ALS Therapy Development Institute
News Article | May 17, 2017
Most research institutions are essentially collections of independent laboratories, each run by principal investigators who head a team of trainees. This scheme has ancient roots and a track record of success. But it is not the only way to do science. Indeed, for much of modern biomedical research, the traditional organization has become limiting. A different model is thriving at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts, where I work. In the 1990s, the Whitehead Institute for Biomedical Research, a self-governing organization in Cambridge affiliated with the Massachusetts Institute of Technology (MIT), became the academic leader in the Human Genome Project. This meant inventing and applying methods to generate highly accurate DNA sequences, characterize errors precisely and analyse the outpouring of data. These project types do not fit neatly into individual doctoral theses. Hence, the institute created a central role for staff scientists — individuals charged with accomplishing large, creative and ambitious projects, including inventing the means to do so. These non-faculty scientists work alongside faculty members and their teams in collaborative groups. When leaders from the Whitehead helped to launch the Broad Institute in 2004, they continued this model. Today, our work at the Broad would be unthinkable without professional staff scientists — biologists, chemists, data scientists, statisticians and engineers. These researchers are not pursuing a tenured academic post and do not supervise graduate students, but do cooperate on and lead projects that could not be accomplished by a single academic laboratory. Physics long ago saw the need to expand into different organizational models. The Manhattan Project, which during the Second World War harnessed nuclear energy for the atomic bomb, was not powered by graduate students. Europe's particle-physics laboratory, CERN, does not operate as atomized labs with each investigator pursuing his or her own questions. And the Jet Propulsion Laboratory at the California Institute of Technology in Pasadena relies on professional scientists to get spacecraft to Mars. In biology, many institutes in addition to the Broad are experimenting with new organizational principles. The Mechanobiology Institute in Singapore pushes its scientists to use tools from other disciplines by discouraging individual laboratories from owning expensive equipment unless it is shared by all. The Howard Hughes Medical Institute's Janelia Research Campus in Ashburn, Virginia, the Salk Institute of Biological Sciences in La Jolla, California, and the Allen Institute for Brain Science in Seattle, Washington, effectively mix the work of faculty members and staff scientists. Disease-advocacy organizations, such as the ALS Therapy Development Institute in Cambridge, do their own research without any faculty members at all. Each of these institutes has a unique mandate, and many are fortunate in having deep resources. They also had to be willing to break with tradition and overcome cultural barriers. At famed research facilities of yore, such as Bell Labs and IBM Laboratories, the title 'staff scientist' was a badge of honour. Yet to some biologists the term suggests a permanent postdoc or senior technician — someone with no opportunities for advancement who works solely in a supervisor's laboratory, or who runs a core facility providing straightforward services. That characterization sells short the potential of professional scientists. The approximately 430 staff scientists at the Broad Institute develop cutting-edge computational methods, invent and incorporate new processes into research pipelines and pilot and optimize methodologies. They also transform initial hits from drug screens into promising chemical compounds and advance techniques to analyse huge data sets. In summary, they chart the path to answering complex scientific questions. Although the work of staff scientists at the Broad Institute is sometimes covered by charging fees to its other labs, our faculty members would never just drop samples off with a billing code and wait for data to be delivered. Instead, they sit down with staff scientists to discuss whether there is an interesting collaboration to be had and to seek advice on project design. Indeed, staff scientists often initiate collaborations. Naturally, tensions still arise. They can play out in many ways, from concerns over how fees are structured, to questions about authorship. Resolving these requires effort, and it is a task that will never definitively be finished. In my view, however, the staff-scientist model is a win for all involved. Complex scientific projects advance more surely and swiftly, and faculty members can address questions that would otherwise be out of reach. This model empowers non-faculty scientists to make independent, creative contributions, such as pioneering new algorithms or advancing technologies. There is still much to do, however. We are working to ensure that staff scientists can continue to advance their careers, mentor others and help to guide the scientific direction of the institute. As the traditional barriers break down, science benefits. Technologies that originate in a faculty member's lab sometimes attract more collaborations than one laboratory could sustain. Platforms run by staff scientists can incorporate, disseminate and advance these technologies to capture more of their potential. For example, the Broad Institute's Genetic