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Crystal HeadshotInvestigator: Dr. Ron Crystal

Disease: Batten Disease

Research Description: Batten Diseases comprise a group of genetic diseases that affect children. Children with these gene defects are missing key enzymes that help to break down waste products in brain cells. These waste products build up, and eventually destroy the brain cells. This causes a loss of function in these children, and eventually death. Dr. Crystal and his team are undertaking a pilot clinical trial to determine the impact of putting a working copy of the affected gene into a virus that can then be injected into the brain. These brain cells would produce the missing enzyme, allowing the brain cells to survive and the children to thrive. If successful, this study could produce a life-saving treatment for children with Batten Disease.

CWR funding role: Primary funder

CWR Start date: November 2010

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Sarah Leary headshotPrincipal Investigator: Dr. Sarah Leary

Disease: Pediatric brain cancer 

Research Description: Pediatric brain tumors are the most common solid tumor malignancy in children, and continue to be a leading cause of pediatric death from disease.  Standard treatment for pediatric brain tumor patients continues to be the chemotherapy and radiation therapy developed decades ago, which results in significant toxicity. Hundreds of new-generation “targeted” cancer therapies have been developed, with success in adults. Over a dozen of these targeted therapies have undergone safety testing in the pediatric population, but none have been successfully incorporated into pediatric brain tumor treatment. This study is a pilot clinical trial in pediatric patients with recurrent brain tumors. Patients whose cancers express a potential target will be treated with a drug developed to inhibit that specific target. All medications prescribed will be FDA-approved drugs for which a pediatric dose has been established, but without indication in pediatric brain tumors.

CWR funding role: Primary funder

CWR Start date: December 2012

Lanctot-KristaPrincipal Investigator: Dr. Krista Lanctôt

Research Institution: University of Toronto

Disease: Alzheimer's

Research Description: Alzheimer’s disease (AD) is commonly associated with behavioral changes such as agitation. Severe agitation is important to treat because it not only increases progression of AD and physical health problems (increased falls and weight loss), but it also increases caregiver stress. Currently prescribed treatments for agitation in AD do not work for all patients. When they do work, the effect is small, and they increase the risk of harmful side effects, including increased risk of mortality. As a result, there is an urgent need for safer medication options. Cannabinoids, such as nabilone, can now be prescribed in capsule form, and their calming, appetite and pain killing effects may have benefits in those with agitation. Through this clinical trial, Dr. Lanctôt hopes to identify the benefits of nabilone in the treatment of agitation in AD.

CWR funding role: Participating funder

CWR Start date: July 2014

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faustman medium 1 Principal Investigator: Dr. Denise Faustman

Disease: Type 1 Diabetes

Research Description: Type 1 diabetes (T1D) is a serious autoimmune disease that occurs when the body mistakenly attacks and destroys the insulin‐producing cells of the pancreas. It affects up to 3 million Americans—many of whom are children—who rely on insulin injections or pumps to live. This project is testing Bacillus Calmette‐Guérin (BCG), an inexpensive generic vaccine, as a treatment for people with TID, including those with long-standing T1D. The team recently completed a Phase I study, and the goal of this Phase II will be to identify a dosage amount and schedule that will put T1D into long‐term remission and allow some level of insulin production to begin again. There is strong scientific evidence that the insulin‐producing cells of the pancreas continue to regenerate in T1D patients, even decades after the disease starts. If the researchers can reduce the immune attack on these cells, it is hoped that they will start to deliver insulin again.

CWR funding role: Participating funder

CWR Start date: January 2015

Rush University Medical Center

Borgia headshotPrincipal Investigator:  Dr. Jeffrey Borgia

Disease: Lung cancer

Research Description: Early detection of lung cancer can save lives. Low‐dose computer tomography (CT) screening in high‐risk lung cancer patients has been shown to reduce mortality. However, only a small percentage of the suspicious lesions identified on CT scans are malignant, leading to unnecessary biopsies or surgeries in many patients. Dr. Borgia and his team are repurposing a blood diagnostic test Dr. Borgia developed with prior Cures Within Reach funding to help clinicians rule out non‐malignant lung cancer cases in other clinical circumstances. This research project will repurpose this diagnostic test to determine if it could help sort out malignant from non‐malignant lesions, thereby improving physician decision making, patient care and outcomes and reducing healthcare costs following a CT screening scan.

