RESEARCH ICIC HAS FUNDED

2015 Research Funding :

*Denotes that all funding listed has been matched by Alex’s Lemonade Stand Foundation, increasing the donation to twice the amount shown.

Pharmacogenomics and Drug Screening Lead to a Novel Targeted Therapy with Potent and Specific Activity Against Mouse Models of MYCN-amplified Neuroblastoma

Virginia Commonwealth University                                   Vrginia Commonwealth

Anthony Faber, Ph.D.

$75,000*

Using a combination of pharmacogenomics, drug screening and gene expression data mining, we have developed a novel targeted therapy combination that is specific for MYCN-amplified neuroblastoma. The goal of this grant is to further develop a combination targeted therapy to treat high-risk neuroblastoma.

Click Here to Read Faber Grant

Preclinical Development of ATR Inhibitor VE-822, Delivered Systemically in Nanoparticles, for Medulloblastoma TherapyUNC

University of North Carolina

Timothy Gershon, M.D./Ph.D.

$75,000*

This proposal will investigate the therapeutic potential of targeting the DNA damage response protein ATR for medulloblastoma therapy, using a small molecule inhibitor, VE- 822, in novel, nanoparticle formulation (pVE-822). Radiation and chemotherapy   significantly extend survival for most patients with medulloblastoma, but produce significant long-term neuro-cognitive side effects.

Click Here to Read Gershon Proposal

 

Small Molecule Inhibitors of ERG for Pediatric Leukemia and Sarcoma

University of Virginia, School of MedicineUVA

John Bushweller, Ph.D

$75,000*

Current treatment for childhood AML, T-ALL, and Ewing’s sarcoma is limited in efficacy and has profound long- term side-effects due to the use of traditional cytotoxic agents rather than targeted drugs inhibiting specific drivers of the diseases. A targeted agent which inhibits ERG, clearly a driver of these diseases, has the potential to improve both survival and quality of life for children with AML, T-ALL, and Ewing’s sarcoma

Click Here to Read Bushweller Proposal

 

2014 Research Funding:MCH_Color

Clinical Trial at Miami Children’s Hospital

John Raghelb, M.D.

$50,000

I Care I Cure’s funding facilitated the clinical trial for 10 children with recurrent brain cancer with an innovative and novel alternative method of giving chemotherapy directly to the site of the tumor. This study will determine if infusion of chemotherapeutic agents directly into brain fluid spaces is a safe and effective method of delivering chemotherapy treatment for children with recurrent malignant brain tumors.

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Development of Targeted Leukemia Inhibitors Using Nanoparticle Delivery

University of Massachusetts Medical School, Lucio Castilla, PhD    UMMS-For-2col-RGB

$100,000*

Research using the targeted therapy of soluble gold nanoparticles as inert and versatile scaffolds to deliver potent leukemia inhibitors aimed at eliminating cancer cells, while having a negligible impact in normal cells.

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Targeted Inhibition of the DNA Damage Response Pathway: A Novel Intervention in the Treatment of Pediatric Leukemia

National Cancer Institute, Andre Nessenzweig, PhDNCI

$100,000*

Research based on DNA damage response proteins to target therapeutic intervention as an inhibitor. These interventions are directed at specific cells, pathways or proteins that are indicative of heightened points of vulnerability in cancers, without the use of broad spectrum approaches.

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*Indicates funds were matched by Alex’s Lemonade Stand Foundation, increasing the donation amount to $200,000 for each research project

2013 Research Funding:

Development of a Pharmacodynamic Marker of EWS-FLII Activity to Aid in the Clinical Translation of Targeted Therapies for Ewings Sarcomalogo-vanderbilt

Vanderbilt University; Patrick J. Grohar, M.D., PhD

$53,000*

Research for a new therapy that targets the dominant oncogene of Ewing Sarcoma, EWS-FLI1, to improve survival rates.

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Development of a Novel Mer TKI for Treatment of ALLUniversity_of_Colorado_Denver_193464

University of Colorado, Denver; Dr. Doug Graham

$53,000*

Research of treatment for acute lymphoblastic leukemia (ALL) by targeting Mer signals (which have been shown to contribute to ALL) with biological agent UNC2025.

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Treatment of Medulloblastomacity of hope

Beckman Research Institute of City of Hope; Margarita Gutova, M.D.

$53,000*

Research for a more effective treatment for medulloblastoma (MB), using neural stem cells that produce anti-cancer drugs localized directly to MB cells to limit toxicity.

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Engineering the Lymph Node Environment with Therapeutic Vaccine Depots to Combat Neuroblastomauniversity of maryland

University of Maryland, College Park; Christopher M. Jewell, PhD

$53,000*

Research combining direct lymph node delivery and vaccines to generate immune responses to combat neuroblastoma.

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*Indicates funds were matched by Alex’s Lemonade Stand Foundation, increasing the donation amount to $106,000 for each research project

2012 Research Funding:

Selective Inhibition of Nuclear Export as a Novel Therapeutic Strategy in AML

Dana-Farber Cancer Institute; Julia Etchin, PhDdanafarberlogo

$50,000*

To examine the current treatments for Acute Myelogenous Leukemia (AML), which often fail to induce long-term remission. Knowledge gained from this study will make a significant contribution toward identifying highly active targeted treatments for AML that are less toxic to normal tissues.

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Identification and Validation of New Therapeutic Targets in Pediatric Leukemia Through an Aneuploidy-based Synthetic Lethality Screen

Dana-Farber Cancer Institute; David Pellman, M.D.danafarberlogo

$50,000*

Research of Trisomy 8, which is abnormal number of chromosomes, that is present in 15-20% of cases of AML and MDS. Data is being analyzed to determine which genes selectively kill trisomy 8 AML cell lines.

