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| Alysia Padilla-Vacarro and daughter, Evangelina, on the day of the baby's life-saving gene therapy treatment at UCLA. UCLA photo |
Numbers speak loudly in America. We focus intently –
sometimes obsessively -- on everything from baseball statistics to the latest count
of illegal immigrants. Numbers appear to create certainty and tend to drive
discussion of public issues.
Which is where the California stem cell agency comes in. Executives at the agency point with pride to
the 10 clinical trials that it has helped to fund. They do not point to the
zero number of therapies that the agency has placed in the public marketplace
despite the apparent promises of the ballot campaign that created the research effort 10 years ago this month.
Nonetheless, agency has chalked up some indirect scores that
are difficult to quantify but deserve ample consideration when evaluating the
California Institute for Regenerative Medicine(CIRM), as the agency is formally
known.
One dramatic example gained national media attention last week. The case involves work at UCLA for what appears to be a cure for
the fatal “bubble boy” syndrome. The term was coined back in the 1970s when stories
involving a youngster in Texas made headlines across the country. The child had to live inside a plastic bubble
because his immune system was compromised. The case even inspired a 1976 movie
called “The Boy in the Plastic Bubble” starring John Travolta. (See here, here and here.)
Enter Donald Kohn, many years later. Kohn is director of the Human Gene Medicine
program at UCLA. And last week, the Los Angeles university announced that Kohn
and his team have developed a treatment that cures the “bubble boy” syndrome by
altering the genes of the afflicted children. In clinical trials, the UCLA announcement
said, the lives of 18 children were saved.
Kohn used a virus delivery system that he first developed in
his lab in the 1990s. But the work on the gene treatment began in earnest in 2009 and
involved two, multi-year clinical trials.
UCLA's Nov. 18 news release described the treatment simply like this:
“During the trials, the patient's blood stem cells were removed from their bone marrow and genetically modified to correct the defect.”
The stem cell agency did not directly fund Kohn’s “bubble”
research, but some of its other funding at UCLA contributed to the effort.
Specifically, the work was performed in the stem cell research facility that
CIRM helped to finance. Kohn’s lab is in the “shared research lab” space funded
by CIRM in still another program. The
research was also aided by persons working in two other separate CIRM training
programs.
The next step for Kohn will be to use the research in proposed
clinical trials involving sickle cell anemia, which are being financed by CIRM
to the tune of $13.1 million. They will also be part of the agency’s Alpha
clinic program.
Steve Peckman, associate director of the UCLA stem cell
center, said the Kohn research “is a perfect example of how CIRM support at all
levels is critical to achieve the goal of driving scientific discoveries to the
clinic.”
Kohn’s work was cited last week at a Los Angeles media event
celebrating CIRM’s 10th birthday. Maria Shriver, the former first lady of
California and whose father died of Alzheimer’s Disease, wrote about the research and linked it glowingly to the stem cell agency in an item on the Huffington Post on Nov.
20. She said,
“The news about 'bubble baby' disease is just the start. I am convinced that stem cell research means we Baby Boomers will be the last generation to have to watch our parents die of Alzheimer's or watch our children die prematurely of sickle cell disease. Proposition 71 (which created the stem cell agency) set this research in motion. Now we have to make sure this research keeps moving forward.”
While CIRM deserves credit for its role in Kohn’s work, it
is only one of 10 entities cited by UCLA as contributing but without providing
figures. Accurately quantifying CIRM’s contribution is
difficult if not impossible. That said, it was significant. The agency’s
programs do ripple out – not only at UCLA but globally. They add to the
bank of knowledge about stem cells, all of which creates an easier path to the
ultimate development of new therapies.
Ironically, one of the CIRM programs -- the "shared labs" -- that helped Kohn has
expired despite pleas by some of the state’s top stem cell scientists. News about that program surfaced again a few days after the Kohn announcement. Without mentioning Kohn, Jeanne
Loring, head of the stem cell program at Scripps, discussed the importance of the "shared labs" in a piece on the blog of UC Davis
stem cell researcher Paul Knoepfler.
Loring said the program has
had “an enormous impact beyond (its) original intention.”
In her “open letter to CIRM,” she said,
“Unwittingly, CIRM’s shared lab program jump-started human stem cell research in California, sending it on a trajectory that has led to stem cell clinical trials in just 6 years….CIRM did not expect that there would be interaction among the labs that would make the whole greater than the sum of the parts. The network of shared labs became our means to communicate and share ideas. It sparked new partnerships between institutions throughout the state, and became a conduit for trainees to move from CIRM’s Bridges internships to graduate school. One scientist described the shared labs as ‘the beating heart of California’s stem cell program.’”
She noted that the program was discussed by the CIRM
governing board in December 2013 at a time when the directors were being told
that the money for new grants would run out in 2017. Today, however, Randy
Mills, the new CIRM president, has reexamined the financial assumptions and
says the money will be around until 2020.
Loring and 11 other researchers are asking the CIRM
governing board at its December board meeting to provide the go-ahead for applications
for another round of funding for what they say is the key maintaining the rapid
pace of stem cell research in California.
Anything less, they say, would impede the development of
stem cell treatments and waste a good portion of the $20 million already spent
on the “shared labs” effort.
Perhaps Donald Kohn would agree as well.
Here is a two-minute CIRM video of Kohn explaining his work.
