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MACC FUND Volume 20 No. 1 Spring 2003 TODAY www.maccfund.org A PUBLICATION OF MIDWEST ATHLETES AGAINST CHILDHOOD CANCER, INC. ‘In our ignorance lies our greatest hope’ Dr. Montgomery leads research on blood coagulation disorders obert Montgomery, M.D., Professor University of Colorado, he went on to a described earlier, such individuals are at and Vice Chair for Research at the Research Fellowship at the Scripps risk of postoperative hemorrhage and RMedical College of Wisconsin’s Research Institute in La Jolla, California, there is even a common childhood tumor, Department of Pediatrics, isn’t exactly sure and it was here that he began his research Wilm’s tumor, that have been shown to where or from whom this quote came on VWD. have acquired VWD until the tumor is from, but he is a believer in the message it successfully treated. sends. "If we knew everything and things were bad, there would be no hope," he "For many patients with tumors, leukemia, says. "Because there are things we don’t or who have had recent bone marrow know, we have an opportunity to make transplants, they will have problems things better." associated with blood clotting – both too little clotting or sometimes too much," he Dr. Montgomery adheres to that said. "There are also situations during philosophy in his work and approach in chemotherapy, for example, when they his specialty of blood coagulation lose many of the precursor cells in the disorders. His specific research is directed bone marrow and they don’t make at a protein known as von Willebrand platelets. If there aren’t any platelets, then Factor (VWF). When this protein is there is nothing for the VWF to work with deficient or abnormal, the disease is called to assist with the control of hemorrhage at von Willebrand Disease (VWD). a site of blood vessel injury. People with VWD may have a normal "VWF isn’t necessarily key to blood clotting system – and a normal understanding cancer or leukemia, but as number of platelets – but when a blood we treat with more aggressive therapies, it vessel is damaged, the platelets don’t means we come face-to-face with more know that they should stop at that injury secondary problems, such as bleeding and clotting." site. This causes a variety of problems, Dr. Robert Montgomery including bruising, or nosebleeds, or Dr. Montgomery has three grants that heavy menstrual periods. Dr. Montgomery came to Milwaukee in support his work on VWF. Dr. Montgomery has been studying this 1980 because of the Midwest Children’s 1) How VWF is synthesized and stored in particular blood disorder for more than a Cancer Center and The Blood Center of the cells of our blood vessels as well as quarter of a century. He first developed Southeastern Wisconsin under the our platelets; his interest in pediatrics as a medical respective leadership of Dr. Bruce Camitta 2) How VWF helps to regulate Factor 8 student at the University of Pittsburgh and Dr. Richard Aster. He helped (FVIII) (a blood coagulation factor); establish a hemostasis reference laboratory from where he graduated in 1969. 3) How the receptors on platelets bind that served the joint needs of the Blood Following his pediatric internship at VWF. Children’s Hospital in Philadelphia and Center and Children’s Hospital and also made available specialized hemostasis the University of Pennsylvania, he worked Dr. Montgomery serves on many national testing that now serves many of the in the Indian Health Service on the Navajo and international committees. He is hospitals in Milwaukee as well as Reservation in Arizona. Besides his Chairman of the VWF Subcommittee of hospitals throughout the United States. clinical work with Navajo children, he also the International Society of Thrombosis did his first research on a viral disease From the beginning of his career, Dr. and Haemostasis, is Chairman-Elect of the called cytomegalovirus – a disease that is Montgomery has focused on VWD, which International Scientific and also a problem for cancer and transplant is the most common hereditary bleeding Standardization Committee, Chairman of patients. He then did his pediatric disorder and affects 1% to 2% of the total the Hemostasis Subcommittee of the residency at Johns Hopkins University population. "Most people who have it American Society of Hematology, and and then decided to further his study on don’t know they have it," Dr. Montgomery serves on the Hematology Study Section hematology. Following his training as a said. Besides the symptoms of VWD of the National Institutes of Health. Fellow in pediatric hematology at the Continued on page 2 Dr. Montgomery, front page "We’re not just interested in the diagnosis of VWD, we want Dear Friend: laboratories throughout the world to offer comparable tests and to elucidate the DNA mechanisms of VWD." The calendar tells