Part the Cloud

2018 Grant Recipients

Stephen Cunnane, Ph.D., University of Sherbrooke (Sherbrooke, Canada)
RCT with a ketone ester drink in MCI
This Phase II clinical trial will determine if a nutrition rich treatment can serve as an alternative brain fuel in people with mild cognitive impairment. Learn more.

Charbel Moussa, Ph.D., Georgetown University (Washington, D.C.)
Bosutinib effects on safety, biomarkers and clinical outcomes in DLB
Bosutinib effects on safety, biomarkers and clinical outcomes in DLB This Phase Ib clinical trial will determine if a FDA-approved leukemia drug is safe in people with Dementia with Lewy Bodies, a type of dementia similar to Alzheimer's. Learn more.

Stefano Sensi, M.D., Ph.D., Universita' degli Studi Gabriele d'Annunzio di Chieti (Pescara, Italy)
Extenzin-based therapy for MCI subjects
This phase II clinical trial will determine if zinc therapy will slow or prevent mild cognitive impairment from progressing to Alzheimer's disease. Learn more.

Dieter Willbold, Ph.D., Research Center Juelick GmbH (Germany)
Placebo controlled multi-ascending dose [targeting protein aggregation] Phase 1 study in healthy volunteers
This Phase I clinical trial will test the safety of D3D, an experimental drug designed to slow harmful beta-amyloid protein accumulation during Alzheimer's disease. Learn more.

Nawaf Yassi, M.D., Ph.D., University of Melbourne (Melbourne, Australia)
S-Adenosyl Methionine for Alzheimer’s disease
S-Adenosyl Methionine for Alzheimer's disease This Phase II clinical trial will examine if boosting the levels of a naturally occurring protein, S-adenosyl methionine (SAMe) helps slow the progression of Alzheimer's disease and reduces the level of harmful tau protein levels in the body. Learn more.

Part the Cloud to RESCUE Brain Cell Degeneration in Alzheimer's Disease

The PART THE CLOUD to RESCUE (REverse, reStore, Cease and UndErstand) Brain Cell Degeneration in Alzheimer's disease program will accelerate the discovery and testing of innovative compounds to be used for interventions in the earliest stages of neurodegeneration-dementia-Alzheimer's disease.

Presently there are no effective interventions to delay or prevent the progression of neurodegenerative processes that underlie the disabling symptoms of Alzheimer's dementia. To date, clinical trials based on current theories of the disease pathogenesis have not resulted in viable treatments. Therefore, the primary focus of PTC to RESCUE Brain Cell Degeneration in Alzheimer's disease program is to encourage the discovery and development of a wide-range of interventions to maintain and/or restore the health and functionality of neurons and/or connectivity of neural networks.

The PTC to RESCUE Brain Cell Degeneration in Alzheimer's disease program will promote human studies to advance innovative ideas for early phase human trials (Phase 1 or Phase 2a proof of concept) that addresses therapies to target neuron health in Alzheimer's disease.

Part the Cloud to RESCUE Grant Recipients

From a pool of 45 letters of intent, 20 applications were submitted for funding through Part the Cloud to RESCUE. These six applications were deemed the most meritorious and will each receive $1 million over the course of the next two years.

Roberta Diaz Brinton, Ph.D., University of Arizona (Tuscon, AZ)
Advancing Allopregnanolone as a Regenerative Therapeutic for Alzheimer's
This phase 2a study will test whether allopregnanolone, an important hormone in brain function, is able to regenerate brain cells and restore function in people with Alzheimer's.

Rafael de la Torre, PharmD, Ph.D., Institute Mar of Medical Investigations (Barcelona, Spain)
Cognitive decline in early stages of AD after EGCG and a multimodal therapy
This phase 1 study will test the potential of reversing cognitive changes in people with early stage Alzheimer's using a high dose of Epigallocatechin gallate, which is found in lower levels in many foods (like green tea).

Xue Hua, Ph.D., Athira Pharma, Inc. (Seattle, WA)
Phase 2a Alzheimer's trials of a novel neurotrophic activator, NDX-1017
This phase 2a study is evaluating a novel compound, NDX-1017, to determine its potential in maintaining and possibly improving the brain cell signalling pathways and benefit brain health in people with Alzheimer's.

Paul A. Newhouse, M.D., Vanderbilt University (Nashville, TN)
M1-PAM VU319 Effects on Network Connectivity in MCI: A POC Study
This phase 2a study will evaluate the potential of a a novel molecule, M1-PAM VU319 to improve brain cell communications in this first-in-human clinical study.

Intent-to-fund: Raymond Tesi, M.D., INmune Bio (La Jolla, CA)
A biomarker directed study to reduce inflammation in Alzheimer's disease
This phase 1 study will evaluate a novel compound, XPRO1595 that aims to reduce inflammation and reverse brain cell death pathways.

Stephen M. Strittmatter, M.D., Ph.D., Yale University (New Haven, CT)
Silent Allosteric Modulation of mGluR5 for Alzheimer's Disease
Targeting a relatively new biology, this phase 1b study will test a new compound for its potential to target brain cell communication systems for improved brain cell health.

