Transplant Community & Sickle Cell Disease Advocates Urge Congress to Advance Policies Supporting Sickle Cell Disease Research and Treatment
Lawmakers, patients, physicians and advocates gather on Capitol Hill to highlight advancements in sickle cell disease care and treatment options including transplant and genome editing.
(WASHINGTON, June 7, 2017) — The American Society for Blood and Marrow Transplantation (ASBMT), American Society of Hematology (ASH), American Society for Gene & Cell Therapy, National Marrow Donor Program®/Be The Match®, Sickle Cell Disease Association of America, Inc. and Pediatric Hospital Sickle Cell Collaborative — in partnership with The House Research & Development Caucus, the Congressional Caucus to Cure Blood Cancers and Other Blood Disorders and the Congressional Sickle Cell Disease Caucus — today hosted a briefing on Capitol Hill to highlight advancements in sickle cell disease (SCD) research and treatment options such as bone marrow transplantation, the one proven cure for SCD, and genome editing, which shows promise for a future cure.
Congresswoman Doris Matsui (CA-6), Congressman Bill Foster, PhD (IL-11) and Congressman Danny Davis (IL-7) took part in today’s event.
“Too often, people with devastating blood disorders like sickle cell disease face significant barriers to treatment. We need to be opening up more options for patients and making the federal investments in research that will accelerate the development of cures. As a co-founder of the Caucus to Cure Blood Cancers and Other Blood Disorders, I will continue to spread awareness about the challenges facing the rare and chronic disease communities so that we can give more families hope for the future,” said Congresswoman Matsui.
“As co-chair of the R&D Caucus, it
was an honor to help bring together these great panelists to help educate
Members of Congress and policymakers about the most recent developments in
sickle cell research. I look forward to continuing to work with researchers and
organizations on this important issue. As soon as technology produces a cure,
we need to fast track it,” said Congressman Foster.
Speakers at today’s briefing spoke about the significant medical discoveries and advances that would not be possible without federal investments in the National Institutes of Health (NIH). NIH funded research has led to improved understanding and treatment of SCD, including the efficacy of transplants in curing SCD and stopping any additional damage to a patient’s organs. Researchers are also exploring genome editing as a tool to repair the gene that causes sickle cell disease, offering another potential cure for the disease.
“Our understanding of basic genetic biology is rapidly translating into promising curative strategies for people with SCD and other inherited genetic diseases,” said ASH President-Elect Alexis Thompson, MD, MPH, of the Ann and Robert H. Lurie Children’s Hospital of Chicago. “While genome editing research is still in early stages, it offers immense promise as a potential cure for SCD. Genome editing, transplant, and other advances in research and treatment give us an enormous opportunity to transform the way we care for people with SCD.”
Bone marrow or umbilical cord blood transplantation is the only curative therapy for SCD patients. Data show that transplant for SCD can lead to improved quality of life. While an increased number of SCD patients have undergone bone marrow or cord blood transplantation in the last decade, experts estimate that less than one percent of the SCD population in the U.S. has received a transplant.
“Patients living with SCD today have a curative treatment path, which until recently, was not available for patients living in chronic pain and facing myriad health challenges. The availability of transplant for this patient population, and the promise of genome editing, are incredibly encouraging improvements for people living with SCD,” said Linda Burns, MD, with the National Marrow Donor Program®/Be The Match®. “While we currently rely on a small, retrospective group of studies to inform SCD treatment, the body of knowledge and treatment strategies continue to grow. Further, it is important to remove barriers to transplant for patients with SCD, especially those patients who rely upon Medicare coverage.”
“I am one of the lucky few SCD patients in the U.S. who has received a bone marrow transplant to cure my SCD. Since my transplant, I am no longer living in constant pain and I am able to live the active, high quality life I always hoped for,” said Constance Benson, a transplant recipient and former SCD patient who spoke at today’s briefing. “I am honored to share my story with lawmakers in Congress today with the hopes it will help more patients access life-changing transplant for SCD.”
“Genome editing approaches hold the promise to cure SCD by rectifying a patient’s own blood cells. Although we have long understood SCD as a ‘molecular disease’, treatment options have remained quite limited and crude. Now we are closer than ever before to developing ‘molecular therapies’ that tackle the root of the problem,” said Daniel Bauer, MD, PhD, of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Harvard Medical School in Boston.
To encourage individuals to register as bone marrow donors, which is critical to expanding the registry and enabling more patients to receive transplants, Be The Match® hosted a registry donor drive in coordination with their briefing on Capitol Hill.
SCD is a genetic blood disorder that is caused by a change in hemoglobin in red blood cells, which helps carry oxygen to all parts of your body. This results in blockages to the blood flow in small vessels, which can cause severe pain in SCD patients. SCD patients are also at high risk for damage to their lungs, brain, kidneys and other organs.
The American Society of Hematology (ASH) ( www.hematology.org) is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment and prevention of disorders affecting the blood. For more than 50 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, which is available weekly in print and online. In 2016, ASH launched Blood Advances ( www.bloodadvances.org), an online, peer-reviewed open-access journal.
The American Society for Blood and Marrow Transplantation (ASBMT) is a professional society of more than 2,200 physicians, investigators and other health care professionals from over 45 countries. The Society is dedicated to advancing the science and clinical care for patients who require blood and marrow transplants for blood cancers and other deadly diseases. Visit www.asbmt.org for more information.
The American Society of Gene & Cell Therapy is the primary professional membership organization for gene and cell therapy. The Society's members are scientists, physicians, patient advocates and other professionals. ASGCT's mission is to advance knowledge, awareness and education leading to the discovery and clinical application of gene and cell therapies to alleviate human disease. For more information, please visit www.asgct.org. American Society of Hematology
Be The Match
For people with life-threatening blood cancers—like leukemia and lymphoma—or other diseases, a cure exists. Be The Match connects patients with their donor match for a life-saving marrow or umbilical cord blood transplant. People can contribute to the cure as a member of the Be The Match Registry®, a financial contributor or a volunteer. Be The Match provides patients and their families one-on-one support, education and guidance before, during and after the transplant.
Be The Match is operated by the National Marrow Donor Program® (NMDP), a nonprofit organization that matches patients with donors, educates health care professionals and conducts research through its research program, CIBMTR® (Center for International Blood and Marrow Transplant Research®), so more lives can be saved. To learn more about the cure, visit BeTheMatch.org or call 1 (800) MARROW-2.
Sickle Cell Disease Association of America, Inc.
The Sickle Cell Disease Association of America, Inc. is a national nonprofit with the mission to advocate for and enhance our membership's ability to improve the quality of health, life and services for individuals, families and communities affected by sickle cell disease and related conditions, while promoting the search for a cure for all people in the world with sickle cell disease. SCDAA serves as the nation’s only nonprofit organization working full-time on a national level to resolve issues surrounding sickle cell disease and trait.
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American Society of Hematology