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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

Start date: November 1, 2010

Most recent report: To date, four children have undergone this procedure without serious side effects.  These children will be monitored to determine whether their disease slows, stops or reverses.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data leveraged into larger philanthropic or government funding for this treatment

 

Raza headshotInvestigator: Dr. Azra Raza and Dr. Naomi Galili

Research Institution: Columbia University

Disease: Myelodysplastic Syndrome

Research Description: Myelodysplastic syndrome (MDS) is a group of bone marrow disorders where the bone marrow cannot produce enough healthy cells. This leads to low blood cell counts and a higher risk for developing acute myeloid leukemia. In the last couple of years, our understanding of the molecular genetic basis of MDS has increased exponentially. This research will look at a specific type called of MDS called RARS, which is found in 10-15% of all MDS patients. The research team will identify mutations unique to MDS‐RARS patients, and use new technologies, such as CRISPR CAs9 gene editing, to see if altering certain genes can increase cell survival. They expect that these results will provide insights into repurposed therapies that can be rapidly developed and used in the clinic.

CWR fuding role: Primary funder 

Start date: August 1, 2012

Most recent report: A mutation found in about 60% of the MDS-RARS patients studied was identified. Patients with the mutation had better survival rates, and it has become the focus of the research project. Results from this research project have already been published in two high-profile scientific journals, PNAS and Molecular Cell. The research team has also begun to study anemia in MDS, which is a defining trait of the disease for many patients.

Anticipated next steps:

→ New data published in a scientific journal and/or presented at a scientific conference 

→ Findings from project led to unanticipated discoveries, now in testing

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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

Start date: December 21, 2012

Most recent report: Seven patients have been enrolled in the clinical study, and enrollment remains open to new patients. Tissue samples have been collected, and two patients are receiving treatment suggested based on analysis of their samples.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data leveraged into larger philanthropic or government funding for this treatment

→ Convert this pilot trial into a nationwide clinical trial for the entire Children's Oncology Group

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

Start date: July 31, 2014

Most recent report: Enrollment in the clinical trial is proceeding on schedule, with 13 patients enrolled as of March 2016. Enrollment is expected to be completed by February 2017, with results available approximately 3 months following.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data leveraged into larger philanthropic or government funding for this treatment

→ Use of this drug off-label for this disease indication in countries where it is available

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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

Start date: January 1, 2015

Most recent report: This trial will shortly begin enrolling patients. 

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data are supporting off-label clinical use

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SarahKeim picPrincipal Investigator: Dr. Sarah Keim

Disease: Prematurity

Research Description: In the U.S., 1 in 8 babies is born prematurely. Children who are born preterm have an increased risk of cognitive deficits and learning disabilities compared with children who are born at term. Several trials have found that dietary supplementation with a fatty acid during infancy moderately benefits cognitive development of preterm children. Studies have not examined whether supplementation during the second year of life is beneficial, which is important since brain development remains very active at this time. Dr. Keim will test whether or not preterm children benefit from supplementation after age 12 months, to inform treatment and dietary recommendations for these children.

CWR funding role: Primary funder

Start date: March 1, 2015

Most recent report: Enrollment for this clinical trial continues, with 60 new participants recruited since July 2015. Data collection is also ongoing, with a survey response rate of 83%. Survey data collected include cognition measures, physical characteristics and diet. Two recent enhancements were added to increase the survey response rate: an initial phone call to introduce the survey before it was sent to participants and an entry into a quarterly drawing for completing the survey.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data leveraged into larger philanthropic or government funding for this treatment

→ Data are supporting off-label clinical use

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pamela itkin ansari profilePrincipal Investigator: Dr. Pamela Itkin-Ansari

Disease: Type 1 Diabetes

Research Description: Type I diabetes is caused when the body’s immune system attacks cells in the pancreas that store and release insulin, thereby reducing or eliminating the body’s ability to secrete insulin in response to glucose. There are several ways to treat the chronic disease, including injecting insulin or transplanting cells, which then requires the patient to take anti‐ rejection drugs. Dr. Itkin‐Ansari and her team have previously placed insulin producing cells inside a repurposed, FDA approved encapsulation device that can implanted just under the skin. The device can serve as an “artificial pancreas” without the need for anti‐rejection drugs, and without the need to check the blood glucose levels or inject insulin. The next step is to “freeze‐dry” the loaded capsule, so that it can be shipped to doctors’ offices around the world. This research seeks to perfect the freeze drying process so the insulin producing cells can be woken up just prior to implantation.

