Receiving a diagnosis of a rare disorder can be not just jarring, but terrifying. In a moment, everything changes, and the world looks a little darker.
How can a patient find the strength to move forward into an uncertain future?
Dr. David Fajgenbaum has insights for people faced with this dilemma, because he’s been there. He was a 25-year-old medical student when he developed Castleman disease — a rare disorder that involves overgrowth of the cells in lymph nodes — and had trouble getting a definitive diagnosis. Later, when he experienced a recurrence after treatment, he dedicated himself to finding a new therapy that would put him back into remission. Now, the doctor, an assistant professor at the University of Pennsylvania, also heads up an organization devoted to accelerating research and treatment to benefit people with Castleman disease.
Fajgenbaum told his story at CURE
magazine’s seventh annual MPN Heroes Awards event, held Dec. 6 in Orlando, Florida, on the eve of the annual American Society of Hematology meeting. In his talk, the doctor expressed a philosophy of hope and determination that he shares with the evening’s nine honorees, who have also dedicated themselves, despite obstacles, to improving life for others.
The eight heroes and one Canadian champion work in support of people with a rare group of blood cancers known as myeloproliferative neoplasms, or MPNs: polycythemia vera, essential thrombocythemia and myelofibrosis. They are clinicians, researchers, patient advocates and caregivers who strive to find new treatments and provide education, emotional aid and care for those with MPNs.
“Heroes, you are turning hope into action, and you are transitioning those hopes into reality,” Fajgenbaum told the evening’s honorees. “Thank you for what you do.”
Erik Lohrmann, vice president of CURE Media Group, which is part of MJH Life Sciences, voiced the organization’s pride in giving the awards.
“We look forward to the opportunity to recognize people who dedicate their careers, their lives, to helping patients,” he said. “It renews our perspective, reminding us why we entered the health care field. The collective passion, dedication and commitment of our 2019 class of heroes is inspirational, to say the least.”
The event was sponsored by Incyte Corporation and eight patient advocacy organizations.
Commitment to the Individual
Four recipients from across the country were honored for their “commitment to the individual.”
Natalie Catalano, a nurse practitioner from Uniontown, Pennsylvania, has been a devoted family caregiver since her daughter, Carmen Orrico, started having unexplained symptoms. Years later, Orrico was diagnosed with essential thrombocythemia (ET) at age 17. Throughout Orrico’s journey, Catalano persistently advocated for her daughter’s care and took on the physical and emotional work associated with being a caregiver to someone with a rare blood cancer. She made it a point to help Orrico maintain her dignity and grace while in the hospital.
Part of an active family, Catalano helps with her daughter’s care on outings and even from afar, now that Orrico is in college.
Orrico nominated her mother, saying that “she never left my side, and she still gives me words of encouragement. She’s my best friend and a superhero in my life.”
Tammy Matuska, B.S.N., RN, is a nurse coordinator in the hematology and oncology department at the Washington University School of Medicine in St. Louis, Missouri. Beyond organizing infusion appointments, scheduling testing and assisting with complicated paperwork for patients with MPNs, Matuska also works beyond her long hospital hours to contact insurance companies and ensure that her patients have access to proper care.
Her dedication to furthering education about MPNs led her to organize an informational dinner for patients and their families. Sponsored by the Leukemia & Lymphoma Society, the event was attended by more than 100 people, creating demand for a second dinner the following year. Now, Matuska plans to start a regularly scheduled support group for people with MPNs.
Her nominator, Dr. Stephen Oh, called her “a lifeline to patients” and said he relies on her compassion and dedication.
Dr. Robyn Scherber is a clinician, hematologist and researcher at Mays Cancer Center at UT Health San Antonio, in Texas, where she works to advance science devoted to MPNs. She helped develop the MPN Symptom Assessment Form (MPN-SAF), which is used to evaluate patients in clinical trials, hospital intake and other medical settings. In addition, her research into chronic MPN-related fatigue and the symptom burden of MPNs has helped thousands of people quantify their symptoms.
Scherber is focused on empowering people living with MPNs to track their own symptoms and evaluate their experiences. To share her knowledge with the MPN community, she has been a part of the Mays Cancer Center family education program and regularly speaks at conferences, such as the MPN Advocacy & Education International’s Women & MPN Conference.
Nominated by Dr. Ruben Mesa, Scherber said she is driven by the satisfaction she feels when helping a patient whose symptoms are interfering with everyday life.
As a research nurse at The University of Texas MD Anderson Cancer Center in Houston, Kurt Schroeder is a passionate advocate for his patients living with MPNs. He strives to ensure that every patient who visits the leukemia department receives excellent care, working with a simple motto: “How would I want my family member to be treated if they came in here?”
Schroeder seeks to include more patients in research studies by pushing for inclusive wording in protocols and less restrictive monitoring. To help patients remain in the studies, he makes creative suggestions to help them comply with their individual care plans.
Schroeder’s advocacy doesn’t stop at the hospital doors — he takes calls during his days off to ensure that his patients find the answers and care they need.
“He’s no doubt a hero to our patients,” said his nominator, Dr. Prithviraj Bose.
Commitment to the Broader MPN Community
A second group of recipients were honored for efforts that have had a significant impact on the broader MPN community.
Madeleine Henriquez is a physician assistant in the leukemia department at MD Anderson Cancer Center in Houston, Texas, who decided to take on additional training as a mental health first-aid professional. Henriquez realized there was a gap in care for patients with MPNs, as the symptom burden of these diseases sometimes created mental health-related issues that went undiagnosed and untreated. So, she joined the Physician Assistant Foundation’s inaugural class of mental health first-aid workers in 2017 and is now serving in this role by using her time off to teach a course for her fellow professionals aimed at raising their awareness of these issues.
