In August of this year, a sample of stem cells kept since 2019 in a private custody bank in Argentina was released, for the treatment of cerebral palsy in a 6-year-old boy. NA spoke with the family of the transplanted child and with specialists.
The stem cell transplant was successfully carried out on 6-year-old Juan Antonio, thanks to the stem cells from the umbilical cord of his sister two years younger, stored since 2019 in the Matercell laboratory and recently released for treatment in the United States .
At birth, Juan Antonio suffered from perinatal hypoxia which led to cerebral palsy. A few months later, his mother, Mirna Ricciari, recalls in dialogue with NA that “a neurologist at the Italian Hospital, where she frequently went for her son’s medical follow-up, recommended that, if they were to have a second child, she keep the stem cells from that umbilical cord for a future treatment for Juan.
At that time, more than four years ago, there was no certainty or even intention, Mirna says, of having a second child. Nor could anyone tell him that after saving those future cells, if that finally happened, when they were used they would be compatible with Juan’s body. The unknowns were great, but there was no doubt that they would try every possibility to improve the quality of life for their young son.
“Several years ago, that doctor told us that treatments with stem cells in cerebral palsy were advanced in the United States and we never forgot that”, slides Mirna.
As Iván Chillik, cardiologist and General Manager of MaterCell, explains to NA, the evolution in the use of stem cells is promising: “Currently the probability that a boy or girl uses stem cells is 1 in 2500. 19 years ago , when MaterCell was founded as the first stem cell bank in the country, the probability of use was 1 in 40,000 and it is estimated that, in the next 10 years, the probability of use will increase 20 times due to the aforementioned advances”.
On this occasion, the Argentine stem cell preservation family bank released its 20th cell for Juan Antonio’s treatment at the request of his family. In August, the sample traveled to Duke University, in the United States, to be used in the treatment of Juan Antonio’s cerebral palsy.
“The process of releasing stem cell samples is a milestone for regenerative medicine, which seeks to provide the population with new proposals for health care,” says Chilik.
At the same time, the specialist explained that of the other 19 samples stored and released over these almost two decades, “5 were sent abroad, for autologous or allogeneic treatment of patients who participated in any of the open clinical trials in Duke University, USA. In these trials, the efficacy of the use of umbilical cord blood or mesenchymal stem cells derived from cord tissue in the treatment of autism, both adults and children, cerebral palsy and other pediatric neurological problems was verified with partial results.
“The remaining samples were used in the country in expanded access protocols for the treatment of pathologies such as cleft lip-alveolus-palatine (FLAP), hydrocephalus, agenesis of the leather callosum, osteoarthritis and oncological diseases”, he pointed out.
The decision to save the stem cells
“Two years later, Amparo, my second daughter, arrived and we never forgot that advice: We decided to look for a bank to store those cells and when we investigated, we opted for Matercell because it was the first in the country and the one with the most experience,” Mirna resumes. with the story of those first difficult years. Until they sent the blood samples of their two children to the Favaloro Hospital, they were not sure if they were compatible. The results were good news and so they were able to take the next step.
The trip and treatment in the United States: On August 17 of this year, they successfully performed the stem cell transplant at the Duke University Children’s Hospital in the United States.
“The treatment was outpatient, it is a transfusion that is done on the same day if there are no complications and it lasts about 15 minutes. In the case of Juan Antonio, what cost us the most were the steps prior to the transfusion at the time of finding the vein. Because something common in most children with cerebral palsy is that they have hypertonia, that is, they have different muscle tones, so when they get nervous or angry they become very hypertonic and that also causes the veins to close, that’s why It took longer than expected since they couldn’t put the track on it.
In turn, Mirna said that in the transfusion “she passed some antiallergics to prevent any type of allergy that could be caused by the preservatives in the cord blood”, and assured that the treatment is safe and that it does not usually cause side effects except, although He heard of some children who had allergies, coughs and rashes.