Perturbation Platform, run by physical chemist David Root, has honed high-throughput methods for RNA interference and CRISPR screens so that they can be used across the genome in diverse biological contexts. Staff scientists make the faculty more productive through expert support, creativity, added capacity and even mentoring in such matters as the best use of new technologies. The reverse is also true: faculty members help staff scientists to gain impact. Our staff scientists regularly win scientific prizes and are invited to give keynote lectures. They apply for grants as both collaborators and independent investigators, and publish regularly. Since 2011, staff scientists have led 36% of all the federal grants awarded for research projects at the Broad Institute (see ‘Staff-led grants’). One of our staff scientists, genomicist Stacey Gabriel, topped Thomson Reuters' citation analysis of the World's Most Influential Scientific Minds in 2016. She co-authored 25 of the most highly cited papers in 2015 — a fact that illustrates both how collaborative the Broad is and how central genome-analysis technologies are to answering key biological questions. At the Broad Institute's Stanley Center for Psychiatric Research, which I direct, staff scientists built and operate HAIL, a powerful open-source tool for analysis of massive genetics data sets. By decreasing computational time, HAIL has made many tasks 10 times faster, and some 100 times faster. Staff scientist Joshua Levin has developed and perfected RNA-sequencing methods used by many colleagues to analyse models of autism spectrum disorders and much else. Nick Patterson, a mathematician and computational biologist at the Stanley Center, began his career by cracking codes for the British government during the cold war. Today, he uses DNA to trace past migrations of entire civilizations, helps to solve difficult computational problems and is a highly valued support for many biologists. Why haven't more research institutions expanded the roles of staff scientists? One reason is that they can be hard to pay for, especially by conventional means. Some funding agencies look askance at supporting this class of professionals; after all, graduate students and postdocs are paid much less. In my years leading the US National Institute of Mental Health, I encountered people in funding bodies across the world who saw a rising ratio of staff to faculty members or of staff to students as evidence of fat in the system. That said, there are signs of flexibility. In 2015, the US National Cancer Institute began awarding 'research specialist' grants — a limited, tentative effort designed in part to provide opportunities for staff scientists. Sceptical funders should remember that trainees often take years to become productive. More importantly, institutions' misuse of graduates and postdocs as cheap labour is coming under increasing criticism (see, for example, et al. Proc. Natl Acad. Sci. USA 111, 5773–5777; 2014). Faculty resistance is also a factor. I served as Harvard University's provost (or chief academic officer) for a decade. Several years in, I launched discussions aimed at expanding roles for staff scientists. Several faculty members worried openly about competition for space and other scarce resources, especially if staff scientists were awarded grants but had no teaching responsibilities. Many recoiled from any trappings of corporatism or from changes that felt like an encroachment on their decision-making. Some were explicitly concerned about a loss of access and control, and were not aware of the degree to which staff scientists' technological expertise and cross-disciplinary training could help to answer their research questions. Institutional leaders can mitigate these concerns by ensuring that staff positions match the shared goals of the faculty — for scientific output, education and training. They must explain how staff-scientist positions create synergies rather than silos. Above all, hiring plans must be developed collaboratively with faculty members, not by administrators alone. The Broad Institute attracts world-class scientists, as both faculty members and staff. Its appeal has much to do with how staff scientists enable access to advanced technology, and a collaborative culture that makes possible large-scale projects rarely found in academia. The Broad is unusual — all faculty members also have appointments at Harvard University, MIT or Harvard-affiliated hospitals. The institute has also benefited from generous philanthropy from individuals and foundations that share our values and believe in our scientific mission. Although traditional academic labs have been and continue to be very productive, research institutions should look critically and creatively at their staffing. Creating a structure like that of the Broad Institute would be challenging in a conventional university. Still, I believe any institution that is near an academic health centre or that has significant needs for advanced technology could benefit from and sustain the careers of staff scientists. If adopted judiciously, these positions would enable institutions to take on projects of unprecedented scope and scale. It would also create a much-needed set of highly rewarding jobs for the rising crop of talented researchers, particularly people who love science and technology but who do not want to pursue increasingly scarce faculty positions. A scientific organization should be moulded to the needs of science, rather than constrained by organizational traditions.
