By Lisa Brown
In early April recent immigrants to the Boston area benefited from a community outreach effort led by the Boston University chapter of the American Student Dental Association (ASDA) and the Global & Population Health office at Boston University Henry M. Goldman School of Dental Medicine (GSDM).
The Notre Dame Educational Center (NDEC) Dental Health Fair offered health promotion, education, age appropriate activities, and referrals to NDEC students. Volunteers gave oral health screenings to 26 attendees of the fair. The event was organized by ASDA Community Outreach Co-Chairs Ingy Alhelawe DMD 15 and Neelam Shah DMD 14, in collaboration with Oral Health Promotion Director Kathy Lituri. GSDM volunteers have held this event biannually for several years.
Founded in 1860, NDEC is a comprehensive adult educational center, which offers many resources for immigrants. Among the programs offered by NDEC are English Speakers of Other Languages (ESOL) classes, a high school diploma program, and a literacy program. They also offer support services, such as career counseling, immigration counseling, tutoring services, and education counseling.
“It was a great event with many participants from NDEC and ASDA volunteers,” said Shah.
The GSDM volunteers were: Ingy Alhelawe DMD 15, Sana Banday AS 15, Yousef Behbehani DMD 16, Rubbiya Charania DMD 16, Justine Karanian DMD 15, Shireen Khan AS 15, Michael Lee DMD 15, Linda Linsinbigler DMD 15, Kathy Lituri, Monica Schmidt DMD 17, Neelam Shah DMD 14, and Jake Ward (pre-dental).
Dean Jeffrey W. Hutter said, “Thank you to Kathy Lituri and all the volunteers that worked to make the School’s outreach at the Notre Dame Educational Center an enduring success.” He continued, “It is through efforts like this that the Henry M. Goldman School of Dental Medicine succeeds in its mission of excellence in community service.”
Submitted by GSDM Communications
On Saturday, May 17, members of the BUSM Class of 2014 gathered to celebrate their graduation from medical school. Congratulations, Grads! View the pics on Facebook
The 167th Boston University School of Medicine commencement opened with an academic procession of 300 members of the faculty lining up on either side of the Class of 2014 to welcome them into the community of scientists and physicians. The ceremony, held at the Agganis arena on May 17, was a celebration of achievement and commitment to medical research and caring for patients.
“We gather together today to publically recognize and celebrate the credentials that these degree candidates have earned, a major life transition,” said Dean Karen Antman, MD. “I speak for the faculty in saying that it has been a great privilege to work with you. You are smart and committed, resilient and adaptive.”
The degrees earned by the Class of 2014 included 52 PhDs, 157 MDs, 12 MD-PhDs, 10 MD-MPHs, and six graduates who received an MSc.
“It has been said that ‘life is what happens when you are otherwise making plans,’” said U.S. Assistant Secretary of Defense for Health Affairs Jonathan Woodson, MD, who delivered the commencement address. “I have found this to be true time and again, and it expresses the need in life to expect the unexpected. Each of you should be prepared to negotiate around obstacles even as you keep your eye on the prize or the long range goals you have set for yourself. It is important to understand that deviations are the friction points in life when we learn the most about ourselves and we grow.
Woodson asked the parents of the graduates to stand to be recognized for their support and dedication that helped make their children’s accomplishments possible.
Speaking for her fellow doctoral students, Ariana Harris, PhD, noted, “To solve the mysteries of the world, we need each and everyone one of us to contribute. In a little while we will received our academic hoods and you will hear the titles of our individual projects, getting a sense of how different they are. This is what makes the scientific community amazing. There is so much to learn and so many of us are eager to figure it out. I am confident that whatever careers we pursue as individuals, our thirst for knowledge will continue. We use our scientific training every day. We search for logic, reason and evidence that support our thoughts and beliefs.”
Referring to the white coats he and his classmates received upon entering BUSM, Brian Curry, MD, speaking on behalf of the medical students, said, “We received these as a symbol of our induction to a calling. We didn’t realize it at the time, they also symbolized the granting of a very special kind of power. One that, just like our white coats themselves, we have spent the better part of four years clumsily trying to grow into and will likely continue to do for the rest of our careers. My message to you, BUSM class of 2014, is simply this: Earn this power. Earn it, but recognize we will never own it. We only can ever be responsible stewards of this power our patients have entrusted to us. Though today marks the retiring of these short white coats, we should never allow ourselves to get fat and happy with the notion that we have somehow grown into them. We must earn it anew every day, with every patient, always.”