For the family from Corrientes, the period prior to treatment was more difficult and distressing than the days they were actually in the United States. “We had to make sure he didn’t get sick before he traveled because he had to be healthy and fever-free in order to receive the stem cell transplant. I think that was more stressful, added to the uncertainty of traveling with him by plane for the first time to an unknown country, but everything went very well, we are happy that we made the decision to move forward.
-What improvements do you expect to happen after the recent transplant?
-As it is still an experimental treatment, there are results that have not yet been scientifically proven in their entirety, but we do bet that the case of our son marks significant differences with respect to boys in the same condition who do not receive this treatment. We hope that Juan has an improvement in the motor part and in the social and communication part. These are two aspects in which improvements have already been seen in other children treated with stem cells and in which they would be of vital help in the case of our son. That he manages to sit alone or walk or that he manages to speak more fluently since today he does it in a very restricted way. In addition, we will continue accompanying his evolution with all the therapies that he performs since he was 6 months old.
In dialogue with the specialist Iván Chilik, NA consulted about the particularities of stem cells:
-Stem cells are defined through two of their properties. The first is that they are capable of self-renewal, that is, they can divide and generate more stem cells of the same type. The second is that they can mature or differentiate into specialized cells capable of carrying out specific functions, such as skin, muscle or blood.
Likewise, stem cells have two distinctive characteristics: they are unspecialized cells that renew themselves unlimitedly; and that, under certain physiological or experimental conditions, they can be induced to become cells with special functions. For example, cardiac muscle cells or pancreatic cells for insulin production. The umbilical cord of a baby contains a large number of these cells in a virgin state, which is why their collection at the time of birth is a unique and important opportunity to take advantage of. Umbilical cord blood and tissue are easy to collect, store, and readily available for transplantation.
-Why is it said that stem cells could be the future of regenerative medicine?
-Because it is the branch of science that applies stem cells for different medical treatments throughout the world with significant advances in the last twenty years.
Since 2003 we have specialized in the processing and cryopreservation of hematopoietic cells from the umbilical cord, later in the mesenchymal cells of the umbilical cord tissue. The former have the ability to differentiate into red blood cells, platelets and white blood cells and have demonstrated clinical utility in 85 diseases (the majority onco-hematological) as a source for a bone marrow transplant, since being from the person (autologous) does not is at risk of rejection. Meanwhile, mesenchymal cells act in the regeneration or repair of damaged tissues and replace cells that die routinely.
There is no doubt that treatment with stem cells from the umbilical cord constitutes the present of regenerative medicine. The most exciting thing is all the research that is currently being carried out worldwide with cord stem cells: Today there are more than 1,300 research centers that are analyzing potential uses with stem cells, which reflects the enormous interest of the scientific community on the subject.
These investigations cover very recent and novel topics such as 3D organ printing to investigations that have been conducted for more than 10 years with evidence built from more than 5,000 patients, such as stem cell investigations for the treatment of autism and cerebral palsy, conducted by Dr. Joanne Kurtzberg of Duke University. All the works are registered and described in www.clinicaltrials.gov, which also includes who leads them, objectives, progress status and publication.
Public umbilical cord guard bench
There is also the possibility of saving cord blood in a public bank for free and for altruistic purposes. In this case, the stem cells in custody will be donated for cases that require it. As when blood is donated to a public bank, stem cells from the umbilical cord can be donated in the same way.
In the country, there is the possibility of doing so at the Regional Center for Hemotherapy – Public Umbilical Cord Blood Bank of National Reference of the Hospital Dr. JP. Garrahan, where the cord blood is stored and is available for any patient who needs it in the future in Argentina or anywhere in the world.
The public bank is authorized by the INCUCAI and at the same time is accredited and approved by international reference entities such as Advancing Transfusion for Cellular Therapies Worldwide, Bone Marrow Donor Worldwide and the National Marrow Donor Program of the USA. (NA)