The drug is effective in treating people with mild to moderate symptoms, but is still far from a cure.
“The problem is that people are suffering, not getting better,” says Dr Peter White, a professor of medicine at Harvard Medical School and director of the National Institute for Mental Health.
And there’s still a long way to go before the drug can be widely used in the clinic.
“We have to develop a way to do this effectively and cheaply,” he says.
To date, there are only two drugs approved for treating fatigue and sleep disorders.
But in recent years, several pharmaceutical companies have developed compounds that target the two most common symptoms: fatigue and chronic fatigue.
One such drug, called Sulfadiazine, was approved in February, and the next is being tested by Pfizer.
In an effort to find an effective drug for chronic fatigue, scientists from Harvard, Harvard-affiliated Brigham and Women’s Hospital, and other institutions used the genetic code of an individual with chronic fatigue to create a synthetic version of the drug.
“It’s an extremely robust approach, very precise, and very precise in its targets,” says John Siegel, a biochemist and co-director of the lab that produced the SulfADIZINE drug.
It works like this: the protein encoded by the genes that make up the gene for the molecule is inserted into the cell membrane, where it binds to a protein on the surface of the cell called a protein kinase.
The resulting molecule then turns on a protein called the receptor.
The receptor binds to the molecule’s protein, causing it to activate a signaling cascade that then leads to the production of an amino acid called cyclic AMP.
SulfAMP is the main drug target for SulfADEZINE.
It binds to two genes, one on chromosome 9 and one on the X chromosome, making it difficult to target by conventional drugs.
So, Dr Siegel’s team used a gene-editing technique to create an alternative version of SulfAMP that also targets the gene on chromosome 2, and it worked by binding to the protein on both chromosomes.
This was the first time that researchers had produced a synthetic Sulfamine drug that also had the ability to activate the same gene on both X and Y chromosomes.
The new Sulf AMP drug has a longer half-life, but it has a better safety profile, says Dr White.
“This drug has been very well tolerated and has been fairly well studied,” he adds.
The drugs are expected to be approved by the Food and Drug Administration (FDA) next year.
Sufficient data is also lacking to make an informed decision about their use in the future, says White.
The main reason is the amount of research required.
The first Sulfamp tests were only conducted in China.
And because the drugs are so expensive, it is hard to develop them quickly in other countries, he says, because their prices are too high.
And, of course, many people are already taking the drugs, which is another reason the FDA has not approved Sulfadezine yet.
Another obstacle is the difficulty of developing the drug in the lab, which means the drugs could be too expensive to be used in clinical trials.
But Dr White says this is unlikely in the near future.
“There are some very good drugs on the market that do a lot of good things,” he explains.
“But they’re expensive and not widely available.
We think the cost-effectiveness of these drugs should be very much in line with the cost of other drugs.”
This article appeared in print under the headline “Cerebral cortex exhaustion and sleep loss”