2017 Grant Recipients

Krista L. Lanctot, Ph.D., Sunnybrook Research Institute (Ontario, Canada)
Linking GSH and Cognitive Response: A Pilot Phase 2a Study of NAC in VCIND
Lanctot and colleagues will evaluate whether a compound known as N-acetylsysteine (NAC), which can boost glutathione production, may improve cognition and brain health in the study's participants. As part of this effort, the participants will receive a series of cognitive tests to determine how NAC treatment may improve cognitive function over time. Learn more.

Manfred Windisch, Ph.D., Neurokine Therapeutics, LLC (Philadelphia, Pennsylvania)
Phase 1 Study of MW150: Novel Stress Kinase Inhibitor Candidate
Windisch and colleagues will devote their research grant to conducting a phase 1 human clinical trial of MW150. The trial will determine what dosage levels of the drug are safe and tolerated with an ascending dose study in humans. It will also clarify how the drug is absorbed and metabolized in the human body. For this effort, the investigators will test their drug on older human volunteers without dementia. The participants will be grouped in cohorts of 10. Eight people in each group will receive the drug, while the other two will receive a placebo. Learn more.

Part the Cloud Challenge on Neuroinflammation

A first-of-its kind competition, the Part the Cloud Neurodegeneration Challenge was created to deepen our understanding of neurodegeneration and accelerate therapeutics to be used in early clinical trials. Scientists throughout the globe were invited to submit proposals that could translate into human trials of treatments targeting neuroinflammation with the goal of improving cognition in individuals with neurodegenerative diseases.

The hope: to fund research that increases our understanding of neurodegeneration and accelerates the development of new therapies to slow, stop, or prevent its progression.

Neuroinflammation is prevalent in many diseases of the brain. Identifying therapies that target this process will have broad implications for treating a large number of the devastating brain diseases including Amyotrophic Lateral Sclerosis, Vascular Dementia, Frontotemporal Dementia, Parkinson's, Huntington's and Alzheimer's.

2016 Grant Recipients

Dr. Linda Van Eldik, University of Kentucky
Phase 1b MAD Study of a Novel Drug (MW189) Targeting Neuroinflammation
Phase 1b multiple dose safety study of a novel drug, MW189, that targets a specific type of immune cell and reduces its activity, targeting inflammation pathways seen in disease.

Dr. Joseph Foss, NeuroTherapia, Inc. (Cleveland, OH)
A Phase I single ascending dose safety and pharmacokinetic study of NTRX-07
Phase I safety study of a single dose for the experimental drug NTRX-07; NTRX-07 targets the cannabinoid type 2 receptor (CBR2) which is involved in the body's innate immunity and thought to be increased in Alzheimer’s.

Dr. Mark Tuszynski, University of California, San Diego
A Clinical Trial of BDNF Gene Therapy in Alzheimer’s Disease
Phase 1 safety study on BDNF (brain derived neurotrophic factor) gene therapy using a novel technology to target delivery and provide nutrients to the brain cells most impacted in Alzheimer’s.

Dr. Frantz Hefti, Proclara Biosciences (Cambridge, MA)
Phase 1 Study with NPT088, a Fusion Protein to Treat Alzheimer's Disease
Phase 1 safety study of novel drug NPT088; NPT088 is thought to target proteins that clump together, including beta-amyloid and tau protein, two hallmark brain changes in Alzheimer’s.

2015 Grant Recipients

Paul A. Newhouse, Vanderbilt University
Phase 1 Testing of a Muscarinic M1 PAM for Alzheimer's Disease
Newhouse and colleagues will test a novel drug, VU0467319, which acts on the muscarinic system in the brain that controls cell-cell communication and nerve cell function. As the first drug of this type to be tested in humans, this study will not only advance a potential therapeutic for Alzheimer's but will also open up the door to this entire class of drugs for future exploration. Learn more.

Mitchel Kling, University of Pennsylvania
A Biomarker-Based Trial of Plasmalogen Repletion in MCI/AD
Kling and colleagues will conduct a Phase 1 clinical study to investigate the man-made lipid, PPI-1011, a type of plasmalogen. This study will not only advance a potential therapeutic for Alzheimer's into Phase 2 but will also advance our understanding of plasmalogen as a marker of biological significance in Alzheimer's. .

Stephen Cunnane, Ph.D., University of Sherbrooke - Québec, Canada
Proof of Mechanism of a New Ketogenic Supplement Using Dual Tracer PET
This Phase 1b/2a clinical trial will examine the safety and effectiveness of a novel dietary supplement as an alternative fuel source for the brain to stabilize or reverse declining memory observed in early stages of Alzheimer's disease. The scientists will use advanced brain imaging to determine if the proposed therapy improves brain blood flow, energy use and helps preserve memory function.

Giulio Maria Pasinetti, M.D., Ph.D., Icahn School of Medicine at Mount Sinai - New York, New York
BDPP Treatment for Mild Cognitive Impairment and Prediabetes
This Phase 1b clinical trial will examine the safety and tolerability of a combination treatment using three grape-derived compounds in people with very early stages of Alzheimer's disease. This novel treatment contains a mixture of polyphenols, which are plant compounds thought to support brain health.

