Schwannomatosis

Pictured: Nonprofit groups parachute into a hospital/Nicole Bean for BioSpace Nonprofit foundations no longer function only as the traditional micro-funders and patient support groups of yesteryear. In 2018, foundations, along with professional societies, collectively devoted $3.8 billion per year to medical research and development in the U.S. That makes them the fourth-largest source of funding, after private-sector biotech firms, the federal government and academic institutions. In other words, healthcare philanthropy by nonprofit organizations now plays a critical role in life sciences innovation. Nonprofits are uniquely positioned to provide funding, build bridges, make connections and even lead efforts to develop new treatments and cures for a variety of illnesses—including rare ones. Foundations share the same sense of urgency to find effective cures as the patients they serve. They’re also well-connected to all the stakeholders involved with particular diseases, sitting at a unique intersection between patients, researchers, clinicians, pharmaceutical companies, funders and regulators. That makes them well-equipped to bridge the “gaps” in drug discovery and development. Consider that basic research leading to medical breakthroughs often begins at university labs, many of which rely on large grants from the NIH or other federal agencies. But to have a shot at winning those grants in the first place, researchers generally need to have preliminary data to show their research is worth funding. That’s where foundations come in. Nonprofits can, and increasingly do, fund the risky, innovative, early-stage research that yields the preliminary datasets that increase scientists’ odds of securing government funding. While academic discoveries can lead to high-impact publications in top journals, translating those discoveries into potential future medicines may require a team of cell biologists, animal pharmacologists, chemists, toxicologists and many others. Here, too, modern nonprofit foundations are “filling the gap” by acting as a superconnecter—funding and managing multidisciplinary teams to ensure the potential medical applications of early-stage discoveries are thoroughly investigated. A Tangible Impact My organization, the Children's Tumor Foundation, is pursuing this path. We focus on the rare genetic disorders neurofibromatosis (NF) and schwannomatosis, which cause tumors to grow on patients’ nerves. To help find a cure, we started a collaborative research effort called Synodos that assembles scientists, clinicians, researchers and patient representatives to solve complex problems. Synodos researchers make their data available in real time through a digital portal. This free flow of information—which is atypical of academic publishing—has the potential to accelerate the translation of research into treatments. The Synodos team has already delivered clinical trial-ready drugs for the treatment of one type of NF. That led to a first-of-its-kind clinical trial, co-funded by CTF and Takeda, which is already achieving incredible results. The Leukemia and Lymphoma Society (LLS) is also playing a key role in bringing drugs to the clinic. LLS brought together world-renowned scientists, drug companies, tech companies and the FDA to launch a precision medicine trial for patients with acute myeloid leukemia, a lethal blood cancer. It was the first time a nonprofit health organization sponsored a clinical cancer trial. LLS also launched PedAL, a global clinical trial for pediatric acute leukemia designed to test new, safer therapies for children. The goal is to prevent childhood cancer survivors from developing chronic health problems as a result of their treatment—which currently happens about 80% of the time. Funding Early-Stage Companies Another gap is in funding for early-stage startups, which has reached an all-time low since 2019. Twenty-eight biotech companies shut down in 2023, compared to just nine in 2021. Often, these companies need investors to fund their R&D and clinical trials, which can cost hundreds of millions for just a single drug. But venture capitalists frequently consider early-stage companies too risky an investment, so many of them go unfunded. Fortunately, foundations are stepping in. Consider the Cystic Fibrosis Foundation, which has shown that investing in high-risk projects can positively impact patients’ lives. Its “venture philanthropy” strategy led to the FDA approval of Kalydeco, the first drug to treat the underlying causes of cystic fibrosis. Or take the groundbreaking CoMMpass study, funded by the nonprofit Multiple Myeloma Research Foundation. CoMpass has over 1,000 patients involved in clinical trials and is delivering its ongoing research to other researchers and biopharmaceutical companies across the country in hopes of accelerating the search for new therapies. Similarly, the I-SPY series of trials—which aims to change the way new breast cancer treatments are developed—has received funding from several nonprofit organizations, including Quantum Leap Healthcare Collaborative, the Breast Cancer Research Foundation, NIH and the William K. Bowes, Jr. Foundation, among others. Research findings from CoMMpass, I-SPY and similar trials could complement biotech firms’ own R&D efforts, ultimately leading to more success stories like Kalydeco. And healthcare foundations are doing much more than writing checks. Increasingly, they're hiring executives with biotech experience and setting up R&D infrastructure of their own. In recent years, nonprofits—like the Multiple Myeloma Research Foundation, Tuberculosis Sclerosis Association, LLS and my foundation—are making significant contributions to R&D, from creating new patient registries and patient engagement programs to establishing clinical care and clinical trial center networks. These sorts of strategic, financial and community-oriented efforts are exactly what patients are counting on. Many life sciences organizations, such as NewYorkBIO (where I am a board member) and the European Federation of Pharmaceutical Industries and Associations, have already recognized as much. It’s time to encourage more life science organizations to view healthcare nonprofits as changemakers sitting at the intersection of research, community and funding and work with foundations to get more medicines to patients. Annette Bakker, PhD is president of the Children’s Tumor Foundation, Chair of CTF Europe and board member of NewYorkBIO.

