Areas of Focus
At the Warren Center for Neuroscience Drug Discovery (WCNDD), we are dedicated to advancing treatments for a range of neurological and psychiatric disorders. Our integrated approach combines cutting-edge research with pharmaceutical-grade infrastructure to address the complexities of these conditions. Below are our primary areas of focus:
-
Problem Statement
Alzheimer’s disease is the most common form of dementia, affecting more than 6 million Americans. It leads to progressive memory loss, cognitive decline, and behavioral changes, posing immense emotional and economic burdens on patients, caregivers, and healthcare systems. (Alzheimer’s Association, 2024 Alzheimer's Disease Facts and Figures.)Current Treatments
Current FDA-approved treatments provide limited symptomatic relief and do not significantly alter disease progression. Most therapies target amyloid-beta or tau proteins, but with modest success in clinical outcomes.WCNDD’s Approach
At WCNDD, we are pioneering new therapeutic strategies that go beyond traditional targets. Our research focuses on modulating muscarinic acetylcholine receptors—key components of the brain’s cholinergic system that are impaired in Alzheimer’s disease.Novel Strategies
We have advanced novel positive allosteric modulators (PAMs) of the M1 receptor into early clinical development. These compounds aim to restore cognitive function while avoiding the side effects of older cholinergic therapies. By enhancing receptor function only in the presence of natural neurotransmitters, our approach offers a more precise and safer path to symptom relief and disease modification. -
Problem Statement
Anxiety disorders affect approximately 12.5% of U.S. adults, leading to significant distress and impairment in daily functioning. (National Institute of Mental Health (NIMH), Any Anxiety Disorder.)
Current Treatments
Treatment options include psychotherapy and pharmacotherapy, such as selective serotonin reuptake inhibitors (SSRIs). Despite these interventions, many individuals continue to experience persistent symptoms or adverse effects from medications.
WCNDD's Approach
We are investigating the neurobiological substrates of anxiety to identify novel therapeutic targets. Our goal is to develop treatments that are both effective and have a rapid onset of action.
Novel Strategies
One innovative avenue involves the modulation of specific ion channels and receptors that regulate neuronal excitability. By targeting these mechanisms, we aim to create therapies that alleviate anxiety with minimal side effects.
At WCNDD, our commitment to translational research drives us to transform scientific discoveries into tangible treatments, addressing the unmet needs of patients with neurological and psychiatric disorders.
-
Problem Statement
Brain tumors are among the deadliest cancers, with glioblastoma having a 5-year survival rate of only 5%. (American Brain Tumor Association; American Cancer Society, Glioblastoma Survival Rates.)
Current Treatments
Treatments include surgery, radiation, and chemotherapy, but outcomes remain poor, especially for aggressive forms of brain tumors.
WCNDD's Approach
We are developing novel immunotherapies and targeted treatments to more effectively treat brain tumors.
Novel Strategies
Our team is investigating the use of oncolytic viruses and immune checkpoint inhibitors to target brain tumor cells with precision.
-
Problem Statement
Depression is a prevalent mental health disorder, with approximately 21.4% of U.S. adults experiencing symptoms in 2022. This condition contributes to significant morbidity and mortality, including an increased risk of suicide. (CDC, National Center for Health Statistics, Mental Health – Household Pulse Survey, 2022.)
Standard treatments encompass antidepressant medications and psychotherapy. However, a substantial portion of patients do not achieve remission with existing therapies, highlighting the need for novel treatment modalities.
WCNDD's Approach
Our research focuses on identifying new molecular targets implicated in depression. By elucidating the underlying pathophysiology, we aim to develop compounds that offer improved efficacy and reduced side effects compared to current options.
Novel Strategies
We are pioneering the development of allosteric modulators that fine-tune neurotransmitter systems involved in mood regulation. This approach holds the promise of more precise therapeutic effects with a favorable safety profile.
-
Problem Statement
Down syndrome affects approximately 1 in 700 live births, causing intellectual disabilities and a higher risk of early-onset Alzheimer’s disease. (CDC, Facts about Down Syndrome; Alzheimer’s Association.)
Current Treatments
There is no cure for Down syndrome, but early interventions such as speech, physical, and occupational therapies can help improve quality of life.
WCNDD's Approach
We are investigating ways to enhance cognitive function and address the neurodegenerative aspects of Down syndrome.
Novel Strategies
Our research focuses on compounds that could enhance brain plasticity and slow the progression of Alzheimer’s-like symptoms in individuals with Down syndrome.
-
Problem Statement
Dystonia is a neurological disorder that causes involuntary muscle contractions, affecting approximately 250,000 people in the U.S. These contractions can lead to abnormal postures, pain, and disability. (Dystonia Medical Research Foundation, Dystonia Overview.)