On Friday, May 16, the BUSM GMS Class of 2014 gathered in Metcalf Hall to celebrate commencement. See them on BUSM Facebook!
Celebrating Success! Division of Graduate Medical Sciences 2014 Commencement
“Your degree empowers you not just as scientists, researchers, clinicians, and health professionals but as critical thinkers, problem solvers and advocates,” said Associate Provost for the Division of Graduate Medical Sciences (GMS) Linda Hyman, PhD, to the 180 master’s degree graduates of 19 of the division’s programs. “This is a great day and we, your parents, friends, colleagues, teachers and mentors are here to celebrate you – your success, hard work, accomplishments, and your efforts to get to where you are today. I hope you leave BU with the knowledge that you have done well, and that you have the confidence to use your talents widely.”
The commencement ceremony, held on May 16 in Metcalf Hall of the George Sherman Union, featured three student speakers. “It has been an honor to be a part of this community of healing these past two years,” Jonathan Waldo, earning a master’s degree in Mental Health Counseling and Behavioral Medicine, told his fellow GMS graduates. “In reflecting on the nature of our field, I’ve recognized that when we invest our time in the lives of others we often gain insight into our own lives, our struggles, our hopes and our strengths. Our horizon beckons, and we are prepared.”
Brian Fry, receiving his Master of Arts in Medical Sciences degree, noted that, “As we move forward with the degrees we have earned today, we’re going to be a part of a rapidly changing landscape of health care, health policy and health sciences research. Many of the people and organizations that will desperately need to change will also be the most resistant to that change. It will be up to us to inspire action and lead by example. It is my hope that we welcome change and personal growth by chasing future, better versions of ourselves. We owe it to our future patients, clients and colleagues to never settle for anything but our very best.”
One of seven graduates of the GMS Bioimaging program and an accepted GMS doctoral student for the fall of 2014, Lauren Zajac reflected on her belief that art and science are the same, noting that in the Bioimaging program students work with images. “We behave as critics, curators, and artists. Interpreting a medical or biological image requires a set of skills similar to those required to interpret a work of art. Both artistic and biological images are representations, carry information and are the products of human experimentation. The process of creating art is a science, and the scientific pursuit of a question is truly an art. The two are intertwined and inseparable,” she said. “Great artists develop methods to produce the works we see in a gallery or museum. In a similar way, great scientists, through trial and error, develop methods to produce work that beautifully demonstrates a particular idea or model.”
Boston University’s Schools of Medicine, Public Health, Dental Medicine and the Division of Graduate Medical Sciences are proudly sponsoring the John McCahan Medical Campus Education Day on Thursday, May 22, 8:30 a.m.-3:30 p.m. in the BUSM Instructional Building, Hiebert Lounge 14th Floor.
“Education Day is a fantastic opportunity to meet and network with innovative educators from across the entire medical campus,” explains BUSM Gross Anatomy Course Director Ann Zumwalt, PhD, who also serves as chairman of the event planning committee. ”The event showcases creative educational initiatives and is consistently inspiring and invigorating for BUMC educators.”
The purpose of the event is to showcase and enhance educational innovations, scholarship and research across the Boston University Medical Campus. All faculty, students, residents, fellows and staff of BUMC are invited to attend.
Register online: http://www.bumc.bu.edu/jmedday/ Registration by May 22 is required for workshops and lunch.
Keynote Lecture: (8:45 a.m.)
The Art and Science of Giving Feedback
Carole Pfeiffer, PhD
University of Connecticut Health Center
Dr. Pfeiffer is a Professor of Medicine and a sociologist who has worked in medical education for three decades. She is the Director of the Clinical Skills Assessment Program at the University of Connecticut School of Medicine and was a founding member of the Association of Standardized Patient Educators. She has a particular interest in communication skills in the clinical encounter.
Poster Session: Posters focus on themes of educational innovations, research and technology. Oral presentations and awards will be given for outstanding student, fellow/resident and faculty abstracts
Luncheon: Pre-registered attendees will be provided lunch. Register
Faculty Workshops: Pre-Registration required. Participants will learn about educational strategies, innovations, research and scholarship by attending the interactive workshops. See workshop details and registration online.
Contact: For more information, contact Liza Young, 638-4799 or firstname.lastname@example.org
John McCahan Medical Campus Education Day
- Thursday, May 22
- 8:30 a.m.-3:30 p.m.
- BUSM Instructional Building, Hiebert Lounge 14th Floor.