News Article | May 26, 2017
Pete Frates, the man who inspired the Ice Bucket Challenge to raise awareness about amyotrophic lateral sclerosis, is now struggling not only with the disease itself but also to pay for his medical bills. His family can no longer keep up with the tremendous financial cost of ALS care, which has now reached $3,000 a day. The former Boston College athlete, now 32, is currently on life support at home. His bedroom has been turned into an Intensive Care Unit, so that his wife and 2-year-old daughter can be constantly by his side. ALS, more popularly known in the United States as Lou Gehrig's disease, is a neurodegenerative disorder that attacks nerve cells. This disease weakens the muscles and eventually leads to total paralysis, making even the simplest things, such as speaking, swallowing and breathing, difficult to perform. This is why Pete is now completely dependent on life support and the care of his family. The 24-hour home healthcare comes with a monthly cost of $85,000 to $95,000, which puts a considerable strain on the family. "Any family would be broke because of this," said John Frates, Pete's father, who disclosed the family has been trying to cope with the enormous expense for two and a half years. Yet the situation has reached a point where "it's become absolutely unsustainable for us," confessed the older Frates. This is why the family is now reaching out for help, so they can continue to afford keeping Pete at home — just like they promised him when he was first diagnosed in 2012. The Frates family is trying to avoid having to commit Pete into a specialized facility away from his wife and daughter. According to the family, it's the two of them that give Pete the resolve to continue fighting this neurodegenerative disease. To help other families dealing with the same situation, the Frateses partnered with philanthropist Rob Griffin, who is also an old family friend, and the ALS Association, in an initiative to raise funds particularly for this type of cases. "We hope to start an initiative to help the Frates family and all families that are going through that, and hope it takes off on national basis," said Griffin. As a first step, the organizers are planning a fundraiser for Pete, which is set to take place on June 5 in Boston. The ultimate goal is to raise $1 million through the Home Health Care Initiative. "I'm just extremely grateful that they are recognizing how significant Pete has been in this community. We just want to keep him at home with his family," said Julie Frates, Pete's wife. Pete, who is a former captain of the Boston College baseball team, first started the Ice Bucket Challenge back in 2014, when he was 29 years old, and it immediately went viral on social media. Besides public awareness, his initiative raised substantial amounts of money for the ALS Therapy Development Institute. In the last three years, the ALS Ice Bucket Challenge inspired donations that totaled more than $250 million. The money funded ALS research and in 2015 helped scientists at Johns Hopkins University investigate the role of the TDP-43 protein in the development of ALS. Last year, the ALS Association reported another breakthrough, when researchers at the University of Massachusetts identified a gene responsible for ALS. Earlier this month, the U.S. Food and Drug Administration approved the first new ALS treatment in more than two decades. The intravenous drug, called Radicava or edaravone, is the second treatment drug to be greenlighted by the FDA for the condition and will be available for ALS patients in the United States starting August. © 2017 Tech Times, All rights reserved. Do not reproduce without permission.
News Article | June 29, 2017
-- Coronado Brewing Company is participating in the fifth annual Ales for ALS™ program. 128 participating breweries from 36 states each brewed a unique beer using a hop blend provided by Loftus Ranches and Yakima Chief - Hopunion (YCH HOPS). Ales for ALS™ has raised more than $1,250,000 for ALS research, with proceeds benefiting the ALS Therapy Development Institute (ALS TDI). The ALS TDI's mission is to discover and develop effective treatments and a cure for amyotrophic lateral sclerosis (ALS); there is currently no cure for the progressive neurodegenerative disease which affects 500,000 people worldwide.Coronado Brewing's Buster 4 IPA is a tribute to Lou Gehrig, who was famously diagnosed with ALS at the height of his baseball career, and subsequently became a face for disease. "We've proudly supported Ales for ALS from the beginning," says Tyler Tucker, R&D Brewer, Coronado Brewing Company. "It's a great cause and we're honored that we can help give back. It's amazing to see the craft beer community rally behind Ales for ALS™."Buster 4 IPA features the unique hop blend provided free-of-charge by YCH HOPS, including Citra, Loral, HBC 472, HBC 344, HBC 568, and HBC 431. "This year's blend is very complex, with layers of tropical fruit, citrus, and noble-like spiciness," says Tucker. "We used 5.6 pounds of hops per barrel, compared to about 2 pounds in a typical IPA. The hops really shine in this beer!" The donated hops were used in the mash, late kettle additions, and dry-hoping for a big tropical and citrus aroma and flavor. Buster 4 IPA comes in at 6.0% and 50 IBUs, with a lingering, dry finish.Buster 4 IPA will debut at the company's Tasting Room on July 4, in honor of Gehrig's retired #4. $1 from each pint sold will directly benefit ALS research. The beer will also be featured at the fourth annual Ales for ALS™ event at Faction Brewing Company, in Alameda, CA on July 22, 2017. Buster 4 IPA is part of Coronado Brewing's ongoing commitment to the community; for more information, visit coronadobrewing.com. Stay Coastal. Cheers.Buster 4 IPA:Style: American IPAABV: 6.0%Bitterness: 50 IBUsHops: Citra, Loral, HBC 472, HBC 344, HBC 568, and HBC 431Package: draft-onlyRelease: 7/4/17 at Coronado Brewing Company Tasting RoomAvailability:California
News Article | December 8, 2016
