Girl whose brain tumor left her paralyzed and blinded "cured" after treatment with an experimental drug

A Maryland teenager whose brain cancer has resisted all standard treatments is in remission after an experimental drug contracted its tumor during a clinical trial.

Kaitlyn Dorman, of Baltimore, was nine years old when doctors discovered a cancerous tumor developing on her brainstem.

They told her family that the tumor was easily treatable – but that nothing seemed to work, and Kaitlyn quickly lost mobility in her left hand, her legs became paralyzed and her vision dimmed in her right eye. .

Doctors at Johns Hopkins Hospital finally offered the Dormans the opportunity to participate in a clinical trial of a new experimental drug that would target the Kaitlyn tumor.

It worked – by reducing the size of the tumor to the size of a grain of rice – and doctors now say they think the 17-year-old officially has no cancer.

"We use the keyword to heal," said Baltimore Sun Dr. Eric Raabe, badociate professor of pediatric oncology at the Johns Hopkins Kimmel Cancer Center, who had been treating Kaitlyn. "It does not come back.

The drug, known as everolimus, was developed by the Swiss pharmaceutical group Novartis and is known by the brand name Afinitor.

It works by preventing a specific protein that cancer cells use to multiply and grow.

In 2016, the US Food and Drug Administration has approved the drug to treat cancers of the brain, bad, kidneys and pancreas, as well as other drugs to prevent the rejection of a kidney or grafted liver.

Novartis has been criticized in the past. In 2013, the National Institute of Health and Clinical Excellence said the cost of Afinitor of about $ 47,000 a year was too high.

And a study published this month found that doctors who had received between 2013 and 2015 payments from a company for a specific drug had increased their prescriptions, one of them being Afinitor for kidney cancer treatment.

With regard to anticancer drugs, double-edged swords are not lacking.

There is a strong demand for new drugs to treat all types of cancer. Behind these drugs: pharmaceutical companies with financial interests and a complex regulatory network, generating costs and access to common problems.

Like many families, Kaitlyn wanted to do everything in her power to save her because they felt their options were dwindling.

In 2011, at the age of nine, Kaitlyn began to feel tremors in her left hand, which worsened progressively.

Her mother, Mary, took her to a pediatrician, who first performed a neurological test and then ordered a scanner.

The family went to Johns Hopkins for the results, where the doctors revealed that Kaitlyn had a brainstem tumor.

& # 39; All I could think was: "What are we doing now?" I wanted to know exactly what we should do, Mary Johns Hopkins Medicine said in an interview.

A biopsy was performed and Kaitlyn was diagnosed with pilocytic astrocytoma, a rare brain tumor in children.

The cancer comes from star-shaped cells in the brain, called astrocytes, but its cause is unknown.

Symptoms include headache, nausea, vomiting, vision problems and balance problems.

According to the National Organization for Rare Diseases, it is estimated that cancer affects 14 people out of a million children under 15 years of age.

The main form of treatment consists of a surgical procedure to remove as much of the tumor as possible, followed by radiation therapy and chemotherapy if the tumor can not be fully removed.

A 2012 study by the Mayo Clinic found that the five-year survival rate of pilocytic astrocytoma was greater than 96% in children and young adults.

The doctors told the Dormans that Kaitlyn simply had to undergo a few chemotherapy sessions and that she would be cured.

But that was not the case. Instead, his tumor did not respond to treatment and began to develop.

Kaitlyn lost a movement in her left arm, then in her left side and, finally, in both her legs.

"I've never been scared," she tells Johns Hopkins. "I just thought it was unreal. I kept wondering, "When can I still move?"

In addition, her right pupil was dilated – making her vision blurred – and she contracted an infection in her stomach during a visit to the hospital, reported The Baltimore Sun .

The doctors tried to be as aggressive as possible with the chemotherapy, but her mother, Mary, according to her father, was in decline.

When it became clear that chemotherapy was not working, her medical team came up with an alternative solution.

At that time, Johns Hopkins was one of many hospitals badociated with a clinical trial on everolimus.

"All we really wanted to know was that our daughter was going to heal," said Mary at Baltimore Sun.

"I was not worried about the clinical trial or even everything we needed to do to collect additional data or monitor it.

"As a parent, I think we would have done everything in her power to survive the tumor that she had."

Kaitlyn also said that she was not worried about participating in the clinical trial of the experimental drug.

"I could not walk and I could not really see," she told the newspaper. "Hearing that there was, for example, something else that could possibly do something that could make everything disappear, I thought," Great. Okay, let's try. "

The drug worked. Kaitlyn slowly began to move in her arms and legs and her vision cleared in her right eye.

Kaitlyn had to stop taking this medicine after it started to touch her liver. But even after you stopped taking this medicine, the tumor began to contract.

Its tumor has shrunk from the size of a walnut to the size of a grain of rice, almost impossible to see on scans, reported The Baltimore Sun.

For eight years the tumor has not grown back.

"I told my parents, 'I'm going back and you can not do anything about it,'" she told Johns Hopkins.