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AML is a rare aggressive cancer of the blood and bone marrow. It is the most common form of acute leukemia in adults with approximately 21,000 people diagnosed each year in the United States.

To recognize World AML Awareness Day, Celgene’s Kim Hodges sat down with Anna Ferguson, RN, BSN, an oncology research nurse at Johns Hopkins Cancer Center for a Facebook Live discussion about acute myeloid leukemia and how it impacts patients as well as doctors, nurses and caregivers. In our discussion, Anna highlighted the basics of the disease and what groups like the first global AML coalition called KNOW AML are doing to raise awareness.

Having started her career 25 years ago, Anna spoke about her personal journey to becoming an oncology nurse and working with patients in clinical trials. She spoke at length about studying the subject of hope over the past five years and the role that it plays in the live and treatments of patients with cancer. “The first person I took care of in the oncology unit had AML and I remember being so struck and moved by the intensity of the illness and the lack of treatments available,” Anna said, “Yet, I was also struck by the amount of hope and joy and perseverance in the patients. It was the first time that I saw that illness and hope could – and should – live side by side.”

Throughout her career, Anna has learned the true meaning of hope through patients’ inspiring perspectives and their stories about why hope matters and the hopes they keep that give them the strength to wake up and battle their disease each and every day. One of Anna’s patients described how she views hope, “I’m only human. Of course, I want to be cured. But the hope for a cure is not what drives me every day. It’s not what gets me out of bed in the morning. It’s not what lights my fire. It’s different, smaller hopes that keep me going every day. The hope of having enough energy to cook a meal for my family, hoping to make it to my daughter’s soccer game, hoping to avoid transfusions for just a week so I can go visit my sister. These are the things I hope for day to day that make the quality of my life better. These are the things I need my treatment team to know, to see if they can help me make them happen.’” For Anna, it is perspectives like this one that helped shape her nursing practice.

Yet, I was also struck by the amount of hope and joy and perseverance in the patients. It was the first time that I saw that illness and hope could – and should – live side by side.

To help support the educational needs of patients and their caregivers, Anna and her team at Johns Hopkins developed a set of important questions for patients to ask themselves in order to help them have an open dialogue with their oncologists. “There are so many things patients discuss at these visits, but in between those things, it is very important to discuss how AML and its treatments are affecting your day to day life and how you’re managing emotionally when it comes to your illness,” remarked Anna.

Join the full discussion on Facebook and share Anna’s message of why hope matters as we recognize AML patients around the world on World AML Awareness Day.

One of Celgene’s defining characteristics has been a unique distributed research model, supporting promising programs from cutting-edge partners to advance new medicines for patients. In this model, Celgene and its partners mutually benefit from the resources, expertise and innovation from each entity.

One of the longest-standing examples of this collaborative strategy at work is the partnership between Celgene and Agios Pharmaceuticals, Inc., which began in 2010. The Celgene and Agios collaboration has encompassed research and development across a range of candidates in metabolic immuno-oncology and, importantly, led to the 2017 U.S. FDA approval of IDHIFA® (enasidenib) –the first approved medicine from the Celgene distributed research model.

Now, IDHIFA has earned another distinction – Prix Galien winner. IDHIFA was named the 2018 Best Pharmaceutical Product at the 12th annual Prix Galien Awards Gala on October 25 in New York City.

Presented by the Galien Foundation, the Prix Galien is an international award that recognizes outstanding achievements in improving the human condition through the development of innovative therapies and is regarded as the equivalent of the Nobel Prize in biopharmaceutical and medical technology research.

“All winners have made crucial contributions to the advancement of scientific understanding and improved outcomes for humankind,” said Bruno Cohen, Chairman of the Galien Foundation in a statement. “We are proud to honor their tireless work and unrelenting spirit.”

The road to this signature award reflects the shared commitment to patients at both Celgene and Agios. Working closely from the discovery stage, the companies evaluated potential candidates, conducted pre-clinical and clinical research, and ultimately achieved a U.S. approval and launch – less than four years from the first patient ever being dosed with IDHIFA. The companies share commercialization of IDHIFA.

“For all of us at Agios, it is truly an incredible honor to receive the 2018 Prix Galien Award with Celgene for IDHIFA for Best Pharmaceutical Product. As one of the highest accolades for pharmaceutical research and development, this award recognizes the extraordinary, collaborative effort that led to the creation of this important medicine,” said David Schenkein, M.D., Chief Executive Officer of Agios. “This feat would not be possible without the support of our partner Celgene, the patients who participated in our clinical trial, and the Agios scientists who made the IDH discovery and created the IDHIFA molecule.”

