David Fajgenbaum was diagnosed with Castleman disease while he was still a medical student. He was perfectly healthy, but in a few days he had organs failing. He was treated with chemotherapy and Sylvant which is the only FDA approved treatment for the disease. But then he relapsed. It was then that he decided to dedicate his life to finding another therapeutic option for himself and the Castleman community.
He now works at the University of Pennsylvania and is the founder of the Castleman Disease Collaborative Network. This network improves collaboration between patients, doctors, and researchers to accelerate the research process.
Fajgenbaum’s most notable work so far is his discovery that Rapamune, a medicine used to prevent the body from rejecting kidney transplants, targeted the same mTOR pathway which is active in Castleman. He made this discovery by examining his very own tissue, and first tried taking the drug himself. Now, this treatment is in clinical trials for Castleman disease. For Fajgenbaum himself, the drug worked for 6 years and it’s estimated that it will work for about a third of patients.
He also recently wrote a book called “Chasing My Cure: A Doctor’s Race to Turn Hope Into Action; A Memoir.” Here, he shares his own personal story and his advice for coping with a rare diagnosis. He says to make the most of every moment, question the status quo, ask for help when you need it, and don’t undervalue hope and humor.
MPN Heroes Awards
On December 6, 2019 in Orlando, CURE Magazine held their 7th annual MPN Heroes Awards event. Fajgenbaum was one of the speakers this year and nine heroes were honored at the celebration. These individuals were nominated for their dedication to improving the lives of those living with myeloproliferative neoplasms (MPNs). MPNs are a group of rare blood cancers. These include essential thrombocythemia (ET), myelofibrosis, and polycythemia vera (PCV). The heroes recognized are caregivers, physicians, researchers, patients, and advocates.
Awards were given for commitment to the individual, commitment to the broader MPN community, and to a Canadian MPN champion. A brief description of each individual’s contribution is noted below.
Commitment to the Individual
Natalie Catalano is a nurse practitioner who lives in Pennsylvania. She is a caregiver for her daughter who was diagnosed at the age of 17 with ET. Natalie has been an advocate for her daughter’s physical and emotional well-being throughout her medical journey.
Tammy Matuska has her BSN and RN. She is a nurse coordinator at Washington University School of Medicine for both hematology and oncology. In addition to this formal role, she is dedicated to supporting patients by providing them information about their condition. She organized a dinner for MPN patients as well as their caregivers. Over 100 people attended the event. Due to its success, there are plans to host the event again. She also is planning on creating a MPN support group which will meet regularly.
Dr. Robyn Scherber works at UT Health San Antonio. She is one of the developers behind the MPN Symptom Assessment Form. She specializes in the chronic fatigue symptoms experienced by patients, helping patients to quantify their symptoms and track their experiences.
Kurt Schroeder is a nurse at The University of Texas in Houston. He aims to treat every patient as he would want his family members to be cared for. He advocates for more inclusive clinical studies and works to improve patient compliance. He even takes calls when he’s not on the clock, as he wants to ensure his patients receive the answers they need, when they need them.
Commitment to the Broader MPN Community
Madeleine Henriquez is a PA at the MD Anderson Cancer Center in Houston. After seeing the gap in the care that MPN patients are receiving in terms of mental health, she became a member of the inaugural class (2017) of first-aid mental health workers for the Physician Assistant Foundation. Now, she is teaching a class for her colleagues which helps to raise awareness of mental health issues of patients. In the first year of this course she trained 30 providers in every department. She’s helping to diminish the stigma surrounding mental health, help patients cope with their diagnosis, and ensure medical professionals are educated on the topic.
Dr. Irum Khan specializes in MPNs at the University of Illinois Cancer Center. She strives to help patients become independent and manage their healthcare themselves. She works with the homeless, the uninsured, and the incarcerated. Collaborating with charities, social workers, and foundations, she also helps patients with the financial burdens of illness. Additionally, she is a strong supporter of the mental health of her patients.
Cannon McMullin is a 11 year old ET patient. Mature beyond his years, he says that he wants to do anything he can to help anyone suffering from his condition. He’s currently a part of a clinical trial and though he’s well aware that the findings will likely not help him, he knows they could help others. His positive attitude is admirable and he refuses to let ET define every aspect of his life.
Dr. Laura Michaelis works at the Medical College of Wisconsin. She speaks regularly at support groups, and is the current editor-in-chief of Hematologist, which its the American Society of Hematology’s magazine.
Canadian MPN Champion
Dr. Brian Leber is a hematologist in Ontario. He studies the biomechanics mechanisms of cancer cell death and has a special interest in the effectiveness of current treatments for MPNs. Leber is also serves the Molecular Hematology Laboratory as the Director.
It is people like these who are changing the lives of MPN patients every day, spreading hope, and advancing therapies.
You can read more about these individuals and their work here.