Henriquez’s nominator, Allyson Price, reports that she is fulfilling a vital gap in the care of patients with MPNs by helping them cope with the realities of their diagnosis. This work has also helped to lift the stigma around mental health issues and furthered a whole-patient approach to treatment of those with rare blood cancers.
“In my first year, I trained more than 30 advanced practice providers across every department,” Henriquez said. “They’re so much more aware that this can be an issue, and they’re not afraid to address it.”
Dr. Irum Khan, a specialist in MPNs and related diseases who works at University of Illinois Cancer Center, has dedicated herself to helping two groups of marginalized patients: people living with MPNs and people without resources to independently manage their own care, including the homeless, incarcerated and underinsured. In her practice, Khan works with social workers, charitable organizations and medical foundations to help her patients manage the hidden costs of extended care like transportation and nutrition. She also conducts research and keeps an eye on the psychological toll of MPNs in her patients, seeking to help them manage issues such as depression and anxiety.
Khan, nominated by Maryam Zia, is active with her local Leukemia & Lymphoma Society and speaks regularly at educational events for patients and their families affected by MPNs.
“It takes a village” to treat a population that includes many underserved patients, “but we have the resources and emotional investment to do it,” Khan said of her team.
Cannon McMullin, of Arkansas, was diagnosed with ET at 8 years old and has been living with the disease for more than three years. He travels regularly for testing and check-ins with his pediatric oncology care team, yet continues to do the activities he enjoys: playing point guard for his school’s basketball team, kayaking with his family and playing video games. He keeps a positive attitude when dealing with his diagnosis and care, refusing to let his illness define his life.
As part of an ongoing clinical trial, Cannon understands that although the research he's contributing to may not help him directly, the findings may help other people with ET in the future. “I have to be a part of this if it can help someone — anyone,” he said.
Cannon was nominated by his mother, Ann McMullin, a nurse.
Dr. Laura Michaelis, a specialist in hematology and oncology at the Medical College of Wisconsin, seeks to deliver superb care in a compassionate, individualized manner. In caring for people living with MPNs, she is a proponent of her hospital’s 24-hour transfusion and evaluation access point — a kind of emergency room designated specially for people with rare blood disorders.
Michaelis regularly speaks to MPN patient support groups, providing information and answers in a more personal setting.
A news reporter before she decided to dedicate herself to medicine, she serves as editor-in-chief of the American Society of Hematology’s member news magazine, Hematologist
“I love my patients and have been humbled by meeting and interacting with them,” said Michaelis, who was nominated by registered nurse Lori Jemison. “That’s precious, and better than any award.”
Canadian MPN Champion
Dr. Brian Leber is a clinical hematologist and professor of medicine at McMaster University Medical Centre and the Juravinski Cancer Centre, both in Ontario, with an interest in the pathophysiology of leukemia and investigational new drug trials. An associate member of the department of biochemistry, he collaborates with David Andrews on studies investigating basic biochemical mechanisms in the death of cancer cells and the effectiveness of specific targeted drugs in treating MPNs. He is also a member of the Hamilton Regional Laboratory Medicine Program and the director of the Molecular Hematology Laboratory.
He was nominated by John Clark.
who heads up the University of Pennsylvania’s Center for Study and Treatment of Castleman and inflammatory Lymphadenopathies, told the audience about his journey with Castleman disease.
In his first experience with the illness, he went from being healthy to having multiple organ failure within a few days. After 11 weeks in the hospital, he was read his last rites, but finally received a diagnosis and chemotherapy that saved his life. Through several relapses, Fajgenbaum was treated with chemotherapy and eventually the one drug approved by the Food and Drug Administration for Castleman disease, Sylvant (siltuximab), but then ran out of options. He vowed to use whatever remaining time he had left to help find additional treatments for the disease.
He started by studying his own tissue and discovering that, in it, the mTOR signaling pathway, a communication line within the immune system, was excessively active. He knew that a 25-year-old drug used to prevent rejection of transplanted kidneys, Rapamune (sirolimus), targeted that pathway, so he tried taking it. The medication has worked for him for about six years.
The drug appears to work in about one-third of patients with Castleman disease — a different one-third than those who respond to Sylvant — and it is being tested in a clinical trial, Fajgenbaum said.
Fajgenbaum also founded the Castleman Disease Collaborative Network, which uses a network of physicians, patients and other stakeholders to crowdsource ideas about what should be studied in clinical trials and who would be best suited to conduct the research.
The doctor — who told his story in a recent book, “Chasing My Cure: A Doctor’s Race to Turn Hope Into Action; A Memoir” — shared with the audience some of the lessons he’s learned from his journey. He suggested making the most of every second; using humor to get through difficult times; questioning the status quo; using hope to motivate actions; and asking for help.
The evening’s awards were handed out by mistress of ceremonies Kristie Kahl, editorial director for CURE Media Group. The judges who selected the winners were David Denny, Mary Dunkle, Dr. Andrew Kuykendall, Julia Olff and Maureen Thyne.
The celebration was made possible with support from Incyte Corporation, a Delaware-based biopharmaceutical company focused on oncology. Also supporting the event were CancerCare
, the Canadian Myeloproliferative Neoplasm Network, Cancer Support Community, the Leukemia & Lymphoma Society, MPN Advocacy & Education International, the MPN Education Foundation, the MPN Research Foundation and the National Organization for Rare Disorders.