Moving Beyond Current CIRM Funding

Delivering on CIRM’s mission of “accelerating stem cell treatments to patients with unmet medical needs” requires multiple stakeholders across the research, development, and commercialization phases of bringing a new product to market. In this post, I am pleased to highlight two recent examples of CIRM-funded projects that have moved beyond CIRM funding by partnering with industry and investors to further develop the underlying technology.

Enhancing healing

In 2000, Dr. Jill Helms, an academic investigator at Stanford University, received a $6.5 million CIRM Early Translational grant. Dr. Helms’ project was titled “Enhancing Healing via Wnt-Protein Mediated Activation of Endogenous Stem Cells,” and the award aimed to develop a novel, protein-based therapeutic platform to accelerate and enhance tissue regeneration by activating adult stem cells. The five-year award achieved several critical milestones, including initiating two preclinical studies to demonstrate the effectiveness of a protein called L-WNT3A in improving spinal fusion surgery success and treating a degenerative bone disease called osteonecrosis, both of which represent unmet medical needs.

Helms_bonegraft
Through CIRM funding, Dr. Jill Helms’ team was able to demonstrate that treatment with a protein called L-Wnt3a regenerates and promotes bone formation in animals models (Figs D,F: untreated; Figs E,G: Wnt3a treated). (image credit: Leucht et al. J Bone Joint Surg Am. 2013;95:1278-88)

Dr. Helms’ work attracted considerable interest from the investor community during her grant, and in the final year of the award, Dr. Helms’ WNT3A technology platform spun out of Stanford into a new company called Ankasa Regenerative Therapeutics. Avalon Ventures, a La Jolla-based life sciences venture capital firm; Correlation Ventures, an analytics-driven venture capital firm; and Heraeus Medical, a diversified global medical device company based in Germany with over $1 billion in annual revenue, provided financial support. Ankasa has raised $8.5 million in the first round of its $17 million Series A financing to continue developing the previously CIRM-funded technology.

Transplanting stem cells in the brain

The second recent example comes from a CIRM Tools & Technology grant to Dr. Daniel Lim, a neurosurgeon at UCSF. Dr. Lim was awarded a $1.8 million grant to develop a more efficient device for transplanting stem cells into the brain, titled “Development and Preclinical Testing of New Devices for Cell Transplantation to the Brain.” Dr. Lim developed a platform technology that enables Radially Branched Deployment (RBD) of cells to multiple target locations at variable radial distances and depths along the initial brain penetration tract, guided by real-time interventional magnetic resonance imaging (iMRI). This technology is a major leap forward from the conventional, crude syringe-and-needle device typically used to inject living cells into the brain.

Moving Radially Branched Deployment_Neurosurgery_Lim
Dr. Daniel Lim’s CIRM-funded BranchPoint Device allows neurosurgeons to deliver cell-based therapies to multiple areas of the brain with just one needle penetration.  (image credit: Silvestrini et al. Stereotact Funct Neurosurg 2013;91:92–103)

Dr. Lim’s work drew Accurexa’s attention. It’s a publicly traded medical device company that licensed the CIRM‑funded technology from UCSF. Under Accurexa’s guidance, the team submitted a 510(k) application to the FDA for the newly named BranchPoint Device. In June, Accurexa raised $2.5 million in equity financing to continue developing and commercializing the device.

Early‑stage stem cell technologies often lack strong industry pull. However, the stories of Drs. Helms and Lim show how CIRM funding can support early research that later attracts investors and commercial partners. These new investors can further develop and advance the technologies beyond CIRM’s early support and, if successful, deliver them to patients with unmet medical needs.

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  1. Pingback: Pushing, pulling and dragging stem cell research forward | The Stem Cellar

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