October 2007 Stem Cell Summit in Boston
Cosponsored by HSCI, the Stem Cell Summit took place October 2–3 at the Hynes Auditorium in Boston. Cosponsors included the Genetics Policy Institute and the Burrill Life Sciences Media Group. In addition to formal presentations, breakfast and luncheon roundtable discussions as well as poster sessions and exhibits by organizations in the field extended the breadth and depth of information available. The audience included advocates and patients, private-sector companies, scientists, and professionals.
Almost 100 speakers, many of them HSCI-affiliated investigators as well as experts from around the world, presented on topics such as the future of stem cell research, philanthropic giving, and collaboration from the vantage point of research hospitals. Panel presentations provided an array of perspectives including representatives from venture capital firms, biotechnology and legal professionals. Attendees could opt for breakout sessions in six disease areas. To achieve a holistic view of a disease area, each discussion included a scientist, a clinician, a disease foundation representative, and a patient advocate. The highlights from two discussions follow.
Summit Breakout Session – Spinal Cord Injury and Paralysis
At a roundtable discussion on spinal cord injury and paralysis, the center aisle was nearly filled with people in wheelchairs. On the dais, about a dozen bottles of pills sat on the table in front of activist Danny Heumann, who was paralyzed from the waist down in a car accident 22 years ago. He related how he depends on the contents of those bottles. He delivered a message to doctors and researchers that was deeply personal: “My goal is to get sensation back.”
All agreed that even that seemingly simple goal will be difficult. Moderator Jeffrey Macklis, MD, PhD, who studies nervous system development and directs HSCI’s Nervous System Diseases program, and John Kessler, MD, director of the Northwestern University Feinberg School of Medicine Stem Cell Institute, outlined how restoring function and sensation following a spinal cord injury is likely to involve repair and regeneration of not only nerve cells but also supporting cells and nonliving extracellular scaffolding. This has the potential to restore an environment that will allow surviving fibers to conduct signals again and transected nerves to grow through the injured area.
Stem cells, embryonic and adult, were seen as important tools in understanding how nervous system cells respond to injury. Kessler cautioned that stem cells, manipulated in culture and then transplanted into patients, were very unlikely to be a “magic bullet” cure that would undo the effects of trauma. “The notoriety of stem cells is actually a problem,” said Peter Kiernan III, chairman of the board of the Christopher and Dana Reeve Foundation, because the hype over stem cells has raised unreasonable expectations.
Faced with these daunting obstacles to complete cures, the concept of a pragmatic cure moved to the fore. “Pragmatic cures are easier,” said John McDonald III, MD, PhD, executive vice president and director of the International Center for Spinal Cord Injury in Baltimore, the world’s first program dedicated to spinal cord injuries in children. He pointed out that if only 20 percent of neurons remain to cross the injury gap, movement or sensation can be preserved. Kiernan reported that the Reeve Foundation is working to find the common denominators of injury, impairment, recovery, and function that are needed to evaluate clinical trials.
To achieve excellence, [Harvard's research] institutions must work hard to create a culture in which teaching programs are viewed as a top priority. Harvard's new interschool Department of Stem Cell and Regenerative Biology is presented with that opportunity.
Despite the many reasons to be pessimistic about finding near term stem-cell-derived treatments for spinal cord injury and paralysis, the discussion ended more hopefully. The encouraging outcome of football player Kevin Everett’s spinal injury during an NFL game in September demonstrated that when treatments are applied in the right way, at the right time, they can be effective at staving off paralysis. Panelists urged patients to challenge obsolete assumptions about the unlikelihood of recovery. “You’d better stay in shape,” said Heumann, who bikes and plays tennis. Added Kiernan, “Don’t write yourself off.”
Stem Cell Research and Opportunity from a Hospital Perspective
Leading off the summit’s second day, a panel of Boston-area research hospital CEOs reflected on competition and collaboration, two essential and inevitable features of stem cell science. David Scadden, MD, HSCI codirector, Harvard professor, and a practicing hematologist/oncologist at Massachusetts General Hospital (MGH), moderated a lively dialogue, as he attempted to tease out answers to difficult questions. He began with the theme of collaboration.
MGH CEO Peter Slavin pointed to the upside of collaboration “but also the temptation to defect,” as he put it, to much laughter from the audience. Factors favoring collaboration include federal funding priorities and the recent California ballot initiative (establishing a state-funded stem cell institute). California sent a lightning bolt, as Slavin put it, “threatening the strategic advantage we have had in stem cell [research].” Collaboration also creates positive conditions for recruiting top scientists. He commented that HSCI has been integral to the creation of research programs across Harvard-affiliated institutions, something that has never occurred before. He acknowledged that donors favor multi-institutional collaboration. But, Slavin cautioned, a collaboration can be fragile, as in any type of relationship.
Gary Gottlieb, CEO of Boston-based Brigham and Women’s Hospital, underscored funding needs. “We need visionary donors,” he said. The huge price tag of stem cell science from the bench to the marketplace is evident to all. Gottlieb commented that generous donors want to know “Why Harvard?” He added that HSCI has raised the visibility of the depth and breadth of research across Harvard. He encouraged participation by all parts of the university and affiliated hospitals, remarking that “big” science is the priority for the next decade.
Scadden noted there would obviously be multiple claims on intellectual property emerging from scientific collaboration. He asked panelists to envision a system in which all institutions benefit. All agreed that one conversation, not many conversations, with commercial interests would be an efficiency. Children’s Hospital Boston CEO James Mandell pointed out that each institution needs to maintain its own scientific oversight; Children’s, like other institutions, also has a rigorous separate and internal ethics committee focused on stem cell research. Brock Reeve, HSCI executive director, reminded the panel, “We want to be sure, even if committees need to be separate, that we coordinate so that no one group reinvents. We can achieve economies of learning and economies of scale by doing this.”
Panelists considered the pervasive issue of safety. Edward Benz, CEO of Boston-based Dana Farber Cancer Institute commented, “In cancer, where we have a long history of experience with bone marrow transplant, major immunological issues emerge around grafting. Secondly, we can’t target therapy until we thoroughly understand how the daughters of stem cells are formed. Generally from a safety perspective, the therapeutic benefit ratio to the risk would be narrow for any therapy. One must be sure the trial is addressing the questions that justify the inherent risk in the trial.”
The hospital leaders affirmed the value of educating young investigators but noted the conflicting demands on scholars around promotion as measured by published papers, funding, and the translation of new discoveries. To achieve excellence, institutions must work hard to create a culture in which teaching programs are viewed as a top priority. Harvard’s new interschool Department of Stem Cell and Regenerative Biology is presented with that opportunity.
Benz pointed out the long road between the lab and the clinic. Institutions must tap and ally with biotechnology companies that have critical resources such as libraries of antibodies and chemical compounds. Mandell pointed out the need for teamwork and for seeking scientists with an entrepreneurial spirit. Slavin termed this being “bilingual in the private business sector and in science.”
This theme was subsequently picked up by the summit’s concluding commentary by experts on the interplay between global regulation and global research collaboration. The broad and ambitious meeting, with HSCI as a galvanizing force, reflects the maturity of the collaborative enterprise at Harvard University and its schools, research centers, and affiliated hospitals. Built on last year’s inaugural summit meeting, the two days of lectures and panel presentations captured some of the new and still-evolving issues at the nexus of policy, stem cell-based treatment of disease, and the patient’s perspective.