| ID | Title | Body | Facebook Description | Facebook image | Facebook Title | Facebook Video | Twitter Description | Twitter Image | Twitter Title | Meta tags |
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| 511 | CMX001 for Treatment of Neonatal Herpes Simplex Virus | In the United States, approximately 1,500 babies are born each year infected with herpes simplex virus (HSV). In newborns, HSV infection, or neonatal herpes, can be life threatening. Untreated, the virus can spread throughout the body, including the brain and spinal fluid, causing seizures and even death. At least 40 percent of babies with virus in the central nervous system (CNS) are left with significant, lifelong neurologic damage. The purpose of this project is to develop a therapeutic regimen to clear HSV from the CNS and avoid this neurologic damage. Scientific Synopsis Despite significant advances in the treatment of neonatal herpes over the past 30 years, approximately 20 percent of babies with disseminated HSV disease die, and at least 40 percent of babies with CNS disease are left with significant, lifelong neurologic damage. Existing antiviral therapeutic options, such as acyclovir, have been maximized in terms of both amount and duration of administration. To accomplish the next series of therapeutic advances, new drugs with improved penetration into the CNS are required. The recent development of CMX001, the lipophilic derivative of cidofovir, provides for the first time a molecule with significant anti-herpetic activity that penetrates the CNS. Before a comparative trial of acyclovir + CMX001 versus acyclovir + placebo can be undertaken in neonatal HSV disease, the correct dose of CMX001 in neonates must be determined. In this project, a multi-institutional team of investigators, known as the Collaborative Antiviral Study Group, will define the pharmacokinetics and concentration response relationship of CMX001 in neonates with HSV CNS disease. TRND researchers will support the preclinical development studies of the pro-drug. Lead Collaborator University of Alabama at Birmingham David Kimberlin, M.D. Public Health Impact Despite significant advances in the treatment of neonatal HSV disease over the past 30 years, approximately 20 percent of babies with disseminated HSV disease die, and at least 40 percent of babies with CNS disease are left with significant, lifelong neurologic damage. This project seeks to develop a therapeutic regimen to clear HSV from the CNS and avoid this neurologic damage. Outcomes This project was carried out in conjunction with NIH’s National Institute of Allergy and Infectious Diseases (NIAID). TRND supported preclinical studies needed to enable successful filing of an Investigational New Drug (IND) application to the Food and Drug Administration (FDA). The FDA has cleared the IND, and NIAID will provide support for the clinical studies in collaboration with Dr. Kimberlin at the University of Alabama at Birmingham. This TRND project is complete. | ||||||||
| 510 | A Potent Oral Therapy for Cytomegalovirus Infection | Cytomegalovirus (CMV) is related to the viruses that cause chickenpox and mononucleosis. Between 50 percent and 80 percent of U.S. adults are infected by CMV by age 40. Most don’t get sick or even know they are infected, but CMV is a major threat to infants and to those with compromised immune systems, especially bone marrow and organ transplant patients. The virus spreads through close contact with body fluids, and once in the body, stays there for life. Transmission from mother to infant occurs in about 40,000 U.S. births each year, and about 10 percent of infected babies die or have severe problems as newborns. Many more have hearing loss or developmental problems later in life. Current anti-CMV agents have severe side effects, such as liver and bone marrow toxicity, so better treatments are needed. These investigators are developing a medication that may inhibit CMV activity but also may be effective against related viruses with few treatment options. Scientific Synopsis CMV infection is a major health concern in the immunocompromised population, especially among recipients of bone marrow and solid organ transplants. The current standard for therapy is ganciclovir (GCV) and its prodrug valGCV, but both can cause bone-marrow toxicity, severe neutropenia and emerging resistance. Alternatives include foscarnet (PFA) and cidofovir, but nephrotoxicity limits their use. Therefore, there is still a major unmet medical need for new anti-CMV agents. Researchers in the laboratory of Jiri Zemlicka, Ph.D., at Wayne State University identified a novel series of purine nucleoside analogs, the methylenecyclopropanes, and showed they are potent inhibitors of human CMV. A second-generation analog, ZSM-I-62, now has been shown to be very potent against murine and human CMV, including GCV- and PFA-resistant clinical isolates, and is less toxic to human bone marrow cells. ZSM-I-62 may offer