Collaboration Will Accelerate Development of Treatments for Neurofibromatosis and Schwannomatosis Patients NEW YORK, Oct. 5, 2023 /PRNewswire/ -- The Children's Tumor Foundation (NEW YORK, NY and BRUSSELS, BELGIUM) and the Global Coalition for Adaptive Research (LARKSPUR, CA) – The Children's Tumor Foundation (CTF) and Global Coalition for Adaptive Research (GCAR), today announced their alliance to accelerate the development of treatments for patients with NF (neurofibromatosis or schwannomatosis) – a group of rare and often debilitating diseases with high unmet medical need. CTF and GCAR are partnering to operationalize a first-of-its-kind clinical trial for patients with NF that was initially designed through the EU-PEARL initiative. The announcement of this strategic alliance follows a 3-year, 26-million-euro investment from the Innovative Health Initiative (IHI), formerly called the Innovative Medicines Initiative (IMI). EU-PEARL was a public-private partnership dedicated to transforming clinical trials by making them more efficient and patient-centered. Through this partnership, leading organizations representing patients, clinicians, researchers, health authorities and industry collaborated to design the first-ever Phase 1 / Phase 2 global platform basket trial for patients with neurofibromatosis type 1 (NF1) or schwannomatosis (SWN), including NF2-related schwannomatosis (NF2-SWN). CTF is the global leader in neurofibromatosis and schwannomatosis research, dedicated to funding and driving innovative research that will result in effective treatments for the 4 million people worldwide living with NF, a group of genetic disorders that causes tumors to grow on nerves throughout the body. One in every 2,000 people is born with neurofibromatosis or schwannomatosis, which may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, or cancer. "Central to CTF's mission is advancing care and treatments for the NF community. The establishment of the NF platform trials will expedite the development of effective treatments for patients," said Dr. Annette Bakker, President of the Children's Tumor Foundation. "We are delighted to partner with GCAR, a leading expert in developing and operationalizing master protocols and adaptive platform trials to actualize this innovative trial." GCAR was formed with the mission to speed the discovery and development of treatments for patients with rare and deadly diseases by serving as the sponsor of innovative trials. Its first initiative was to implement the clinical trial GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) into a self-sustaining engine of therapeutic and biomarker development for patients with glioblastoma. Since the launch of GBM AGILE, GCAR also initiated U.S. sponsorship for innovative global trials in COVID-19 and is advancing platform trials for other rare and deadly diseases such as high-grade ovarian cancer. "We are excited to work with the Children's Tumor Foundation to build a patient-centric clinical trial that will rapidly and efficiently evaluate multiple investigational therapies for the treatment of NF," said Dr. Meredith Buxton, CEO & President of the Global Coalition for Adaptive Research. "GCAR is committed to leading the way in clinical trial innovation by uniting stakeholders to advance new therapies for patients. Our alliance with CTF exemplifies a collaboration that places patients and the community at the forefront of clinical research." The NF platform trials will be built to complement CTF's NF Preclinical Hub, a comprehensive infrastructure to accelerate the availability of clinical trial-ready treatments, including gene therapy. The preclinical hub will provide access to a pipeline from concept to clinic, pre-negotiated master service agreements, protocols for compound testing, as well as access to testing models, data tools, libraries, and biomarkers. The plan is to launch an NF1 platform trial for four different manifestations including plexiform neurofibroma, cutaneous neurofibroma, optic pathway glioma, and low-grade glioma, with future plans to build a platform trial to support SWN patients. Additionally, the Foundation's NF Patient Registry provides dependable access to NF patients available to participate in NF clinical trials and research studies. With the establishment of the NF platform trials (an NF1 trial followed by an SWN trial), pharma companies are invited to explore testing of their therapies, as well as investors interested in the opportunity to fuel the progress to approved treatments. About the Children's Tumor Foundation (CTF) The Children's Tumor Foundation (CTF) is the world's leading nonprofit dedicated to funding and driving innovative research resulting in effective treatments for the millions of people worldwide living with NF. Guided by a bold and innovative strategic plan, CTF is laser-focused on delivering therapies (right treatments at the right time) to patients who are accurately diagnosed. In addition to driving bench to bedside research, CTF manages global public-private multi-disciplinary consortia, and has invested in a gene therapy and small molecule company. The Foundation is now actively working with ten pharma/ biotech companies to advance therapies for NF, and is inviting other companies to join. CTF recognizes the unique niche role of disease foundations in the R&D ecosystem, involving the patient voice in every initiative. While being committed to building