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Over the past decade, researchers have begun to uncover the complex biology behind myelofibrosis. Nearly 5,000 people in the United States are estimated to be diagnosed with this blood cancer each year.



“We do not yet fully understand the biology behind why myelofibrosis advances in patients,” explained Ruben Mesa, M.D., director of UT Health San Antonio MD Anderson Cancer Center. “Improving our understanding can help us advance treatment beyond the current approved and investigational therapies, which are still being examined to determine how they might slow progression of disease.”

What Is Myelofibrosis?

Myelofibrosis is a rare blood cancer that starts in the stem cells of the bone marrow, leading to the production of faulty blood cells that don’t mature or function properly. Eventually, the disorder results in scarring of the bone marrow, further stifling its ability to produce healthy blood cells.

While some patients may live years without symptoms, others see their disease progress rather quickly. Without enough healthy red blood cells, these patients can develop anemia, leading to fatigue, weakness and shortness of breath. Myelofibrosis can also cause an enlarged spleen and the disorder has a higher incidence in people over age 50.

In addition to monitoring patients closely for any signs of disease progression or other conditions, physicians face the challenge of tailoring treatment. “Myelofibrosis is a variable disease,” Mesa said. “A better understanding of the role of genetic mutations that cause the cancer and refining the prognostic scores could help doctors have a much more detailed assessment of prognosis and determine which treatment options are best for each individual patient.”

Understanding Options

While a stem cell transplant is potentially curative in some cases of myelofibrosis, doctors reserve it for patients fit enough to endure the procedure due to the risk of life-threatening complications. Eligibility depends on several factors, including prognosis, age, overall health and the availability of an appropriate donor.

“The average age of diagnosis for myelofibrosis is about 60 years old, which is toward the latter end of when transplantation is considered safe or effective,” Mesa said. “So it’s not an appropriate therapy for all patients.”

For many patients, the goal of treatment is focused on relieving symptoms, reducing an enlarged spleen and improving blood cell counts. Therapy options for those who are ineligible for a transplant include JAK inhibitors, chemotherapy, immunomodulators and corticosteroids. Not all of these agents target the signs and symptoms associated with myelofibrosis, and researchers are working to find additional options.

There are many reasons to be hopeful in myelofibrosis research – a better understanding of the disease, better understanding of its genetic drivers and better therapies.

Hope for the Future

As with many rare diseases, several unmet needs in myelofibrosis are attracting attention, according to Mesa. “Currently, there are very few targeted therapies approved for myelofibrosis, and we do not have effective therapies for patients with the most advanced forms of the disease.”

There is hope. At December’s American Society of Hematology (ASH) Annual Meeting, researchers presented data from more than 20 clinical trials in myelofibrosis. Treatment options being investigated include JAK inhibitors and combinations that incorporate other agents, like immunomodulatory therapies which adjust the body’s immune responses to disease. Researchers are also exploring treatment strategies that target multiple disease-signaling pathways, which are showing promise in early studies, according to Mesa.

These are positive developments for patients with a disease like myelofibrosis. More targeted therapies may one day allow doctors to tailor a patient’s treatment to their specific symptoms and unique genetic makeup of the disease.

“There are many reasons to be hopeful in myelofibrosis – a better understanding of the disease, better understanding of its genetic drivers and better therapies,” Mesa said.

To learn more about Celgene’s commitment to myelofibrosis and other rare diseases, read “Supporting Research to Find Cures for Rare Diseases.”

While Blood Cancer Awareness Month provides an opportunity to raise awareness and understanding of the various forms of blood cancer, it also serves as a reminder of the progress that has been made in treating these diseases. This progress would not have been possible without the numerous individuals who are bound together to improve the lives of people diagnosed with blood cancers.

A blood cancer diagnosis significantly alters the lives of not only patients but also their caregivers, friends and family. In 2012, more than 900,000 people worldwide were diagnosed with blood cancer. In the United States, someone is diagnosed with blood cancer approximately every three minutes.

Explore a model “Day in the Life” of various individuals through the following infographics to better understand the effect blood cancer has on their daily lives.

Blood Cancer Awareness Month
Multiple Myeloma


February 28, 2018 marks the 11th annual Rare Disease Day, dedicated to raising awareness of the impact of rare diseases worldwide. This year’s theme – research – highlights the many advances that have been made in the treatment of rare diseases, while emphasizing remaining research gaps.

This year, and every year, Celgene joins with the many supporters of Rare Disease Day as we all strive toward the ultimate goal: cures.

A rare disease is one that affects fewer than 200,000 individuals in the United States and less than one in every 2,000 in Europe. As a whole, though, rare diseases are hardly insignificant; there are approximately 7,000 different rare conditions affecting more than 300 million people globally.

Over the decades, regulatory initiatives have helped to encourage more innovation and the generation of much-needed therapies for these patients. The U.S. Food and Drug Administration’s (FDA) Orphan Drug Act of 1983 spurred significant progress by creating incentives for research and allowing for expedited approval of new therapies. While fewer than 10 treatments for rare diseases were approved from 1973 to 1983, more than 400 medicines and biologic products for rare diseases have been approved since the Act was passed.

More recently, provisions of the 21st Century Cures Act of 2016 have helped to streamline FDA review of rare disease therapies. And in December 2017, the Rare 2030 pilot program was adopted by the European Commission to research sustainable policies, address challenges and identify opportunities in the field of rare diseases from 2020-2030.

And yet despite these efforts, approved therapies are available for only five percent of rare diseases. Clearly, we still have a long way to go for these patients.

With clinical trials in more than 45 rare cancers and immuno-inflammatory diseases, Celgene recognizes the significance of medical innovation to address these often overlooked diseases. Celgene’s research efforts center on patient need, including rare diseases.

Clinical trials are key to making progress, but for rare diseases, enrolling a clinical trial can be a challenge, given the limited number of patients. To find the most patients that can participate in a trial, Celgene believes a critical first step involves carefully examining and engaging the appropriate trial locations, for instance, by establishing a presence in countries where a rare disease is more common.

Driven by patient and physician insights, Celgene also leads research efforts that go beyond traditional trial programs. For instance, the Journey Pro app, launched by Sage Bionetworks with financial support from Celgene, uses patient-reported data collected through mobile and wearable technologies to help quantify the daily burden of chronic anemia, which affects patients with rare diseases such as myelodysplastic syndromes, myelofibrosis and beta-thalassemia. The app provides direct and immediate information to research participants to help them manage their health, with a long-term goal of developing a tool to evaluate new treatments.

In multiple myeloma, Celgene has collaborated with the UAMS Myeloma Institute and Dana-Farber Cancer Institute to create the Myeloma Genome Project, a global initiative compiling the largest set of genetic profiling data associated with clinical outcomes. The Project aims to develop a genetic classification system, and eventually, relevant tests for genetic mutations that could improve the diagnosis and prognosis of multiple myeloma while driving more personalized, targeted approaches to treatment.

In addition to these and other research endeavors, Celgene’s collaborative efforts with patient and professional advocacy organizations through the Patients’ Partners program, launched in 2011, explore new ways to strengthen patient support from diagnosis through treatment.

As research efforts in rare diseases continue to expand across the healthcare ecosystem, the progress being made drives Celgene to keep pushing forward in an effort to deliver new solutions that may improve patients’ lives.