Current Treatments
Treatments typically include botulinum toxin injections and oral medications such as anticholinergics and muscle relaxants, which offer partial relief but are not curative.
WCNDD's Approach
WCNDD is exploring new molecular targets to improve treatment outcomes for dystonia patients. Our team is investigating gene therapy and neuromodulation techniques as innovative approaches for managing dystonia symptoms.
Novel Strategies
We are developing a novel neuromodulatory treatment aimed at controlling the excessive neural signaling that causes muscle contractions. This therapy promises to offer greater precision and fewer side effects than current treatments.
-
Problem Statement
Epilepsy affects about 3.4 million people in the U.S., with a lifetime prevalence of 1 in 26 people. Despite effective treatments, one-third of patients experience uncontrolled seizures. (CDC, Epilepsy Data and Statistics.)
Current Treatments
Current treatments for epilepsy include anticonvulsant drugs and surgical interventions, but many patients still suffer from breakthrough seizures.
WCNDD's Approach
Our research focuses on identifying new drug targets for the treatment of epilepsy, with an emphasis on the development of fast-acting and long-lasting therapies.
Novel Strategies
We are investigating the use of ion channel modulators to control seizure activity at the molecular level, offering potential for more precise and effective treatment.
-
Problem Statement
Antimicrobial resistance is a growing threat, with resistant infections contributing to millions of deaths annually. (CDC, Antibiotic Resistance Threats in the United States, 2019; WHO.)
Current Treatments
Existing antibiotics are becoming less effective against resistant bacteria, leading to the urgent need for new treatments.
WCNDD's Approach
We are focused on discovering novel antibacterial agents that target unique bacterial mechanisms.
Novel Strategies
Our research focuses on developing compounds that disrupt bacterial communication and virulence, making it harder for bacteria to develop resistance.
-
Problem Statement
Kidney disease affects approximately 37 million Americans, with chronic kidney disease leading to end-stage renal disease in many cases. (CDC, Chronic Kidney Disease in the United States, 2021.)
Current Treatments
Current treatments include dialysis and kidney transplantation, but these options are not always viable or effective.
WCNDD's Approach
We are exploring regenerative medicine and novel drug candidates to prevent or reverse kidney damage.
Novel Strategies
Our researchers are investigating compounds that stimulate kidney regeneration, potentially offering a new path forward in treatment.
-
Problem Statement
Neurological diseases such as Alzheimer's, Parkinson’s, and multiple sclerosis involve progressive neuronal damage.(NIH/NINDS, general literature; Alzheimer’s and Parkinson’s foundations.)
Current Treatments
Existing treatments primarily focus on symptom management rather than halting or reversing the disease process.
WCNDD's Approach
We are developing neuroprotective therapies aimed at preserving neuronal function and slowing disease progression.
Novel Strategies
Our research focuses on compounds that can modulate neuroinflammation and protect against oxidative stress, crucial factors in neurodegenerative diseases.
-
Problem Statement
Chronic pain affects over 50 million adults in the U.S., with opioid addiction contributing to an ongoing public health crisis. (CDC, Chronic Pain Among Adults — United States, 2019.)
Current Treatments
Opioid medications are widely prescribed but carry significant risks of addiction and overdose. Non-opioid treatments include NSAIDs, but they often provide insufficient relief.
WCNDD's Approach
We are researching non-opioid alternatives by focusing on novel pain receptors and pathways that mediate pain, aiming to create safer, more effective treatments.
Novel Strategies
One promising strategy involves targeting TRPV1 receptors to modulate pain without the risk of addiction associated with opioids.
-
Problem Statement
Obesity is a growing global epidemic, affecting over 42% of adults in the U.S. and contributing to numerous health complications, including cardiovascular disease and diabetes. (CDC, Adult Obesity Facts.)
Current Treatments
Current interventions include lifestyle changes, medications (such as orlistat), and bariatric surgery. However, weight loss is often difficult to maintain, and treatments can have side effects.
WCNDD's Approach
Our team is working on developing metabolic modulators that target the brain's regulation of hunger and energy expenditure.
Novel Strategies
We are exploring the use of neuropeptide Y antagonists to control appetite and improve metabolism, potentially offering a more effective long-term solution for obesity.
-
Problem Statement
Parkinson's disease affects approximately 10 million people globally. Beyond motor symptoms, about one-third of these individuals experience significant anxiety, which severely impacts their quality of life. (Parkinson’s Foundation, Statistics; NIH/National Institute of Neurological Disorders and Stroke.)
Current Treatments
While treatments like deep brain stimulation (DBS) are employed to manage motor symptoms, options for addressing non-motor symptoms such as anxiety are limited and often inadequate.
WCNDD's Approach
Our team is investigating the neural mechanisms underlying anxiety in Parkinson's disease. Recent studies have identified specific brain wave patterns associated with anxiety levels, providing new targets for therapeutic intervention.