CWR funding role: Primary funder

CWR Start date: July 2015 

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Principal Investigators: Dr. James Young, Dr. Max Fitzgerald

Disease: MS, Parkinson's, stroke, traumatic brain injury

Research Description: Neurological conditions (including MS, Parkinson’s disease, stroke and traumatic brain injury) cause mobility impairment that is described by patients as the most intrusive symptoms impacting their quality of life. A form of electric stimulation administered with a device placed on the tongue has been studied for many years in patients with a variety of neurological disabilities. A portable version of the device, referred to as the Portable Neuromodulation Stimulator or PoNS, is being repurposed experimentally to improve gait and balance difficulties for people with these neurological disabilities. In this pilot study, Drs. Young and Fitzgerald are evaluating the effectiveness of combining the PoNS device with customized exercise/therapy routines to improve gait and balance for MS and Parkinson’s patients with moderate difficulties with mobility. They also expect to see other potential benefits to mood, quality of life and other functional aspects of daily living.

CWR funding role: Primary funder

CWR Start date: October 2015

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Logan Wink HeadshotPrincipal Investigator: Dr. Logan Wink

Disease: Autism

Research Description: One in 68 children is diagnosed with an Autism Spectrum Disorder (ASD). Individuals with ASD endure lifelong deficits in social communication and interaction, with restricted patterns of behavior or interests. They often also exhibit associated symptoms including hyperactivity and irritability. Historically, treatment for ASD in children has been most successful in treating these associated symptoms. To date, no medication has been shown in controlled trials to improve the core defining features of ASD. As the population of children, adolescents, and adults with ASD grows, there is a pressing medical need to develop effective and safe treatment for the core features of this disorder, thereby improving outcomes. Dr. Wink and her team will conduct a pilot clinical study of intranasal ketamine, a generic drug, in individuals with ASD to determine its effect on the core features.

CWR funding role: Participating funder

CWR Start date: 2016

 

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Riggins HeadshotPrincipal Investigator: Dr. Gregory Riggins

Research Institution: Johns Hopkins University 

Disease: Medulloblastoma, Glioblastoma

Research Description: Medulloblastoma is the most common malignant brain tumor in children, and brain cancers are the second leading cause of pediatric cancer-related deaths. Dr. Riggins’s recent studies have shown the antiparasitic drug mebendazole to be effective in animal models of aggressive brain tumors, including advanced gliomas and medulloblastomas. Because mebendazole has a history of safe use in patients, it has a relatively short pathway to new clinical trials. Dr. Riggins and his team have improved the formulation and anticancer performance of oral mebendazole, and this Phase I trial will examine dosage levels in recurrent pediatric brain cancers resistant to therapy. They hope to demonstrate not only safety, but to eventually provide the evidence and rationale for future Phase II studies.

CWR funding role: Participating funder

CWR Start date: 2016

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Kahn HeadshotPrincipal Investigator: Dr. Stacy Kahn

Disease: Clostridium difficile infection

Research Description: Fecal microbiota transplantation (FMT) is the repurposing of human stool to deliver healthy bacteria to the gastrointestinal tract of individuals with disease, most notably Clostridium difficile infection (CDI). CDI is an increasing health care burden and is the leading cause of hospital-associated gastrointestinal illness. FMT has become a cost effective and life-saving treatment for patients with recurrent and refractory Clostridium difficile infection who have not responded to conventional antibiotic treatment. Dr. Kahn will create a pediatric patient registry to study FMT in children. This patient registry will be used to study the natural history of CDI and the safety and efficacy of treatment with repurposed stool through FMT. Dr. Kahn will also pursue a proof concept clinical trial testing swallowed human microbiota capsules to treat CDI infections in children.

