Sickle Cell Disease Research Among Top 10 Clinical Research Achievements of 2012
Pioneering research led by Johns Hopkins scientists on the use of partially matched bone marrow transplants to wipe out sickle cell disease has been selected as one of the Top 10 Clinical Research Achievements of 2012 by the Clinical Research Forum. The success of a preliminary clinical trial of the so-called haploidentical transplants has the potential to bring curative transplants to a majority of sickle cell patients who need them, eliminating painful and debilitating symptoms and the need for a lifetime of pain medications and blood transfusions.
The NHLBI and Sickle Cell Disease
The NHLBI is supporting research on more and better treatments to ease the burden of sickle cell disease on those affected. Trials using new bone marrow and stem-cell transplant procedures have cured a small number of some adults with sickle cell disease. More research is needed to understand possible health implications of sickle cell trait.
Could an Old Antidepressant Treat Sickle Cell Disease?
An antidepressant drug used since the 1960s may also hold promise for treating sickle cell disease, according to a surprising new finding made in mice and human red blood cells by a team from the University of Michigan Medical School.
What is a clinical trial?
Clinical trials help researchers assess the effectiveness of various types of treatments, be they new medications, medical devices or physical therapies – all in a carefully controlled setting. An important goal of the Children’s Sickle Cell Foundation is to help families find effective treatments for SCD. We hope to achieve this goal by connecting families who are interested in participating in clinical trials with researchers who are recruiting patients.
Why are clinical trials important?
Clinical research trials, typically just referred to as clinical trials, play a critical role in the advancement of medical knowledge.
The prospect of participating in a clinical treatment trial that has the potential to improve function, prognosis, and quality of life can give families and individuals living with SCD a lot of hope. But with that hope may come an emotional rollercoaster that brings elation and anxiety as families learn about study eligibility and what is involved in participation. A family’s best approach is education about the research goals, eligibility, process, and pros and cons of study participation. Such information comes from resources like CSCF, Inc. and from open communications with the study team.
Much of today’s medical care is based on the results from past clinical studies. Clinical trials help researchers assess the effectiveness of various types of treatments, be they new medications, medical devices or physical therapies – all in a carefully controlled setting.
In addition to helping others by contributing to the advancement of medical knowledge, participants in clinical trials can play a more active role in their own health care. They can also gain access to new treatments before they are widely available.
By conducting clinical trials we can:
Learn how a new therapeutic medicine or treatment works in humans
Learn which treatment strategies work well and which do not
Discover new and better ways to prevent, diagnose, and treat complications of sickle cell disease
Get closer to discovering a cure for sickle cell disease and related hemoglobinopathies that will work for a majority of patients
You don’t have to do it alone.
We offer tons of great programs and support.
Children’s Sickle Cell Foundation, Inc. knows how hard it can be to live with Sickle Cell. We offer a variety of family and individual programs and services to help with everyday needs and to provide support.