Russell Swerdlow, M.D., University of Kansas Medical Center - Fairway, Kansas
Trial of Oxaloacetate in Alzheimer's Disease (TOAD) Study
This Phase 1b clinical trial will examine the safety and effectiveness of oxaloacetate as an alternative energy source to improve brain function in individuals with Alzheimer's disease. Providing the brain an alternative fuel source may help slow or prevent brain changes associated with the disease process.
Update: Dr. Russell Swerdlow, PTC 2015 awardee, discusses possible biological pathways important for Alzheimer's disease in recent PRI interview

Tim West, Ph.D., C2N Diagnostics - St. Louis, Missouri
A Single Ascending-Dose, Double-Blinded, Placebo-Controlled Study of an Anti-Tau Antibody
This Phase 1 clinical trial will examine the safety and effectiveness of an antibody against tau to treat various dementias, including Progressive Supranuclear Palsy (PSP) and Alzheimer's disease. The results will inform future clinical trials designed to determine if the antibody removes tau from the human brain and results in improved cognitive function.
Update: PTC 2015 awardee C2N Diagnostics partners with AbbVie for advancing tau antibodies in clinical trials for Alzheimer's disease and Progressive Superior Palsy (PSP)

Whitney Wharton, Ph.D., Emory University - Atlanta, Georgia
Mechanistic Potential of Antihypertensives in Preclinical Alzheimer's
This Phase 1b clinical trial will determine if the FDA-approved antihypertensive drug, perindopril, may work by mechanisms other than lowering blood pressure to ultimately reduce Alzheimer's risk in African-Americans with a family history of Alzheimer's disease.

2013 Grant Recipients

Adam L. Boxer, M.D, Ph.D., University of California, San Francisco
Phase I Multiple Ascending Dose Trial of the MT Stabilizer TPI-287 for AD
Dr. Boxer and his team are working to moderate the stability of microtubules — small, tube-like structures that act like a skeleton inside cells, maintain cell structure and help to transport nutrients throughout the cell —potentially decreasing the abnormal buildup of tau protein into tangles, one of the hallmark brain changes of Alzheimer's disease.
Update: UCSF team – led by Adam Boxer - launched the safety clinical study with TPI-287. Further, Dr. Boxer received a significant award to establish a clinical research team to study loss of brain cell function in dementias. Dr. Adam Boxer is highlighted as part of the international tau partnership supported by Richard Rainwater.

Frank Longo, M.D., Stanford University and Anne Longo, Pharmatrophix, Inc.
Phase I Trial for P75 Receptor Ligand
This study aims to test a therapy that targets the cell death pathway associated with Alzheimer's disease by inhibiting a known component. The researchers hope to ultimately determine whether this decreases the detrimental effects of Alzheimer's in the brain.
Update: Dr. Frank Longo and his group at Pharmatrophix are gearing up to launch their next clinical study thanks to the success of their initial safety study, partially funded by PTC.
Dr. Frank Longo, PTC 2013 recipient, is part of team investigating underlying biology of why stroke may be a risk factor for dementia.

Ahmad Salehi, M.D., Ph.D., Palo Alto Institute for Research & Education, Inc.
Improving ß2 Adrenergic Signaling in Alzheimer's Disease
Researchers at the Palo Alto Institute for Research & Education, Inc., are exploring potential therapeutics to improve the health of brain connections thought to play an early role in the disease processes.

Dale E. Bredesen, M.D., Buck Institute for Research on Aging
An Exploratory Safety, PK/PD, and Preliminary Efficacy Study of F03 in MCI
This study strives to impact the activity level of Amyloid Precursor Protein (APP), the complex responsible for the production of the beta amyloid — thereby potentially influencing the amount of beta amyloid produced in the brain.
Update: Part the Cloud awardee Dr. Dale Bredesen published a report on 10 individuals who saw reversal of cognition issues following a personalized lifestyle-intervention.

Keith Vossel, M.D., University of California, San Francisco and Gladstone Institute for Neurological Disease
Phase 2a Levetiracetam Trial for AD-Associated Network Hyperexcitability
Led by Dr. Keith Vossel, these scientists are working to moderate the amount of underlying electrical activity of the brain cells associated with Alzheimer's disease to potentially impact the rate an individuals' cognition declines.

Mike Weiner, MD., University of California, San Francisco
The Part the Cloud initiative also supported, in part, Mike Weiner, MD (principal investigator of ADNI) and the Whole Genome Sequencing Alzheimer's Disease Neuroimaging Initiative (WGS-ADNI), a project to sequence the whole genome of more than 800 individuals. The genome data will be available to researchers around the world this summer, enabling further study of the genes related to Alzheimer's.
Update: The competitive renewal of the Alzheimer's Disease Neuroimaging Initiative (ADNI), where Mike Weiner, MD serves as principal investigator, got a very good score after review at the National Institutes of Health (NIH) and will be funded for 5 more years, making it the largest funded program on Alzheimer's in the United States.

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