DUBLIN--(BUSINESS WIRE)--A decade after their work sparked a revolution in patient empowerment and patient-centered medicine, PatientsLikeMe co-founders Jamie and Ben Heywood were awarded the 2016 Humanitarian Award by the International Alliance of ALS/MND Associations. Inaugurated in 2000, the Humanitarian Award recognizes and encourages contributions to the fight against Amyotrophic Lateral Sclerosis/Motor Neurone Disease (ALS/MND) and is awarded to those whose work is of international significance for people affected by ALS/MND. In presenting the award, the Alliance’s citation acknowledged the founding of both the patient network PatientsLikeMe and the ALS Therapy Development Institute (ALS TDI), the world’s first non-profit biotechnology company. “When their brother was diagnosed with ALS at the age of 29, James Allen Heywood and Benjamin Heywood were devastated at his prognosis and at the lack of effective treatments for the disease. They saw firsthand how isolating ALS/MND can be. They took up these challenges as a family and, with family and friends, founded two organizations: ALS TDI, tasked with finding effective treatments for the condition, and PatientsLikeMe, to tackle social isolation and to collect data on what other drugs, interventions or supplements might make a difference to those with ALS/MND.” Jamie Heywood thanked the association on behalf of his family and PatientsLikeMe members and staff and said the company is ready to lead the next decade of advancements in research and medicine, with and for patients. “We changed the rules by helping patients digitize and share their experience so they could make more informed decisions about how to live with and treat their condition. Now we’re embarking on the next stage of the journey by piloting biomarker discovery in ALS and other conditions. We hope many more patients will join us as we work together to find new answers.” More information about how to be part of PatientsLikeMe's upcoming research in ALS/MND is available at www.patientslikeme.com/advanceals. About PatientsLikeMe PatientsLikeMe is a patient network that improves lives and a real-time research platform that advances medicine. Through the network, patients connect with others who have the same disease or condition and track and share their own experiences. In the process, they generate data about the real-world nature of disease that help researchers, pharmaceutical companies, regulators, providers, and nonprofits develop more effective products, services, and care. With more than 400,000 members, PatientsLikeMe is a trusted source for real-world disease information and a clinically robust resource that has published more than 85 research studies. Visit us at www.patientslikeme.com or follow us via our blog, Twitter or Facebook.
News Article | September 7, 2016
In 1996, Kathy Giusti was diagnosed with multiple myeloma, a rare and often fatal cancer. Her first bone-marrow biopsy took place on a Friday night. Outside the room, a group of scientists waited with an ice chest to take her bone-marrow sample. She told her doctor that she was surprised to see them working late. “He said my tissue was precious,” she recalls. For precision medicine to live up to its potential, millions of people must share their genomic data, their health records, and their experiences. To researchers, all of it is precious. The richer the databases, the better patient care will become. This need gives ordinary people more power in medical research — not only to improve research quality by participating in greater numbers, but by speaking up and influencing what questions are asked in the first place. Despite lingering concerns over privacy (see page S70), it is patients and their loved ones who have been pushing for changes to the medical system that will enable personalized medicine, says Guisti, who founded the Multiple Myeloma Research Foundation with her sister in 1998. For individual patients, empowerment takes a lot of time, work, education and economic resources. If programmes such as the United States' planned million-volunteer Precision Medicine Initiative Cohort Program are to succeed (see page S69), they must build people's trust and bring in not only highly motivated, seriously ill people, but healthy volunteers too. Early patient-centred research projects are already showing that this can pay off for researchers, for drug companies and, more importantly, for patients. It can be difficult for even well-educated, financially secure people living close to major US medical centres to access the best medical care. In 1998, Marty Tenenbaum was diagnosed with melanoma that had metastasized to his liver, “which in those days had zero survivors”, he says. After a lot of searching, he was lucky enough not just to get onto a trial for an experimental therapy and surgery, but to be one of the few who responded. The trial failed, but Tenenbaum is still in remission. This experience, and the genome-sequencing boom, led Tenenbaum — a computer scientist and former professor at Stanford University — to found a personalized-medicine consultancy called CollabRX in 2008. The company used bioinformatics to suggest therapies to its wealthy customers at a cost of US$35,000–50,000. “My vision was to use information technology to close the loop between cancer research and clinical care,” he says. When CollabRX was acquired by Tegal in 2012, Tenenbaum wanted to start a non-profit organization that could serve more people. So he set up Cancer Commons, whose goal is to make the expertise of the best clinicians available to more cancer patients — especially those who cannot afford to travel to the best medical centres or pay for a personal consultation. Tenenbaum sees buried treasure in the scientific discussions that take place behind closed doors on tumour boards — the groups of doctors, geneticists and researchers who discuss individual cases and decide on the best course of therapy. There are millions of possible combinations of drugs, many more than could ever be tested in clinical trials. Instead, the best oncologists and tumour boards are in effect experimenting on their patients, says Tenenbaum, trying new drug cocktails and seeing what happens. Yet little is learned: the deliberations and failed hypotheses of the tumour boards are not included in individual patients' records, and none of