a promising alternative that has broad activity across members of the beta- and gamma-herpesviruses, for which few therapeutic options exist. The project goal is to complete the ZSM-I-62 preclinical toxicology and safety pharmacology studies, file an Investigational New Drug (IND) application for the treatment of CMV-mediated disease and initiate a human clinical phase I safety evaluation. The requested BrIDGs ZSM-I-62 synthetic route improvements, bulk Good Manufacturing Practice (GMP)-grade synthesis and formulation support will greatly facilitate this goal. Lead Collaborator Wayne State University School of Medicine, Detroit Jiri Zemlicka, Ph.D. Public Health Impact CMV infection continues to be a major cause of morbidity and mortality in immune-suppressed patients, especially recipients of solid organ or bone marrow transplants. The five drugs that have been approved for use in patients with CMV infection have severe limitations that preclude their long-term use. These include poor oral bioavailability, dose-related toxicity and selection of drug-resistant viral mutants. Therefore, there is an urgent medical need for more effective and safer therapies. Outcomes Work on this project is complete. The investigator successfully filed an IND application using BrIDGs data. Project Details Synthesis of GMP and non-GMP material Formulation development | ||||||||
| 509 | Use of Retinal Progenitor Cells for the Treatment of Retinitis Pigmentosa | Retinitis pigmentosa (RP) is a severe form of hereditary blindness characterized by progressive damage to and loss of the light-sensing cells of the retina. Most patients have night blindness in their early teens, typically progressing to legal blindness by age 40. There are no approved treatments for RP. The lead collaborator has identified an innovative approach to save the light-sensing cells of the eye. The purpose of this project is to support the development of these retina-derived cells as a transplantable treatment to stop the cellular damage that leads to blindness in RP patients. Scientific Synopsis RP is a rare genetic disease of the eye characterized clinically by the loss of rod photoreceptor cells, followed by cone-cell degeneration. As rod cell function degenerates, impaired night vision typically appears as an initial symptom. When rod photoreceptors die, cone photoreceptors are destabilized, resulting in progressive loss of visual acuity and color/central vision. The principal investigators have developed a cell-based therapeutic approach to stop retinal cell degeneration. Work in animal models has demonstrated the ability of grafted retinal-derived, lineage-restricted progenitor cells (RPCs) to promote survival and function of dystrophic host photoreceptors. The principal investigators and others have demonstrated that injected donor RPCs survive in the vitreous and migrate to the retina, where they integrate with the host cellular architecture. As lineage-restricted progenitor cells, these engrafted RPCs are able to differentiate further into rod photoreceptors, providing additional support to the dependent cone cells. The therapeutic hypothesis is that the release of trophic factors and engraftment by donor RPCs should rescue host cone cells, improving existing vision and delaying or preventing total blindness. In a pre-Investigational New Drug (IND) meeting, the Food and Drug Administration (FDA) identified additional studies needed to support submission of a full IND package. Lead Collaborator University of California, Irvine Henry Klassen, M.D., Ph.D. Public Health Impact There are no approved treatments for retinitis pigmentosa. The disease typically results in legal blindness by age 40. Outcomes TRND researchers and the collaborator performed IND-enabling studies to support submission of a full IND package. TRND researchers completed a biodistribution study of the formulated RPCs (jCell) in pig eyes. The results of these studies were incorporated into an IND filing by the collaborator’s start-up company, jCyte, Inc. The FDA cleared the IND to begin human clinical trials. jCyte will now be able to continue development of jCell using internal resources. This TRND project is now complete. | ||||||||
| 508 | Use of Rapamycin for the Treatment of Hypertrophic Cardiomyopathy in Patients with Noonan Syndrome with Multiple Lentigines (NSML) | LEOPARD syndrome (LS) is a rare genetic disease affecting only about 200 patients worldwide. Nearly all cases of LS result from mutations in a single gene, PTPN11. In the heart, the most common manifestation of LS is hypertrophic cardiomyopathy (HCM), a thickening of the walls of the heart. There is no existing treatment for LS patients who have HCM, and end-stage heart failure can lead to early death. The lead collaborator has shown that rapamycin can prevent and reverse HCM in animal models of LS. The purpose of this project is to develop rapamycin or similar compounds as effective HCM therapies for LS