San Diego-based project gets $2.4 million from CIRM for preclinical work
Mike Abrams, who has Parkinson's disease, hikes the strenuous Clevenger Canyon Trail near Ramona with the non-profit group Summit for Stem Cell. — Charlie Neuman
Advancing a years-long quest for a stem cell treatment for Parkinson's disease, a San Diego nonprofit has received a $2.4 million grant from California's stem cell agency.
The group, Summit for Stem Cell, proposes to replace brain cells destroyed by Parkinson's with replacement brain cells. These cells make the neurotransmitter dopamine, which enables movement. Success could relieve symptoms for many years, giving patients a normal life.
Cheers erupted from group members after the California Institute for Regenerative Medicine's governing board approved the funding and other research grants at its meeting Thursday in La Jolla. California voters created CIRM in 2004 under Proposition 71, appropriating $3 billion in bond money to fund its work.
"I've wanted to believe this was going to happen for so long," said group member Cassandra Peters, who has Parkinson's.
"Today, I'm going to go home, look in the mirror, and say, 'Parkinson's, your days are numbered in this house."
The grant enables the group to get near the point where it can ask regulatory permission to begin clinical trials, said Scripps Health neurologist Dr. Melissa Houser, who leads clinical development. Stem cell researcher Jeanne Loring of The Scripps Research Institute leads the basic research.
Summit for Stem Cell is a volunteer non-profit partnership including patients, patient advocates and scientists. It is led by scientists and and medical professionals from Scripps Health and The Scripps Research Institute. It can be reached at summit4stemcell.org.
The nonprofit has raised money on its own since 2011 with fund-raising events such as climbs to Mt. Kilimanjaro and Base Camp at Mt. Everest. Its members, including some Parkinson's patients, are training to climb Machu Picchu in Peru this October.
With the funds and volunteer support, researchers have created the replacement neurons, derived from artificial stem cells called induced pluripotent stem cells. Those IPS cells were grown from the Parkinson's patients themselves. Because the cells came from the patients to be treated, they are genetically matched, which should reduce the risk of immune rejection.
Safety testing of the transplants have been performed in animals, but more work is needed to satisfy concerns of the U. S. Food and Drug Administration, Houser said. The group has had very early discussions with the FDA, and more discussions lie ahead before the group can file an application asking authorization.
"CIRM has granted us the ability to further refine our cells, make sure our cell lines are pure, and get them to the point where we feel they're safe for implantation into the human brain," Houser said. “We will get to the point with the FDA where we will find out what we need to guarantee safety.”
If all goes well, the group will be ready to file an application in about a year, she said. More money will be needed, and the group will be fund-raising the whole time, as with the Machu Picchu expedition. Members of that expedition must pay their own way, in addition to raising funds for the group.
After the CIRM meeting, the group met with its president, C. Randal Mills, who reassured members that the agency is committed to helping the Parkinson’s project succeed.
Mills responded to a question from TSRI’s Loring, who asked how CIRM could better inform funding applicants about what is needed to meet the FDA’s criteria.
“It would be great if we could have more advice ... if we could get feedback from other people who have had different views of the FDA, for example,” Loring said.
Mills replied that the group will get that advice, now that it’s a CIRM-funded program.
“The good news is now, we can,” Mills said. “We’re not a consulting agency to everyone. That’s not a possible thing for us to do. But for the programs that are ours -- they’re ours. We’re all in on that.”
CIRM has internal milestones based on how well its funded programs progress, Mills said.
“Once you get in, we want you to go,” he said.
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