CWR funding role: Primary funder

CWR Start date: 2016

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Havelka photo copyPrincipal Investigators: Dr. George Havelka, Dr. Darwin Eton

Disease: Chronic limb-threatening ischemia

Research Description: Patients with Chronic Limb‐threatening Ischemia (CLI) suffer from impeded blood flow to their limbs. They present with foot pain, ulceration and/or gangrene. They require risky and costly revascularization operations to avoid amputation, although these surgeries are ineffective in many cases and amputations are sometimes required. Drs. Havelka and Eton have found that combining a compression device with a drug originally approved for use in cancer patients restored vascularization, significantly improves patient outcomes, eliminates the need for surgery and reduces amputation rates. This clinical study of CLI patients will describe the molecular effects of the repurposed treatment.

CWR funding role: Primary funder

CWR Start date: 2016

Halatsch HeadshotPrincipal Investigator: Dr. Marc-Eric Halatsch

Research Institution: Ulm University 

Disease: Glioblastoma 

Research Description: For decades, new experimental therapies have repeatedly failed to improve the outcome of patients relapsing after initial treatment of the brain cancer glioblastoma. The chemotherapy drug temozolomide is used for the initial treatment of these brain cancers. Dr. Halatsch and his team surveyed the research literature looking for approved non-cancer drugs that inhibit key cellular pathways in glioblastoma. Using selection criteria including low likelihood of increasing patient side effects, good quality of life maintenance and adequate clinical experience with the drug, they identified nine repurposed drugs. These drugs will be given together in combinations with low-dose, uninterrupted, daily temozolomide. The primary objective is to assess the safety and tolerability of the combination treatment in patients with recurrent glioblastoma. The secondary objectives are to evaluate overall survival, progression-free survival after six months and best tumor response.

CWR funding role: Participating funder

CWR Start date: 2016

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Cox headshotPrincipal Investigator: Dr. Zachary Cox

Disease: Heart failure

Research Description: Approximately 90% of heart failure admissions result from too much fluid in the blood. Loop diuretics are often used to treat this condition. However, some patients develop a resistance to loop diuretics, resulting in a recurrence of symptoms. Yet despite this frequent resistance, current international guidelines provide no clear guidance on the best method to restore diuretic efficacy. While the combination of loop diuretics with oral thiazides is the common practice, the current data on this treatment is limited. Off-label use of other diuretics, such as intravenous chlorothiazide or tolvaptan, in combination with loop diuretics might lead to better outcomes with less adverse events. This proof of concept clinical trial will compare the efficacy and safety of new diuretic combination therapies to overcome loop diuretic resistance in patients with heart failure.

CWR funding role: Participating funder

CWR Start date: 2016

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Benjamin KimInvestigator: Dr. Benjamin Kim

Disease: Macular Degeneration

Research Description: The retina is the light sensing tissue that lines the inside of the back of the eye. In the geographic atrophy type of age-related macular degeneration (AMD), areas in the central retina waste away or develop “atrophic” lesions. These lesions lead to blind spots in the patient’s vision, which interfere with many important activities for these patients such as reading, driving, watching television, and recognizing faces. These lesions are responsible for 20% of the legal blindness in North America, and there currently is no treatment at all. Thus, there is a critical need to find a cure for this disease. This study will investigate the use of Alpha Lipoic Acid (ALA), a nutraceutical, to treat geographic atrophy from AMD. ALA was approved years ago in Germany for the treatment of diabetic peripheral neuropathy, is available over-the-counter and has an excellent safety profile in humans. We hypothesize that oral ALA supplementation reduces the rate of enlargement of the blinding lesions. Given that there is no treatment for these blinding retinal lesions, the potential impact of this study is tremendous, and the implementation of ALA as a treatment would be expected to be relatively quick.

