February 27, 2024

Second trial with embryonic stem cells

To cure blindness
The US government has approved a second
clinical trial of a treatment using human embryonic stem cells to cure a
rare disease that causes serious vision loss. Advanced Cell Technology,
a Santa Monica-based biotechnology company, said the research is set to
begin next year, after it was approved by the US Food and Drug
Administration. Another biotech company, Geron Corp, started a clinical
trial for spinal cord injury with hESCs.

ACT’s test will focus
on Stargardt disease, which affects just 30,000 Americans. The company
hopes the same approach will work for similar and more common eye
disorders such as age-related macular degeneration, which impacts the
lives of millions. Stargardt is an inherited disorder typically
beginning in adolescence that attacks central vision which is used for
tasks like recognising faces. Some Stargardt sufferers go completely
blind or lose all peripheral vision. Others are severely impaired and
can only detect light or see their hands moving in front of their eyes.

The key problem with
Stargardt is that impaired scavenger cells fail to remove toxic
byroducts from the eye, allowing them to accumulate, killing other
cells. No proven treatment exists. In ACT’s new study, 12 patients will
be treated with healthy scavenger cells, created in a laboratory from
hESCs. This preliminary phase of treatment is for testing the safety of
various doses, injecting just one eye on each patient. “We’re also hoping to
see some improvement in visual acuity, but that’s a bonus,” Dr. Robert
Lanza, ACT’s chief scientific officer, said Monday. ~ AP, Nov 22; BioEdge, Oct 15

Jared Yee
clinical trials
stem cells