Doctors of the University of Illinois Hospital & Health Sciences System have successfully conducted a stem cell transplant to cure a rare blood disorder, named congenital dyserythropoietic anemia (CDA). It’ a rare condition, in which the body does not create enough red blood cells, which leads to organ damage and even early death.

This is the story of David Levy, who started experiencing the unbearable pain of CDA when he was 24. The pain was so severe that he had to leave his studies. He stated, “I spent the following years doing nothing – no work, no school, no social contact -because all I could focus on was managing my pain and getting my health back on track.”[1]

David Levy used to receive regular blood transfusions for more than 30 years, so that his tissues and organs keep receiving enough oxygen. When he was 32, he lost his spleen and developed an enlarged lever. As the results of regular blood transfusions, he was suffering from other illnesses such as fatigue, palpitations, and iron poisoning. At last, a desperate David wrote to Dr. Damiano Rondelli, the Michael Reese Professor of Hematology at the University of Illinois at Chicago.

He recollected, “It was bad. I had been through enough pain. I was angry and depressed, and I wanted a cure. That’s why I started emailing Dr. Rondelli.”[2]

Dr. Rondelli stated that many institutions had rejected the possibilities of stem cell transplant on Levy just because of his physical condition was not stable enough to agree to chemotherapy and radiation. But Rondelli performed transplant on Levy in 2014. He said, “This procedure gives some adults the option of a stem cell transplant which was not previously available.”[3]

He also stated that Levy’s condition created the optimum situation for the transplant. He added, “For many adult patients with a blood disorder, treatment options have been limited because they are often not sick enough to qualify for a risky procedure, or they are too sick to tolerate the toxic drugs used alongside a standard transplant.”[4]

After the surgery, Levy, now 35, is back to his normal life, even though he still feels the pain. He is pursuing his doctorate in psychology and runs group therapies at a behavioral health hospital. He said, “The transplant was hard, and I had some complications, but I am back to normal now. I still have some pain and some lingering issues from the years my condition was not properly managed, but I can be independent now. That is the most important thing to me.”[5]

The specialty of the transplant is, no chemotherapy and radiation were administrated in order to prepare the body for the surgery. The process involves the donor’s cells taking over the patient’s bone marrow slowly to avoid usage of any toxic agents to discard the patient’s cells. Rondelli said, “The protocol can be used even in patients with a long history of disease and some organ damage because of the minimal use of chemotherapy.”[6]

Rondelli believes that the procedure has a great potential to treat severe congenital anemias. He concluded “The use of this transplant protocol may represent a safe therapeutic strategy to treat adult patients with many types of congenital anemias – perhaps the only possible cure.”[7]

The story of David Levy truly evokes a raw of hope for many such patients!

Sources:

[1] First patient cured of rare blood disorder  – https://eurekalert.org/pub_releases/2017-03/uoia-fpc032017.php

[2] First patient cured of rare blood disorder  – https://eurekalert.org/pub_releases/2017-03/uoia-fpc032017.php

[3] First patient cured of rare blood disorder  – https://eurekalert.org/pub_releases/2017-03/uoia-fpc032017.php

[4] First patient cured of rare blood disorder  – https://eurekalert.org/pub_releases/2017-03/uoia-fpc032017.php

[5] First patient cured of rare blood disorder  – https://eurekalert.org/pub_releases/2017-03/uoia-fpc032017.php

[6] First patient cured of rare blood disorder  – https://eurekalert.org/pub_releases/2017-03/uoia-fpc032017.php

[7] First patient cured of rare blood disorder  – https://eurekalert.org/pub_releases/2017-03/uoia-fpc032017.php

The post Stories of Hope – David Levy’s Win Against a Rare Blood Disorder appeared first on Stem Cell Banking Guide and Pregnancy Tips For New Parents.

Stem cells have opened so many gateways to personalized medicine that no one could ever imagine a few years back. Not only the traditional transplants, with stem cells, medical practitioners are now able to treat many rare conditions. Thanks to the researchers and their novel techniques, involving stem cells.

This is a story of Javier Tan, a 9 year old boy, who was suffering from a rare genetic illness, Fanconi anaemia, which led his blood count drop to a significantly low levels. He required a stem-cell transplant; so that his body would be able create new blood cells

He received his first stem cell transplant when he was 7. The surgery was meant to save his life but call it irony of fate that it turned his life into a nightmare. Instead of healing, he contracted a bone infection. His condition was so severe that his doctors anticipated he would lose the limb.

In that scenario, the optimum option was to wait for another donor, which might have taken several months. Instead his doctors administrated another transplant and that too within weeks. When his doctors realized that they would require the donor to be only a 50% and not 100%, his father became the donor, who was earlier rejected.

Human leukocyte antigen (HLA) matching is important in both cord blood and bone marrow transplant. It’s a protein, which is found in cells, used to match a donor’s stem cells with that of the patient’s. However, this time Javier’s doctors went for a different method, called haploidentical transplant. In this process, the T-cells, the immune cells that are responsible for attacking foreign elements in the body were removed. So in Javier’s case, the T-cells could not attack the newly transplanted cells.

After seeing the immense improvement, Javier’s mother is very happy. Mrs Vivian Tan recalled the day when doctors told her that they might need to amputate his son’s leg. And now look at him. He is a healthy student of Primary 3, excelling in English and Chinese. His surgery was done in January and Javier was back in school in May. She said, “I am definitely relieved and thankful that he has his health back.”

Dr Rajat Bhattacharyya, the consultant at the department of paediatric sub-specialities at KK Women’s and Children’s Hospital (KKH) confirmed that the new transplant method is versatile enough to find anyone the suitable donor. However, the technique has been in existence since 2010. The National University Hospital (NUH) successfully conducted the procedure successfully on 38 children. The National University Cancer Institute, Singapore (NCIS) has administrated 8 transplants on adults. KKH doctors have treated 4 kids with this technique since October 2014. 10 adult leukaemia patients have been treated with this method by Singapore General Hospital (SGH) since 2004.

The number of patients undergoing haploidentical transplant is gradually increasing. And why not! Without the procedure, the chances of finding the most suitable donor might take several months or year. The haploidentical transplant offers hope for patients, who suffer from rare conditions and are left with minimum hope to live a healthy life ahead.

The post Novel Stem-Cell Procedure Saves Boy’s Leg appeared first on Stem Cell Banking Guide and Pregnancy Tips For New Parents.