Seeking to speed its stem cell-based Parkinson’s therapy to patients, a San Diego nonprofit has moved out of a lab where it has incubated for the last several years.
Summit for Stem Cell, an alliance of patients, scientists, doctors and supporters, had been working out of The Scripps Research Institute. This week, the nonprofit is moving into newly subleased space nearby.
The new location on Torrey Pines Mesa brings scientific and financial advantages as the group prepares to apply for clinical testing.
The group has leased about 5,000 square feet, doubling its space, and as a parting gift received nearly $1 million in donated equipment from Scripps Research. At the same time, its overhead is dramatically reduced, said Jeanne Loring, the group’s lead scientist and professor at Scripps Research.
A $2.4 million grant the group received from California’s stem cell agency ran out earlier this year, Loring said. Now the group can step up fundraising and add staff. It’s also soliciting more donated equipment.
Assuming all goes well, Summit for Stem Cell expects to get federal authorization to try its therapy in about 18 months, Loring said. Volunteer patients taking part in the effort will be treated first. If the therapy proves effective, the group plans to make it available to other Parkinson’s patients.
The group also plans to establish a for-profit company to get funding from investors interested in generating returns, Loring said. By drawing investments as well as philanthropy, the project gains access to capital not otherwise available.
The group was inspired by the example of the Cystic Fibrosis Foundation, Loring said. In 2000, the foundation gave San Diego’s Aurora Biosciences $40 million to develop a cystic fibrosis drug.
Aurora was later acquired by Vertex Pharmaceuticals, which turned Aurora into its San Diego operations. In 2000, Vertex received approval from the Food and Drug Administration for Kalydeco, the first drug to treat the underlying genetic cause of the disease.
Summit for Stem Cell’s goal is to directly fix the cause of Parkinson’s disease. The movement disorder arises from the destruction of brain cells that secrete the neurotransmitter dopamine.
Using stem cell technology, replacement dopamine-making brain cells can be produced directly from patients. This procedure is now refined enough that high-quality cells genetically compatible with patients can be made at will, Loring said.
But these cells must be surgically transplanted into the brains of patients, requiring approval from the Food and Drug Administration. It also takes millions to complete the required preclinical safety work and for long-term observation and care of treated patients.
Raising that money has been difficult, in large part because of the overhead issue, Loring said.
Scripps Research imposes overhead of up to 94 percent, meaning that every dollar donated to the group required an additional 94 cents for the institute, Loring said. At the new location close by on Torrey Pines Mesa, overhead is roughly 10 percent.
Lower overhead means the group can now apply for philanthropic grants that previously weren’t practical, she said, because donors prefer as much as their money as possible go directly to the cause.
For more information about the group, go to https://www.summitforstemcell.org or call 858-759-1610.
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