“The Celgene and Agios teams have spent years working together toward a common goal,” said Nadim Ahmed, President of Hematology/Oncology for Celgene. “The Prix Galien award is a recognition that our shared commitment has a meaningful impact on the lives of patients.”

“The Celgene and Agios team represent the very best attributes of collaboration,” said Krishnan Viswanadhan, Vice President of Global Alliances for Celgene, who accepted the Prix Galien on behalf of the team alongside Dr. Schenkein. “The combination of Agios’ pioneering science and Celgene’s deep experience in blood cancer research created tremendous potential for innovation, and the drive to help patients constantly pushed us to deliver.”

The Prix Galien award for IDHIFA recognizes, first and foremost, the significant potential to help change outcomes for patients, but also the value and importance of great partnerships.

Please see full Prescribing Information, including Boxed WARNING, for IDHIFA®

In 2009, a patient with acute myeloid leukemia (AML) was the first person with cancer to have his or her whole genome sequenced, helping scientists to learn more about the molecular drivers of the disease. Despite the knowledge gained, researchers have struggled to develop therapies that specifically shut down those drivers.

But this year brings hope for patients with AML, with the approvals of several new treatment options, including therapies that target specific molecular mutations. Dr. Gwen Nichols, chief medical officer for the Leukemia & Lymphoma Society (LLS), believes that these targeted therapies are helping to usher in the era of precision medicine in AML. As we recognize Blood Cancer Awareness Month, Dr. Nichols explains the challenges of translating knowledge into treatments and why she is excited about the future of precision medicine in AML.

Dr. Gwen Nichols, chief medical officer for The LLS, is hopeful about the future of precision medicine in AML.

Dr. Gwen Nichols, chief medical officer for The LLS, is hopeful about the future of precision medicine in AML.

Why has treating AML remained a challenge?

“AML is a complex and dynamic disease that really needs a precision medicine approach to treat appropriate patients. Some patients diagnosed with AML will respond to standard chemotherapy regimens, but most will relapse. Chemotherapy targets highly proliferating cells but may be missing the cells that initiated the AML. Those cells remain behind, recover and can cause the disease to come back in AML patients. This is one reason why the five-year survival rate for AML patients remains low at just 27 percent.

Why has it been challenging to develop targeted therapies for AML?

“When the AML genome was sequenced, researchers thought they were going to find single mutations that drive the disease. They believed that if you got rid of this single molecular abnormality, you could get rid of the disease. We have found a few of these mutations in other cancers, such as in the Bcr-Abl tyrosine kinase in chronic myeloid leukemia. But over the last decade, we’ve learned that some cancers, including AML, are more complex and driven by multiple factors. So an effective therapy targeting one mutation won’t be the end of the story because it’s only one piece of the puzzle. As we work toward the future of precision medicine, we need to look at multiple targeted therapies in combination.”

 AML is a complex and dynamic disease that really needs a precision medicine approach to treat appropriate patients.

What type of diagnostics would you like to see to facilitate precision medicine in AML?

“In a perfect world where it costs nothing and can be done rapidly, you would sequence a patient’s genome as frequently and as completely as possible. The targeted sequencing that doctors are doing for AML patients today makes the most sense because that information can help determine diagnosis and prognosis. But I fear that we may be missing valuable information by not sequencing more of our patients’ genomes. We also need to sequence at intervals to make sure the disease has gone away and again when there’s evidence that the disease is coming back. We can’t assume that it’s the same [form of the] disease when it returns.”

How do the clinical trial designs need to change for precision medicine?

“In diseases such as AML, it’s clear that there are subsequent mutations as the disease progresses and that the disease becomes more complex as it evolves. Most therapies are first tested in patients with relapsed or refractory disease, but you cannot expect a targeted agent to be effective when other driving mutations have arisen. This is a recipe for failure. We may be throwing out therapies that could benefit patients because we are testing them at a time when the disease is so complex that there’s little hope for a single therapy to be effective. That’s why the LLS’ Beat AML Master Trial is focused on newly diagnosed AML patients.”

What needs to happen to truly enable precision medicine in AML?

