Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not available to him. In 2022, London-based Orchard Therapeutics stopped investing in an experimental treatment for the condition, Wiskott-Aldrich syndrome. And there are no gene therapy studies he can join.
Robin Alderman, right, and her son, Camden Alderman, 21, pose for a portrait near their home in Greensboro, N.C., Wednesday, June 12, 2024. Camden, 21, was diagnosed as a baby with a rare disease called Wiskott-Aldrich syndrome, which is caused by a mutated gene on the X chromosome. It primarily affects boys – up to 10 out of every million — and can cause frequent infections, eczema and excessive bleeding.
Collectively, about 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual disorders affects perhaps a few in a million people or less. There’s little commercial incentive to develop or bring to market these one-time therapies to fix faulty genes or replace them with healthy ones. This leaves families like the Aldermans scrambling for help and some trying to raise money themselves for cures that may never come.
A major Italian charity announced in February that it’s taking over the Wiskott-Aldrich treatment Orchard had been pursuing. And an arm of the charitable Foundation Fighting Blindness helped launch a company, Opus Genetics, to advance gene therapy work by University of Pennsylvania researcher Dr. Jean Bennett and a colleague.“Some of them have bake sales. One family mortgaged their house to give some money for a study for their rare disease,” Bennett said. “I just feel responsible to help them.
Today, Camden Alderman is a rising senior at North Carolina Agricultural and Technical State University. He takes penicillin daily and gives himself weekly immunoglobulin infusions under his skin, which help fight infection. Still, he’s landed in the hospital a few times in recent years and has developed a kidney problem.
For a while, it seemed gene therapy for Wiskott-Aldrich was on track for wider availability. Genethon, a French nonprofit research organization, sponsored promising clinical trials but didn’t have funding to continue development, CEO Frédéric Revah said. Lacey Henderson’s daughter, 5-year-old Estella, has alternating hemiplegia of childhood, a neurological condition that affects 300 people in the U.S. Estella is cognitively delayed, has limited use of her hands and becomes temporarily paralyzed in part or all of her body, Henderson said. Medications can curb symptoms, but there’s no cure.
Ultimately, most biotechnology companies become public and must focus on shareholder profit, said Francois Vigneault, CEO of the Seattle biotech Shape Therapeutics.
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