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Fred Hutch researcher Rainer Storb receives $12.9 million to study immunotherapies, gene therapies to improve treatments for sickle-cell anemia, “bubble boy” disease and related conditions

Research to improve treatments for sickle-cell anemia, “bubble boy” disease and related conditions

Seattle – July 7, 2015 – Rainer Storb, M.D., head of the Transplantation Biology Program at Fred Hutchinson Cancer Research Center, has received a $12.9 million grant from the National Heart, Lung, and Blood Institute to improve treatments for patients with inborn diseases of the immune system and red blood cells.

Storb and collaborators will launch a five-year, bench-to-bedside research program that seeks to refine treatments based on blood stem cell transplantation for immune deficiencies, such as the condition commonly known as “bubble-boy disease”, and noncancerous disorders of red blood cells, such as sickle-cell disease. Their goal is to make transplantation safer and more widely available to people facing these diseases.

“Current approaches at cell and gene therapy for lethal noncancerous diseases of the blood and immune systems have inherent toxicities that may affect patients for the rest of their lives,” Storb said. “The targeted therapies proposed under this grant aim to eliminate these toxicities”.

Storb will lead the clinical-research portion of the program together with colleagues Ann Woolfrey, M.D. and Lauri Burroughs, M.D., testing less-toxic approaches that could prevent dangerous side effects in patients receiving transplants of blood stem cells for their diseases. The program’s preclinical research, which will eventually feed into the clinic, will be led by Fred Hutch colleagues Brenda Sandmaier, M.D., and Hans-Peter Kiem, M.D., Ph.D, with support from resource cores led by Oliver Press, M.D., Ph.D., and David Rawlings, M.D.

The research will test numerous cutting-edge methods. For example, one set of experiments will test the use of targeted immune system molecules, called monoclonal antibodies, to shut down harmful immune responses that could cause the patient’s body to reject partially matched transplants. (Partially matched transplants are often the only option for patients of racial or ethnic minorities who cannot find a fully matched donor, but they come with a risk that they will be rejected or even begin to attack the patient’s body.) In another set of studies, a genetic “safety switch” is inserted into donor immune cells prior to transplant, ensuring they can be turned off if they start to go haywire.

This program builds on almost 35 years of large-scale research funding from NHLBI that Storb, 80, received to develop and improve transplantation of blood stem cells for noncancerous blood diseases.

As one of the founding scientists of Fred Hutch, Storb worked with E. Donnall Thomas, M.D., the Nobel Prize-winning bone marrow transplant pioneer, to develop transplantation as a curative therapy for patients with leukemia and other blood diseases. In the mid-1990s, Storb developed a less-toxic type of transplantation called a “mini-transplant” which greatly expanded the upper age limit for transplant recipients.

40 YEARS OF CURES 1975-2015

At Fred Hutchinson Cancer Research Center, home to three Nobel laureates, interdisciplinary teams of world-renowned scientists seek new and innovative ways to prevent, diagnose and treat cancer, HIV/AIDS and other life-threatening diseases. Fred Hutch’s pioneering work in bone marrow transplantation led to the development of immunotherapy, which harnesses the power of the immune system to treat cancer with minimal side effects. An independent, nonprofit research institute based in Seattle, Fred Hutch houses the nation’s first and largest cancer prevention research program, as well as the clinical coordinating center of the Women’s Health Initiative and the international headquarters of the HIV Vaccine Trials Network. Private contributions are essential for enabling Fred Hutch scientists to explore novel research opportunities that lead to important medical breakthroughs. For more information visit or follow Fred Hutch on FacebookTwitter or YouTube.