On May 26, Tracy Atteberry checked in to the hospital at the National Institutes of Health in Bethesda, Md. The 57-year-old has an ultrarare inherited disease that hobbles his immune system so that the most innocuous of germs could kill him. Now he was going to receive a potential cure in the form of a gene editing treatment that had never before been tried in patients.
But, in a tale that gene therapy experts say is too common, Mr. Atteberry and one other patient, a Canadian teenager, may be the only ones ever to get this treatment. As has happened over and over when gene therapies are developed, the company that made it, Prime Medicine, is no longer offering it.
The problem is economics. The science is there to cure Mr. Atteberry’s condition, a subtype of chronic granulomatous disease, or CGD. But it costs so much to develop a treatment and get it approved that even when a company decides to start along this path, it often abandons the effort.
In part, that is because there are so few patients. Only about 200 people in the United States share Mr. Atteberry’s genetic condition, according to Prime Medicine’s chief medical officer, Dr. Mohammed Asmal. But only about 50 of them might be eligible for gene editing, Dr. Asmal said.
He added that the company — before it ran out of money for the study — planned to treat six to 12 patients at a cost of $20 to $30 million.
As gene therapies are developed, then discarded, the result is dashed hopes for many of the 400 million people in the world with rare genetic diseases.
Thank you for your patience while we verify access. If you are in Reader mode please exit and log into your Times account, or subscribe for all of The Times.
Thank you for your patience while we verify access.
Already a subscriber? Log in.
Want all of The Times? Subscribe.
The post These Patients Got the Cure. Then It Went Away. appeared first on New York Times.
