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Doctors develop "treatment" for children with a "bubble" disease

However, because of his illness, going out to the streets and in the crowds of people has posed a serious threat to lethal infections, and Hawkins accepted this as a reality Ja & # 39; Ceon – still.

Christine Simpson, like any mother, would want her child, Omarion Jordan, to grow into a healthy boy who could go out and not risk a serious illness.

Now, she says, he can.

If Ceon and Omarion were diagnosed as children with a rare genetic disorder called X-linked Severe Combined Immunodeficiency or SCID-X1.
  Treatment for UCSF last year
Heavy combined immunodeficiency or SCID refers to a group of rare disorders caused by mutations in genes that affect the development and function of important immune defects cells Infants born with SCID may sound healthy, but very susceptible to severe infections – so that the cold can kill them.

Because of Hollywood's passion for this disorder, many people may think of SCID as a disease noted in 1976 by John Travolta's "The Boy in a Plastic Bubble" movie, or the All-All, 2017 film, featuring Amanda Stenberg.

However, some common misconceptions about the condition are depicted on the big screen, and they live in a plastic bubble or locked in your home – as in these films – has never been the standard way of treating this disease, according to the Immunodeficiency Foundation.
Bone marrow transplantation is commonly used to treat SCID in the early stages of life, before infection can occur, and transplantations in the first three months of life have the highest levels of success. Transplants from the respective brothers and sisters are preferred, but if they are not available, parents or unidentified donors may enter. Although transplants save lives, they often only partially restore the patient's immunity.

Now it turns out that there is another way of treating the disease – and doctors call this new therapy "treatment".

A team led by researchers at the St.Jude Hospital in Memphis has developed experimental gene therapy, which includes the collection of SCT-X1 baby bone marrow stem cells by inserting a normal gene that is absent from these patients into these cells, and then injected them back into the baby so that the cells re-settled and restored the immune system.

  Tod Lubitch (played by John Travolta) 1976 film

It is difficult to b • lifting eyebrows in a medical field

There are several types of SCID based on which gene defect is involved; Mutations in the IL2RG gene on the X chromosome cause an X-linked SCID or SCID-X1, which primarily affects infants. According to the National Medical Library of the United States, this condition is likely to affect at least 1 in 50,000 to 100,000 newborns.
If it is not treated, a child with SCID-X1 will rarely live after her second birthday, say doctors. In the United States, all newborns are screened for SCID.
"From a physiological point of view and from a" quality of life "to these patients, this is a medicine. The question is whether this will be a solid treatment. Will it last 10, 20, 50 years for these children, and only time will show," Dr. James Downing, president and chief executive officer of the St.Jud Children's Research Hospital, said on Tuesday.

"I felt the complete restoration of the immune system, which made it possible for these children to get out of isolation," he said. "So, we believe that at this time we are confident that this treatment. Only time will tell if it will be a solid, life-long treatment."

Recently developed gene therapy, conducted in combination with Busulfan chemotherapy, is described in a study published in the journal New England Journal of Medicine on Wednesday.

Both Ja & # 39; Ceon and Omarion took part in a clinical trial for therapy testing, and this proved to be successful in the treatment of SCID-X1 in both.

"Children will be cured because, for the first time, we were able to restore all three types of cells that make up the complete immune system," said Dr. Evelina Mamkarz, first author of the study and researcher in Saint-Jude

including T- Cells, B cells and NK cells

"We will continue to monitor these patients, but the results are only that these patients have complete immunity restoration without immediate side effects," Mamkarc said.

"Since this therapy is still in the early stages of development, until it is too early to evaluate the general but we want it to be affordable for any child who needs it, "she said." Most patients need only one infusion, and since we were able to fully restore their immune system, they did not need additional treatments as in transplantation. "

When JaCheon Golden was born in New Mexico in 2016, he never had the opportunity to return home from the hospital. His newborn screening tests have shown that he has a SCID-X1. When the doctors told his aunt, Danny Hawkins, about his test results, she did not know what to think.

  Jaon Ceon, in 3 months, with doctors at the University of California, San Francisco.

"I was like this:" Well, what is it? "And their answer was to me:" Well, have you ever seen the movie "The Boy in the Bubble"? And I was such, "yes," and they said, "Well, that's what he has." At this moment, all my stuff passed through my mind, "Hawkins said with CNN alone.

Hawkins, guardian Jaa Keon, learned more about the mess and how to care for I'm Keon while he was in a hospital, including a permanent washing hands so as not to expose him to the microbes and heat the water in front of his baths so that the water is sterile.

  Jaon Ceon and his aunt, Dannie Hawkins.

The doctors then told Hawkins that she could go to San Francisco to receive treatment for Ja & # 39; Ceon, and she was informed of a study for a new gene therapy.