CWR funding role: Primary funder

Start date: March 1, 2015

Most recent report: The researchers are currently developing new, proprietary technology for the freeze-drying process.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Pre-clinical findings lead to human clinical trial

→ Data leveraged into larger philanthropic or government funding for this treatment

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

Start date: July 1, 2015 

Most recent report: A significant number of patients are enrolling in this screening study and blood samples are being collected. A maximum of 200-300 patients are expected to enroll per month. The hope is to screen up to 10,000 patients having CT scans for lung cancer, in order to produce robust results.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data leads to a change in clinical practice to improve patient outcomes

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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

Start date: October 1, 2015

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data supporting marketing approval of this device for at least one of these disease indications

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Chani Traube HeadshotPrincipal Investigator: Dr. Chani Traube

Disease: Pediatric delirium

Research Description: Delirium is disturbed consciousness, cognition and/or acute change in mental status and inattention. Delirium is recognized as a significant complication of critical illness, associated with increased mortality in adults. A growing body of pediatric literature suggests that delirium is a serious and under-recognized problem in critically ill children, yet little research has focused on treatment. Quetiapine, an FDA approved atypical antipsychotic, may be repurposed as an effective off-label treatment option for pediatric delirium. The proposed clinical research trial will test the drug in children diagnosed with delirium in the pediatric intensive care unit.

CWR funding role: Primary funder

Start date: October 1, 2015

Most recent report: Patient recruitment and data collection for this clinical trial are currently underway.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data are supporting off-label clinical use

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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

Start date: Started in early 2016

Most recent report: Patient enrollment is proceeding on track, with three patients enrolled so far and a waitlist established.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data are supporting off-label clinical use

→ Data leveraged into larger philanthropic or government funding for this treatment

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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

Start date: Starting in 2016

Most recent report: Enrollment will begin soon, with an expected rate of 1-2 patients per month.

Anticipated next steps 

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data leveraged into larger philanthropic or government funding for this treatment

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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

Start date: Starting in 2016

Most recent report: Creation of the registry will begin in 2016.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data are supporting off-label clinical use in the pediatric population

→ Data leveraged into larger philanthropic or government funding for this treatment

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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

Start date: Starting in 2016

Most recent report: Patient enrollment will begin in mid 2016.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data leveraged into larger philanthropic or government funding for this treatment

→ Data are supporting off-label clinical use

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

Start date: Starting in 2016

Most recent report: Enrollment will begin in 2016

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data are supporting off-label clinical use

→ Data leveraged into larger philanthropic or government funding for this treatment

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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

Start date: Starting in 2016

Most recent report: Patient enrollment will begin in 2016.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data are supporting off-label clinical use

→ Data leveraged into larger philanthropic or government funding for this treatment

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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 nutriceutical, 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

Start date: May 2016

Most recent report: Recruitment for this study has begun, with six patients enrolled as of September 2016. Recruitment is expected to increase rapidly, as more clinical sites become involved in the process.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference

→ Data are supporting off-label clinical use

→ Data leveraged into larger philanthropic or government funding to further investigate this potential treatment

 

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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

Start date: November 2016

Most recent report: Patient enrollment is expected to begin before the end of 2016

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference

→ Data leveraged into larger philanthropic or government funding for this treatment

 

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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

Start date: December 2016

Most recent report: Project will begin enrolling patients and analyzing blood samples in December 2016.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference

→ Data are supporting off-label clinical use

→ Data leveraged into larger philanthropic or government funding for this treatment 

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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

Start date: December 2016

Most recent report: Tests of different doses of pindolol or buspirone plus an SSRI in mice will begin in late 2016.

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference

→ Pre-clinical findings lead to human clinical trial

→ Data leveraged into larger philanthropic or government funding for this treatment

 

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Rafael DavalosDavalos  RCGM Picture

Investigator: 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

Start date: November 2016

Most recent report: Tests on human pancreatic cancer cells to establish the optimal levels of IRE treatment are expected to begin before the end of 2016. 

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference→ Data leveraged into larger philanthropic or government funding for this treatment

→ Data leveraged into larger philanthropic or government funding for this treatment

→ Findings from project lead to additional discoveries for future testing

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