the data are shared outside the hospital. “There's all this experimentation and no learning,” he says. “Every patient presents a vastly complicated data set that we're barely able to interpret,” says C. Anthony Blau, an oncologist at the University of Washington in Seattle who specializes in finding therapies for people with difficult-to-treat breast cancer. The ability to search a large pool of data on what has been tried with others would help oncologists to find the right treatment faster. Tenenbaum is promoting this data-sharing vision through an online portal called Ask Cancer Commons. Patients or their care-givers can upload whatever medical records or genetic tests they have and give a description of their case. A group drawn from more than 100 volunteer oncologists and geneticists, including Blau, then reviews the case and gives feedback, serving as a virtual tumour board. In the short term, Tenenbaum hopes that this will provide a lifeline for people who cannot access top-quality cancer care locally. But as more people take part, the database will grow. Patient data and the reports of the virtual tumour board are fed into a database that doctors can use to help future patients. Cancer Commons follows up to find out what the doctors did with their feedback, and how well it worked. Hospitals are also contributing to the growth of the database. In a pilot test in 2015, 50 cases from three tumour boards were summarized by volunteers and verified by doctors. Eventually, Cancer Commons will have enough of these hand-annotated data to construct algorithms that are capable of picking out important information from complex patient records and tumour-board deliberations without the need for volunteers at all. The project, known as the Insight Network, is currently fundraising and is expected to shift into full gear in the next few years. As such projects grow, the need for people to take charge of their own care will diminish. “Avid patients will lead the way,” says Blau. “The knowledge gained through them will be applicable to the population as a whole.” One of the largest online data-sharing health projects, PatientsLikeMe, began in 2004 with a focus on neurological disorders. Since then, it has expanded to include 2,500 diseases. PatientsLikeMe now has about 500,000 users, most of them seriously ill. Through the website, these people can track their symptoms, join discussions and complete research surveys. Jamie Heywood, a mechanical engineer and co-founder of the site PatientsLikeMe, calls it “a prospective epidemiology platform”. So far, PatientsLikeMe has published more than 75 studies, mostly in collaboration with academic or corporate researchers. It has partnerships with the US Food and Drug Administration and drug company AstraZeneca among others, and it is financed by sharing patients' data with drug companies and researchers. Heywood's younger brother, Stephen, was diagnosed with amyotrophic lateral sclerosis (ALS) in 1998 at the age of 29, spurring Jamie to start the ALS Therapy Development Institute. It took the unusual approach of publishing results in real time as it screened drugs in mice and conducted a stem-cell trial with three participants, including Stephen. People with a serious disease can feel very alone, says Heywood. They want to be treated like partners, not subjects, and this is what PatientsLikeMe tries to do. Sharing data and experiences that may help others, and knowing that they are going to help researchers and drug companies, can make people feel heard and empowered ( et al. Patient http://doi.org/bpqw; 2016). “My brother died eight years ago and he's still helping people,” says Heywood. To build trust and encourage people to share data, the site is designed to be as accessible as possible. Instead of medical terms, patients use phrases such as 'brain freeze' to describe how they feel; the vernacular is then automatically matched with the standard medical code. The company also gets to know its users and adapts how it interacts with them accordingly. Some need to make a decision quickly after a diagnosis, for instance, whereas others with degenerative diseases have more time. Unlike conventional top-down studies, in which data are collected on rigid timetables, PatientsLikeMe offers its users the flexibility to add data whenever they want, but this makes it less statistically rigorous than traditional research. “Real-world observational studies will never be able to match double-blind prospective studies in their ability to examine causality,” says Heywood, and understanding biases in data contributed by users is a big challenge for epidemiologists and others at the company. But when patients are in charge, he says, they supply data that researchers do not normally have access to. As an example of how patient-centred research can yield insights that benefit both patients and drug companies, Heywood points to a collaboration on insomnia between PatientsLikeMe, Northwestern University in Evanston, Illinois, and US drug company Merck & Co. After developing a new sleep drug, surovexant, Merck approached PatientsLikeMe and asked it to look at the sleep patterns of members. Based on an initial survey of 75,000 users, Heywood says that his organization came up with about 50 hypotheses. The team then narrowed both the set of questions and the study group. The resulting survey of just over 5,000 users in 2013 showed that only 13% had been diagnosed with insomnia, but that 73% of those who were undiagnosed also reported symptoms ( et al. Sleep Med. 16, 1332–1341; 2015). The data suggest that many people with serious illnesses have trouble sleeping — something that can exacerbate their condition, and doctors should be aware of the need to manage it. As the five-year study continues, participants will receive information about their individual sleep patterns. Another of the site's self-tracking tools has been particularly useful for Allison Silensky, who has been using PatientsLikeMe since 2008. Silensky, who has a form of bipolar disorder, is a member of the company's user advisory board. The site's mood tracker has helped Silensky to notice and remember trends in her mood that she might otherwise