Author: Nancy Meredith
35th Anniversaries of NORD and The Orphan Drug Act
Many mesothelioma patients may not know that every day the National Organization for Rare Disorders has a team of people fighting for them. NORD was established in 1983 to improve the lives of individuals and families affected by rare diseases. This month the organization announced it will be spending 2018 marking 35 years of advocacy.
Not coincidentally, the Orphan Drug Act, that provides incentives to the pharmaceutical industry to invest in developing therapies to help small patient populations, has also reached the 35-year milestone. The Act was created and passed by the hard work of an ad hoc coalition of parents of children with rare diseases who lobbied Congress and the medical and pharmaceutical communities to call attention to rare diseases. After the successful passage of the law, the coalition formed NORD.
“The advances in diagnosis, treatments and care for patients with rare diseases have been remarkable, but we still have many challenges ahead of us,” said Peter L. Saltonstall, President and CEO of NORD, in a Jan. 4 press release announcing the milestone. “The Orphan Drug Act and the creation of NORD brought national attention to rare diseases. The joint anniversaries are an appropriate time for us to recognize the progress made and rededicate ourselves to the needs of the patient community.”
A disease is designated as rare or orphan when less than 200,000 Americans are affected by the disease at any given time. Over 7,000 diseases affect close to 30 million Americans. Mesothelioma is responsible for approximately 3,000 new cases of cancer each year in the U.S. Although there is no cure for mesothelioma, it can be treated with varying degrees of success through the use of surgical procedures, chemotherapy and radiation.
While there is ongoing research to find new treatments or a cure for these orphan diseases, research requires significant amounts of time and money. Since many of these illnesses affect so few people, researchers and pharmaceutical companies do not find it beneficial to expend the time, effort or money to find treatments and cures. Although the Orphan Drug Act has helped in the development of some treatments, still less than 500 of the orphan diseases have an approved treatment, according to NORD.
Some of the milestones NORD highlights from its 35-year history include:
- Named a 4-star charity by Charity Navigator, one of the 100 Best Charities by Worth Magazine, and “Charity of the Week” by The Week Magazine;
- In at least two instances, NORD’s rare disease research grant program has resulted in FDA-approved orphan therapies;
- NORD established a Natural History Study program and entered into a cooperative agreement with FDA to further rare disease research.
Since inception of the Orphan Drug Act, the FDA has approved more than 600 orphan products and 4,000 orphan drug designations. The Orphan Drug Act, according to NORD, is credited with helping to drive innovation in cancer treatment and gene therapies.
“The Orphan Drug Act is just as important today as it was in 1983,” Saltonstall said. “This year, we will be saluting the progress made to date, we will also be focusing on the fact that much remains to be done.”
Improving Immune System’s Memory May Lead to New Mesothelioma Treatment
Mesothelioma patients often lose ground when fighting back against the deadly cancer due to a compromised immune system. Immunotherapy is one way oncologists and researchers use to awaken the immune system so it can successfully fight the cancer. Now, researchers report they may be able to stimulate the immune system’s ability to “remember” infections leading to new treatments that may better help fight disease.
Researchers from Monash University in Australia report they have discovered a “novel molecular ‘blueprint’” that could be the key to immunological memory, according to a Dec. 20 press release from Monash University’s Biomedicine Discovery Institute. The team found that when the immune system fights an infection, the B and T cells create memory cells. This memory is how immunity is developed and how vaccines work by mimicking that immunity.
B cells generate antibodies, whereas T cells are known as killer cells and destroy the invading sick cells. Using a strain of the flu, the researchers found the T cells, that the team called hit men, “are genetically wired to behave” the way they do. This finding broke new ground in understanding the changes T cells undergo when fighting an illness.
“What we found is there’s a specific sort of wiring that seems to characterise this type of immunological memory – it’s like an electrical circuit,” Professor Turner said. “We were able to identify specific ‘switches’ that are used in parts of the genome that actually give T cells their function.”
Mesothelioma, a signature cancer of asbestos, is extremely aggressive, and the cancer cells continue to grow and multiply as the patient’s immune system is compromised. Finding a way to wake up the immune system is critical for extending survival. Immunotherapy has been used successfully in some mesothelioma patients, however, this research points to another way to engage the immune system. In fact, Turner said, the “blueprint could serve as a benchmark for research, and potentially be used to hone and personalise therapies.”