patients. Scientific Synopsis LS is named for its presenting manifestations: multiple lentigines (L), electrocardiographic conduction abnormalities (E), ocular hypertelorism (O), pulmonic stenosis (P), abnormal genitalia (A), retardation of growth (R) and sensorineural deafness (D). Despite the fact that pulmonic stenosis is part of the LS acronym, the most common cardiac manifestation is HCM, occurring in approximately 70 percent of LS patients. To determine the biological and functional mechanisms in LS, the lead collaborators generated an LS mouse model harboring one of the two most common mutations in the human disease, the Y279C mutation. These mice recapitulated nearly all major aspects of the human LS disorder. The investigators identified a hyperactivation of the Akt/mTor signaling pathway as the mechanism by which Y279C causes HCM in LS, implicating rapamycin as a potential pharmacologic intervention. LS is one of several autosomal dominant disorders associated with RAS/MAPK pathway genes (RASopathies). Rapamycin already is approved for treatment of renal cancer and is undergoing clinical trials for polycystic kidney disease. The goal of this project is to leverage TRND support to develop the necessary preclinical package to support filing an Investigational New Drug (IND) application with the Food and Drug Administration (FDA) and clinical trials for HCM in LS patients. Lead Collaborator Beth Israel Deaconess Medical Center, Boston Maria Kontaridis, Ph.D. Public Health Impact There is no treatment for LS, and hypertrophic cardiomyopathy often leads to early death among LS patients. Developing a better understanding and innovative therapeutic approach to LS may have broader application to other congenital heart defects. Outcomes To facilitate therapeutic development, the TRND team further characterized the NSML mouse models using magnetic resonance imaging (MRI) to measure overall changes in heart structure and function, in comparison to echocardiography, to better inform clinical endpoints. TRND scientists conducted additional animal efficacy studies with the lead molecule and another mTOR inhibitor. As milestones of in vivo efficacy were not met in the mouse models tested, the project did not continue into further development. | ||||||||
| 507 | Repurposing an EU Therapeutic for Hemoglobinopathies | The most common global genetic diseases — beta-thalassemia and sickle cell disease — share a biochemical basis. Both of these disorders are caused by defects in one part (beta-globin) of hemoglobin, the protein in red blood cells that carries oxygen throughout the body. These hemoglobin disorders, called “hemoglobinopathies,” can result in moderate-to-severe anemia, with symptoms ranging from weakness and fatigue to damage to the heart, brain, lungs and other organs. These symptoms can cause chronic disabilities and early death. No drugs are approved to treat the underlying causes of these disorders. The lead collaborator has identified drug that is currently approved in the European Union to treat another condition that has the potential to treat beta-thalassemia and sickle cell disease. The goal of this project is to develop this existing drug as an effective therapy targeted at the underlying cause of both blood disorders. Scientific Synopsis The oxygen-carrying hemoglobin protein (Hemoglobin A or HbA) is a tetrameric molecule, comprising two paired alpha-chain/beta-chain protein dimers. Beta-thalassemia is caused by mutations that result in insufficient production of the beta-globin protein, whereas sickle cell disease is caused by a point mutation in the beta-globin protein that causes the hemoglobin tetramers to form rigid polymers that deform the red blood cell, causing early cell death, abnormal adhesion to blood vessels and resulting widespread organ damage. HbA is the “adult” form of hemoglobin, produced mostly after birth. At earlier developmental stages, “fetal” forms of hemoglobin (HbF) are expressed. Importantly, these fetal globin proteins can replace the abnormal adult beta-globin proteins found in sickle cell disease and compensate for the absence of adult beta-globin proteins in beta-thalassemia. Although fetal globin expression stops shortly after birth, the drug PB-04 can reactivate expression of the fetal-stage beta-globin gene. This activation may lead to production of sufficient levels of HbF to mitigate the beta-hemoglobinopathies. The goal of this project is to repurpose this drug, currently in use in the European Union and Canada for another indication, for treatment of the hemoglobin disorders beta-thalassemia and sickle cell disease. Lead Collaborator Phoenicia Biosciences, Inc., Weston, Massachusetts Susan Perrine, M.D. Public Health Impact Although both are orphan conditions in the United States, beta-thalassemia and sickle cell disease affect millions worldwide, are increasing in frequency in the U.S. and are classified by the World Health Organization as a growing