An experimental stem cell treatment has, according to doctors, saved the life of a seven-year old Romanian girl with a deadly bone marrow disease. The treatment, called PLacental eXpanded (PLX) (note: correct spelling) cell therapy, was used as a last resort after two bone marrow transplant operations failed and now the little girl is getting ready to return home from hospital.
JERUSALEM (MAY 9, 2012) (REUTERS) -If it wasn't for the surgical mask, seven-year-old Bianca would seem like any other child playing a game.
Bianca came to the Hadassah Medical Centre in Jerusalem from Romania in July 2011, suffering from severe Aplastic Anemia - a deadly condition where bone marrow produces insufficient cells to replenish cells in the blood. After two bone marrow transplants failed to improve her condition and caused severe infections, doctors believed she had only a few months left to live.
But Professor Reuven Or, Director of Bone Marrow Transplantation Department at Hadassah Medical Centre in Jerusalem, wanted to give her body one last chance to overcome the disease with a previously untried treatment. It's called PLacental eXpanded (PLX) cells therapy, developed by Israel's Pluristem Therapeutics Inc. Cells are extracted from human placenta and processed in a laboratory before being incorporated in the treatment.
As the first procedure on a bone marrow patient in the world, it required a special permit from Israel's health ministry.
According to Professor Or, the treatment on Bianca has been successful. Within ten days of the final round of treatment, he says her her body was able to restart its production of red and white blood cells, as well as blood platelets.
"We see it as a miracle but this miracle is based on a lot of work, a lot of brain input, human brain, and we believe that we have a new tool for the benefit of many patients in the future," said Or.
Pluristem's PLX cells are designed to release a cocktail of therapeutic proteins into the patient's organs, targeting inflammatory and ischemic diseases. Ischemia is a restriction in blood supply to tissues, causing a shortage of oxygen and glucose needed for cellular metabolism - keeping tissue alive. Unlike other medical procedures such as bone marrow implantation, PLX cells are grown in a lab and are an 'off-the-shelf' product that require no tissue matching prior to administration.
In Bianca's case, Pluristem says it appears that the PLX cells worked by stimulating the recovery of the hematopoietic stem cells contained in the second bone marrow transplant that she had received more than two months earlier.
"This treatment is based on biological treatment, it's cell therapy. We use here human cells that are universal, that can be given to patients regardless the typing or matching," said Or.
Zami Aberman, Pluristem's Chairman and Ceo, believes the therapy offers help to patients suffering from a wide range of conditions.
"We have animal studies that demonstrate that our cells can be good to treat patients with Multiple Sclerosis or inflammatory bowel disease. We can inject the cells after injuries in order to improve the recovery of the patient like in hip replacement and so we believe that in the future cells will be used in medicine exactly as blood samples of blood doses are used in conventional treatment," he said.
Pluristem recently received clearance from the U.S. Food and Drug Administration (FDA) to begin a Phase II clinical trial of its product for the treatment of Intermittent Claudication (IC), a vascular disease that causes a narrowing of arteries to the legs.
For Bianca and her mother Simona, who has already lost one child to the same disease, cell therapy was a last resort.
Doctors needed to file for a special permit from Israel's health ministry to perform the unprecedented procedure.
"The treatment I gave to my child was her last chance and really helped her and I recommend everyone to allow their children to have the same chance," she said.
Bianca still requires regular check-ups and is not yet able to return to Romania but with her condition improving, doctors hope she will soon be able to have a normal life, ending almost a year of hospitalisation.