NEW YORK—In 2002, Don Brockman was diagnosed with amyloidosis. The following year, he and his wife launched the nonprofit Amyloidosis Foundation, and in 2007, fellow patient Dennis Krysmalski merged his Amyloidosis Support Network with the foundation.
For the past 20 years, Brockman’s widow, Mary O’Donnell, has led the small charity from Clarkston, Michigan. Today, as executive director and CEO, she supervises 3 employees and an annual budget of around $500,000.
Among other things, the group exhibits at 5 medical conferences throughout the year, including those held by the American Society of Hematology, the American Association of Nurse Practitioners, the American College of Cardiology, and the Heart Failure Society of America.
Mary O’Donnell, executive director and CEO of the Amyloidosis Foundation (Photo courtesy of the Amyloidosis Foundation)
“At the beginning, our primary mission was to support medical and scientific research for amyloidosis,” O’Donnell said. “Over the years, we started putting more emphasis on advocacy and patient support. We still have a primary role in providing research grants, but we also provide help for patients and their caregivers.”
One of the group’s grantees is Heather Landau, MD, a board-certified oncologist and hematologist at Memorial Sloan Kettering (MSK) Cancer Center on the Upper East Side of New York City.
In 2015, the foundation awarded Dr. Landau a $50,000 research grant to study gene expression changes in light chain, or AL, amyloidosis. In early 2025, MSK won a $125,000 fellowship to support the training of a hematologist in the field of amyloidosis diagnosis and treatment.
An expert on amyloidosis, Dr. Landau recently spoke to Rare Disease Advisor from her 22nd-floor office on the eve of National Amyloidosis Awareness Month. Every March, patient advocates mark the month with a “Light the Night” campaign by illuminating hundreds of porches, bridges, and buildings in red to raise awareness about this complex, often misunderstood disease.
Dr. Landau said the future appears bright for treatment of amyloidosis, which is diagnosed in at least 4000 Americans per year. Two especially rare forms of this disease are transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloid polyneuropathy (ATTR-PN).
“Finally, pharmaceutical companies are partnering with us to try to develop drugs in this space,” she said. “For many years, because of the risk of sudden cardiac death in patients who presented with advanced cardiomyopathy, nobody wanted to study their drugs in a patient population who had that risk. That’s why we’ve lagged behind the drug development paradigm, compared to multiple myeloma.”
According to a 2019 article in the Journal of Cardiac Failure, 1 of 25 Black Americans carries the genetic mutation that can lead to ATTR-CM. But that doesn’t mean all such carriers will develop the condition. A separate type of the disease, known as wild-type ATTR, occurs for no apparent reason and is more likely in men and people more than 65 years old, according to the Cleveland Clinic.
Heather Landau, MD, is an amyloidosis expert at Memorial Sloan Kettering Cancer Center in New York City (Photo by Larry Luxner)
“When I came here, I was very interested in pursuing a career in blood cancers,” she said. “My mentor, Dr. Ray Comenzo, who had an infectious enthusiasm for amyloidosis, did some of the pioneering work in stem cell transplant for this disease. He introduced me to this topic, and I felt there was no better way to spend my career than to make sick people well—and these were very sick patients that could ultimately benefit from our care.”
Dr. Landau received her medical degree from Upstate Medical University in Syracuse, New York, followed by a residency in internal medicine from the University of Colorado Health Sciences Center, and later, a fellowship in hematology-oncology from MSK.
Until relatively recently, she said, AL amyloidosis was the most common amyloid disease in the developed world, because the transthyretin (TTR) amyloid cases weren’t being diagnosed due to little awareness. But that’s changing.
“Awareness is driving the incidence of diagnoses substantially,” she said. “Now that we have drug development in this space, this may be even more common than light chain amyloidosis because as we age—in mostly older men—their liver takes a wild-type or a normal transthyretin protein that begins to misfold, similar to a man developing prostate cancer. If you live long enough, you may develop wild-type TTR.”