global health burden. Beta-thalassemia has no approved drug therapy, and the single approved therapy for sickle cell disease is the anticancer agent hydroxyurea, which is approved only for use in adults and has undesirable side effects. Thus, there is a high-priority unmet medical need to develop a treatment specific for these diseases. Outcomes The TRND team formalized and initiated a comprehensive preclinical project plan with a primary focus on beta-thalassemia as the first indication. Cell-based and animal efficacy studies confirmed key data generated previously by the collaborator. Further pharmacology, toxicology and chemistry, manufacturing and controls studies enabled the collaborator to file an Investigational New Drug (IND) application with the US Food and Drug Administration and a Clinical Trial Application (CTA) with Health Canada. Both the IND and CTA were cleared, allowing the collaborator to initiate clinical trials in the US and Canada. See ClinicalTrials.gov, NCT04432623. | ||||||||
| 506 | Repurposing of a Long-Acting Parathyroid Hormone Analog for the Treatment of Hypoparathyroidism | Hypoparathyroidism is a rare hormone deficiency syndrome in which the body lacks parathyroid hormone (PTH). Due to PTH’s central role in maintaining the balance of calcium and phosphate in the blood, symptoms of hypoparathyroidism include muscle cramping, convulsions, intellectual disabilities, cataracts and abnormal heart rhythm. Hypoparathyroidism can occur after an injury to the parathyroid glands or following surgery or radiation treatment for thyroid cancer. It also may occur as a consequence of other rare genetic disorders or toxic exposures. Attempts to replace PTH have shown limited usefulness. Due to a persistent lack of calcium, patients must receive high-dose calcium supplements, which can have negative effects on the kidneys. The goal of this project is to develop a PTH replacement that will demonstrate a more normal, stable physiological level of PTH activity and lessen the need for chronic high-dose calcium supplements. Scientific Synopsis The primary symptoms of hypoparathyroidism are due to low serum calcium (hypocalcemia). Replacement of PTH has been explored to remedy the calcium deficiency, but maintaining an optimal calcium level has proven problematic because hypercalcemia can occur as a result of excess PTH. Multiple efforts are under way targeting either full-length PTH (PTH 1–84) or the active amino-terminal domain (PTH 1–34), but these molecules have undesirable pharmacokinetic properties for chronic daily management of calcium levels in patients with hypoparathyroidism. Eli Lilly scientists have identified a long-acting PTH receptor modulator (PTH-RM) that can normalize serum calcium. At fairly low doses, the PTH-RM was shown to normalize calcium levels in parathyroidectomized rats. The investigators are collaborating with TRND to develop this PTH-RM toward a Phase 2 proof-of-concept study for hypoparathyroidism by leveraging the existing data package. Lead Collaborator Lilly Research Laboratories, Eli Lilly and Company, Indianapolis Henry U. Bryant, Ph.D. Public Health Impact Insufficient PTH causes a range of symptoms, which must be managed in part through lifelong, high-dose supplements of calcium and vitamin D. Hospital and emergency room visits are common, and the high-dose supplements can cause kidney damage. Outcomes TRND scientists, in collaboration with researchers from Eli Lilly and Company, have further developed and validated the rat thyroparathyroidectomy model of hypoparathyroidism to generate robust efficacy data, including development of bioanalytical methods and assays. If all preclinical milestones are achieved, TRND plans to support the preparation and filing of the Investigational New Drug application with the Food and Drug Administration, as well as partnering with NIH clinical experts to support subsequent clinical trials in patients. | ||||||||
| 505 | Inhaled GM-CSF Therapy for Autoimmune Pulmonary Alveolar Proteinosis | Autoimmune pulmonary alveolar proteinosis (PAP) is a rare disease marked by an accumulation of surfactant (proteins and lipids) in the narrow gas exchange pockets of the lung, leading to respiratory failure. Patients generate antibodies against a normal protein, Granulocyte Macrophage Colony Stimulating Factor (GM-CSF), neutralizing its ability to signal to important immune cells (macrophages), whose function is critical for the proper clearance of accumulated surfactant. Current therapy requires lifelong, periodic washing of the lungs (whole lung lavage, or WLL) under general anesthesia, a risky and invasive procedure that is particularly problematic in children. The purpose of this project is to develop an inhaler-based formulation of the GM-CSF protein to stimulate PAP patients’ own immune cells to properly clear the lungs and thus avoid WLL. Scientific Synopsis In autoimmune PAP, patients generate antibodies against the GM-CSF