Aspen Neuroscience, a new San Diego biotech company working on stem cell treatment for Parkinson’s disease, has come out of stealth mode and raised $6.5 million to pursue clinical testing for its therapy.
Co-founded by well-known stem cell scientist Jeanne Loring, Aspen Neuroscience proposes creating stem cells from modified skin cells of Parkinson’s patents via genetic engineering.
The stem cells, which can become any type of cell in the body, then would undergo a process that makes them specialize into dopamine-releasing neurons.
People with Parkinson’s lose a large number— up to 50 percent at diagnosis — of specific brain cells that make the chemical dopamine.
Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems.
Once these modified skin cells have been engineered to specialize in producing dopamine, they can be transplanted into the Parkinson’s patient to restore the types of neurons lost to the disease.
“The reason we called it Aspen is because l was raised in the Rocky Mountain states,” said Loring. “When there is a forest fire in the Rockies, the evergreens are wiped out but the aspens are the fist that regenerate after the burn. So it is a metaphor for regeneration.”
Aspen still has a long way to go before its proposed therapy would be available to Parkinson’s patients. It has been meeting with the U.S. Food and Drug Administration to provide animal trial data and other information in hopes of getting permission to start human clinical trials.
But the company expects the earliest it would get the go-ahead from FDA to start human trials would be 2021.
Loring has been working on the therapy for eight years. She is professor emeritus and founding director of the Center for Regenerative Medicine at the Scripps Research Institute.
Loring co-founded the 20-employee company with Andres Bratt-Leal, a former post-doctoral researcher in Loring’s lab at Scripps.
Joining them as Aspen’s Chief Executive is Dr. Howard Federoff, former vice chancellor for health affairs and chief executive of the University of California Irvine Health System.
Federoff said the company is the only one pursuing the use of Parkinson’s patient’s own cells as part of neuron replacement therapy.
Aspen’s proprietary approach does not require the use of immuno-suppression drugs, which can be given when transplanted cells come from another person and perhaps limit the effectiveness of the treatment.
Aspen’s approach “is a therapy that is likely to benefit from the fact that your own cells know how to make the best connections with their own target cells in the brain, even in the setting of Parkinson’s disease,” said Federoff. “So when transplanted it is able to set back the clock on Parkinson’s.”
In addition to Aspen’s main therapy, it is researching a gene-editing treatment for forms of Parkinson’s common in certain families.
Aspen’s research work up to now has been supported by Summit for Stem Cell, a non-profit on which provides a variety of services for people with Parkinson’s disease.
The new seed funding round was led by Domain Associates and Axon Ventures, with additional participation from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32.
“Aspen’s financial backing, combined with its experienced and proven leadership team, positions it well for future success,” said Kim Kamdar, a partner at Domain Associates. “Domain prides itself on investing in companies that can translate scientific research into innovative medicines and therapies that make a difference in people’s lives. We clearly see Aspen as fitting into that category, as it is the only company using a patient’s own cells for replacement therapy in Parkinson’s disease.”
