Philadelphia, Pennsylvania —(Map) Scientists have created the first gene-editing medicine made for a single patient.
Test showed that KJ’s DNA had a mistake which made it hard for his body to get rid of a dangerous chemical called ammonia.
But because of the problem with his genes, KJ’s liver couldn’t get rid of ammonia.
Scientists at Penn Medicine had been working on the idea of creating medicines to fix the genes of a single patient.
The researchers studied KJ’s DNA.
Map of Philadelphia, Pennsylvania.
The first gene-editing medication for a single patient has been developed by scientists. The patient, a young infant who was ill due to a genetic issue, is significantly better now and should return home shortly.
Doctors were aware of KJ Muldoon’s difficulties shortly after his birth. According to the test, KJ’s body had trouble eliminating the hazardous chemical ammonia because of a mistake in his DNA. The brain and other organs may suffer harm from excessive ammonia. The majority of children born with this issue pass away while still infants.
Ammonia is produced by the human body as it breaks down proteins. The liver aids in the removal of this ammonia in a healthy individual. But KJ’s liver was unable to eliminate ammonia due to a genetic issue.
KJ was too small and young to receive a liver transplant, and it would have been extremely risky to wait for one. KJ’s parents were asked if they would be interested in trying a novel approach by the doctors at Children’s Hospital of Philadelphia (CHOP): a medication specifically designed for KJ. His parents accepted.
Penn Medicine researchers had been developing the concept of developing medications to correct a single patient’s genes. They now had the opportunity to give it a serious try. Nevertheless, there was limited time to assist KJ, so the pressure was on.
Research was done on KJ’s DNA. The issue was comparable to correcting a single misspelled letter in a three billion-letter word. They quickly created a CRISPR tool for editing KJ’s DNA with the assistance of numerous scientists who volunteered and put in long hours.
They then used mice and monkeys to test the new medication. Positive outcomes showed that the new treatment benefited the animals and seemed to be safe. The FDA only took six months to approve the new medication.
KJ had been receiving medication and special care to help lower his ammonia levels during this time. The new medication, which was intended to reach his liver and cause a single, minute alteration in his DNA, was first administered to KJ when he was six months old.
Shortly after the initial treatment, KJ was able to consume protein without increasing his ammonia levels. Due to the favorable outcomes, KJ’s doctors decided to give him two additional doses. KJ is now much healthier and has put on some weight. The hospital anticipates that he will return home for the first time in his life shortly.
KJ is not entirely recovered. One day, he might require a liver transplant and need to continue taking medication. However, he is in much better physical condition now than he was at birth.
The findings have scientists and physicians giddy with excitement. A unique DNA-editing medication for a single patient was developed in just six months by the researchers and the businesses they collaborated with. That work would have taken ten years or more in the past. For more than just KJ, the project is a success. Millions of other people with conditions that could be treated with similar techniques are also given hope by it.
