Scientists use incurable HIV to ‘cure’ deadly childhood disease

By Sola Ogundipe

Whoever finds a cure for the Human Immunodeficiency Virus (HIV) deserves the Nobel Prize in Medicine & Physiology. The reason is clear. HIV is one of the most intelligent viruses known. It has evaded cure for more than three decades simply because it is incredibly smart, so it’s going to take someone that is very clever indeed, to outsmart it.

•A researcher at St Jude Children’s Research Hospital in Memphis, Tennessee, where edited DNA was used to fix genetic illness into patients using modified HIV

Although genetically simple, HIV demonstrates remarkably complex behaviour. It is a master of invasion and evasion. Despite its comparatively small “genetic brain,” the virus is able to evade sophisticated detection mechanisms of the immune system.

It also counters attacks of the vastly more complex human cells, reach into the cell’s nucleus and replace the human genome with its own viral DNA. By attacking the immune system directly, HIV weakens the entire body. After taking over invaded cells, the smart HIV transfers itself between multiple cells.

Enormous research has been done on HIV, yet much more is yet to be known about the incredibly adaptive virus that continues to face human onslaught.

Certainly, scientists have not given up in the quest to overcome the smart virus and while a cure may have not been found, cure research remains promising as scientists continue work on a functional cure and a sterilising cure.

While the search continues, significant discoveries about the human immune system made on the road to finding a cure remain paramount. The efforts to improve treatment, prevention and awareness tools are continuing to have a positive impact on the lives of many.

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One of the most profound outcomes of application of HIV research occurred lately when doctors at St Jude’s Children Research Hospital in Memphis, Tennessee, reported how they “used” HIV to “cure” children with a rare immune system abnormality.

The children were all born with a condition known as Severe Combined Immunodeficiency (SCID)—commonly known as Bubble Boy Disease—that makes it impossible for their bodies to make white blood cells and they were at risk of dying as infants.

Ten of the affected children were treated with an experimental gene therapy which made eight of them to produce vital white blood cells for the first time. The scientists “edited” DNA to fix the fault which causes the genetic illness and delivered the “cured” genes into the children’s body using HIV. The altered genes used to fix the children’s defective DNA were delivered into the body using damaged HIV viruses.

Experts working on the project say the breakthrough is a first for children with this rare and life-threatening illness, and say only time will tell if it’s a permanent cure.

Each of the patients, according to the MailOnline,  has X-linked severe combined immunodeficiency (SCID-X1), a condition present from birth which stops a child’s immune system working.

The illness is known as ‘bubble boy disease’ because patients have to be kept in isolation tanks to avoid common infections like colds, which could kill them.

While the therapy is said to have cured the patients, Dr Ewelina Mamcarz, one of the scientists, notes that only time will tell if the cure is permanent.

“These patients are toddlers now who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives. This is a first for patients with SCID-X1.”

Without treatment, children with bubble boy disease usually die when they’re one or two because their body can’t fight off even the simplest of infections.

•A boy born with the bubble boy disease but can now live a normal life because the gene-editing therapy has restored his immune system…

According to The Bubble Foundation UK, bubble boy disease is caused by a defect in the IL2RG gene. “It means sufferers are born without an immune system and cannot fight infections—as a result the common cold can be fatal.

“It occurs in around one in 200,000 births—and usually affects boys.The only way of keeping these children safe from infection is to care for them in a bubble, a high-tech sterile room.

They can survive if they have a bone marrow transplant, giving them a new immune system—however this can take up to two years and is not successful in all patients.

“If a transplant fails to improve a sufferer’s immunity, their organs can become irreversibly damaged, which can be life-threatening. Many children with the illness die in the first few years of their life.

The condition was brought into the public eye by a boy called David Vetter, who was born in Texas in 1971. David could only have been cured by a bone marrow transplant but he died shortly after having one, when he was just 12 years old.

He spent his life living in protected environments—in a large tank or with a plastic bubble on his head to keep germs out—but he died of cancer in 1984.

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His lymphoma was later found to have been caused by the Epstein-Barr virus, one of the most common human viruses and one which the immune system usually controls.

After the new treatment, which is combined with chemotherapy to help the edited DNA to multiply once inside the patient’s body, most patients were able to leave the hospital within a month.

The disorder has been tried on 10 children so far in Tennessee and UCSF Benioff Children’s Hospital in San Francisco.

The results of eight patients were published in the New England Journal of Medicine and revealed they all now have working immune systems.

The therapy meant their bodies could, for the first time, create their own crucial white blood cells called T cells, B cells and natural killer cells.

It worked by scientists creating the gene required to fix sick patients’ DNA in a lab, then injecting it into their bodies inside weakened HIV viruses.

Because HIV  can to hijack DNA and spread through the body, this allowed the “cured” genes to multiply and kick-start the immune system.

Once the genes had been delivered, the modified HIV viruses self-destructed inside the patient’s blood.

All but one of the patients had working immune systems within three months of their first round of gene therapy—the final patient needed a second round before it succeeded.

Researcher, Dr Mort Cowan said: “While longer follow-up is needed to assess any late effects of treatment, these results suggest most patients treated with this gene therapy will develop a complete durable immune response without side effects.”

Omarion Jordan, who turns one later this month, had the therapy in December.

His mother, Kristin Simpson, said: “For a long time we didn’t know what was wrong with him. He just kept getting these infections.” She said learning that he had SCID “was just heartbreaking… I didn’t know what was going to happen to him.”

Thanks to the therapy, Omarion now has a normal immune system.

“He’s like a normal, healthy baby,” Ms Simpson said. ‘I think it’s amazing.”