protein normally found in circulation. GM-CSF plays an important role in the development and activation of macrophages. In the lungs, surfactant proteins and lipids facilitate gas exchange in the alveoli. Alveolar macrophages are responsible for maintaining surfactant homeostasis. If circulating autoantibodies prevent functional GM-CSF from reaching the alveoli, alveolar macrophages fail to clear accumulated surfactant. If not properly cleared, excess surfactant accumulation can lead to shortness of breath, decreased oxygenation, and increased risk of lung infections. The goal of this project is to develop inhaled recombinant human GM-CSF as therapy for autoimmune PAP and directly deliver GM-CSF to the lungs. As GM-CSF is currently used as an intravenous treatment for other conditions, the Food and Drug Administration (FDA) has required the completion of a formal toxicology study to ensure safety via the inhalation route before proceeding to further clinical trials in humans for PAP. Lead Collaborator Cincinnati Children’s Hospital Medical Center Bruce Trapnell, M.D. Public Health Impact Current WLL treatment for PAP requires general anesthesia and hospitalization. It is a risky and invasive procedure, particularly for children with the disease. No other therapeutic treatments are available for this rare disease. Inhaled GM-CSF would be a major improvement over standard care. Outcomes A comprehensive project plan was developed by TRND researchers and the lead collaborators at Cincinnati Children’s Hospital. The team subsequently entered into collaboration with Genzyme Corp., which provided essential research materials to the partnership. TRND scientists supported extensive preliminary toxicology and dosing studies in primates, which were necessary to demonstrate the safety of using inhaled GM-CSF. To ensure appropriate translation to the clinic, the TRND team obtained concurrence from Food and Drug Administration to conduct a limited Phase 1 PK/PD study in patients, the results of which will support the ongoing, Investigational New Drug-directed preclinical toxicology work in primates. The lead collaborator subsequently secured additional resources through the NCATS Division of Clinical Innovation, Clinical and Translational Science Awards Program to support a Phase 1 clinical evaluation of inhaled GM-CSF in autoimmune PAP patients, utilizing existing infrastructure provided by the Rare Lung Diseases Consortium. This TRND project is complete. | ||||||||
| 504 | NIH-Industry Partnerships Frequently Asked Questions | Overview How is this model different from other public-private partnerships for research that support the discovery and development of new medicines? How is the program speeding therapeutic development and saving money? What happens at the conclusion of the research project? How will new discoveries actually be turned into new medicines? Industry-Provided Assets Have all the assets been shelved by the company? What are the asset selection criteria? Are these assets approved by the Food and Drug Administration (FDA) for clinical use? Budget/Funding Will the pharmaceutical companies involved in each project be able to offer supplemental funding and resources if needed to complete the project? Overview How is this model different from other public-private partnerships for research that support the discovery and development of new medicines? Some other public-private partnerships enabled agreements between a single company and a single investigator or biomedical research institution. A key feature of this program is the involvement of multiple pharmaceutical companies and the potential for any U.S. researcher to participate. The program provides model template agreements between NIH and the pharmaceutical company and between the company and the biomedical research partner. The template agreements streamline and limit the amount of negotiation that is required before a project can begin. This program could serve as a model for similar collaborations among government, biomedical research organizations and industry. The program also provides open access to a limited amount of confidential information about partially developed therapeutic candidates (referred to as Assets) from participating pharmaceutical companies. How is the program speeding therapeutic development and saving money? A novel drug can take 10 to 15 years and more than $2 billion to develop, and failure rates occur in about 95 percent of human studies. This failure rate means that many existing therapeutic candidates could be repositioned for a new use and advanced to clinical trials more quickly than starting from scratch. Existing candidates already have undergone significant preclinical and Phase I safety testing and are ready for additional testing in humans. With the promising assets available to the entire research community through this program, investigators with great ideas have the opportunity to test hypotheses for