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Potential autism breakthrough as three-year-old boy’s symptoms are reversed using cheap drug

Potential autism breakthrough as three-year-old boy’s symptoms are reversed using cheap drug


A nonverbal autistic child said his first words after taking a cheap drug normally given to cancer patients.

Mason Connor of Arizona was diagnosed with autism at two-and-a-half-years-old after his mother noticed he hadn’t started talking.

After years of failed therapies and treatments, Mason’s parents met with a doctor researching experimental new therapies for autism.

Dr Richard Frye, a pediatric neurologist at Rossignol Medical Center in Arizona, suggested the family try Leucovorin, a generic drug derived from folic acid. 

Three days after starting the $2.50 tablets, Mason said his first words at three years old. 

Folic acid – also called vitamin B9 – is required for DNA synthesis and repairs. It’s naturally occurring form, folate, is found in leafy vegetables, peas, lentils, beans, eggs, yeast, and liver. 

Leucovorin is typically given to cancer patients to help protect their red blood cells from chemotherapy. 

But research suggests some children with autism don’t get enough of folate supply to their brains due to a blockage, which could cause communication issues. 

Mason Connor (pictured here) was diagnosed with autism at age two and a half after he failed to start talking

Mason Connor (pictured here) was diagnosed with autism at age two and a half after he failed to start talking

Leucovorin is thought to help bypass that blockage. 

Dr Frye told CBS News: ‘[Leucovorin] could really have a substantial impact on a very good percentage of children with autism.’ 

According to the latest CDC data, one in 36 children in the US have autism, or just under 2million.

Generally, most with the disorder are diagnosed by age five, though some can be tested as young as age two. 

According to the National Institutes of Health, about seven in 10 autistic children have folate receptor autoantibodies, a type of antibody that targets healthy areas of the body. 

Folate receptor autoantibodies prevents folate from reaching the brain, which could cause language delays.

In one of Dr Frye’s studies, 44 autistic children with these autoantibodies were given 50 milligrams of Leucovorin per day for four months. 

All of the patients had improvements in their language, behavior, hyperactivity, mood, attention, and aggression.

Another 2018 study from Dr Frye’s team found that children with autism and language delays treated with Leucovorin all experienced ‘significant’ improvements in language, as well as irritability, hyperactivity, and lethargy.

Mason, pictured here as a toddler, started speaking at age three. Now age five, his family plans to enroll him in mainstream kindergarten next fall

Mason, pictured here as a toddler, started speaking at age three. Now age five, his family plans to enroll him in mainstream kindergarten next fall

Leucovorin, which costs about $30 for 12 pills, is typically given to cancer patients to help protect their red blood cells from chemotherapy. However, research suggests it may clear chemical blockages in autistic children's brains, improving their language skills

Leucovorin, which costs about $30 for 12 pills, is typically given to cancer patients to help protect their red blood cells from chemotherapy. However, research suggests it may clear chemical blockages in autistic children’s brains, improving their language skills

Leucovorin comes in tablets, injections, and infusions. 

Dr Frye said: ‘We’ve done the science, and the next step is that we want to get more funding so we can actually get it FDA approved.’ 

Previous research has shown that some children with autism have antibodies in their blood that prevent folate (vitamin B9) from getting to the brain, causing a deficiency.

Leucovorin is a reduced folate that can bypass those antibodies, allowing it to reach the brain. 

Leucovorin is only FDA approved for chemotherapy patients and those with some forms of anemia, though doctors can prescribe it off-label for autism.

However, it’s still unclear how many doctors do this. 

Dr Frye fears that the drug’s cheap cost means there is no impetus to invest in studying it.

Previous studies have indicated that pregnant women who take folic acid could reduce their child’s risk of autism by 40 percent. 

Now five years old, Mason is still on Leucovorin. Once nonverbal, his parents now expect to enroll him in traditional kindergarten this fall. 

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