Medical College of Wisconsin
Amyloidosis Research Progress Report
Camille A. Lonstorf Trust
Hazelyn and Harrold McComas Charitable Trust
The Medical College of Wisconsin is grateful to the Camille A. Lonstorf Trust and the Hazelyn and Harrold McComas Charitable Trust for support of research about amyloidosis, a rare disease that occurs when abnormal proteins called amyloid build up in the body.
These donations were used to support amyloidosis research by Parameswaran Hari, MD, MRCP, MS, the Armand Quick – William Stapp Professor of Hematology, and Director, Adult Blood and Marrow Transplant Program, and Anita D’Souza, MD, MS, an assistant professor at MCW who is focusing on amyloidosis in her research and patient care. Three projects were identified and initiated for support from these two sources. This report has been developed to update Mr. Paul McComas MA, trustee of the Camille A. Lonstorf Charitable Trust, on progress made on these three projects during 2018.
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 and Amyloid Research Consortium
Patient-oriented outcomes, specifically disability and health related quality of life, are extremely important 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 continues to grow. As of November 2018, 30 patients are enrolled at Froedtert, and 21 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:
- Patient-Reported Outcomes in Light Chain Amyloidosis. Oral presentation of poster board at 2018 PROMIS Outcomes Meeting Symposium held in Dublin, Ireland.
- 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 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 compares favorably to prior reports. Plans are underway to expand the study, as these initial findings warrant a randomized, multicenter study.
These results will be presented on December 1 at the 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.
Froedtert & the Medical College of Wisconsin welcomes the opportunity to develop a private reception when amyloidosis patients could meet Michael York, critically acclaimed film and stage actor and Paul McComas, MA, a Milwaukee native and award-winning author and performer. Copies of The Readiness Is All, Mr. York’s book about his experience with amyloidosis, would be distributed to guests. We are happy to discuss hosting this event at a time convenient for Mr. York and Mr. McComas.
Froedtert & the Medical College of Wisconsin is grateful for philanthropic support from the Camille A. Lonstorf Trust and the Hazelyn and Harrold McComas Charitable Trust to advance research and develop new treatment options for amyloidosis patients. At this time, we are requesting continued support from both trusts 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 past generous support and for your thoughtful consideration to continue to partner with us in this important research.