“The last couple of months have been exciting with several new therapies introduced for AML. We are seeing real progress toward that now with this first wave of targeted therapies. With over 700 clinical trials active or recruiting in AML, there is certainly more to come. But the hope would be to have several different therapies available that target all the drivers of AML. These therapies will not be developed on their own. We need to think about the best way to help facilitate the future of precision medicine through novel trial design and combinations.”

For more information on the progress of precision medicine, read “Getting Patients Access to ‘Precision’ Medicine Is Crucial.”

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For 25 years, Dr. Linda Burns, MD, cared for patients with acute myeloid leukemia (AML) at the University of Minnesota in Minneapolis. She strived to give patients the best care possible. But too often she made decisions based on what treatments her patients’ insurance would cover.

Now, Burns is furthering the conversation about the value of AML treatments. Her goal is to find opportunities to reduce costs without restricting access to appropriate treatments.

“AML is an aggressive disease, and patients will die in weeks or months without proper treatment,” said Burns, who is now vice president and medical director of Health Services Research at the National Marrow Donor Program/Be The Match in Minneapolis. “If a treatment can help these patients, it is creating value for those patients. Cost alone should not be guiding treatment decisions.”

Dr. Linda Burns, MD, vice president and medical director of Health Services Research at the National Marrow Donor Program/Be The Match, believes that costs alone should not guide treatment decisions for AML patients.

Dr. Linda Burns, MD, vice president and medical director of Health Services Research at the National Marrow Donor Program/Be The Match, believes that costs alone should not guide treatment decisions for AML patients.

In reality, even those costs are not well understood. Physicians and patients lack access to the prices of treatments and procedures. To shed light on the costs for AML treatments, Burns and her colleagues analyzed the Truven MarketScan database of private insurance claims from 2007 to 2011 for patients aged 50 to 64 who were treated with either chemotherapy or chemotherapy and stem cell transplantation. Their recently published findings demonstrate the expense involved in treating this rare disease.

There are two primary treatment options for AML: chemotherapy alone or in combination with a stem cell transplant. While stem cell transplants cure more patients than chemotherapy alone, the procedure in combination with chemotherapy costs over $540,000 on average.

Patients who undergo this aggressive treatment option need lengthy hospitalizations that drive up the total costs. On average, patients had five hospitalizations, 92.5 inpatient days and 74.5 outpatient visits.

Cost of Care for Acute Myeloid Leukemia

“The treatment weakens the patient’s immune system and their ability to fight infections, so they need to be hospitalized,” Burns explained. “And we know that hospitalization is very, very expensive.”

The study provided a price benchmark for AML treatment costs. Now, further information about how the treatment improved or extended the patient’s life is needed to understand the value that those treatments create for patients. To this end, Burns and her colleagues are linking cost data to patient health and outcomes information.

It’s important that we understand the value of AML therapies within the current treatment landscape and ensure patients have access to them as well.

The question of value will only grow in importance as new options for AML become available. Progress in AML treatment has been slow over the past 40 years, but clinical trials are exploring therapies that target specific molecular genetic changes in the disease. As we continue to better understand AML and can more precisely target the disease, new treatments have the potential to improve outcomes and, therefore, may help to relieve burdens on the healthcare system.

“Bring them on,” Burns said. “We need new treatments, and targeted therapies would add to the care we can provide to AML patients. It’s important that we understand the value of all AML therapies within the current treatment landscape and ensure patients have access to them as well.”

Last month, James Fitzgerald celebrated his wedding day. It was an event that he never thought he would see when he was diagnosed with acute myeloid leukemia (AML) six years ago. He was immediately hospitalized, and his liver and kidneys failed within 24 hours. His doctors put him into a medically-induced coma for a week before starting his treatment, which included an intensive combination of chemotherapy and radiation. It was one of the only treatment options for AML at that time.

But that’s changing for AML patients like Fitzgerald. Therapies that target some of the specific molecular genetic changes in AML are being developed, and that’s making mutational profiling more important than ever. That’s good news for Fitzgerald who is currently in remission but is concerned that his disease may one day come back.

“When I was diagnosed, I didn’t know to ask my doctors about the different molecular mutations or whether that would even make a difference in my treatment,” Fitzgerald said. “It was only after my treatment that I started asking questions and learning about the genetics of AML.”