have neglected to mention to her doctor. If she feels great at the doctor's office, she says, “I don't realize that the three weeks prior were horrible because I'm living in the moment.” At first her doctors warned her against getting involved with something on the Internet, she says, but they soon came to see the benefit. After a few years, Silensky saw a trend that she had not noticed before: all her hospitalizations were in spring. Now her doctor adjusts her medication in January, and her therapist checks on her more frequently in spring. She has not been hospitalized since. People with serious and rare diseases, and their families, may be highly motivated to participate in data-sharing projects. But personalized medicine also needs healthy volunteers if researchers are to understand how diseases emerge. “This is a national movement, and we need everyone to participate,” says Bray Patrick-Lake, director of patient engagement at the Duke Translational Medicine Institute in Durham, North Carolina. Research and anecdote alike suggest that the possibility of helping others motivates people to share their data. Unpublished findings from a survey funded by the US National Institutes of Health suggest that proponents of precision medicine will have to convince people that they are contributing to the public good by sharing their data. Sandra Soo-Jin Lee, a biomedical ethicist, is looking at how diverse communities feel about projects that link electronic health records with biobanks for research. Her group at the Stanford Center for Biomedical Ethics in California is still analysing the results, which are based on surveys of 20 focus groups, including Hispanic, Asian and African American people, but already she has found “a tension” in people's attitudes. There is excitement that the data might lead to fresh targeted therapies or discoveries that are possible only with large pools of data. But some worry that the information will be used by the government for non-medical purposes, and others, says Lee, simply feel “a loss of control”. And many are nervous about who will profit. “There's concern about who's actually going to use the data,” she says — particularly that a third party will use the information to develop an expensive therapy. Donating data and tissue for the public good is one thing, but often the benefits are not distributed equally. The HeLa line of immortal cancer cells, for instance, derived without consent from the ovarian tumour of African American woman Henrietta Lacks, has long been a workhorse for cancer researchers. But the benefits of this research have not been distributed equally: cancer mortality rates for African Americans are still higher than those of any other ethnic group in the United States. “In a fundamentally unequal health system, it's harder to argue that everyone should share,” says Barbara Koenig, a medical anthropologist at the University of California, San Francisco. She studies the limits of informed consent in large public data-sharing projects. Without automatic enrolment for enterprises that serve the public good, most people will be motivated to opt into data sharing only when tragedy strikes in the form of a diagnosis for themselves or a loved one, says Koenig. “If people know they will benefit, they will share.” Heywood thinks that the people behind large government projects are misguided if they believe that “because they're trying to do the world good, the world will follow them”. Building trust with people takes time, he says. Continuing to carry out “onerous, top-down recruitment for clinical studies” is not working, says Sharon Terry, chief executive of the Genetic Alliance, a non-profit health advocacy organization. One of its projects, PEER, is an online resource that not only allows patients to make their information available to researchers, but also gives them control over how much of their health information is shared, and with whom, on a case-by-case basis. “Our experiment is to use the tools of social media to engage people,” says Terry. She thinks that the health-care industry does not do outreach as well as community organizations. So the Genetic Alliance is trying to learn from people in education and social services. Today, precision-medicine portals, whether patient-driven or not, are fragmented, and that can make it hard to reach people, admits Giusti. But once people believe they are “on the path to a cure”, they want to participate, she says. The progress made by the Multiple Myeloma Research Foundation towards treating that particular cancer is due in part to the patients who willingly donated their tissue to a biobank in the hope of accelerating research. That project, called CoMMpass, was launched in 2011, and since then has validated several drug targets and treatment strategies. It has shown, for instance, that people treated with a combination of three drugs live longer without the disease progressing than those who are given only two. But these projects are expensive: CoMMpass cost more than $40 million. “We struggle to see how this can be sustained,” says Terry. Large national, and ideally international, projects are the only way to make precision medicine work, says Kathryn North, leader of the Australian Genomics Health Alliance — and most people in the field accept that, she adds. Projects such as the US Precision Medicine Initiative are a great start, but they are only a start. There are economic, political and bureaucratic barriers to overcome, and probably only patients can make it happen. “The biggest advocates for this are the patient groups, because they can see how it transforms health care,” says North. The question facing patient advocates who want to see personalized medicine, says Terry, is this: “Can we impact the culture of large academic institutions, behemoth drug companies, and staid federal agencies?” Even if they want to change, there must be incentives driving them to do so. “I keep banking on public pressure, interest and rallying,” she says. If precision medicine one day comes to benefit broad swathes of the population, it may well be thanks to a few patients who took it on themselves to push for that kind of future.
