Nadia Houri is a Business Development Analyst with the Centre for Commercialization of Regenerative Medicine (CCRM).
Last month, I had the pleasure of attending The Business of Regenerative Medicine: Cells at Work course, hosted by Georgia Tech, in Atlanta. With two and a half very full days of lectures and networking opportunities, cells weren’t the only things working hard. There were many great talks, but due to space constraints I’ll discuss only a few here.
Course founder Dr. Arnold Caplan (Case Western Reserve University) launched the event with a talk on the state of the science of Mesenchymal Stem Cells (MSCs). He spoke about pericytes (cells on capillaries and microvessels) as MSCs in response to injury. In this scenario, pericytes come off the injured blood vessel and become MSCs after which they mediate: 1) an immunomodulatory function as a first line of defense to stop the immune system from surveying and interrogating the tissue left behind, and 2) trophic activity to stop scars from forming. As these activities are distinct from the definition of stem cells, Dr. Caplan proposed that a better name for MSCs is “Medicinal Signaling Cells”.
Dr. Frank Berry (REMEDI, Ireland) presented on the major logistical questions in the “translational roadmap” of MSCs, which were: 1) potency, 2) mechanism of action, 3) immunomodulation, 4) new isolation methods, including selection and scalability, 5) xeno-free culture, and 6) better delivery in patients (targeted, tissue-specific). Dr. Berry also spoke about REMEDI’s development of xeno-free media formulations for supporting MSC growth, two of which are superior to the standard 10% fetal bovine serum (FBS). Due to supply issues and risk of disease transmission, it is important for the field to move towards growth media free of animal origin materials. Also, from an economic perspective, the use of sera can have significant impact on the Cost of Goods Sold (COGS).
VetStem Biopharma stood out as the only veterinary medicine company presenting at the course. At CCRM, our focus is 100% on human therapeutics, so I haven’t been exposed much to the veterinary space in regenerative medicine (RM). However, as the owner of a senior dog, this presentation caught my attention. VetStem’s first product is an autologous cell therapy for the treatment of orthopedic injuries and diseases using adipose-derived stem cells (84% response rate according to founder and CEO Dr. Bob Harman). The company is currently developing an allogeneic, off-the-shelf product for the treatment of canine osteoarthritis and is pursuing FDA approval. This product will be more affordable than the autologous approach (<$1,000 vs. $3,500). With an estimated 10 million dogs in the U.S. suffering from osteoarthritis, and only a small fraction receiving treatment, the company is in a position to benefit from this under-tapped market.
As a neuroscientist, I was excited to see a session on the state of neural stem cell (NSC) science and presentations by companies in the neuroscience space. Dr. Evan Snyder (Sanford-Burnham Medical Research Institute) discussed neurological disease and disability from an NSC perspective (for example, using NSCs to predict therapeutic response and restore neuronal function), as well as the challenges of studying chronic diseases/injuries and recreating their complex environments.
Dr. John Sinden, CSO of ReNeuron, gave an update on the company’s activities and shared some great news for 2015: an ongoing phase II clinical trial in stroke disability following a recent successful phase I trial; FDA approval of an investigational new drug (IND) for a phase I/II clinical trial in retinitis pigmentosa (therapy candidate granted Fast Track designation too); and, a successful fundraising round (£68.4 million).
Dr. Jane Lebkowski, CSO of Asterias Biotherapeutics, presented on AST-OPC1, the company’s allogeneic, embryonic stem cell-derived product consisting mainly of oligodendrocyte and neural progenitors. AST-OPC1 has been shown to produce neurotrophic factors, induce demyelination and induce vascularization. With regards to clinical stage, the most advanced indication for AST-OPC1 is spinal cord injury (SCI) (a phase 1/2a trial is currently enrolling patients with complete cervical SCI). Other indications such as multiple sclerosis and stroke are also being explored in preclinical studies. Neurological diseases, disorders and injuries are devastating to patients and their families, and place a large burden on the health-care system. I look forward to seeing how the promise of RM delivers on curing or mitigating these conditions in the future.
