Physicians at the University of Illinois Hospital & Health Sciences System have cured 12 adult patients of sickle cell disease using a unique procedure for stem cell transplantation from healthy, tissue-matched siblings.
The transplants were the first to be performed outside of the National Institutes of Health campus in Maryland, where the procedure was developed. Physicians there have treated 30 patients, with an 87 percent success rate. The results of the phase I/II clinical trial at UI Health, in which 92 percent of treated patients were cured, are published online in the journal Biology of Blood & Marrow Transplantation.
The new technique eliminates the need for chemotherapy to prepare the patient to receive the transplanted cells and offers the prospect of cure for tens of thousands of adults suffering from sickle cell disease.
About 90 percent of the approximately 450 patients who have received stem cell transplants for sickle cell disease have been children. Chemotherapy has been considered too risky for adult patients, who are often more weakened than children by the disease.
“Adults with sickle cell disease are now living on average until about age 50 with blood transfusions and drugs to help with pain crises, but their quality of life can be very low,” says Dr. Damiano Rondelli, chief of hematology/oncology and director of the blood and marrow transplant program at UI Health, and corresponding author on the paper.
“Now, with this chemotherapy-free transplant, we are curing adults with sickle cell disease, and we see that their quality of life improves vastly within just one month of the transplant,” said Rondelli, who is also the Michael Reese Professor of Hematology in the UIC College of Medicine. “They are able to go back to school, go back to work, and can experience life without pain.”
Sickle cell disease is inherited. It primarily affects people of African descent, including about one in every 500 African Americans born in the U.S. The defect causes the oxygen-carrying red blood cells to be crescent shaped, like a sickle. The misshapen cells deliver less oxygen to the body’s tissues, causing severe pain and eventually stroke or organ damage.
Doctors have known for some time that bone marrow transplantation from a healthy donor can cure sickle cell disease. But few adults were transplanted because high-dose chemotherapy was needed to kill off the patients’ own blood-forming cells — and their entire immune system, to prevent rejection of the transplanted cells, leaving patients open to infection.
In the new procedure, patients receive immunosuppressive drugs just before the transplant, along with a very low dose of total body irradiation — a treatment much less harsh and with fewer potentially serious side effects than chemotherapy.
Next, donor cells from a healthy and tissue-matched sibling are transfused into the patient. Stem cells from the donor produce healthy new blood cells in the patient, eventually in sufficient quantity to eliminate symptoms. In many cases, sickle cells can no longer be detected. Patients must continue to take immunosuppressant drugs for at least a year.
In the reported trial, the researchers transplanted 13 patients, 17 to 40 years of age, with a stem cell preparation from the blood of a tissue-matched sibling. Healthy sibling donor-candidates and patients were tested for human leukocyte antigen, a set of markers found on cells in the body. Ten of these HLA markers must match between the donor and the recipient for the transplant to have the best chance of evading rejection.
In a further advance of the NIH procedure, physicians at UI Health successfully transplanted two patients with cells from siblings who matched for HLA but had a different blood type.
In all 13 patients, the transplanted cells successfully took up residence in the marrow and produced healthy red blood cells. One patient who failed to follow the post-transplant therapy regimen reverted to the original sickle cell condition.
None of the patients experienced graft-versus-host disease, a condition where immune cells originating from the donor attack the recipient’s body.
One year after transplantation, the 12 successfully transplanted patients had normal hemoglobin concentrations in their blood and better cardiopulmonary function. They reported less pain and improved health and vitality.
Four of the patients were able to stop post-transplantation immunotherapy without transplant rejection or other complications.
Read more: UI Health validates cure for sickle cell in adults
The Latest on: Sickle cell disease
[google_news title=”” keyword=”Sickle cell disease” num_posts=”10″ blurb_length=”0″ show_thumb=”left”]
via Google News
The Latest on: Sickle cell disease
- Exa-Cel Eliminates Vaso-Occlusive Crises in Most Sickle Cell Patientson April 25, 2024 at 11:30 pm
For almost all patients with sickle cell disease, treatment with exagamglogene autotemcel (exa-cel) eliminates vaso-occlusive crises, according to a study published in the April 24 issue of the New ...
- Walgreens expands specialty pharmacy with unit dedicated to cell and gene therapieson April 25, 2024 at 6:38 am
Walgreens Boots Alliance (WBA) expands specialty pharmacy services with dedicated unit for gene and cell therapy. Read more here.
- Researchers publish final results of key clinical trial for gene therapy for sickle cell diseaseon April 25, 2024 at 6:14 am
In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY ...
- CT Up Close: Students with sickle cell disease share key takeaways from visit to NASA space centeron April 25, 2024 at 5:06 am
Last month, about 20 local students with sickle cell disease traveled to NASA’s Goddard Space Center in Maryland to learn more about STEM careers and what they can ...
- CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patientson April 24, 2024 at 11:33 pm
In a landmark study, an international consortium led by researchers at (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the ...
- 10-year-old survivor uses literature to fight sickle cellon April 24, 2024 at 2:16 pm
Cai Stoudemir is a 10-year-old Author and Sickle Cell Survivor from Milwaukee. She wrote this book, Cai hopes to educate kids and adults about the disease.
- 2025 Cell & Gene Therapy Reimbursement Outlookon April 24, 2024 at 1:00 pm
CMS proposed Medicare’s CGT reimbursement policy matches Medicaid’s sickle cell disease policy, forming a unified government approach.
- Alteration of brain network condition could predict painful vaso-occlusive crisis in patients with sickle cell diseaseon April 24, 2024 at 12:16 pm
A new study led by Indiana University School of Medicine researchers has found that a brain network condition called "explosive synchronization" could be the cause of extreme pain crises in people ...
- Tulsa Woman Organizes Event To Support Caregivers, People Living With Sickle Cell Diseaseon April 24, 2024 at 6:50 am
Velvet Brown Watts gave birth to her son who was diagnosed with Sickle Cell Disease. She said it’s been a challenging journey, but now she is working to raise awareness of this blood disorder within ...
- Young New Orleans sickle cell patient is leaving pain behind after a bone marrow transplanton April 22, 2024 at 5:44 am
At only 12 years old, Kelsey Farris already has endured periods of searing pain, endless rounds of medical tests and several lengthy stays at Children’s Hospital in New Orleans. But ...
via Bing News