News Article | February 15, 2017
DALLAS, Feb. 15, 2017 (GLOBE NEWSWIRE) -- ClubCorp – The World Leader in Private Clubs® (NYSE:MYCC) – today announced that through its Charity Classic events the company raised $3,053,312 in 2016, breaking its previous all-time fundraising record set in 2015. The Charity Classic, ClubCorp’s major philanthropic effort benefiting over 100 national and local charitable organizations, Augie’s Quest/ALS Therapy Development Institute and ClubCorp’s E.P.C.F., has now raised more than $20.3 million since its inception in 2007. A photo accompanying this announcement is available at http://www.globenewswire.com/NewsRoom/AttachmentNg/0f032b6e-a58d-4706-8b69-606e4b37114a Across the U.S. throughout much of 2016, nearly 15,000 people participated in more than 160 ClubCorp clubs open-to-the-public Charity Classic events. The events, consisting of golf and tennis tournaments, concerts, galas, auctions, 5K runs and unique dining experiences, all benefited the following deserving non-profits: “The Charity Classic has reached this $20 million milestone because of the hard work and dedication of our clubs, their members and employee partners and those in their communities who come together each year to support these very important causes,” said Eric Affeldt, ClubCorp CEO. “I am so proud of what the Charity Classic has become and would like to thank every participant for their support.” Augie Nieto, founder of Augie’s Quest and person with ALS says, “The partnership between Augie’s Quest and ClubCorp continues to exceed expectations. Each club’s team works tirelessly on the events and we are so grateful for the continued support.” The official date for the upcoming 11th annual ClubCorp Charity Classic event is Sept. 29, 2017, with events throughout the country to be held from February through November. For more information on the ClubCorp Charity Classic, visit www.clubcorpcharityclassic.com. About ClubCorp (NYSE:MYCC) Since its founding in 1957, Dallas-based ClubCorp has operated with the central purpose of Building Relationships and Enriching Lives®. ClubCorp is a leading owner-operator of private golf and country clubs and private business clubs in North America. ClubCorp owns or operates a portfolio of over 200 golf and country clubs, business clubs, sports clubs, and alumni clubs in 26 states, the District of Columbia and two foreign countries that serve over 430,000 members, with approximately 20,000 peak-season employees. ClubCorp Holdings, Inc. is a publicly traded company on the New York Stock Exchange (NYSE:MYCC). ClubCorp properties include: Firestone Country Club (Akron, Ohio); Mission Hills Country Club (Rancho Mirage, California); The Woodlands Country Club (The Woodlands, Texas); Capital Club Beijing; and Metropolitan Club Chicago. You can find ClubCorp on Facebook at facebook.com/clubcorp and on Twitter at @ClubCorp.
