When the biotechnology trade association began holding annual conferences in 1993, they drew 1,400 to the first event. This year BIO International expected nearly 20,000 here in San Diego. Among the dozens of concurrent sessions each day of this four-day scramble, stem cells got one track on one day this year. But listening to the progress being made by our presenters yesterday, our field is set to grow at the pace this meeting has—and could dominate the medical sessions here within the next decade.
After setting the scene with our opening panel yesterday, four subsequent panels confirmed the vast near-term potential painted by the opening speakers. They revealed a field maturing rapidly and starting to be a valued research tool of the bigger companies that have dominated the biotech industry, at the same time it is starting to deliver therapies to patients.
The second panel displayed the robust power of stem cells to model disease better than animal models ever could. These cells also let researchers dive much deeper into the genetic causes of disease, particularly diseases with multiple genes involved. Anne Bang from the Sanford-Burnham Institute mentioned her role in a consortium organized by the National Institutes of Health that is looking at the many genes involved in a type of heart weakening called left ventricular hypertrophy. Because different ethnicities tend to respond differently to drugs used for the condition, the consortium teams are creating iPS-type stem cell lines from 125 Caucasian patients and 125 African-American patients with various forms of the condition.
Their goal is to personalize and improve therapy across both patients groups. The way cells behave in the lab can tell the researchers much more relevant information than most animal models, so drugs developed based off their discoveries should have a better chance of success. All four panelists agreed that the field needs enough drugs developed with these tools to show that they do indeed have a better success rate. That track record should start to develop over the next few years.
The third panel talked about the shift in the medical mindset that will happen when genetically modified stem cells can change the care of chronic diseases from daily therapy to cures. Louis Bretton of Calimmune discussed how his company is trying to do this for HIV, which we blogged about yesterday when they announced promising first phase results from their first four patients. Faraz Ali of bluebird bio showed that his company has already made this life-changing shift for two patients with the blood disorder Beta Thalassemia. Like most patients with the disease they had been dependent on regular transfusions to survive, but when they received transplants of their own stem cells genetically modified to produce the correct version of a protein that is defective in the disease, they were able to live without transfusions.
The fourth panel provided proof that the field is maturing in that they discussed the many hurdles and pitfalls in taking those final steps to prepare a cell therapy to be a commercial product. The three big hurdles—financing, regulatory approval and reimbursement by insurers—all required creativity by the companies outlined in the two case studies. They are working through them but it is anything but a straightforward path. This is the area I hear the most hand wringing about in the halls of meetings in our field.
The last panel showed that one way around some of those end stage hurdles is to reach across borders. Four panelists discussed specific examples of ways international collaborations have accelerated their work toward developing therapies. CIRM has more than 20 collaborative agreements with funding agencies around the world, many of them painstakingly nurtured by our former president Alan Trounson. He gave the final presentation of the panel talking about one of his new projects, building an international stem cell bank with enough cell lines that almost everyone could get donor cells that were immunologically matched.
Our board chair, Jonathan Thomas, moderated the last panel and ended with a tribute to Alan noting that his build-out of our international program would be one of his many lasting legacies.
Don Gibbons
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