4-Amino-2-(piperidin-3-yl)isoindoline-1,3-diones as Anti-inflammatory Agents for Systemic Degenerative and Neurodegenerative Disorders

Summary:

The National Institute on Aging (NIA) seeks research co-development partners and/or licensees for the pre-clinical and clinical development of the compounds as anti-inflammatory therapeutics for systemic degenerative and neurodegenerative disorders.

Peanut therapeutics and diagnostics to treat severe food allergies

Up to 10% of the US population suffers from food allergies, with more than 40% of those experiencing life-threatening anaphylaxis. Peanut is one of the most common food allergens that give rise to persistent IgE-mediated food allergy. Oral immunotherapy (OIT) is used to reduce sensitivity to an allergen through repeated, small-dose exposure to the allergen. However, only a subset of patients develop a sustained response to the allergen and OIT carries notable side effects. 

Gene Therapy Vector for the Treatment of Glycogen Storage Disease Type Ia (GSD-Ia)

GSD-Ia is an inherited disorder of metabolism associated with life-threatening hypoglycemia, hepatic malignancy, and renal failure caused by the deficiency of glucose-6-phosphatase-alpha (G6Pase-alpha or G6PC). Current therapy, which primarily consists of dietary modification, fails to prevent long-term complications in many patients, including growth failure, gout, pulmonary hypertension, renal dysfunction, osteoporosis, and hepatocellular adenomas (HCA).

Assay to Screen Anti-metastatic Drugs

Scientists at the NCI developed a research tool, a murine cell line model (JygMC(A)) with a reporter construct, of spontaneous metastatic mammary carcinoma that resembles the human breast cancer metastatic process in a triple negative mammary tumor. The assay is useful for screening compounds that specifically inhibit pathways involved in mammary carcinoma and can improve clinical management of of triple negative breast cancer that are greatly refractory to conventional chemo and radiotherapy.

Novel Biased Potent Opioid-Like Agonists as Improved Medications to Treat Chronic and Acute Pain

There are no analgesics to ameliorate chronic pain without adverse side-effects (e.g., respiratory depression, gastrointestinal effects, tolerance, dependence), thus forcing patients into a difficult choice of negative impacts on quality of life. Most of the analgesics used for chronic and acute pain are drugs such as oxycodone, morphine, oxymorphone, and codeine. All of these opioids have been subject to misuse; prescription drug abuse is a severe problem worldwide, causing high mortality and greatly increased emergency room visits.

Therapeutic Antitumor Combination Containing TLR4 Agonist HMGN1

Immune checkpoint inhibitors (e.g. CTLA-4, PD-L1) have recently shown significant promise in the treatment of cancer.  However, when used alone, these checkpoint inhibitors are limited by the absence or repression of immune cells within the targeted cancer.  For those cancers associated with these limited immune systems, there remains a need for effective therapies.  Agents capable of recruiting and activating immune cells to these types of cancers could extend the overall and complete response rates of combination therapies within the immunooncology domain. 

Methods For Treating or Preventing Inflammation and Periodontitis

Bone-loss-related diseases, such as periodontitis, are characterized by an imbalance between the formation and activity of osteoblasts and osteoclasts, leading to bone loss. There are several signaling pathways that participate in the osteoclastogenesis process. Finding inhibitors of these pathways and other osteoclastogenesis-related pathways may have an effect on bone-loss diseases.