Medical College of Wisconsin
Amyloidosis Research Progress Report
Camille A. Lonstorf Trust
Hazelyn and Harrold J. McComas Charitable Trust

In Memory of Sheldon I. Miller, MD

Cancer-Center2The Medical College of Wisconsin is grateful to the Camille A. Lonstorf Trust and the Hazelyn and Harrold J. McComas Charitable Trust for support of research about amyloidosis, a rare disease that occurs when abnormal proteins called amyloid build up in the body. Donations were made at the direction of Paul McComas, trustee of the Lonstorf Trust, who developed the Renewal Initiative in 2014 to create awareness of this devastating disease.

Donations support amyloidosis research by Parameswaran Hari, MD, MRCP the Armand Quick – William Stapp Professor of Hematology, and Chief, Division of Hematology/Oncology, and Anita D’Souza, MD, MS, Associate Professor of Hematology/Oncology at MCW who is focusing on amyloidosis in her research and patient care. Three projects were initially identified from this support. This report provides an update on recent progress on these projects.

Project 1: Cardiac Amyloid Imaging
This project involves a pilot trial of the use of new nuclear medicine agents active in Alzheimer’s amyloid plaque to image AL amyloid. The clinical trial protocol for this study recently was approved by the Institutional Review Board. Enrollment of patients will begin soon. Christopher Mueller, MD, FACC, an assistant professor of medicine in the division of cardiovascular medicine at MCW, will serve as the Principal Investigator on the project.

Project 2: Patient-Centered Outcomes in AL Amyloidosis
Patient-oriented outcomes, specifically disability and health-related quality of life, are vital but poorly studied issues in AL amyloidosis. Froedtert & the Medical College of Wisconsin is prospectively collecting these outcomes at multiple time points in the first year after the diagnosis of AL disease. Delineating these important effects of AL amyloidosis has immediate clinical and research implications.

Enrollment in this study was recently completed. As of April 2019, 35 patients are enrolled at Froedtert, and 26 patients are enrolled at the Mayo Clinic, under the direction of MCW.

To share this information with other researchers and invite their input and collaboration, Dr. Anita D’Souza has presented this research at several key conferences, including:

  1. Patient-Reported Outcomes in Light Chain Amyloidosis. Oral presentation, 2018 PROMIS Outcomes Meeting Symposium held in Dublin, Ireland.
  2. Patient-Reported Outcomes in the Amyloid Clinic: A Clinician’s Perspective.
    Symposium presented at the 25th Annual Conference of the International Society of Quality of Life held in Dublin, Ireland, in October 2018.

Project 3: Elimination of Tissue Amyloid Using Doxycycline
DUAL (Doxycycline to Upgrade response in light chain [AL] amyloidosis) is a prospective phase 2 clinical trial using Doxycycline as a fibril disrupting agent. It has successfully completed enrollment of a total of 31 patients with support from the American Cancer Society pilot research grant, the Camille A. Lonstorf Trust and the Hazelyn and Harrold J. McComas Charitable Trust.

This research showed that in newly diagnosed systemic AL amyloidosis, doxycycline was safe with concurrent chemotherapy. The low one-year early mortality of 20 percent and autologous stem cell transplant rate of 60 percent compared favorably to prior reports. Plans are under way to expand the study, as these initial findings warrant a randomized multicenter study.

These results were presented at the 2019 American Society of Hematology Annual Meeting in San Diego. This society is the world's largest professional society serving both clinicians and scientists who are working to conquer blood diseases.

SheldonMillerMDIn Memoriam
Generous donations to this initiative have been and continue to be made in memory of Sheldon I. Miller, MD, a renowned professor of psychiatry and behavioral sciences. A founding father of the subspecialty of addiction psychiatry and an early identifier of Fetal Alcohol Syndrome, Dr. Miller was an impassioned advocate for mental health care. He continued his work and advocacy late into life, even as the challenges of his amyloidosis mounted. He was a dear friend of Mr. McComas, who is dedicated to increasing awareness about amyloidosis in Dr. Miller’s memory through the Renewal Initiative.

Moving Forward
Froedtert & the Medical College of Wisconsin is grateful for philanthropic support to advance research and develop new treatment options for amyloidosis patients. We welcome your support for this important work. Ultimately, amyloidosis patients and their families will benefit from improved medical care and advances in scientific knowledge.

Thank you for your thoughtful consideration to partner with us in this important research.