September 19, 2019

The road ahead for #regenmed

This fall, it will be 15 years since regenerative medicine faced a crisis of funding and support in the United States — and the public stood up to pave a path forward. Now, at this milestone, it’s time to look ahead, and think about how we continue to deliver on that vote of confidence.

In 2004, federal lawmakers were threatening to put severe legal and funding restrictions on stem cell research. California voters, in response, passed the landmark California Stem Cell Research and Cures Act, creating a $3 billion dollar fund and a state oversight agency, the California Institute for Regenerative Medicine. I was honored to co-author that initiative and help steer its passage, and then serve as the first CIRM Chief of Staff. 

When we set out to protect advanced therapies, we couldn’t foresee all the ways in which regenerative medicine would expand. Now, the field includes cell therapies, gene therapies, and cancer vaccines. There are two approved CAR-T cell therapies, approved gene therapies for beta-thalassemia and hereditary blindness, and more than 1,060 clinical trials underway world-wide. (For more detail on the scientific progress since Prop. 71, please see a recent guest op-ed I authored in DDNews.)

But new challenges have arisen as well. At last week’s CAR-TCR Summit conference in Boston, a frequent topic of conversation was “The road ahead is hard.” Impressive progress on the scientific side is inspiring — and has also outpaced adoption of supporting technologies. I was fortunate to have the chance to join one of Vineti’s ecosystem partners, Lonza Pharma & Biotech, in a presentation to discuss one solution — how a pre-built integration between our systems will support scalable cell therapy manufacturing that opens up access for more patients.

And at an open house this week at innovation powerhouse Allogene Therapeutics, excitement about the development of allogeneic, donor-based cell therapies was mixed with discussion about how best to deliver them. With autologous, patient-based therapies up and running, the ecosystem now needs to expand further for allogeneic approaches and the workflows they require.

How does the sector keep growing? Fundamentally, we all need to focus on our core competencies and work together if regenerative medicine is to continue to expand. An entire infrastructure is being laid to industrialize this new, personalized medical landscape that is truly regenerative and patient-specific. Vineti’s software platform for personalized therapeutics is a key piece of this foundation.

In my DDNews piece, I outlined a few recommendations, including:

• “Standards. Without standardization, we cannot industrialize or scale regenerative medicine. Each patient has her own unique biology, and each therapeutic approach brings innovations and nuances. But underlying these essential differences are critical harmonizing drivers, such as digital infrastructure, industry guidelines, and regulations, that will make these therapies more predictable, repeatable, and scalable. The Standards Coordinating Body, an independent non-profit organization doing FDA-funded work in this area, can be looked to as a source of information, standards development, collaboration, and progress.
• Integration. For the regenerative medicine ecosystem to flourish, it must truly operate as an ecosystem. In a patient-centric value chain, all parts must be connected to provide timely, reliable, cost-effective care. We all must work together to enable progress, and we must connect our systems to operate this ecosystem on behalf of as many patients as possible.
• Outreach. Other industries offer valuable best practices to draw from. Digital innovation in other industries, for example, offers critical lessons on simplicity and usability that are already helping make cell and gene therapy operations and delivery simpler and more reliable for healthcare providers. As our field matures, we should actively collaborate with other industries that have already mastered scale.”

In 2004, we were just getting started. In the next 15 years, we need to take regenerative medicine mainstream. As author Siddhartha Mukherjee noted recently in The New Yorker, this will require pursuing kaizen, or a mindset of continuous improvement,  in balance with scientific and medical innovation.  

Thank you for reading. You can learn more about Vineti and how our software solution supports advanced therapies here. The Vineti team and I are excited to be a part of regenerative medicine’s next 15 years!

Amy DuRoss is the CEO and Co-founder of Vineti. If you’d like to learn more about how Vineti’s Personalized Therapy Management (PTM) is helping to standardize and industrialize workflows for personalized therapies, please contact us.