an enterprise that will benefit patients with NF, CTF hopes that the model can act as a blueprint to accelerate drug discovery and development in the larger rare disease community. CTF acknowledges Goodwin for its continuous pro bono support. For more information, please visit: ctf.org. About Global Coalition for Adaptive Research (GCAR) The Global Coalition for Adaptive Research (GCAR) unites physicians, clinical researchers, advocacy and philanthropic organizations, biotech/pharma, health authorities, and other key stakeholders in healthcare to expedite the discovery and development of treatments for patients with rare and deadly diseases. As Sponsor of innovative trials, including master protocols and adaptive platform trials, GCAR is dedicated to the advancement of science by modernizing clinical trials that support more efficient, less costly drug development. Adaptive platform trials can accelerate the time from discovery in the lab to implementation in the clinic resulting in better treatments and lives saved. To learn more about GCAR and its initiatives, visit gcaresearch.org. View original content to download multimedia: SOURCE Children's Tumor Foundation

Hosted by the Children's Tumor Foundation, NF Summit and NF Conference are the largest gathering of the NF community in the world NEW YORK, June 22, 2023 /PRNewswire/ -- From June 21 through June 27, the largest gathering of NF researchers, clinicians, and patients in the world will take place at the Fairmont Scottsdale Princess in Scottsdale, Arizona. NF is a group of genetic disorders that causes tumors to grow on nerves throughout the body, and affects 1 in 2,000 births of all populations equally. As such, NF affects millions worldwide, but is underrecognized. While there is one approved treatment for a small subgroup of NF patients with plexiform neurofibromas, there is no cure yet, and the vast majority of NF patients face serious health issues because of the condition. This gathering in Arizona, comprising two meetings, offers novel insights into NF research, generates enthusiasm that effective therapeutics are on the horizon, and fosters a deeper sense of community. - NF Summit: patients, families, and caregivers will convene for three and a half days (June 21-24) to hear about promising research, meet with world-class NF experts, and participate in patient empowerment sessions. All forms of NF will be covered – neurofibromatosis type 1 (NF1) and all forms of schwannomatosis (SWN), including NF2-related schwannomatosis (NF2), as well as quality of life issues for the myriad manifestations of NF, which can cause deafness, blindness, bone abnormalities, learning disabilities, disabling pain, and cancer. Notable sessions at the NF Summit include: Clinical trials in nutraceuticals that are leading to less toxic treatment and prevention strategies through a specific dietary component. Panel discussion on alternative & traditional approaches to chronic pain Seminar for parents and other caregivers about navigating chronic illness; this session will explore the emotional and psychological impact of NF on parents, siblings, and other family members, as well as the practical challenges that arise when a family member has a chronic illness. The NF Clinical Trials Consortium session will review the structure, discuss trials completed or ongoing, and plans for the future of the Department of Defense-funded program. - NF Conference: this four-day global meeting (June 24-27) attracts leading specialists from across a wide range of scientific disciplines and research and clinical backgrounds, all focused on improving outcomes for NF patients. This annual event is the most important date on the NF research calendar, and is critical to consensus building and advancing basic, translational, and clinical research in NF and related fields. Among the key advances to be presented at the NF Conference are: dedicated afternoon to gene therapy in NF with a targeted focus on developing curative therapies that address the underlying genetic abnormalities in NF patients focus on pain, from basic science to preclinical and translational testing & development to novel imaging biomarkers to clinical management a full day of programming dedicated to all types of schwannomatosis, which includes NF2-related schwannomatosis (NF2), covering alternative cell models, new therapeutic approaches, and microenvironment of nerve tumors and the role of neurons in tumor growth For more information about NF and the Children's Tumor Foundation, visit . For more information about the NF Summit, visit . For more information about the NF Conference, visit . ABOUT THE CHILDREN'S TUMOR FOUNDATION: The Children's Tumor Foundation is the world's leading nonprofit dedicated to funding and driving innovative research that will result in effective treatments for the millions of people worldwide living with NF, a group of genetic conditions that causes tumors to grow on nerves throughout the body. One in every 2,000 people is born with some type of neurofibromatosis or schwannomatosis, which may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, or cancer. NF affects all populations equally, and while there is no cure yet, the Children's Tumor Foundation mission of driving research, expanding knowledge, and advancing care for the NF community fosters our vision of one day ending NF. For more information, please visit: ctf.org. SOURCE Children's Tumor Foundation