OMRF ADVANCES:

Diagnostics And Treatments

Scientists at the Oklahoma Medical Research Foundation are making incredible strides in developing experimental treatments and diagnostics for conditions ranging from heart disease to cancer and osteoarthritis.

Here are some recent breakthroughs that have the potential to transform patient care:

Treating Rare Conditions

Drs. Wan Hee Yoon and Lijun Xia have used gene editing technology to reverse genetic mutations that cause severe neurodevelopmental issues, metabolic issues and skeletal dysplasia in children. Meanwhile, Dr. Courtney Griffin has identified a potential therapeutic for eye diseases characterized by blood vessel overgrowth in premature infants and people with diabetic retinopathy. Finally, Dr. Rheal Towner has developed an antibody that reduces tumor volume and increases cell death in glioblastoma multiforme, a rare and highly malignant brain tumor.

DIAGNOSING SJÖGREN’S DISEASE

Sjögren’s disease affects as many as 4 million Americans. Current diagnostics for the painful autoimmune condition rely on biomarkers associated with Sjögren’s that fail to identify close to 40% of those with the disease. Drs. Darise Farris and Umesh Deshmukh have identified a distinct panel of biomarkers that stand to improve the diagnosis of the disease.

Healing Hearts

Heart disease is the number one cause of death globally, with patients with diabetes being particularly at risk. In OMRF’s Cardiovascular Biology Research Program, Dr. Chi Fung Lee has shown that inhibiting an NAD-degrading enzyme can elevate NAD+ levels and improve metabolic cardiomyopathy. In neighboring labs, Dr. Sathish Srinivasan has discovered that the absence of a gene causes mitral valve regurgitation and that inhibiting PDGF-β signaling can partially reverse this dysfunction, and Dr. Jasim Ahamed has identified a potential therapy for aortic stenosis by blocking the activation of a platelet-derived protein called TGF-β1.

Progress On Osteoarthritis

Despite the 32 million Americans living with osteoarthritis, no disease-modifying drugs exist for the debilitating condition. Scientists’ inability to accurately subset patients as progressors or non-progressors leads to failed clinical trials. At OMRF, Dr. Matlock Jeffries has identified novel epigenetic biomarkers that can predict OA progression. His work on OA-protected mice has also suggested that gut microbiome transplantation is a potential therapy for the condition.

Intervening In Als

Dr. Rheal Towner has discovered that OKN-007, an experimental anti-inflammatory compound, can protect motor neurons and prevent the late-stage progression of amyotrophic lateral sclerosis, better known as ALS. The drug also has potential to treat diseases such as Parkinson’s and Alzheimer’s and solid tumors like endometrial, colon and pancreatic cancers. It is currently in phase 2 trials to treat patients with glioblastoma, an aggressive brain cancer.

Treating Muscle Loss

As people age, they experience a loss of muscle mass and strength, which can become more pronounced after age 65. This can lead to difficulty performing daily tasks, making older adults more susceptible to falls. Dr. Holly Van Remmen’s research has shown that the reduced function of a calcium pump contributes to age-related muscle atrophy and that pharmacological stabilization of this pump can reverse these effects.