new uses. This approach avoids research duplication and reduces the time and money required to determine if these well-developed assets can be used to treat a variety of important medical conditions. In addition, use of NCATS’ template agreements has shortened the amount of time it takes for each of the partners to negotiate the terms for research collaborations. Specifically, the time to establish collaborations between industry and academia for this program is only about three months, whereas typically it can take nine months to one year. These delays can result in a failure to launch the project when the science moves forward at a faster pace than the legal negotiations. What happens at the conclusion of the research project? How will new discoveries actually be turned into new medicines? NIH will support studies through Phase II clinical trials. The pharmaceutical company collaborator will have the first option to license the academic research partners’ new intellectual property arising out of the research. In cases where the pharmaceutical company collaborator owns active patents on an asset, the company will decide whether to advance the asset through further clinical studies to commercialize the new indication or to enable another company to do so. In cases where there are no longer active patents covering an asset and the pharmaceutical company passes on its commercialization option, the biomedical research partner is free to find another commercial collaborator. Commercial options and licenses also are covered in the collaborative research agreements on the Template Agreements page. Back to top Industry-Provided Assets Have all the assets been shelved by the company? Some assets are proprietary drug candidates that failed to show efficacy for the original indication or were deprioritized for business reasons and no longer are being pursued for their original therapeutic indication. Others are under active investigation for specific indications. What are the asset selection criteria? Assets selected for the program have advanced to clinical studies, and they have a safety profile, which allows further clinical investigation for other potential therapeutic uses. The mechanism of action for each compound is known, and pharmacokinetics is suitable for exploring the mechanism for a new indication. Are these assets approved by the Food and Drug Administration (FDA) for clinical use? None of the assets used in these studies are FDA-approved drugs. However, before any assets will be used in clinical studies, each investigator will file an investigator-sponsored Investigational New Drug application with the FDA to conduct the proposed clinical trials. Back to top Budget/Funding Will the pharmaceutical companies involved in each project be able to offer supplemental funding and resources if needed to complete the project? Yes. | ||||||||
| 503 | Development of Malaria Transmission-Blocking Drugs | Malaria is a parasitic disease that spreads through the bite of an infected mosquito. Malaria affects an estimated 250 million people worldwide, particularly in the tropical regions of Sub-Saharan Africa. The disease affects multiple organs in the body, and symptoms include cycles of chills, fever and sweating along with headaches, tiredness, muscle pain, vomiting and diarrhea. Current therapies generally lead to complete recovery, but approximately 650,000 patients die each year. Even while on current therapies, patients remain infectious for a period of time, allowing further mosquito-borne transmission to others. The purpose of this project is to develop a novel class of drugs that will not only prevent infection and relieve symptoms but also block mosquito-borne transmission from person to person. Scientific Synopsis Control of parasite transmission is critical for elimination and eradication of malaria. However, most antimalarial drugs are not active against sexual stage P. falciparum parasites — called gametocytes — which are responsible for the spread of malaria from person to person via mosquitoes. To begin to fill this void, investigators screened 5,215 known bioactive compounds and approved drugs for gametocytocidal activity. One compound with favorable pharmacokinetics, Torin2, was selected as the first candidate for further evaluation, including testing in an in vivo rodent malaria transmission model. Two 4 mg/kg doses completely blocked parasites’ ability to infect mosquitoes, and a 2 mg/kg dose gave a partial blockade, confirming the transmission-blocking activity of Torin2. Preliminary data indicate that the Torin2 target in P. falciparum is distinct from the mammalian target, which means researchers can design malaria-specific derivatives. Investigators used a gametocyte viability assay, a cellular mTOR assay and an in vivo rodent malaria transmission model to identify new malaria-specific Torin2 analogues. The goal of this project is to further optimize the Torin2 series and advance a drug