CWR funding role: Participating Funder

CWR Start date: May 2016

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Desmond OathesInvestigator: Dr. Desmond Oathes

Disease: Depression and Posttraumatic stress disorder

Research Description: Repetitive transcranial magnetic stimulation (TMS) is a non-invasive FDA approved treatment for depression that does not respond well to medication. However, recent research suggests that using information from a patient’s brain images can help to improve TMS treatment outcomes. In addition, TMS has not yet been established for patients suffering from posttraumatic stress disorder (PTSD). We will use our combined expertise in brain imaging techniques and in TMS to treat two specific areas in the brains of patients who suffer from either depression, PTSD or both. Our expectation is that TMS treatment to our new brain target will improve symptoms in depression patients, compared to the current FDA approved treatment. We also expect that symptoms of PTSD will be improved with our targeted treatment. Therefore, the results from our study could form the basis of a new treatment for both PTSD and depression.

CWR funding role: Primary Funder

CWR Start date: November 2016

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Investigators: Dr. James Dowling and Dr. Carsten Bönnemann

Disease: Myotubular Myopathy

Research Description: X-linked myotubular myopathy (MTM) is a rare disorder that affects males and is caused by mutations in the MTM1 gene. It is one of the most severe neuromuscular diseases, with 1/4 to 1/3 of patients dying in the first year of life. The affected survivors have significant physical impairments and typically die before reaching adulthood.  Currently, there are no cures or even disease-modifying therapies for this devastating disorder. The research team has discovered that the anti-cancer drug tamoxifen promotes a significant reduction of MTM symptoms in mice.  The researchers will conduct a clinical trial to test the ability of repurposed tamoxifen to improve motor function in individuals with MTM. The trial will include 5 patients at the Hospital for Sick Children, and 5 patients at National Institutes of Health. This research will be supported through a collaborative funding effort between Cures Within Reach and the Canadian Institutes of Health Research, designed to fund proof of concept repurposing clinical trials in rare diseases and to promote collaboration between U.S. and Canadian research institutions.

CWR funding role: Participating funder

CWR Start date: August 2017

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Michael Levine

Levine Sochett headshotInvestigators: Dr. Michael Levine and Dr. Étienne Sochett

Disease: CYP24A1 mutations, hypercalcemia, hypercalciuria

Research Description: Patients with loss of function mutations in the CYP24A1 gene are unable to degrade vitamin D byproducts. This leads to clinical consequences that include low levels of calcium, osteoporosis and renal stones. There is currently no accepted treatment that can reverse the effects of this mutation. A low-calcium diet is the only current option, but it is mostly ineffective. The research team will investigate if repurposing rifampin, a commonly used antibiotic, can provide a highly beneficial and long-term treatment for these patients. They will conduct a clinical trial among 5 patients with CY24A1 mutations. This research will be supported through a collaborative funding effort between Cures Within Reach and the Canadian Institutes of Health Research, designed to fund proof of concept repurposing clinical trials in rare diseases and to promote collaboration between U.S. and Canadian research institutions.

CWR funding role: Participating Funder

CWR Start date: December 2016

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Kristen HollingerInvestigator: Dr. Kristen Hollinger

Disease: Pediatric Depression

Research Description: Suicide due to major depressive disorder (MDD) has simultaneously become a leading cause of death around the world (particularly among children, adolescents and young adults) and a growing silent epidemic for which we have made little to no progress for decades. The current standard of care for severe MDD includes antidepressant drug treatment, such as a selective serotonin reuptake inhibitor (SSRI), often in combination with cognitive behavioral therapy. It takes approximately 4 weeks for SSRIs to produce mood-lifting effects, and during that time, particularly in pediatric populations, an increase in suicidal behaviors can be seen. Our research suggests that a rapid drop in serotonin release during acute exposure to antidepressant treatment could account for this increase in suicidal behaviors. To overcome the initial drop in serotonin, we propose the use of repurposed drugs, pindolol and buspirone, to be delivered in combination with a SSRI antidepressant for the first month of treatment. Pindolol and buspirone block a specific serotonergic receptor in the brain, thereby allowing more serotonin to be released during acute antidepressant treatment. This combination has the potential to prevent suicidal behaviors following SSRI treatment. Preclinical studies in mice will evaluate the efficacy and appropriate doses of pindolol or buspirone to be translated to human studies.

