Are those children who receive gene therapy okay after 10 years?

More than a decade ago, scientists at the University of California at Los Angeles (UCLA) used the gene therapy they developed to treat 10 children born with rare and fatal immune system diseases
.

How are these children now?

Screenshot source: Blood

Recently, an article published in "Blood" reported the long-term effects of these children who received gene therapy
.

The study pointed out that the effect of this therapy seems to be long-lasting.

These children suffer from adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID), an autosomal recessive genetic disease caused by mutations in the gene that produces ADA (adenosine deaminase)
.

Adenosine deaminase is very important to the normal function of the immune system.
If the synthesis of adenosine deaminase is abnormal, it will cause severe immunodeficiency in children
.

For babies suffering from this disease, even if they are exposed to everyday bacteria, it can be fatal.

At present, the treatment methods for ADA-SCID include ADA enzyme replacement therapy (ERT), allogeneic hematopoietic stem cell transplantation (HSCT), or gene-corrected cell autologous hematopoietic stem cell transplantation (gene therapy).
ERT has been approved by the US FDA and gene therapy has been approved.
Approved in the European Union
.

About 10 years ago, the UCLA team included 10 children aged 3 months to 15 years with ADA-SCID in a Phase 2 clinical study of gene therapy, most of them infants
.

In the treatment, the researchers first remove hematopoietic stem cells from the bone marrow of each child, and then use a specially modified virus (ADA gamma retroviral vector [gRV]) originally isolated from mice to guide health The ADA gene enters the DNA of stem cells

These patients are monitored in a long-term follow-up program of 8 to 11 years
.

It was found that 9 out of 10 patients had sufficient immune reconstitution to prevent serious infections and did not need to resume ADA enzyme replacement therapy or undergo secondary gene therapy

The study author, Professor Donald Kohn of UCLA, explained: "This shows that gene therapy is most effective for young children
.

"

But the researchers did find that children who were also successfully treated had significant differences in their immune systems
.

They observed that in some cells, hematopoietic stem cells containing the corrected ADA gene were nearly a hundred times higher than other cells, and there were more gene copies in each cell

Professor Donald Kohn pointed out: “Those who have more copies of ADA genes have better immune function, and some people with lower levels of gene copies need regular infusion of immunoglobulin to maintain the normal function of the immune system
.

Over time Over time, this process may lead to improper activation of genes and make cells cancerous

Finally, the paper concluded that the study proved the lasting effect of gene therapy on ADA-SCID, but also emphasized the genotoxicity risk of ADA gamma retroviral vectors
.

"In the beginning, we saw that gene therapy was effective.
Now, we can say that it is not only effective, but it can be effective for more than 10 years.
Hopefully one day we can see that the results can last for 80 years
.

" The author of the study, UCLA's ElineT Dr.

Reference

Reference

[1] Bryanna C.

[1] Bryanna C.
Reinhardt et al, Long-term Outcomes after Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency (ADA SCID), Blood (2021).
DOI: 10.
1182/blood.
2020010260

[2] A decade after gene therapy, children born with deadly immune disorder remain healthy.
Retrieved OCTOBER 15, 2021, FROM https://medicalxpress.
com/news/2021-10-decade-gene-therapy-children-born.
html

[2] A decade after gene therapy, children born with deadly immune disorder remain healthy.
Retrieved OCTOBER 15, 2021, FROM https://medicalxpress.
com/news/2021-10-decade-gene-therapy-children-born.
html

[3] New gene therapy for the treatment of children with ultra-rare immune disorder recommended for approval.
retrieved 01/04/2016, from https:// -treatment-children-ultra-rare-immune-disorder-recommended-approval

[3] New gene therapy for the treatment of children with ultra-rare immune disorder recommended for approval.
retrieved 01/04/2016, from https:// -treatment-children-ultra-rare-immune-disorder-recommended-approval