The springboard was an announcement that the Food and Drug Administration (FDA) had granted a "rare pediatric disease designation" to a therapy that the agency has backed with nearly $52 million.
The FDA action comes as the agency is in what some call its "last stage." The agency, formally known as the California Institute for Regenerative Medicine (CIRM), expects to run out of cash for new awards in about three years. No additional funding appears readily available for the agency, which was created by voters in 2004. It has yet to fulfill voter expectations that it would produce a stem cell therapy that is widely available.
The agency this week, however, touted the FDA designation, which involves a treatment for what is commonly known as "bubble baby syndrome." The treatment is called OTL-101 and is being developed by Orchard Therapeutics Ltd, which is based in the United Kingdom The firm is working with Donald Kohn of UCLA, who has been researching in the area for decades.
Writing on the $3 billion agency's blog, The Stem Cellar, Kevin McCormack, senior director for communications, said,
"The treatment) involves taking the patient’s own blood stem cells, genetically modifying them to correct the SCID mutation, and then returning the cells to the patient. Those modified cells create a new blood supply, and repair the child’s immune system."He wrote,
"The importance of the rare pediatric disease designation is that it gives the company certain incentives for the therapy’s development, including priority review by the FDA. That means if it continues to show it is safe and effective it may have a faster route to being made more widely available to children in need."McCormack continued,
"This is the second time in less than two weeks that a CIRM-funded therapy has been awarded rare pediatric disease designation. Earlier this month Capricor Therapeutics was given that status for its treatment for Duchenne muscular dystrophy.
"Two other CIRM-funded clinical trials – Humacyte and jCyte – have been given regenerative medicine advanced therapy designation (RMAT) by the FDA. This makes them eligible for earlier and faster interactions with the FDA, and also means they may be able to apply for priority review and faster approval.
"All these are encouraging signs for a couple of reasons. It suggests that the therapies are showing real promise in clinical trials. And it shows that the FDA is taking steps to encourage those therapies to advance as quickly – and safely of course – as possible."
McCormack added, however,
"Getting these designations is, of course, no guarantee the therapies will ultimately prove to be successful. But if they are, creating faster pathways means they can get to patients, the people who really need them, at a much faster pace."