CWR funding role: Primary funder

CWR Start date: December 2016

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Rafael DavalosDavalos  RCGM PictureInvestigator: Dr. Rafael Davalos (Virginia Tech)

Co-investigator: Dr. Robert C.G. Martin, II (University of Louisville)

Disease: Pancreatic Cancer

Research Description: Irreversible electroporation (IRE) is a non-thermal, focused procedure that relies on the delivery of short, yet intense, electrical pulses to destroy the target tissue. These pulses damage the cell membrane, which in turn kills the cells. IRE is ideally suited to treat patients with tumors that cannot be removed via surgery (unresectable), because it destroys the tumor without damaging the surrounding tissue. The use of IRE has been shown to dramatically improve the lifespan of thousands of patients suffering from unresectable prostate, liver, pancreatic and adrenal cancers. This clinical project will use an IRE device to treat pancreatic cancer patients and will track the immune response to this IRE treatment. The IRE treatment will be optimized to increase the immune response in patients, which then could be used to help fight the cancer. Results from this study will be used to effectively combine IRE with immunotherapy in subsequent studies, with the hope of improving patient outcomes in pancreatic cancer.

CWR funding role: Primary funder

CWR Start date: November 2016

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Nadler headshotInvestigator: Dr. Evan Nadler

Disease: Biliary Atresia

Research Description: Biliary atresia (BA) is a rare disease, occurring in approximately 1 in 10,000-20,000 live births. It is the leading cause of chronic end-stage liver disease in children, and the leading indication for liver transplantation in pediatric patients. In BA, obliteration of the biliary tract and complete obstruction of bile flow leads to progressive liver fibrosis and cirrhosis. BA is lethal, with a median survival of 10 months. Prompt surgical treatment by 2 months of age relieves biliary obstruction and improves BA mortality. Yet long-term survival after surgery without liver transplantation is low. The majority of surgical patients eventually require liver transplantation. Despite current treatment efforts, BA is the most serious and costly liver disease in infants. Granulocyte-Colony Stimulating Factor (GCSF), a drug used in cancer treatment, has been shown to improve liver function in mice and in other adult liver conditions. These data suggest that GCSF may be used as innovative support therapy for BA, both in surgical patients and in patients who did not have the benefits of surgery. This project will test the dosing and safety of repurposed GCSF therapy in newborns and infants with BA, and will be the basis for a future multi-national, multi-institutional trial of GCSF therapy in children.

CWR funding role: Primary funder

CWR Start date: January 2018

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Swiecicki WhiteCoatInvestigator: Dr. Paul Swiecicki

Disease: Thyroid Cancer

Research Description: Thyroid cancer is the sixteenth most common malignancy in the world. Although most patients are cured with a combination of surgery, radioactive iodine therapy, and thyroid stimulating hormone suppression, approximately 15% of patients will develop treatment-resistant (refractory) disease. In the U.S. alone, roughly 9,400 patients fall into this category each year. Currently, the treatment options for refractory thyroid cancer are limited and are mostly palliative in intent. Previous molecular and genomic analyses in thyroid cancer have identified potential targets for repurposed therapeutics. The research team looked at other cancers with similar targets, and saw they responded to a combination of the generic drugs cyclophosphamide and sirolimus. The team tried this combination in patients with aggressive thyroid cancer and anecdotally had success. They now propose a clinical trial repurposing cyclophosphamide and sirolimus to evaluate if this is a safe and effective combination therapy to treat refractory thyroid cancer. This is of profound interest given the lack of current treatment options in the patient population and potential healthcare cost savings with this repurposed combination.

CWR funding role: Participating funder

CWR Start date: January 2018

 

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