Oral Iron-Chelator Therapy for Treating Developmental Stuttering

This technology discloses the use of small-molecule iron chelators—drugs that bind and remove excess iron—for the oral treatment of developmental stuttering in children and adults. Mouse models carrying human stuttering mutations show both elevated striatal iron and impaired vocalization; daily low-dose deferiprone reverses these speech-like deficits while normalizing brain-iron MRI signals.

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.

A Protocol to Enhance Therapeutic Effects of Transcranial Magnetic Stimulation and the Methods to Realize It

Summary: 

The National Institute on Drug Abuse (NIDA) seeks research co-development partners and/or licensees for a high-powered electronic device and coil that delivers Transcranial Magnetic Stimulation (TMS) pulses as well as the software that controls the device for treating treatment resistant depression, substance use disorders and other CNS disorders.

Description of Technology: 

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. 

Polypeptides for Stimulation of Immune Response (Adjuvants)

HMGN polypeptides belong to the high mobility group (HMG) family of chromosomal binding peptides. HMGN polypeptides typically function inside the cell nucleus to bind to DNA and nucleosomes and regulate the transcription of various genes. HMGN polypeptides also can be released by peripheral blood mononuclear cells. However, the extracellular release of a HMGN polypeptide initiates activation of the immune system. Therefore, it has potential use as a biological therapeutic for stimulating an immune response.

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.