In 2017 The year when in San Francisco JaChawn was treated for his illness. As soon as Hawkins heard that the treatment was successful, she first took Ja & # 39; Ceon outside the window and things like that, because I would put he was up to the window of the hospital so he could see people walking and the cars are going, "she said. "Just to see him looking at the car and just a smile, it was a blessing."

  Je " Ceon with his hair braided.

In the following days, Hawkins was still nervous about the fact that the immune system of her nephew was completely restored.

"I still had this fear that if he got sick?" You know, because he never left the hospital before, "she said.

Then – we went to the Giants game, and his immune system could cope with it, with all the people who were there, and then I realized that it worked, "she said.

  Jaon Ceon in a skateboard, now as a kid.

During the game, Hawkins took Joe Keona, a baseball fan, to meet Barry Bonds, a former star-player in San Francisco. In addition to Hawkins and a medical specialist, he was the first person to be held by Ja & # 39; Ceon.

"He never knew the state of I'm Keon, because that's not what I talked to him, but he had to keep it, and we took him," said Hawkins, "it was the third Day of leaving the hospital.

"I really prayed for a miracle, and I think it happened"

  Omarion as a child from SCID-X1.

As for Omarion Jordan, he is now a loving, happy and healthy baby with a golden fish – but this was not always the case.

When Omarion was born almost a year ago in the state of Indiana states did not test newborns for SCID, and his seven did not know about his condition. As a baby, he often encountered health problems such as rashes or scaly patches on the skin, but nobody knew why.

"They probably guessed that it was not like they told me he had a cradle, he had eczema, he had an allergy to milk," said his mother, Christine Simpson.

"I just felt that nobody took me seriously, and that it was much more serious than anyone who gave it," she said. .

  Omarion with an infection.

Once Simpson was enough. She took her son to see her primary pediatrician, who then sent Omarion to a neighboring hospital.

At the hospital, "they said he had pneumonia, he had a viral infection, he had a fever, it was difficult for him to breathe, and they just continued to do more and more tests," Simpson said. [Then I remember how they told me he had a cross-sickness sick of X.] Omarion was at the age of about 3 months, and Simpson had never heard of this disorder.

  Omarion and his mother, Christine Simpson. and his mother, Christine Simpson.

Once Omarion was diagnosed, Simpson and his doctors make efforts not to expose him to any microbes, and he was not allowed to go out into the street for months. He was then enrolled in a study for new gene therapy.

After that, Simpson was informed that her son was successfully treated.

"By entering into this, I honestly expected the worst," she said. ] "I really prayed for a miracle, and I think it happened. I do not think it's something like that miracle," she said. "As soon as we learned that he was cured, he actually snorted, and we woke him to take him outside, and then we were all out on the street until he fell asleep."

Next for gene therapy

Ja & # 39; Ceon and Omarion were two of the eight infants of patients who were involved in a new study. Patients who were middle-aged 3½ months and recently diagnosed with SCID-X1 were treated with gene therapy. The therapy included collecting stem cells from their bone marrow and then using the virus as a vector to insert a healthy copy of the IL2RG gene into the patient's cells before putting these cells back into their bodies. "The process takes about 10 days," she said.

"Before correctional gene stem cells were injected back into patients, the children received two days of low dose bosulfan, which was the most commonly used chemo-therapeutic agent. In bone marrow transplantation, to make space in the bone marrow for stem-cell stem cells The result was really outstanding for our patients, "said Mamkarz

or HIV, but patients" can not get HIV, "said Dr. Morton Covan, a pediatrician from the University of California, San Francisco, who was an author to "It came out as HIV, but it's actually no longer HIV when it's inserted," he said. The virus was designed in such a way that its active parts were removed, making it safe to use.
"This virus is able to effectively deliver a healthy copy of the gene in the stem cell in a manner that was not possible earlier. "This type of virus is safer and more effective for gene therapy," said Dr. Steven Gotsschalk, chair of the bone marrow transplant and cell therapy department at St.Jude's Children's Research Hospital and research author on Tuesday. Such genetic treatments may also have a risk of cancer, since the delivery of the IL2RG gene to the patient's cells may affect neighboring genes, but Gothschalk said that the HIV vector was designed to include isolates that block the activation of surrounding genes.

"The goal is to prevent the unintended occurrence of leukemia in gene therapy," he said.

All patients survived the treatment and went well on average 16.4 months after treatment, according to a study. Additional studies are needed to determine if the effects will continue when patients become older.

Researchers explore how this gene therapy can be used to treat elderly patients and other types of diseases.

For example, "the same vector that was made here in Saint-Jude is currently used by the National Institutes of Public Health for the treatment of older children who have failed bone marrow transplantation," Mamkarc said.

"We are investigating other immunodeficiencies, but this vector can also be potentially used to treat sickle cell disease, which is a life-threatening disorder of blood that is very common throughout the world," she said.
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