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Tufts Medical Center sponsors national ‘RAIN’ trial for rare blood cancer


Myeloma and amyloidosis expert Ray Comenzo talks with a patient in Boston at Tufts Medical Center.BOSTON (July 17) – The John Conant Davis Myeloma and Amyloid Program in the Division of Hematology/Oncology at Tufts Medical Center announced that it will begin recruiting patients with a rare blood cancer, called light-chain or AL amyloidosis, for a clinical trial using a new monoclonal antibody, NEOD001, produced by Prothena Biosciences. 

AL amyloidosis, which afflicts 3,000 patients a year in the U.S., causes destruction of key organs in the body, such as the heart and kidneys. The ‘RAIN’ trial – which stands for Renal AL-amyloid Involvement and NEOD001 (NCT03168906) - is aimed at patients with kidney damage who have a high likelihood of needing dialysis in several years. Tufts MC is the sponsor of the multi-center trial, a center of excellence for the care of AL patients, and a key member of the collaborative network of the Amyloidosis Research Consortium (ARC).

“The RAIN trial shows how dedicated physicians committed to clinical and translational research can capitalize on the outstanding infrastructure available at Tufts MC to perform important multi-center studies that aim to greatly benefit patients,” said Andrew M. Evens, DO, MSc, Director of the Tufts Cancer Center and Chief of the Division of Hematology/Oncology at Tufts Medical Center.

The RAIN trial is the brainchild of AL and myeloma expert Raymond Comenzo, MD, Director of the Blood Bank and Stem Cell Processing Laboratory and Director of the John C. Davis Myeloma and Amyloid Program at Tufts MC.

“Our sponsorship of the RAIN trial demonstrates the commitment of the Medical Center to clinical and translational research that can change the practice of medicine,” said Dr. Comenzo. “There are AL patients across the country who depend on efforts such as RAIN to provide them with hope and the possibility of long-term disease control.”

Medical Oncologist Cindy Varga, MD, is the Principal Investigator for RAIN at Tufts Medical Center and is also the lead translational researcher collaborating with the Clinical Director of the ARC, Kristen Hsu, on a major grant submission that will support intensive histologic and molecular studies of kidney biopsies from AL patients on RAIN.

“In this trial, patients will be randomized to receive the anti-amyloid antibody NEOD001 or placebo, and will be followed by their doctors to see if kidney function improves, worsens or stabilizes,” said Dr. Varga. “The translational research we will conduct will help us understand why some patients respond to the antibody while others don’t, and why some patients progress to dialysis in months and others remain stable for years.”

“The future of clinical and translational research at Tufts MC is embodied in this trial,” said Errol Norwitz, MD, PhD, Chief Scientific Officer and Chairman of Obstetrics and Gynecology at Tufts Medical Center. “It is a collaborative process involving our physician investigators, Tufts MC Research Administration, and the Neely Center for Clinical Cancer Research in the Tufts Cancer Center. In RAIN, the Neely Center is demonstrating its capability to direct and oversee a multi-center trial as an academic clinical research organization.” 

Key collaborating renal physician-investigators on the grant submission for RAIN ancillary studies are Dr. Agnes Fogo (Vanderbilt) and Dr. Matthias Kretzler (University of Michigan). Dr. Varga is joined at Tufts MC by Lesley Inker, MD, Director of the Kidney and Blood Pressure Center, who will be overseeing novel renal clearance studies on AL patients in the Tufts Clinical and Translational Research Center (CTRC) and other RAIN- participating centers.

The other centers involved in the trial include the three centers in the Mayo Clinic Health System (Minnesota, Arizona, Florida), Memorial Sloan-Kettering Cancer Center (New York), University of Pennsylvania, Karmanos Cancer Institute (Michigan), Vanderbilt (Tennessee) and University of California at San Francisco. 

Patients and families interested in learning more about RAIN can telephone the clinical project manager, Mr. George Mensing, at 617-636-2683, or Dr. Cindy Varga at 617-636-8148.        


About Tufts Medical Center and Floating Hospital for Children

Tufts Medical Center is an exceptional, not-for-profit, 415-bed academic medical center that is home to both a full-service hospital for adults and Floating Hospital for Children. Conveniently located in downtown Boston, the Medical Center is the principal teaching hospital for Tufts University School of Medicine. Floating Hospital for Children is the full-service children's hospital of Tufts Medical Center and the principal pediatric teaching hospital of Tufts University School of Medicine. Tufts Medical Center is affiliated with the New England Quality Care Alliance, a network of more than 1,800 physicians throughout Eastern Massachusetts. For more information, please visit

Media Contact: Jeremy Lechan
(617) 636-0104