Ribbon Cuttings Mark Official Openings for Two New GSDM Department of Restorative Sciences & Biomaterials Labs
Two ribbon cutting ceremonies marked the official opening of two new laboratory spaces for the Department of Restorative Sciences & Biomaterials at Boston University Henry M. Goldman School of Dental Medicine (GSDM) in March. One of the new labs is located on the Second Floor of the Evans Biomedical Research Center at 650 Albany St. (X-2) and the other is on the Fifth Floor of the Housman Medical Research Center at 780 Harrison Ave. (R-5).
The Department of Restorative Sciences & Biomaterials at GSDM is at the forefront of evaluating and developing materials for computerized fabrication of restorations. The faculty has developed new concepts and techniques for analyzing the interaction between biomaterials and cells at the molecular and genetic levels. The department is strategically positioned to create, analyze and test novel synthetic materials for tissue replacement and prosthetic therapy.
The ceremonies began at the X-2 laboratory space, where Dean Jeffrey W. Hutter led a ribbon cutting with Dr. Karen Antman, Provost of Boston University Medical Campus (BUMC) and Dean of the Boston University School of Medicine (BUSM); Dr. Ronald Corley, Associate Provost for Research at BUMC and Professor and Chair of Microbiology at BUSM; Dr. Dan Nathanson, Professor and Chair of the Department of Restorative Sciences & Biomaterials; Dr. Maria Kukuruzinska, Associate Dean for Research and Professor in the Department of Molecular & Cell Biology; and Dr. Lee Chou, Professor of Biomaterials in the Department of Restorative Sciences & Biomaterials.
The guests and dignitaries then walked over to R-5, where Dean Hutter led a second ribbon cutting with Provost Antman; Associate Provost Corley; Dr. Kukuruzinska; Dr. Nathanson; and Dr. Russell Giordano, Associate Professor in the Department of Restorative Sciences & Biomaterials and Director of Biomaterials. Prior to cutting the ribbon, Dean Hutter, Dr. Nathanson, and Provost Antman each briefly addressed the attendees.
Dean Hutter said, “Our Department of Restorative Sciences & Biomaterials proudly conducts basic science and applied research; trains pre- and post-doctoral students and residents in research methodology and biomaterials; and educates dental students with respect to novel materials and procedures that may improve patient care.” He continued, “I know that these new research laboratories will only add to the quality of our research and the education we are able to provide our students and residents.”
Submitted by GSDM Communications.
Benjamin Wolozin, MD, PhD, professor of pharmacology and neurology, was awarded the Alzheimer’s Association Zenith Fellows Award, a $450,000 grant dispersed over three years. Initiated in 1991, the award provides support for cutting edge basic science or biomedical research that addresses fundamental problems related to early detection, etiology pathogenesis, treatment and/or prevention of Alzheimer’s disease (AD).
The Wolozin Lab won the award with its proposal “It Takes TIA to Tangle: The Role of RNA Binding Proteins in AD.” The lab already has discovered a RNA binding protein that induces tau misfolding, one of the essential steps that leads to cognitive loss in AD. This award will allow the Wolozin Lab to experimentally induce the misfolding, investigate the factors that regulate the misfolding and in the future, potentially design therapeutics to prevent the misfolding.
“Dr. Wolozin’s investigation represents the exciting and promising research that is very much needed if we are to eventually find effective treatments for Alzheimer’s. For the more than five million Americans with Alzheimer’s, we are pleased to be a partner in that work,” said Jim Wessler, president/CEO Alzheimer’s Association, MA/NH Chapter.
“Trauma Informed Services: Implications for Healthcare Providers and Systems”
Carole Warshaw, MD
Director, National Center on Domestic Violence, Trauma and Mental Health
Executive Director, Domestic Violence Mental Health Policy Initiative
Tuesday, May 13
BUSM Instructional Building, L-110
This annual memorial lecture is in honor of Lynne Stevens, LICSW, BCD (1946-2009), the director of the Responding to Violence Against Women Program and an Assistant Professor in the Department of Family Medicine. She was a clinician, activist and researcher who worked locally, nationally, and internationally, specializing in evaluation of the quality of care offered to women who were victims of violence.
Attendees are cordially invited for lunch at 11:45 a.m. and a Q & A discussion from 1-2 p.m. with Dr. Warshaw following the lecture.
According to a study released April 17 in the American Journal of Public Health, dental-related Emergency Department (ED) visits increased up to 14 percent following Massachusetts Medicaid cuts.
Findings are based on data from Boston Medical Center, an urban safety-net hospital in Boston’s South End.
