The NMDP and other organizations are working together on a clinical trial for children (ages 3-16) with SCD. A study like this has never been done before and it aims to determine the effectiveness of unrelated marrow and cord blood transplants in children.
The trial is facilitated by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) and is funded by multiple organizations, including the National Heart, Lung, and Blood Institute; National Cancer Institute; National Marrow Donor Program; Sickle Cell Disease Clinical Research Network (SCD CRN) and the National Center on Minority Health and Health Disparities. Potential transplant candidates and their families can visit http://www.marrow.org/ for more information. Referring physicians can visit http://www.bmtctn.net/.
Currently, marrow or blood stem cell transplantation is the only potential cure for severe SCD. Most people with SCD who have undergone a transplant have received stem cells from family members, usually siblings. In this nationwide, multicenter clinical trial, researchers will evaluate the role of unrelated donor transplants in treating severe SCD and the effectiveness of a less-intensive regimen of chemotherapy to prepare patients for transplant.
The study is looking for 45 children with SCD from around the country. There are 20 different trial sites in the US participating.
Please click HERE for more info, and have your doctor contact the study if you are interested in joining. The more participants, the better chance we have for a cure.
Contact Antoine LaFromboise for more info.
612-455-1724
alafromboise@psbpr.com
2 comments:
I read about this last year. What I read did not make it sound as if they were still in trial phase. I wondered why this information wasn't more widely available. Thank you for spreading the word. It would be nice to someday read about the curable disease, SCD. When I was young (1984), my biology book indicated that no one with SCD lived past the age of 40. It scared me and made that birthday very special for me. I am now 43 and doing well. I later found out that I have sickle beta thalessemia minor but get sick quite often now that I have passed the 40 mark.
Last year I was almost fired for being out for 6 weeks. I also developed rheumatoid arthritis and problems with my lungs and suffered a lot last year. It would be nice if SC was considered a disability. It would be easier on those of us who work.
Thanks for your insight. It helped me feel better last year and I enjoy reading the information you provide.
Dear AnnKura, Congratulations on 43. May you have many more 43 years to come. Thank heart, in the last 3 years, the pace of genetic research to find a cure for this 100 year old disease (at least that is what the Western literature will have us believe) has picked up pace. Recently, Dr Krishnamurti published successful bone marrow transplant in 6 of 7 sickle cell patients with reduced intensity chemotherapy (See http://www.http://livingwithsicklegenes.com/blogs/posts/DrDon). This and the trial you are talking about should make this cure more readily available. Additionally, a group of scientist at Yale has shown that they can (theoretically) take you bone marrow cells, correct the sickle cell gene and put it back in you. It is not too long now before a universal cure will be available. Meanwhile, take good care of yourself. One reason why people with sickle cell may get worse with age is because over the years they would have accumulated many insults that may have damaged internal organs (many manifest as painful crises or no pain at all), that now shows up as one gets older. Work with your healthcare provider to make sure you are in perfect health and await the cure. We have suffered too long!
Stay blessed!
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