Researchers at Necker Hospital were able to increase the height of a young woman by over 10 centimeters with Infigratinib, a drug already used to treat bladder cancer.
Man With Achondroplasia. Image Courtesy of Richard McCoy
It is a hope for a family and for thousands of people. On March 30, the trial of a new drug began for nine-year-old Amandine, who suffers from achondroplasia, a form of dwarfism. Infigratinib the drug being tested is already used to treat bladder cancer, but a team of scientists at Necker Hospital, has discovered its value in treating this abnormality.
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More than 20 years of research
In 1994, Dr. Laurence Legeai-Mallet and her team discovered the gene responsible for achondroplasia. It is associated with a mutation of the FGFR3 gene, which then produces the FGFR3 protein in excess. This protein is present in bone and cartilage cells and, when produced in excess, interferes with bone growth and ossification. At the Imagine Institute, located on the Necker Hospital campus, the team has been “constantly exploring the mechanisms that are dysregulated by the disruption of this gene and have developed cellular and animal models to test molecules and develop new therapeutic options,” according to a press release.
A promising drug
It took 20 years to find a potential treatment. In 2016, Dr. Laurence Legeai-Mallet published the final results of a study conducted with Infigratinib. “This molecule reduces the phosphorylation of FGFR3, which is responsible for its hyperactivity, and corrects abnormal growth in our animal models,” she explains. We have shown that a low dose injected subcutaneously is able to penetrate the growth plate of these models and modify their organization.” A patent has been filed in France, but it is a U.S. laboratory that manufactures the drug.
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The first administration was done in Australia
In July 2020, a young Australian started this treatment. In France, nine-year-old Amandine is the first person to benefit from the treatment. She will have to take seven tablets a day for at least two years. They could prevent the onset of complications such as ENT problems, orthopedic or back problems. In addition, the girl could become 10 to 20 cm taller than the average female with the same disorder. People afflicted with achondroplasia typically reach 1,24 m in adulthood at most. “This is the only chance for Amandine to grow taller,” her mother Valerie confesses. It’s not a miracle, it won’t reverse the disease, but it will help her live a more normal life: Every centimeter gained is a victory.” The treatment will also soon be tested on a young boy in France. If these various trials are successful, adults could also benefit. In the US up to 1 in 15000 people suffer from achondroplasia.
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