Hello, my name is Dr. Philip McCarthy. I work at Roswell Park Comprehensive Cancer Center. I'm here today at ASH 2025 to discuss work that was conducted by my colleague, Janine Joseph. She's the first author. Yann Solengas is the senior author, and I'm presenting the poster on their behalf on Monday night regarding amyloidosis and immune profiling in AL amyloidosis as well as ATTR, which is transthyretin amyloidosis. And when I first started doing this a few years ago, not many people knew what ATTR amyloidosis is, and it's a very complex set of diseases, both hereditary as well as acquired with age, where there's a buildup of a protein called amyloid, and in many cases it's in the heart or in the nerves. And that's different from amyloid that's due to light chain deposition, which is associated with plasma cell disorders. And for people who are aware of plasma cell disorders, there's a syndrome called monoclonal of undetermined significance, and its AL amyloidosis is kind of like evil MGUS, where it deposits in the kidneys and the heart and requiring patients to receive therapy. Several years ago, patients with amyloidosis didn't have many therapeutic options. Now what's very exciting is in addition to early diagnosis, patients with AL amyloidosis receive therapy that's similar to the treatment for multiple myeloma, is directed against the plasma cells to prevent further deposition and prevent worsening renal failure or cardiomyopathy. With ATTR, it's often the hematologist who may get the referral because the patient has amyloidosis, perhaps seen on cardiac echo or on heart biopsy, but on workup they don't have a monoclonal protein. So initially there wasn't a lot that could be done for these patients, but now there are several drugs that are either in development or FDA approved that prevent the deposition of the ATTR, or of the TTR in ATTR, and it allows patients to live longer lives because the disease is better controlled. So what did we find? We found, most of our surprise, that patients with ATTR, we know that they have inflamed hearts because the protein gets deposited there and causes the immune cells to come in and cause damage. But we also found that the peripheral blood contains a lot of activated cytotoxic T cells and what are called exhausted T cells, which means they've been inflamed for so long, they become exhausted. And we found that a bit surprising in that AL amyloidosis was showing some degree of activation and these are all compared to healthy people who don't have any inflammation. ATTR was more inflamed and interestingly there were more B cells in ATTR than there were R in AL amyloidosis, which is a B cell disorder. So it led to a lot of questions on our part and we're further diving into why are these differences seen and is this a sign along with thinking about the disease and the causes early on that will allow us to be able to modify the course of both AL amyloidosis as well as ATTR. If our videos have helped you in any way and you're able to, please consider making a donation to help us continue this important work. Your gift will go three times as far when we reach $500,000 by the end of the year. Every contribution, big or small, makes a difference and we're deeply grateful for your support.
HealthTree • December 20, 2025