Understanding immunological memory may help them improve existing treatments or create new ones that use the immune system to fight cancer, the researchers concluded.
The team will continue this research and reports the next step is to use the human influenza virus next.
Read the full study in the Dec. 19, 2017, issue of Cell Reports.
Libby, Montana Mesothelioma Doctors Contribute Knowledge to Medical Journals
The doctors at Libby, Montana’s Center for Asbestos Related Disease (CARD) know a thing or two about mesothelioma. The town is home to a vermiculite mine blamed for widespread contamination from asbestos exposure and for hundreds of cases of mesothelioma, and CARD has been the hub for care and screening of many of the patients. Now, the medical team is sharing their knowledge of this deadly disease through collaboration on articles for scientific journals.
Dr. Brad Black and Dr. Gregory Loewen, both physicians at CARD, were among the authors in two separate studies published recently in the peer-reviewed journals Inhalation Toxicology and the Journal of Occupational and Environmental Medicine (JOEM), according to a Dec. 29 article in The Western News. The two provided their expertise into the studies looking at the pulmonary function of miners at Libby’s vermiculite mine.
In the study of 256 individuals who worked at the mine for a minimum of six months, the researchers concluded “eighty-seven percent of miners exposed to Libby Amphibole had pleural abnormalities on CT [computed tomography].” The study was published in JOEM.
“… most doctors probably don’t know about this unique type of asbestos related disease and wouldn’t be able to recognize it in their patients if they didn’t hear about it,” said Dr. Black on why the study is important.
The W.R. Grace vermiculite mine and mill located in Libby, closed since 1990, was found to have an asbestos deposit in the mine and is the site of significant asbestos exposure. According to reports, nearly 3,000 residents and former miners have been diagnosed with an asbestos-related disease, and over 400 have died from mesothelioma. Contamination from the site led the federal government to declare Libby a health emergency.
Asbestos is a known carcinogen and is proven to cause mesothelioma, a serious cancer caused by breathing in the asbestos fibers that then become lodged in the thin membrane that lines and encases the lungs.
The second study followed one individual who worked at the mine from 1969 to 1990 and had no other known exposure to asbestos. The person developed “asbestos-like” pathological features and eventually an adenocarcinoma. According to the authors, “This is to our knowledge the first time such an extensive evaluation has been conducted in a vermiculite miner from Libby, Montana.”
About CARD
CARD’s primary goal is to provide specialty healthcare and screening to those affected by Libby Amphibole Asbestos. CARD’s secondary goal is to stimulate research from around the country to gain further understanding of disease mechanisms, improve early disease and cancer detection and intervention, and develop effective health management strategies in hope of finding answers to improve health outcomes for individuals and communities.
Find out more about the Center for Asbestos Related Disease on its website.
Read the studies in the Dec. 1 issue of the Journal of Occupational and Environmental Medicine and in the Oct. 17 issue of Inhalation Toxicology.
Sources:
- Center for Asbestos Related Disease
http://www.libbyasbestos.org/ - Inhalation Toxicology
http://www.tandfonline.com/doi/abs/10.1080/08958378.2017.1372536 - Journal of Occupational and Environmental Medicine
http://journals.lww.com/joem/pages/default.aspx
- The Western News
http://www.thewesternnews.com/front_page_slider/20171229/libby_asbestos_clinic_contributes_research_to_two_journals
Make the Most of the New Year
During the holidays, “Happy New Year!” gets repeated countless times throughout the day. Whether it is from the barista at the local coffee shop or the staff at the medical center, this is not the message a mesothelioma patient or his or her family wants to hear. It is doubtful that anyone with mesothelioma is happy.
But today is the start of a new year, and mesothelioma patients and their families can make the most of every day. In 2018, consider looking at “happy” another way and change the way you look at the new year.
Help others in the mesothelioma community by offering them insights from your experiences.
Always stay positive.
Praise your caregivers and let them know they are appreciated and valued.
Take Pleasure in the little things and look forward to whatever the next day may bring.
Your medical team members are your partners and advocates. Insist on the best care from them, and make sure they know what you want.
Just like looking at “happy” one letter at a time may help you change your perspective, take 2018 one day at a time and make the most of the new year.
Top Mesothelioma Stories of 2017
Day after day, countless researchers from across the U.S. and the globe are driven to find an effective treatment, if not a cure, for mesothelioma. It is this dedication to research that brought some significant breakthroughs for mesothelioma care in 2017.