Spot the Difference in AML

Enabled by breakthroughs in genome sequencing and analysis over the past decade, researchers have learned more about the genetics that drive AML. In 2008, an AML patient was the first cancer patient to have her genome sequenced, enabling researchers to discover 10 molecular mutations related to the disease.

Since then, scientists have identified as many as 80 potentially disease-associated mutations in many genes, including NPM1, FLT3-ITD, IDH2, DNTM3A, KIT, IDH1, and CEBPA. They have now found genetic differences between the 34 subgroups of AML, confirming the idea that not every AML case is the same.

This diversity can make treatment a challenge. But scientists and patients are hopeful that a better understanding of these molecular changes will improve outcomes. That’s particularly good news for a disease that has not seen substantial improvements in 20 years.

I hope doctors start learning more about the mutations in AML and start talking with their patients about the genetics of the disease.

The current standard of treatment — an intense combination of chemotherapy — is not particularly easy for patients to tolerate, something Fitzgerald can attest to. Eventually, he received a bone marrow transplant and has been in remission ever since.

“It certainly wasn’t fun,” said Fitzgerald. “I had a 106-degree fever at one point, and I was in the hospital for three months. It was definitely tough for me, but at least I had that option.”

Given the severity of treatment-related adverse events, the standard treatment regimen is often not prescribed for older or unhealthy patients. So while half of AML patients under age 60 years live five years or longer, only up to 15 percent of older patients, who more often cannot receive intense chemotherapy, reach the same milestone.

Mutational Profiling May Help Inform Therapeutic Decisions

Researchers are turning their focus to develop therapies that target the specific molecular mutations that drive AML. A recent analysis of 200 patients showed that more than 99 percent of patients had at least one mutation associated with the disease.

As new targeted therapies are being explored, an understanding of the underlying mutations in each patient becomes increasingly important for doctors and patients to decide on an appropriate treatment plan. The Leukemia & Lymphoma Society is exploring the possibility of bringing a personalized approach to AML treatment through its Beat AML clinical trial. With such initiatives in progress, molecular profiling is on its way to becoming a standard part of disease classification and treatment for AML patients.

“I hope doctors start learning more about the mutations in AML and start talking with their patients about the genetics of the disease,” said Fitzgerald. “I can only hope AML patients don’t have to go through what I faced in my treatment.”

To learn more about how clinical trials are bringing a precision medicine approach to AML, read “Bring Patients Closer to Personalized AML Treatments.”


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There has been limited advancement in the treatment of acute myeloid leukemia over the past 40 years, with most patients undergoing intensive, toxic chemotherapy. Today, AML remains a deadly blood cancer, taking the lives of more than 10,000 patients each year, and less than 30 percent of patients live five years or longer after being diagnosed.

As the blood cancer community recognizes April 21 as the second annual AML Awareness Day, an innovative approach to clinical trials holds promise to overcome the research stagnation and explore tailored alternatives tailored for individual patients.

“A high unmet medical need exists for AML treatment,” Amy Burd, Ph.D., vice president of Research Strategy at The Leukemia & Lymphoma Society (LLS), said. The limitations of chemotherapy are especially notable for the elderly, she explained. “Older patients — those who are the most likely to be diagnosed with AML — cannot tolerate the standard chemotherapy regime. It’s just too toxic for them.”

One reason treatment remains challenging is that AML is not a single disease but a complex group of more than 10 different subtypes, some of which are more aggressive than others. And not all types respond to treatment equally.

In 2008, researchers sequenced the genome of AML tumor cells, improving the understanding of how the disease develops and, potentially, treatments it might respond to. With this knowledge in hand, scientists have developed targeted therapies that open up the possibility to tailor treatments for individual patients.

AML Treatment Milestones Timeline

Last October, the LLS launched a unique clinical trial initiative called the Beat AML Master Trial, which matches patients with an investigational drug or drug combination potentially best suited to attack the specific mutations causing their cancer.

Their goal is to create nothing short of a paradigm shift in how AML is treated, moving away from the current one-size-fits-all approach.

But that shift requires a change in the clinical development culture — shaking up business as usual for clinical trials. A typical trial focuses on one or two therapies; but the Beat AML Master Trial is examining multiple treatments — including small molecule therapies, immunotherapies and checkpoint inhibitors — at multiple medical research centers across the country. The LLS has been coordinating the activities with the U.S. Food and Drug Administration, multiple biopharmaceutical companies, and at least 10 trial centers. LLS plans to enroll approximately 500 patients in this trial

We’re trying to put all these pieces together in a way that is focused on what is best for each patient. This is a new approach for clinical trials.

