company receiving an audit option must announce to the public that an auditor has found sufficient evidence that there is doubt about the company being able to continue operations. The audit option announcement does not change its Annual Report for the fiscal year that ended on July 31st, 2008.

Frogs Used to Develop Mesothelioma Drug

In order to gain approval for any new drug, it must undergo years of testing and clinical trials. The newest drug to treat mesothelioma , Onconase (ranpirnase), proved to be no different in this requirement. Onconase is a drug created by the pharmaceutical company Alfacell. It has been derived from the egg cells of Northern Leopard Frogs (Rana pipiens), and in the experiments done using the drugs on tumors, it seems to have an effect on halting their growth. The last stage of clinical trials before approval, Phase III, showed that this drug proved to be effective in patients with malignant mesothelioma whose cancer failed to be slowed by chemotherapy. Scientists wanted to know why Onconase seemed to work better than other treatments in these patients.

Many possibilities for Onconase’s effectiveness were tested in a study whose results were recently published in Cell Cycle. It seemed that the drug had an anti-tumor property and worked by targeting the small interfering RNA (siRNA). RNA forms play an integral role in gene and cell replication, which runs out of control in tumors. By slowing or stopping the cell replication of tumors, cancers can be halted in their progression. In the study, by honing in on siRNA, Onconase was able to prevent the quelling of a common gene –glyceraldehyde 3-phosphate dehydrogenase (GAPDH). This led the scientists to conclude that Onconase would indeed by effective in controlling the siRNA agent during tumor cell replication and growth. This study further enforces the studies behind the clinical trials which led to Onconase’s approval.

Mesothelioma Patients Sought for Ongoing Clinical Trial

A new device for treating mesothelioma is being studied by the Swiss Group for Clinical Cancer Research. To be conducted, the trials will need patients suffering from mesothelioma to volunteer. The device — the Electronic Tool for Monitoring Symptoms and Syndromes Associated With Advanced Cancer (nicknamed E-MOSAIC) — is being tested to see whether or not its use in monitoring mesothelioma symptoms helps to control them. The goal of the study is to determine if the E-MOSAIC is an effective means of communication of symptoms between doctor and patient. If it is, better courses of treatment can be sought. Most drug trials examine products to treat or cure a disease, but the E-MOSAIC is intended to be tested as a palliative treatment.

Palliative treatments are those that seek to improve quality of life in patients undergoing other treatments for their disease. The E-MOSAIC is being considered as a means of reducing pain and suffering. Ideal candidates for the study will be those 18 years old or older, suffering from an advanced cancer that results in pain, appetite loss, weight loss, fatigue, depression, and/or anxiety. Patients must also be taking palliative anticancer treatment continuously every week or every other week as an outpatient. Health professionals conducting the E-MOSAIC trial will rule on final eligibility. Those interested should contact Dr. Florian Strasser, MD, at the Kantonsspital in St. Gallen, Switzerland. The study will consist of two randomly assigned groups. Both will use an electronic hand-held device, but one group will use it to monitor their symptoms and diet. The second group will record diet and specific symptoms such as pain, shortness of breath, loss of appetite, fatigue, nausea, depression, and overall well-being. Weekly evaluations by nurses will also be made of both groups. An increase in quality of life for mesothelioma patients and those suffering from other forms of cancer is the goal of the study. The trials are expected to be finished by July of 2010.

Eli Lilly Mesothelioma Drug Approved by the FDA

Alimta, a new first-line drug to treat non-squamous mesothelioma , has been approved by the United States Food and Drug Administration. Its approval is based upon its combination with cisplatin to aid those with locally advanced, metastatic non-small cell lung cancer (NSCLC). Those with squamous non-small cell forms of lung cancer do not have FDA approval to take the drug, but the majority of new lung cancer cases are NSCLC. Use of Alimta as a first line treatment of non-squamous, advanced NSCLC is the third use for the drug to receive U.S. FDA approval. It has been used since 2004 in conjunction with cisplatin for patients with unresectable malignant pleural mesothelioma . In 2004, Alimta was also approved in the US for use as a single-agent for second-line advanced or metastatic NSCLC patients. This has been changed by the FDA to be only for those with non-squamous forms of lung cancer.

