AL amyloidosis was placed in the spotlight at the 59th American Society of Hematology (ASH) annual meeting in Atlanta, Georgia when Dr. Giampaolo Merlini, from the University of Pavia, Italy, gave the prestigious keynote Ham-Wasserman lecture on the first day. Speaking to an audience of several thousand hematologists, Dr. Merlini provided an overview of the recent advances in several key areas in AL amyloidosis. These included; a better understanding of the molecular mechanisms that give rise to AL amyloidosis; the importance of early diagnosis; the need to tailor treatment to individuals; and the role of cardiac biomarkers at critical stages of the disease particularly at diagnosis, when risk stratifying patients and measuring response to treatment. Dr. Merlini also discussed the exciting developments in the drug pipeline. He explained how different approaches were being used to target not only the cause of AL amyloidosis but also its consequences, noting how new drugs aimed at accelerating the breakdown and removal of amyloid deposits were showing promising results in clinical trials.
Dr. Vaishali Sanchorawala, our Director of the Amyloidosis Center, presented the results from the first trial noting that patients achieved rapid reduction in free light chain levels after the first dose of daratumumab and further reductions with subsequent doses. Patients also showed improved kidney and heart function with continued treatment up to six months. Side-effects associated with daratumumab were mild and easily managed.
Results from the second trial presented by Dr. Murielle Roussel, from the University Cancer Institute of Toulouse, Oncopole, France also demonstrated that patients achieved rapid responses with daratumumab. Further analysis showed that those who responded well after the first dose were more likely to achieve a complete or near complete response and have better outcomes.
In other talks, data from a Phase I trial of the novel drug CAEL-101 also showed promised. CAEL-101 is one of a new generation of drugs which works by breaking down the amyloid deposits. When given to AL amyloidosis patients who had already received plasma cell-directed treatment but had persistent organ dysfunction, rapid, early and sustained improvements in organ function were observed. Plans were underway to further investigate CAEL-101 in more patients.
Vaishali Sanchorawala, Director of the Amyloidosis Center at BUSM said, “We’re extremely grateful that amyloidosis was featured at ASH to raise awareness among fellow clinicians.”
Amyloidosis Research Consortium. (2017, Dec 14). Highlights from ASH 2017: Research Update [Blog post]. Retrieved from http://www.arci.org/blog-1/2017/12/14/highlights-from-ash-2017-research-update