Novel Strategies
Building on these findings, we are exploring the potential of adaptive deep brain stimulation techniques. These approaches aim to modulate aberrant brain activity in real-time, offering a personalized treatment strategy for alleviating anxiety in Parkinson's patients.
-
Problem Statement
PWS is a rare genetic disorder that causes severe obesity, developmental delays, and cognitive impairment. Approximately 1 in 15,000 to 20,000 births are affected by PWS. (Prader-Willi Syndrome Association USA, About PWS; NIH Genetic and Rare Diseases Information Center (GARD).)
Current Treatments
There are no FDA-approved treatments for the underlying genetic causes of PWS. Symptomatic treatments include growth hormone therapy to improve height and body composition, but they do not address the root cause.
WCNDD's Approach
We are working on genetic therapies that target the specific genetic mutations in PWS, aiming to restore normal function in the hypothalamus, the brain region responsible for regulating hunger and metabolism.
Novel Strategies
Our researchers are developing a targeted RNA therapy that could potentially correct the genetic imbalance in PWS, offering a more sustainable and effective solution than current symptomatic treatments.
-
Problem Statement
PAH is a rare, life-threatening disease affecting the lungs, with an estimated 100,000 people diagnosed in the U.S. (Pulmonary Hypertension Association, About PH; NIH GARD.)
Current Treatments
Current treatments focus on dilating blood vessels and reducing pulmonary pressure, but they do not address the root causes of PAH.
WCNDD's Approach
Our team is working on developing targeted therapies to address the underlying molecular causes of PAH.
Novel Strategies
We are investigating the use of gene therapy to repair the genetic mutations that lead to vascular remodeling in the lungs.
-
Problem Statement
Rett syndrome is a rare genetic disorder that affects mostly females, causing severe cognitive and physical impairments. The incidence is approximately 1 in 10,000 to 15,000 live female births. (Rett Syndrome Research Trust; NIH GARD, Rett Syndrome.)
Current Treatments
Current treatments are primarily focused on symptom management, such as physical therapy and medications to control seizures. There is no cure.
WCNDD's Approach
We are exploring gene-based therapies to restore normal neural function and potentially reverse the course of Rett syndrome.
Novel Strategies
Our team is investigating a gene-editing approach to correct the mutations causing Rett syndrome, offering hope for a disease-modifying treatment.
-
Problem Statement
Schizophrenia is a severe mental disorder affecting approximately 24 million people worldwide. In the United States, prevalence estimates range from 0.25% to 1.6% of the adult population, translating to about 3.8 million individuals. This condition significantly impairs cognitive and emotional functioning, leading to challenges in daily living and social interactions. (World Health Organization (WHO), 2022; National Institute of Mental Health (NIMH), Prevalence of Schizophrenia.)
Current Treatments
Existing antipsychotic medications primarily target positive symptoms such as hallucinations and delusions. However, these treatments often come with substantial side effects and offer limited efficacy against negative symptoms like social withdrawal and cognitive deficits.
WCNDD's Approach
Our researchers have developed a novel compound in collaboration with Boehringer Ingelheim that shows promise in treating both positive and negative symptoms of schizophrenia. Preclinical studies in rodent models have demonstrated the compound's potential efficacy, marking a significant advancement in therapeutic options.
Novel Strategies
In partnership with Neumora Therapeutics, we have advanced a potential schizophrenia treatment into Phase 1 clinical trials. This investigational drug, discovered at WCNDD, represents a new class of therapeutics aimed at addressing unmet clinical needs in schizophrenia care.
-
Problem Statement
Scleroderma is a rare autoimmune disease that causes hardening and scarring of the skin and internal organs, affecting an estimated 240,000 Americans.(Scleroderma Foundation, About Scleroderma; NIH GARD.)
Current Treatments
There are no cures for scleroderma, and treatments focus on managing symptoms, such as immunosuppressants and antifibrotic therapies.
WCNDD's Approach
We are exploring new therapies to target the fibrotic processes in scleroderma, aiming to slow disease progression and improve organ function.
Novel Strategies
Our researchers are focusing on developing small molecules that block fibrosis-promoting signals in the body.
-
Problem Statement
Substance use disorder affects millions globally, with alcohol, nicotine, and illicit drugs like opioids being the most commonly abused substances. This disorder leads to severe social, physical, and psychological consequences. (National Institute on Drug Abuse (NIDA), Substance Use in America.)
Current Treatments
Current treatments for substance use disorder include behavioral therapy and medications like methadone and buprenorphine, but relapse rates remain high.
WCNDD's Approach
At WCNDD, we are focused on developing novel pharmacological agents that target the neural circuits involved in addiction.
Novel Strategies
Our researchers are exploring the potential of targeting the brain's reward system with selective modulators that offer more precision and fewer side effects compared to current treatments.