A phase III trial resulted in the FDA’s approval for the new use of Alimta. The trial compared the use of combining Alimta with cisplatin against a combination of Gemzar with cisplatin (GC). Results of the study showed an increase in survival rates by one month in those with non-squamous NSCLC cancer against the Gemzar combination. In the group with squamous NSCLC, the survival rates were better by one month with the Gemzar and cisplatin. Patients using the Alimta-cisplatin combination showed fewer toxins in their blood, required fewer blood transfusions, and lowered the use of growth factors when compared to Cisplatin-cisplatin. Common side effects in those taking the Alimta-cisplatin regime included: vomiting, neutropenia, leukopenia, anemia, stomatitis/pharyngitis, thrombocytopenia, and constipation. This new approval adds to the ever-growing list of available treatments for many malignant and often deadly forms of cancer including pleural mesothelioma.

Insurance Lawyer Claims: Pleural Plaque is a “Good Thing”

Dr. Pamela Abernethy, an insurance lawyer with the Forum of Insurance Lawyers, claimed that pleural plaques were a “good thing”, and that they showed the efficiency of the body’s natural defense system to prevent foreign particles in the lungs from doing damage to the rest of the body. Pleural plaques are a scarring of the lungs’ pleural linings, most often caused by asbestos inhalation. Dr. Abernethy was presenting her expert advice to the Scottish Parliament in discussions concerning passing legislation to assist those afflicted with pleural plaques. Concerns that the pending legislation would send the “wrong message” to the public led to the presentation from Dr. Abernethy. The arguments from the insurance lawyer held that the pleural plaques develop to trap foreign particles to prevent them from doing further damage to the lungs. Dr. Abernethy is quoted as saying, “My submission is that plaques are a good thing — they don’t cause harm. Harm is something which is pathological in the body, when you get damage and you usually get symptoms.

These plaques are markers of exposure to asbestos .” She holds that it is the same testimony she gave to the House of Lords when they passed legislation preventing those with pleural plaques from being compensated. The Scottish Parliament’s justice committee’s hearing is seeking to overturn that law with the Holyrood bill. P Proponents of the Holyrood bill to allow payments to those with pleural plaques insist that the plaques are evidence that the patient has been exposed to asbestos which could later develop into a life threatening disease such as asbestosis or mesothelioma. Most diseases caused by asbestos exposure do not manifest themselves until 30 to 40 years later, the pleural plaques can develop much sooner. Many believe that the plaques can be indicators for an increased risk of developing future illnesses caused by asbestos.

US FDA Grants Orphan Status to Mesothelioma Drug

A new drug – MolMed’s NGR-hTNF (trade name Arenegyr)- has been granted orphan status by the United States Food and Drug Administration for the treatment of the rare cancer, mesothelioma . It already received the same designation in June of 2008 from the European Union. Orphan drugs are those which treat rare diseases whose sufferers have few or no other options. The orphan status allows for the manufacturer to exclusively market the drug for seven years in the United States and 10 years in the European Union, providing an incentive for drugmakers to produce these pharmaceuticals despite the small market for them. Mesothelioma is a cancerous growth affecting the membrane of the lungs – the mesothelium. The disease almost always results from inhaled asbestos fibers, particles, or dust. Asbestos was once a popular construction material, used in everything from ceiling tiles to insulation.

Decontamination of buildings which had used asbestos is an ongoing problem to this day. While the absolute numbers have been low, incidences of mesothelioma have been increasing over the last few years. This phenomenon is likely due to patients who were exposed to asbestos years ago finally developing the disease. Unlike many other diseases, it can take decades after exposure before a patient manifests symptoms of mesothelioma. This makes pinpointing the exact source of the asbestos exposure difficult. Current therapies for mesothelioma – radiation, chemotherapy, and surgery – have largely proven ineffective. The life expectancy after a malignant pleural mesothelioma diagnosis is only 6 to 12 months. The orphan status was granted to NGR-hTNF after it demonstrated a record of safety and efficiency during its Phase II trials. Finalized results of those trials is expected in December of 2008, but initial findings show that when it us used in patients who have also been treated with chemotherapy, survival rates improve and disease progression is controlled. The drug is designed to destroy tumors in the mesothelium, thereby killing the cancer. The drug is also currently undergoing Phase I testing as a combination with other drugs to treat other forms of cancer.