SCID

According to US CDC guidelines, severely immunocompromised people may get a fourth COVID-19 vaccine six months after their third dose.

According to the CDC, small studies have found that fully vaccinated immunocompromised people accounted for about 44% of the breakthrough cases that led to hospitalization. And one Johns Hopkins University study found that vaccinated immunocompromised people were 485 times more likely than most vaccinated people to be hospitalized with COVID-19 or die from the disease.

Immunocompromised may need a fourth Covid-19 shot, CDC says People with health conditions that make them moderately or severely immunocompromised may get a fourth mRNA Covid-19 shot, according to updated CDC guidelines.

A way to restore immune function in infants with one form of SCID!

Newborn screening of TREC is now used in the US to detect severe combined immunodeficiency before infections occur . But that still leaves us with the need for effective treatment.

Treating infants with X-linked severe combined immunodeficiency with low-dose chemotherapy followed by gene therapy gave the children the ability to make the cells needed to mount a normal immune response, in the New England Journal of Medicine. The finding marks a milestone in the long effort to use gene therapy for the devastating condition, also known as bubble boy disease, which requires untreated patients to be isolated in order to protect them from life-threatening infections. Experts caution that longer follow-up is needed to determine whether the gene therapy–treated patients are truly cured.

They were able to remove the protective isolation within three to four months post gene therapy and send the babies home to their families. They are all toddlers now, exploring life, attending daycare.

People with severe combined immunodeficiency (SCID) have mutations in genes needed for immune cell function, leaving them vulnerable to infection. In the most common form of the disease, X-linked SCID (SCID-X1), the gene at fault is IL2RG, which codes for a piece of the cytokine receptors needed for the normal development of several different kinds of immune cells, including T cells, B cells, and natural killer cells.

The standard treatment for the condition is a transplant of bone marrow tissue that can make normal immune cells, but finding an immunological match for patients can be a challenge.

But, only about 20 percent of SCID-X1 patients have a matched sibling, and receiving a transplant from another donor carries a risk of graft-versus-host disease.

Efforts to treat SCID-X1 with gene therapy began two decades ago. While they initially produced promising results, the earliest therapies appeared to cause leukemia in some patients.

The gene therapy in the new study delivers a functioning copy of IL2RG into patients’ extracted bone marrow cells using a lentiviral vector modeled on HIV’s shell. The IL2RG sequence comes with an insulator sequence at the end designed to prevent it from turning on nearby genes in the area of the genome it lands in, given that switching on the wrong gene could lead to cancer.

In addition to using a different vector from previous gene therapies, the study protocol called for a novel step, devised by researchers at the University of California, San Francisco (UCSF): pretreatment of patients with a low dose of the chemotherapy drug busulfan, which kills off immune precursors. Its aim was to create space around the bone marrow for the gene-edited cells to get in and take hold.

Ten infants were treated in the study, half at UCSF and half at St. Jude’s (the study reports results for the first eight). They ranged in age from 2–14 months at the time of treatment, and none had a matched sibling donor available. Doctors extracted bone marrow cells from each patient and sent them to a lab at St. Jude’s for editing. They then treated the babies with one to two doses of busulfan, and injected the gene-edited cells back into them.

The researchers monitored levels of the patients’ immune cells for a median of 16 months; all but one of the first eight saw climbing levels of T cells, B cells, and natural killer cells following the treatment. (The nonresponsive patient was re-treated with the gene therapy, which increased his T cell count.) Four of the patients received vaccinations against tetanus, diphtheria, pertussis, polio, and pneumonia, although only two of those infants mounted a normal immune response to all of the vaccines, as measured by counts of cells and other markers in their blood

Gene Therapy Effective for Severe Combined Immunodeficiency Researchers report they’ve found a way to restore immune function in infants with one form of "bubble boy disease."

We want to provide you with new information from IDF!

One of the biggest things that IDF has done this year is to create a whole new program and website for patients/families with SCID. The following link to the SCID website has a wealth of information.

There are also videos, recorded IDF Forums, blogs, updates and more regarding COVID-19 in the COVID-19 portal.

Also see the new IDF Spanish Link that contains all kinds of information of PI information in Spanish:

Events | Immune Deficiency Foundation [view:events=page]