“Putting all the pieces together is the biggest challenge with this initiative,” Burd said. “We have the DNA sequencing technologies, genomic analysis and targeted therapies. But how do we get to the point where they are working together in a way that is focused on what is best for each individual patient? This is a new approach for clinical trials and we think this Master Trial has the potential to stand as a model for future cancer clinical trials.”

Since the launch, they’ve already exceeded the targeted preliminary enrollment of 25 patients and provided those patients with personalized treatment plans. Enthusiasm among investigators and patients has been high, according to Burd.

“It speaks to the high unmet need in AML and desire for patients and doctors to have better treatment options,” Burd said. “Our mission has always been to put patients first. So we’ve incorporated patient-reported outcomes into the study to ensure they are getting the results that matter to them — whether that’s being able to walk around the block or run a marathon.”

AML survivor Vonnie Sullivan stands with her two daughters, Alexandra (left) and Cameron (right).


In December 2007, just days before Christmas, Vonnie Sullivan told her two daughters, Cameron and Alexandra, who were 8 and 13 at the time, that she was leaving them for six weeks to be treated for cancer.

Sullivan, who was in her 40s, had just been diagnosed with acute myeloid leukemia (AML), a blood cancer in which the body makes too many immature white blood cells. She was treated with intensive chemotherapy with the hope of eliminating every cancer cell in her body. The treatment would knock out her immune system, leaving her susceptible to infections. Seeing her children would be out of the question.

“Leaving my daughters was one of the hardest things that I’ve ever had to do in my life,” said Sullivan who at the time was in disbelief about her diagnosis. “When you think of leukemia, you think it’s something that young children get, not adults. Most people don’t know much about AML; I certainly didn’t.”

Lack of awareness is one reason The Leukemia & Lymphoma Society (LLS), MDS Alliance and Patient Power have partnered to launch the first annual AML Awareness Day on April 21, 2016. The goal is to help patients and caregivers better understand this disease and highlight the need for more research.

Progress in AML has been slower than in other blood cancers over the last decade. Common treatment options, which include chemotherapy and stem cell transplants, have changed little over the past 40 years, leaving much work to be done.

Why We Need Better Treatments for Acute Myeloid Leukemia

Lee Greenberger, Ph.D., LLS chief scientific officer, is guiding the organization’s AML research projects. Source: LLS


“The AML patient journey is a difficult one,” Lee Greenberger, Ph.D., LLS chief scientific officer, said. “You can’t work when you are being treated, so patients often require long-term assistance and significant time off.”

For patients who are older or in poor health, these treatments are often too intense for their bodies to handle. Considering the average age of AML patients is 67, some are left with few treatment options.

For younger patients or anyone else who has had successful treatment, many will eventually relapse. By the time the disease returns, new mutations may have developed that may be resistant to current treatment options.

But there are many success stories as well, giving patients a reason to remain positive. It has been eight years since Sullivan was diagnosed, and she’s been in remission since her first and only round of chemotherapy. She has since achieved her long-standing dream of starting her own small business when she opened a home design store in Larchmont, New York in 2014.

AML survivor Vonnie Sullivan became a business owner when she opened her home design store in 2015.


“I have been blessed and am doing very well,” Sullivan said. “I know others who have not been so lucky. It’s a little disheartening that more progress hasn’t been made over the past decade. We need more research.”

Sullivan’s message is being heard. LLS is investing in research that will help doctors determine the most appropriate treatments—either alone or in combination—based on a patient’s particular AML mutations. Meanwhile, several new therapies are being investigated, and some are close to the finish line. This new batch of treatments includes novel approaches such as therapies that target specific AML-related mutations.

Every improvement in the survival of AML patients is important, even small ones.

“Every improvement in the survival of AML patients is important, even small ones,” Greenberger said. “We need new therapies and approaches that can better control this disease and eventually get rid of every AML cell in the body.” 

Patients play an important part in the search for a cure. Many clinical trials in AML are ongoing, and LLS specialists can help patients find a clinical trial to enroll in.

“For patients who are diagnosed with AML, or any type of leukemia, knowing the resources available is important,” Sullivan said. “Hopefully, we can make some real progress in this disease.”

This article was originally published April 20, 2016.