As 2017 comes to an end, Mesothelioma Help looks back at some of the biggest stories that brought excitement and hope to all of the mesothelioma community.
FDA Approvals
Anytime the U.S. Food and Drug Administration approves another anti-cancer therapy, the mesothelioma community takes notice. This year, two landmark approvals took the cancer world by storm: gene therapy and an approval based on a biomarker and not a tumor type.
FDA Commissioner Scott Gottlieb said in Dec. 7 remarks before the U.S. Senate Committee on Health, Education, Labor & Pensions Hearing:
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm588046.htm
“We’ve seen two recent approvals of CAR-T therapies for cancer, where a patient’s own immune cells are re-engineered – using the tools of gene therapy – to target a patient’s individual cancer. This form of gene therapy represents a whole new paradigm in treating cancer. And the early results are changing the way we treat serious tumors.
Over the next several years, we’ll see this approach become a mainstay of treating, and probably curing, a lot of our most devastating and intractable illness. At FDA, we’re focused right now on establishing the right policy framework to capitalize on this scientific opening.”
Read about the first-ever gene therapy approval.
In another first, the FDA approved an anti-cancer drug based on a biomarker and not cancer type. The FDA granted accelerated approval to Keytruda as a treatment based solely on the genetic mutations of a cancer and not on the type of cancer.
The FDA’s Richard Pazdur, M.D. announced in a May 23 press release:
https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm560167.htm
“This is an important first for the cancer community. Until now, the FDA has approved cancer treatments based on where in the body the cancer started—for example, lung or breast cancers. We have now approved a drug based on a tumor’s biomarker without regard to the tumor’s original location.”
Keytruda is also approved in the U.S. for use in melanoma and lung cancer patients whose cancer continues to grow after a prior round of chemotherapy failed to stop the progression. The immunotherapy drug was wildly successful for Mavis Nye of England, who is now an eight-year mesothelioma survivor. She recently launched her Mavis Nye Foundation to give back to the community that supported her throughout her journey.
Read about the first-ever approval for an anti-cancer drug for a biomarker.
Clinical Trials
The National Cancer Institute promoted its nationwide clinical trial that is open to thousands of cancer patients for treatments based on the genetic makeup of their tumors. For patients suffering from cancer that continues to grow, despite previous treatment, the NCI-Molecular Analysis for Therapy Choice (NCI-MATCH) clinical trial offers hope. The trial was developed to determine whether treating patients with drugs that target the gene abnormalities believed to be driving their cancer will shrink their cancer regardless of the cancer type.
Read more about the NCI-MATCH trial.
To make the list of the top mesothelioma stories of 2017 doesn’t always mean it is good news. One of the big stories this year, unfortunately, is that the number of mesothelioma cases continue to rise. In its report “Malignant Mesothelioma Mortality — United States, 1999–2015,” published March 3, the U.S. Centers for Disease Control and Prevention reports that despite a decline in asbestos exposure due to regulatory actions and the decline in the use of asbestos, the number of mesothelioma deaths each year is still rising.
Read more about the report here.
http://www.asbestosdiseaseawareness.org
Asbestos Related News
None of the breakthrough drugs would be needed if people were not exposed to asbestos in the first place. Yet, exposure continues to be a real threat, and scientists, environmentalists and physicians continued their call for a ban on the toxic mineral.
At least one country recently took action to stop the spread. Brazil, the world’s third highest producer of asbestos, recently banned asbestos in the country.
Many in the U.S. mesothelioma community hope that this action opens the eyes of U.S. government officials and a U.S. ban won’t be far behind. Linda Reinstein, President/CEO and co-founder of the Asbestos Disease Awareness Organization, is doing her part and saw progress when the Senate passed the Alan Reinstein Ban Asbestos Now Act (ARBAN) of 2017.
Read more about what ARBAN could mean for Americans.
About Mesothelioma
Mesothelioma is a deadly cancer of the lungs, heart or abdomen, caused by past exposure to asbestos. Mesothelioma has a long latency period where those exposed to asbestos may not exhibit symptoms for decades after exposure. Mesothelioma can be treated with surgery, chemotherapy and radiation, however, the likelihood of recurrence of the cancer is high. There is no cure for mesothelioma.
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