In this retrospective study of existing data, researchers looked at dental-related ED visits and costs for three years before and two years after Massachusetts Health Care Reform, which reduced Medicaid coverage for adult dental care in July 2010. The study looked at adults 21 and older and ED use from July 1, 2007 to June 30, 2012.
“Dental-related ED visits increased two percent the first year and 14 percent the second year after Medicaid cuts,” said Dr. Judith Jones, one of the study’s authors and Director of the Center for Clinical Research at Boston University Henry M. Goldman School of Dental Medicine. “Percentage increases were highest among older adults, minorities, and persons receiving charity care, Medicaid, and Medicare.”
Related costs of care rose significantly, too. The average cost per patient per visit increased seven percent in 2010–2011 and 27 percent in 2011–2012.
The study found that most people using the ED for dental care reported caries (cavities) and soft tissue issues, conditions that are best treated in the dentist’s office.
In short, “Use of EDs for dental care points to an inappropriate use of resources and lack of continuity of dental care,” according to Jones.
“The findings highlight the need for primary dental care among the poor, racial/ethnic minorities, and adults of all ages, especially older adults.”
The idea for the study came from Dr. Pushkar Mehra, chair of the Department of Oral & Maxillofacial Surgery, who noted that, “after MassHealth Dental for adults was reduced, the residents in the BU/BMC Oral & Maxillofacial Surgery program were spending more time in the ED seeing patients with dental needs.”
Dean Jeffrey W. Hutter added, “I am proud of the leading role the Boston University Henry M. Goldman School of Dental Medicine is taking in identifying and addressing access to care issues in the state of Massachusetts through our research, in our Patient Treatment Centers, and at our Dental Health Center locations.”
Massachusetts Health Care Reform cut Medicaid spending on adult dental care by nearly 50 percent—from $139.4 million/year from July 1, 2007 to June 30, 2010, to $67.2 million/year from July 1, 2010 to June 30, 2012. In 2012, Medicaid partially restored dental care coverage for adults in Massachusetts. The study authors recommend further research to determine whether ED visits declined as a result.
Additional authors include Drs. Martha Neely, Lillelenny Santana Gutierrez, and Sharron Rich, MPH.
Submitted by GSDM Communications.
Gerard Doherty, MD, chair of the Department of Surgery at Boston University School of Medicine (BUSM) and chief of Surgery at Boston Medical Center (BMC) and has been elected as President of the American Association of Endocrine Surgeon (AAES). He will serve as President through 2015.
As president, Doherty will preside at council assemblies and the annual members’ assembly. He will appoint members to all committees, serve as an ex-officio member of each, appoint successors to open positions, and deliver the 2015 Presidential Address.
Endocrine surgery is the discipline of surgical management of endocrine disorders, including the understanding of the disease process, and comprehensive care of surgical endocrine disease of the neck and abdomen. The AAES is dedicated to the science and art of endocrine surgery, and maintenance of the highest standards in clinical practice.
“Being chosen by my colleagues to fulfill this role is a distinct honor. The AAES has been my professional home throughout my career, and I admire the many achievements that we have made as a group to improve the care of patients, to advance the field, and to train the next generation of practitioners.”
Doherty’s clinical focus is endocrine oncology and comprises surgical diseases of the thyroid, parathyroid, endocrine pancreas and adrenal glands, as well as the surgical management of multiple endocrine neoplasia (MEN) syndromes.
Prior to coming to BUSM and BMC in 2012, Doherty spent a decade as chief of General Surgery at the University of Michigan Health System. He is the immediate past-president of the Michigan Chapter of the American College of Surgeons and has held multiple leadership positions in national and international professional groups, including the Board of Directors of the American Thyroid Association.
A graduate of Holy Cross and Yale School of Medicine, Doherty completed his residency at the UC-San Francisco, including Medical Staff Fellowship at the National Cancer Institute. He joined the faculty at Washington University School of Medicine in 1993 and became professor of Surgery there in 2001.
Egos in BU center take a backseat to sharing, progress, and promise
In the video above, CReM founders Darrell Kotton, Gustavo Mostoslavsky, and George Murphy discuss how they use iPS cells to study disease development, conduct drug screenings, and eventually to correct genetic mutations, with the goal of producing healthy tissues and organs for transplantation. Photo by Jackie Ricciardi
“Our mission is to decrease the burden of human suffering on the planet, help patients, and advance new knowledge.” —Darrell Kotton
With the motto Advancing Science to Heal the World the BU stem cell scientists who founded the Center for Regenerative Medicine (CReM) could be pegged as starry-eyed idealists or scientific superheroes. Or perhaps a bit of both.