us spring has sprung, He is also working with the National Institutes of Health to but the unpredictability of Mother create the National Center for The Molecular (DNA) Diagnosis Nature often spins a different tale. of VWD with this center being located here in Milwaukee. However, one thing for certain is that spring is the season of hope. And hope "This program is only possible because of the close collaboration between the Medical College of Wisconsin, Children’s Hospital is what the MACC Fund offers and truly of Wisconsin, and The Blood Center," said Dr. Montgomery, who what we’re all about. serves as Director of Research at Children’s Hospital and as Senior Investigator of The Blood Center. The MACC Fund is the "Hope Through Research." You have heard it many times College’s largest donor. "These three organizations, working with the MACC Fund. Your support in 2002 led to $3.6 synergistically, really enable the creation of something better than any one organization could have done on their own." million in research awards for the MACC Fund-supported investigators at the Midwest Children’s Cancer Center. Dr. Montgomery stresses the need to educate clinicians about These researchers were also awarded 10 National Institute VWD. "It’s important for clinicians to not only understand of Health grants. This certainly brings the promise of a bleeding disorders, but, in addition, to have laboratories that are capable of making the correct diagnosis. cure that much closer. It has been your loyal and generous support of the MACC Fund that has made this possible. So "It is also important for doctors taking care of children with many people from so many different walks of life have complex diseases including tumors, leukemia, or even other come together to help children with cancer. It is both complex problems, that may be associated with hemorrhage (too little clotting) or thrombosis (too much clotting). humbling and exciting to witness. "What we have in the MACC Fund Research Center– in the The MACC Fund and everyone associated with our hematology, oncology, and transplant groups -- is a group of organization are very proud of the work being done and researchers and physicians who are trying to identify what complex clinical disorders for which we don’t have easy the progress being made. Your charitable dollars help fund answers today and then to pursue answering questions in the our primary beneficiary, the Midwest Children’s Cancer laboratory, so that we can offer treatment or better treatment Center of the Medical College of Wisconsin, as well as tomorrow. The University of Wisconsin’s Comprehensive Cancer "Many times questions that one investigator is pursuing in his or Center. Both institutions are leaders in the field of finding a her individual laboratory may offer opportunities to others cure for childhood cancer. working in an entirely different field of investigation. We need to be able to provide state-of-the-art treatment today but also We have many success stories. Just think of all of the pursue the hope for the future of things being even better. children who had cancer, beat it, and who are now grown "While I may be studying ways to put a better protein into a cell up and preparing to graduate or get married this spring. to correct a bleeding disorder, a similar approach might be used to introduce therapy that might cause a tumor cell to be We are always mindful of those who have left us before destroyed." they could achieve these milestones. Yet we persevere and The same cells that Dr. Jim Casper and Dr. Dave Margolis isolate with your help we march together toward our goal of "Life to perform bone marrow transplants for leukemia patients might for All Kids." be used in the future as a means of gene therapy for children with bleeding or clotting disorders. "If we want to have the best We wish you and yours all of the best as the cool breezes of physicians providing the best possible care for our children, they also need to be working in an environment that has phenomenal spring give way to warm summer days. On behalf of all the curiosity so that our treatment tomorrow can even be better than children with cancer, thank you. Thank you for giving the our treatment is today. children and their families a chance to enjoy the change of seasons together…and for giving them hope. “The MACC Fund has have been responsible for bringing to Milwaukee, physicians who have a lot of curiosity and also a lot of compassion." maccfund.org Jon McGlocklin, President The MACC Fund TODAY is the official newsletter of the Midwest Athletes Against Childhood Cancer, Inc. The MACC Fund supports pediatric cancer research primarily at the Midwest Children’s Cancer Center, with research conducted in the MACC Fund Research Center of the Medical College of Wisconsin.

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