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Development of An Innovative Treatment Strategy for Neuroblastoma and Other Pediatric Tumors: A Novel Phospholipid Ester Analogue and its Multifunctional Cancer-targeting Nanoconstructuwlogohome

University of Wisconsin-Madison; Mario Otto, M.D., PhD

$50,000*

Study will look at the molecule NM404 to ascertain if NM404 can target and kill neuroblastoma cells or other forms of childhood cancer cells, and to evaluate if this compound could be a new potent treatment drug.

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Development of Targeted Therapies in High Risk Childhood Acute Lymphoblastic LeukemiaCHOP

The Children’s Hospital of Philadelphia; Sarah Tasian, M.D.

$50,000*

Research will focus on studying blood and bone marrow samples from children with certain types of high-risk Acute Lymphoblastic Leukemia (ALL) with the goal of improving our understanding of the functional consequences of specific genetic mutations. Through this work, she hopes to improve understanding of the critical biologic mechanisms involved in these leukemias to help identify new targeted therapies for children with high-risk ALL.

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*Indicates funds were matched by Alex’s Lemonade Stand Foundation, increasing the donation amount to $100,000 for each research project

2011 Research funding:

131I-MIBG Imaging and Therapy for Neuroblastoma

University of California San Francisco; Kate Matthay, M.D.UCSFBCHLogo_2clr

$62,500*

Research to use a new tumor-targeted therapy for neuroblastoma—131I-MIBG which can deliver radioactivity directly to the tumors. This method is more effective and less toxic than current methods which do not deliver directly to the tumors.

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Mechanisms of Oncogenic Transformation by TLX1/HOX11um_seal

Regents of University of Michigan; Mark Yat-fung Chiang, M.D., PhD

$40,000*

To advance the understanding of the biology of TLX1-driven leukemogenesis so as to identify important genes that may lead to more effective and less toxic therapies as more than one third of ALL survivors suffer long-term consequences such as second cancers.

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Targeted Polymeric Parthenolide Carriers to Treat Childhood AMLrochester

University of Rochester; Danielle Benoit, PhD

$40,000*

Research to develop more effective treatments for children with AML by employing a polymer delivery system that would increase the solubility of parthenolide (PTL). This would allow it to be used for leukemia therapy, as it is only soluble at around 1 mg/ml in aqueous solutions. Polymer nanoparticles used for loading a leukemia therapeutic have been successfully created and thoroughly analyzed in vitro for their potential as therapeutically relevant drug carriers in mice.

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Targeting Polycomb-mediated Epigenetic Silencing in T cell Acute Lymphoblastic Leukemialogo-vanderbilt

Vanderbilt University Medical Center; Emmanuel Volanakis, M.D.

$40,000*

Research to understand the molecular signals the allow leukemia cells to thrive at the expense of normal cells by observing certain signaling mechanisms.

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Development of Childhood Leukemia Translational Research Programdanafarberlogo

Dana-Farber Cancer Institute; Lewis B. Silverman, M.D.

$62,500*

Research to identify, develop, and test novel treatments and outcomes for children and adolescents with ALL and AML.

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*Indicates funds were matched by Alex’s Lemonade Stand Foundation, increasing the donation amount to either $80,000 or $125,000, depending on the grant amount, for each research project

2010 Research Funding:

131I-MIBG Imaging and Therapy for NeuroblastomaUCSFBCHLogo_2clr

University of California San Francisco; Kate Matthay, M.D.

$62,500

Research to use a new tumor-targeted therapy for neuroblastoma—131I-MIBG which can deliver radioactivity directly to the tumors. This method is more effective and less toxic than current methods which do not deliver directly to the tumors.

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Discovery of Inhibitors of MLL Fusion Proteins as Targeted Therapies for Pediatric Acute LeukemiaDupont_hospital_for_children_logo

Alfred I DuPont Hospital for Children; Andrew Napper, PhD

$100,000

Research of selective killing of leukemia cells with MLL fusions with a cell-penetrating AF4 peptide; study extended to leukemia engrafted in mice.

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Targeted Polymeric Parthenolide Carriers to Treat Childhood AMLrochester

University of Rochester; Danielle Benoit, PhD

$40,000

Research to develop more effective treatments for children with AML by employing a polymer delivery system that would increase the solubility of parthenolide (PTL). This would allow it to be used for leukemia therapy, as it is only soluble at around 1 mg/ml in aqueous solutions. Polymer nanoparticles used for loading a leukemia therapeutic have been successfully created and thoroughly analyzed in vitro for their potential as therapeutically relevant drug carriers in mice.

2009 Research Funding:

Targeted regulation of Acetylation as Novel Therapy for Ewing’s Sarcoma

Georgetown University; Jeffrey Torestky, M.D.georgetown

$100,000

Research found that EWS-FLI1 in Ewing sarcoma cells is only minimally or not at all regulated by acetylation. Cell lines continue to be tested for sensitivity and causes of cell death.

2008 Research Funding:

Targeting the FLT3 Signaling Pathway in MLL-AF9 LeukemiaCincinnatiChildrensHospital

Cincinnati Children’s Hospital; Dr. Kevin Link

$25,000

Anticipate a new treatment based on research that has been successful to date.

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Role of Notch1 in T-cell Development and Leukemianyu_logo (1)

NYU; Dr. Severine Chathelin

$25,000

New discovery about survival and self-renewal of stem cell; studies have opened the way for pharmacological targeting.

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