CReM codirectors Darrell Kotton, Gustavo Mostoslavsky, and George Murphy have established themselves as venturesome researchers who are willing to share their discoveries with almost anyone. And they do it for free—bucking the prevailing trend to patent, publish, and protect scientific breakthroughs. The trio’s “open source biology” is just one of the things they teach to the next generation of stem cell researchers at CReM.
Open source biology can seem antithetical to scientists in an extremely competitive field. One young researcher training at CReM recently approached Kotton, a School of Medicine professor of medicine, seeking advice on how to answer an outside request for a vial of stem cells that took several years and hundreds of thousands of dollars in federal grants to develop. The obvious answer, the trainee assumed, was to tell the researcher to wait until the discovery was published.
Kotton saw it differently. “Our mission is to decrease the burden of human suffering on the planet, help patients, and advance new knowledge,” Kotton reminded him. “If this competitor of ours has the same goal, then we’re obligated to share this cell vial with them, because that’s going to achieve our mission.…which is not to get credit and to stroke our egos.”
A naïve response? “Here’s the thing that nobody talks about,” says Kotton. “If you behave in this way, people in our community quickly get the idea that the BU-BMC CReM are the good guys of science. At some point, the equation gets so lopsided that people almost feel embarrassed that they’re not sharing with you and so they tell you stuff, and the whole field starts to move forward.”
In fact, CReM founders say that increasing numbers of researchers are asking CReM to collaborate on grants, and foundations have begun to recognize that funding a CReM project very probably means that resulting knowledge, expertise, and reagents will be shared with other academic or nonprofit laboratories without restriction or exclusivity.
“I can’t emphasize enough how unique that was for the community,” says Mostoslavsky, a MED assistant professor of gastroenterology. “We have dozens of emails that testify to that, saying, ‘I must tell you this is the first time in my 30 years of being a scientist that someone replied and sent me stuff the same week of asking for it.’”
Still, Mostoslavsky says, there is a “fine balance that is not easy to achieve” between freely sharing their work and protecting it once research has advanced to the clinical stage. “That is a major undertaking,” he says. “It’s very expensive—no academic institution can support it—so we do need a company to move forward,” which also means they’ll need patented protection of intellectual property.
Scientific soul mates
Kotton, Mostoslavsky, and Murphy met as Harvard postdoctoral fellows in the lab of renowned stem cell scientist Richard Mulligan, who is famous for his rigorous research and forthright style of mentorship. “It was more of a sink-or-swim methodology, where you really had to prove yourself,” says Murphy, a MED assistant professor of medicine. “Coming out of there, we were battle-tested and bombproof.”
The three gravitated toward each other as “scientific soul mates,” Mostoslavsky says. Long after fellow researchers had left the lab, they would gather for late-night pizza and animated discussions, probing one another’s data to test the strength of their work. “We were each other’s worst critics as well as biggest fans,” Kotton says. It was around that time that they began toying with the idea of conducting science their way—in a meticulous, yet open and collegial manner.
After completing his Harvard fellowship, in 2006 Kotton returned to MED, where he had done a fellowship previously, to launch his own lung stem cell lab. He confesses to “putting psychological pressure” on his friends to follow him to BU, and he is not at all unhappy that it worked. In 2008, Mostoslavsky came aboard, creating his own lab. He was followed soon after by Murphy.
Several events conspired to launch CReM on the Medical Campus. The founders discovered a strong advocate in David Coleman, Wade Professor and chair of the MED department of medicine, who emphasized the importance of a robust research presence on the Medical Campus. Kotton, Mostoslavsky, and Murphy had followed closely the rapid advance of stem cell biology since 2006, when scientists at the University of Kyoto developed induced pluripotent stem cells (iPS) by reprogramming an adult differentiated cell. A tinkerer at heart, Mostoslavsky was fascinated by the Kyoto process, but felt he could go one better. In 2008, he developed a more efficient tool to generate stem cells, called the stem cell cassette (STEMCCA). BU patented the tool, which has become industry standard.
In 2010, with STEMCCA and multiple publications under their belts, the trio established a virtual Center for Regenerative Medicine, with its own website, seminar series, and iPS cell bank carrying branded labels. All this was accomplished while working in separate labs, with Murphy’s and Mostoslavsky’s divided by floors within a building, and Kotton’s located across the street.
