WASHINGTON (Reuters) - Advanced Cell Technology, a small Massachusetts-based biotechnology company, said on Thursday it has asked for approval to test human embryonic stem cells in treating a rare cause of blindness.
The company said it filed an IND, an investigational new drug application, with the U.S. Food and Drug Administration to use the stem cells to treat patients with Stargardt’s macular dystrophy.
If approved, it would be the second U.S. approval to test human embryonic stem cells in human patients.
Geron Inc won approval in January to test human embryonic stem cells to treat paraplegic patients who can use their upper bodies but cannot walk. That trial is on hold while the company and FDA check on some side-effects seen in animals.
ACT has previously reported it used human embryonic stem cells to make retinal cells. They have reversed blindness in rats.
“The treatment for eye disease uses stem cells to re-create a type of cell in the retina that supports the photoreceptors needed for vision,” ACT’s scientific director, Dr. Robert Lanza, said by e-mail.
“These cells, called retinal pigment epithelium (RPE), are often the first to die off in Stargardt’s macular dystrophy.”
Stem cells are the body’s master cells. Embryonic stem cells, taken from a days-old embryo, are the most pliable as each one has the power to morph into any of the body’s cell types.
Companies like Geron and ACT are trying to use them as the basis of a new field called regenerative medicine. Embryonic stem cells can be used for tailor-made treatments that a patient’s body is less likely to reject.
U.S. President Barack Obama lifted some of the restrictions on their use soon after he took office.
“It has been over a decade since human embryonic stem cells were first discovered. The field desperately needs a big clinical success,” Lanza said.
“After years of research and political debate, we’re finally on the verge of showing the potential clinical value of embryonic stem cells. Our research clearly shows that stem cell-derived retinal cells can rescue visual function in animals that otherwise would have gone blind. We are hopeful that the cells will be similarly efficacious in patients.”
If approved, the trial would be a phase I/II study looking at safety and for some sign that it is working. Twelve patients would be recruited to try it.
Researchers have learned to create embryonic-like stem cells called induced pluripotent stem cells from ordinary skin cells. But this technology is far newer than embryonic stem cell technology and scientists are not entirely sure it is safe.
Editing by John O'Callaghan