Dr. Dolores Baksh, Innovation Leader, Cell Therapy Technologies at GE Healthcare, gave an engaging talk on the industrial cell therapy manufacturing ecosystem, its challenges, the market opportunity, and GE’s progress towards a “future factory:” a closed, physically integrated, automated and integrated system for cell manufacturing. There are also ongoing efforts at GE Healthcare to embed future factories in the health-care system, linking them to logistics and distribution. To learn more about cell therapy manufacturing and GE’s efforts, I recommend this post by my colleague Mark Curtis.
Dr. Chuck Wilson, President and CEO of immunotherapy company Unum Therapeutics, presented interesting data on the company’s Antibody-Coupled T-Cell Receptor (ACTR) technology. ACTR is a cellular immunotherapy platform combining the benefits of engineered T cells and monoclonal antibodies using constructs that are universal and not limited to a specific set of cell surface targets. The safety of Unum’s antibody-directed T cells can be controlled via antibody dosing. Dr. Wilson described Unum’s vision for curing a wide set of cancers, advancing beyond the safety and efficacy limitations of current generation T-cell therapies, scaling up via industrial manufacturing, and leveraging existing clinical-stage antibodies. As part of that leverage, Unum will be collaborating with Seattle Genetics, Inc. (SGI) to develop ACTR products that incorporate SGI’s antibodies. It is a great time to be in the cancer immunotherapy space. These drugs (mainly checkpoint inhibitors and vaccines) generated $41 billion in 2014 (nearly 50% of the overall oncology drugs market). Cell-based approaches like CAR-T have generated strong efficacy results in clinical trials, and it is exciting to see companies like Unum continuing to innovate on these approaches.
From an investor point-of-view, Chris Fair, Founder of Medtown Ventures, gave a light-hearted talk on how investors evaluate opportunities as well as some pointers for entrepreneurial success. A few quotable words of advice for entrepreneurs:
- The Monet Rule: If you do not have a solid plan on how to spend AND make money, stop and figure it out.
- The Field of Dreams Rule: Just because you made it, doesn’t mean people will buy it. The sales and marketing strategy is as important to value creation as product development.
- The Fight Club Rule: First rule is we don’t talk about our exit. Focus on building a profitable business.
- Self-Awareness Rule: Know what you are good at and own it. Recognize your shortcomings, and hire qualified people to fill those gaps.
Last, but certainly not least, was the Organ-on-a-Chip session. Dr. Geraldine Hamilton, President and CSO of Emulate, Inc., gave an engaging talk on the company’s efforts to identify more predictive and human-relevant models for use in drug development. The development of new drugs is a capital-intensive process, costing between $500 million and $2 billion USD per new chemical entity (NCE). Over the last decade, development times have increased by 20% while the approval rates for NCEs have dropped by 30%, resulting in a stagnant rate of 20-30 new drug approvals per year. Organs-on-a-chip will hopefully mitigate the high failure rate of drugs and the high costs of R&D. Dr. Hamilton presented data on some of the organs-on-a-chip in development at Emulate, including airway-on-chip, gut-on-chip, and thrombosis-on-chip. For example, a lung model was used to demonstrate white blood cells’ response to bacterial infection. Emulate has collaborated with various pharmaceutical companies, who like to see models that correlate well with human physiology, an advantage that the company believes it has.
Beyond the great talks, I enjoyed the intimate atmosphere of the course, which allowed for ample opportunity to get to know other attendees. As someone who is relatively new to the exciting world of RM, I appreciated the opportunity to interact with some of the brightest minds in the field.
Next year’s Business of Regenerative Medicine course will be hosted by the Harvard Stem Cell Institute (HSCI) in partnership with Case Western, Georgia Tech and CCRM. See you there!
 Kevin Stafford, The Welfare of Dogs (2006).
 Mapping Connections: An Understanding of Neurological Conditions in Canada (2014). http://www.phac-aspc.gc.ca/publicat/cd-mc/mc-ec/assets/pdf/mc-ec-eng.pdf
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 Market Report: New Insights into Toxicity and Drug Testing, InTech (2013).