As the number of stem cell biologists, physician-researchers, and biomedical engineers grew on both BU campuses, the affectionately labeled CReM brothers felt it was time to pitch a physical center to BU President Robert A. Brown, who firmly backed the idea. Boston University and Boston Medical Center invested jointly in the endeavor, and in November 2013, CReM opened in its newly remodeled space on the second floor of 670 Albany Street.
In the video above, CReM founders discuss how they use induced pluripotent stem (iPS) cells in their labs.
Clinical trial in a test tube
CReM’s mission is to advance stem cell research and regenerative medicine for the treatment patients, in particular those at BMC, with diseases such as cystic fibrosis, emphysema, sickle cell anemia, and amyloidosis. Investigators collect blood or skin cell samples, usually from patients at the Alpha-1 Center, the Center of Excellence in Sickle Cell Disease, and the Amyloid Center, and reprogram them into iPS cells.
“Any cell can be reprogrammed,” says Mostoslavsky. “It’s a true biological rejuvenation. The cells really go back in time.” Researchers now have the ability to coax iPS cells—which uniformly carry a patient’s genetic mutations—into their desired cell line, such as lung, liver, or blood. (CReM’s iPS cell bank stores at least 13 such cell lineages.) Mostoslavsky says the resulting cells are “still a work in progress,” compared to those found in nature, but the process allows researchers to watch how an iPS-derived lung cell develops the early stages of cystic fibrosis. What took years to unfold in a patient takes days in the lab.
CReM investigators can screen drugs against patient cell lines to determine which medications are most effective for a specific genetic mutation—the “clinical trial in a test tube,” as Murphy calls it. “In theory,” he says, “you could develop therapies that are molded specifically for a particular patient with a particular disease.”
Developmental biology and drug screenings are now CReM’s bread and butter, but its founders keep in mind what they call their long-term “Apollo projects,” such as genetically engineering iPS cells to correct patients’ mutations. The resulting healthy cells could be cultured and multiplied to regenerate a transplantable organ that wouldn’t be rejected by the patient’s body. That particular medical breakthrough would mean Kotton’s wife would need to look for a new job. Camille Kotton is a transplant infectious disease physician at Massachusetts General Hospital, and Kotton jokes that his “goal is to put her out of business.”
Kotton has been collaborating with a team of MGH researchers using a technology called lung decellularization and recellularization, which strips the organ of its cells and repopulates it with genetically engineered copies that lack the patient’s original mutation. Theoretically, the reprogrammed cells could multiply, fill the lung’s scaffolding, and someday be used for transplantation.
Kotton, who thinks it will be possible eventually to re-create lungs via 3-D printing technology, is collaborating on this with Christopher Chen, a College of Engineering professor of biomedical engineering.
Meanwhile, Murphy dreams of brewing blood. “The human body makes 2.5 million red blood cells every second of every day,” he says. “How does one contend with that from a research platform?” He thinks they will be able to “harness molecular cues” that exponentially increase the amount of artificial blood they can produce in vitro. Such a discovery could eliminate the need for blood donation in the United States and—even more urgent—in Third World countries, where the practice is not widely accepted.
Another of Murphy’s Apollo projects is boutique blood, or the development of small batches that could be produced for people who suffer from sickle cell anemia or the blood disorder beta thalassemia and require transfusions of rare blood types.
Researcher as healer
In the past four years, word has spread about the CReM founders’ work to the point that as well as the emails from potential collaborators who want access to their work, they now get email from patients who either suffer from the particular disorders they study or have children who do.
The latter are heartrending. “‘We will sign whatever you want us to sign, and we don’t care how experimental your platform is, we would still like to use it to save our kid,’” Murphy recalls reading. He explains that although his team is working hard to find new treatments, right now there are no current stem cell therapies for their child’s condition. Asked when they might be available, he replies, “‘Sooner than you think.’ That’s vague, but it’s hopeful. And it also is the way that we see things.”
Kotton likes to tell a story that underscores CReM’s potential. He and his colleagues were approached about a child who suffered from severe cardiac arrhythmias. Their labs developed iPS cells from the boy’s skin cells and differentiated them into heart muscle cells, then sent them to researchers in New York City, who screened them against a series of drug regimens until they found a winning combination. After three months on the medication, the boy’s arrhythmia decreased from 100 incidents per month to zero.
“This success story represents, I believe, the first human being on planet Earth to be helped by the new iPS cell technology,” Kotton says. “If there is one patient who can benefit from these cells, then surely there are myriad more for generations to come.”