candidate through preclinical development and early clinical development. Lead Collaborator Uniformed Services University of the Health Sciences, Bethesda, Maryland Kim Williamson, Ph.D. Public Health Impact Although current antimalarial treatments are effective, malaria still causes considerable numbers of deaths each year. Moreover, resistance to current therapies is a growing concern. Global health authorities have placed a high priority on developing new strategies for the control and elimination of malaria parasites. Developing a new class of drugs that not only treat the disease but also prevent its patient-to-patient spread would be an important step toward the goal of eradication. Outcomes Pilot studies between the lead collaborator and NCATS scientists resulted in identification of a series of compounds suitable for lead optimization. The TRND team initiated a comprehensive preclinical project plan, performing medicinal chemistry optimization to identify a lead candidate for further development. Currently, the project is in late-stage lead optimization, with the goal of identifying a compound with single-dose activity against all stages of the disease. Publication In vitro evaluation of imidazo[4,5-c]quinolin-2-ones as gametocytocidal antimalarial agents, Bioorg Med Chem Lett., 2016 Jun 15, 26(12):2907-11 | ||||||||
| 502 | New Therapeutic Uses in Action | Through its New Therapeutic Uses program, NCATS aims to improve the process of developing new treatments and cures for disease by finding new uses for existing therapies that already have cleared several key steps along the development path. Many existing or partially developed therapeutic candidates can be repurposed for use in new disease indications. Bringing together the best assets from pharmaceutical companies with the best new ideas from academic researchers could produce new treatments much more quickly than starting from scratch. Read the latest news about these partnerships below. September 2018 Templates for Success: Speeding the Formation of Public-Private Partnerships NCATS’ New Therapeutics Uses program is helping to speed the formation of innovative private-public partnerships through the development, demonstration and dissemination of template agreements, enabling smarter, faster science. NCATS Funding Available to Repurpose Existing Drugs On Sept. 6, 2018, NCATS announced two new funding opportunities for researchers to use partially developed therapeutic candidates to identify treatments for a broad range of diseases. April 2018 NCATS Funds Drug Repurposing Projects, Seeks New Industry Partners NCATS is seeking additional industry partners to participate in future New Therapeutic Uses funding opportunities. February 2017 NCATS Announces Funding Opportunities to Repurpose Existing Drugs Through Public-Private Partnerships In collaboration with AstraZeneca and Janssen Research & Development, LLC, NCATS is seeking applications to explore new treatments for patients using existing experimental drugs or biologics. Through its NIH-Industry Partnerships initiative, NCATS will support public-private partnerships by making a selection of industry assets available to test ideas for new therapeutic uses. August 2016 Bench Testing Therapeutic/Indication Pairing Strategies (UH2/UH3) NCATS announces the Bench-to-Clinic Repurposing initiative and related funding opportunity. The goal is to support investigators to repurpose — or identify new uses for — existing experimental drugs or biologics, as well as Food and Drug Administration-approved therapies already on the market. July 2015 NCATS Matches Researchers with Pharmaceutical Industry Assets to Test Ideas for New Therapies NCATS announces nearly $3 million to fund four drug repurposing projects aimed at finding therapies for type 2 diabetes, glioblastoma, acute myeloid leukemia and Chagas disease. March 2015 NCATS Support Leads to Clinical Trial to Test Repurposed Cancer Treatment as Alzheimer’s Therapy NCATS announces that scientists at Yale University have found that a compound originally developed as a cancer therapy could potentially be used to treat Alzheimer's disease. The team has begun testing the compound's effectiveness in humans. May 2014 NCATS Announces Funding Opportunities to Repurpose Drug Candidates from Industry NCATS announces the next round of New Therapeutic Uses funding opportunities to repurpose drug candidates from industry. For the first time, assets to treat pediatric indications will be available. July 2013 New Therapeutic Uses Projects NCATS releases video and audio clips of New Therapeutic Uses-funded investigators discussing the program and their research. May 2012 NIH Launches Collaborative Program with Industry and Researchers to Spur Therapeutic Development NIH, including NCATS, launches the Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program at the National Press Club in Washington, D.C. |
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