News Article | February 22, 2017
Orangetheory® Fitness, with 21 locations in the Tampa Bay area, announced it has partnered with Augie’s Quest, a foundation dedicated to finding a cure for amyotrophic lateral sclerosis (ALS) disease, with the goal of raising $1 million in two weeks. The company’s first national fundraiser, named the #IBurnForALS campaign, will take place from Feb. 20 to March 5. During the two-week period, more than 570 Orangetheory Fitness studios around the country will encourage members to donate money to support Orangetheory’s quest to reach $1 million. All money raised will be donated to Augie’s Quest in support of ALS research. The #IBurnForALS fundraising campaign will consist of Orangetheory Fitness coaches challenging participants in each class during the two week period to donate at least $1 per splat point achieved. Splat points are attained for every minute a participant spends in the ‚orange zone,’ which is their target-training zone of 84 percent to 91 percent of their heart rate. The orange zone stimulates metabolism and increases energy. In addition, on Saturday, February 25, Orangetheory will host Augie-thon, a specialty donation session at every studio. Augie-thon is a unique 90-minute Orangetheory class that members and non-members can attend for a donation of $20. The first 45 participants at each studio will receive a special black or orange sweatband. Tampa Bay however has added to this special initiative. Coaches will do workouts, rowing and burpees corresponding to dollars donated, and members can create their own class playlist with a $500 donation, or host their own VIP class with a $1000 donation. “ALS is a disease that with the proper research and funding, we can prevent. The Tampa Bay stores have employees and members that have been affected and we are committed to this National Partnership, as we want to exceed fundraising goals, to help Burn for ALS.” Says Don Allen, CEO of Tampa Fitness Partners. Augie’s Quest was co-founded by Augie Nieto and his wife, Lynne. Nieto is known for his role in shaping today’s fitness world as the co-founder and CEO of Life Fitness, Lifecycle and Octane Fitness. He was diagnosed with ALS in 2005 and has been dedicated to finding treatments and cures for the disease ever since through events, partnerships and fundraising activities around the globe. Augie’s Quest directly funds the ALS Therapy Development Institute (ALS TDI), the world’s foremost drug discovery center focused solely on finding a cure for ALS. The Institute’s innovative science and cutting edge approach has resulted in the identification of AT-1501, a promising treatment for ALS. ALS TDI also pioneered the ALS Precision Medicine Program, the world’s premier program and partnership with ALS patients to discover additional potential treatments. ALS, also known as Lou Gehrig’s disease, is a disorder that affects the function of nerves and muscles. More than 6,000 people in the U.S. are diagnosed with ALS each year. The life expectancy of a person with ALS averages two to five years from the time of diagnosis, however there is evidence that people with ALS are living longer, at least partially due to clinical management interventions, riluzole and other compounds and drugs under investigation. For more information about the #IBurnForALS campaign, please visit your nearest Tampa Bay Orangetheory Fitness studio. To learn more about Augie’s Quest, please visit AugiesQuest.org. Members and non-members alike can make donations at AugiesQuest.ALS.net/OTF. About Orangetheory Fitness: Orangetheory® Fitness (http://www.orangetheoryfitness.com) is a scientifically designed, one-of-a-kind, group personal training workout broken into intervals of cardiovascular and strength training. Backed by the science of excess post-exercise oxygen consumption (EPOC), Orangetheory’s heart-rate-monitored workouts are designed to get participants within the target-training zone of 84 percent to 91 percent of their heart rate, which stimulates metabolism and increases energy. Led by highly skilled coaches, each Orangetheory Fitness workout incorporates endurance, strength and power elements through a variety of equipment including treadmills, rowing machines, TRX® suspension training and free weights. The end result is more energy, visible toning and the ‚Orange Effect’ – where participants keep burning calories for up to 36 hours post-workout (the Orangetheory Fitness ‚afterburn’) for an average of 500+ total calories burned per every 60-minute workout. The company was ranked #60 in Inc. magazine’s Fastest Growing Private Companies List and #225 in Entrepreneur’s 2016 Franchise 500® list of the top franchises in the world.
Lincecum J.M.,ALS Therapy Development Institute |
Vieira F.G.,ALS Therapy Development Institute |
Wang M.Z.,ALS Therapy Development Institute |
Thompson K.,ALS Therapy Development Institute |
And 10 more authors.
Nature Genetics | Year: 2010
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive loss of motor neurons. Using unbiased transcript profiling in an ALS mouse model, we identified a role for the co-stimulatory pathway, a key regulator of immune responses. Furthermore, we observed that this pathway is upregulated in the blood of 56% of human patients with ALS. A therapy using a monoclonal antibody to CD40L was developed that slows weight loss, delays paralysis and extends survival in an ALS mouse model. This work demonstrates that unbiased transcript profiling can identify cellular pathways responsive to therapeutic intervention in a preclinical model of human disease. © 2010 Nature America, Inc. All rights reserved.
Als Therapy Development Institute | Date: 2012-11-29
Methods and therapeutic compositions are disclosed for treating neurodegenerative disorders and, in particular Amyotrophic Lateral Sclerosis, using sphingosine1-phosphate receptor modulators, such as fingolimod or a pharmaceutically acceptable salt, hydrate, or solvate thereof.
Als Therapy Development Institute | Date: 2012-02-23
Methods and therapeutic agents are disclosed for treating neurodegenerative disorders by depletion of CD8 positive T cells by using antibodies, FAb fragments of antibodies or similar agents that sequester, neutralize or deplete the CD8+ cytotoxic T cells.