A UCSF spinout is growing neuronal stem cells to transplant into the brain, for potential use in treating epilepsy, spinal cord injury, Parkinson’s and Alzheimer’s disease – and investors are listening. Because one thing that differentiates Neurona Therapeutics is that its stem cells turn exclusively into interneuron cells – which are less likely to be tumorigenic than other IPS cells.
The company has raised $7.6 million of a proposed $24.3 million round, according to a regulatory filing. But the company’s staying a touch under the radar – it lacks a website, and ’tis the season for calls to the company to remain unanswered.
But funding for the six-year-old company comes from 11 investors. Listed on the document’s contact pages are Tim Kutzkey and David Goeddel, both partners at early stage healthcare venture firm The Column Group – giving some insight into who the startup’s investors are.
Also listed is Leo Guthart, a managing partner at New York private equity firm TopSpin Partner, and Arnold Kriegstein, director of the UCSF developmental and stem cell biology program.
Kriegstein and his UCSF colleagues filed a patent for the in vitro production of medial ganglionic eminence (MGE) precursor cells – which are, in essence, immature cells that morph into nerve cells. The work that led to the patent was funded by the California Institute of Regenerative Medicine, the NIH and the Osher Foundation.
“We think this one type of cell may be useful in treating several types of neurodevelopmental and neurodegenerative disorders in a targeted way,” Kriegstein said in a UCSF statement last year.
Neurona Therapeutics’ scientific backers collaborated on a paper on these MGE cells in Cell Stem Cell, finding that mouse models closely mimicked human cells in neural cell development – and that human cells can successfully be transplanted into mouse brains. UCSF writes:
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Kriegstein sees MGE cells as a potential treatment to better control nerve circuits that become overactive in certain neurological disorders. Unlike other neural stem cells that can form many cell types — and that may potentially be less controllable as a consequence — most MGE cells are restricted to producing a type of cell called an interneuron. Interneurons integrate into the brain and provide controlled inhibition to balance the activity of nerve circuits.
To generate MGE cells in the lab, the researchers reliably directed the differentiation of human pluripotent stem cells — either human embryonic stem cells or induced pluripotent stem cells derived from human skin. These two kinds of stem cells have virtually unlimited potential to become any human cell type. When transplanted into a strain of mice that does not reject human tissue, the human MGE-like cells survived within the rodent forebrain, integrated into the brain by forming connections with rodent nerve cells, and matured into specialized subtypes of interneurons.
