Vascarta is currently clinically focused on 2 conditions with great unmet medical need, Sickle Cell Disease and Osteoarthritis. However, Vascarta and collaborators also have conducted initial research showing the therapeutic potential of its Vasporta™ and anti-cancer linker technologies in 5 additional conditions. These 5 conditions are areas of unmet medical which are important on their own. Moreover, early research in 3 of these conditions also supports Vasceptor’s benefits for our clinical focus conditions of Sickle Cell Disease and Arthritis.
The table below provides links to promising early research in each of the 5 additional conditions:
VASCARTA PRE-CLINICAL RESEARCH IN OTHER CONDITIONS
| Platform/Drug Candidate | Indication | Also Supports Sickle Cell & Osteoarthritis Clinical Focus? |
|---|---|---|
| VASPORTA™ | NA | Yes |
| VAS-101 (Vasceptor®) | Red Blood Cell Preservation & Storage | Yes |
| VAS-101 (Vasceptor®) | Hypertension | Yes |
| VAS-101 (Vasceptor®) | Anti-Aging/Dementia | Yes |
| Quesceptor™ | Hypertension | Yes |
| STO-1 | Brain Cancer – Glioblastoma | No |
| STO-2 | Pancreatic Cancer | No |
Red Blood Cell Preservation and Storage
Condition Background
- Having healthy red blood cells is essential to treating many disease conditions including anemia, sickle cell anemia, and acute injuries.
- In the United States, about 15 million red blood cell (RBC) units are transfused each year. Worldwide, about 85 million RBC units are transfused each year. It is typically a challenge to obtain enough high quality blood to meet these transfusion requirements.
Vascarta Red Blood Cell Preservation and Storage Research – Key Findings to Date
- Stored red blood cells can undergo biochemical and structural changes called storage lesions which reduce their safety, therapeutic efficacy, and circulation lifetime when transfused. Recently, scientists from the Food & Drug Administration’s Laboratory of Biochemistry and Vascular Biology at the Center for Biologics Evaluation and Research, along with colleagues from the University of California San Diego and Albert Einstein College of Medicine found that these storage lesions can be mitigated by adding Vasceptor® to stored red blood cells.
- As visualized below, this published study shows that addition of a single dose of Vasceptor® (Curcumin) to stored red blood cells reduces oxidative damage by about 50%. Note the reduction in Reactive Oxygen Species (ROS) in the 14-day curcumin group versus the 14-day control (Con) group:
Vasceptor® reduces oxidative damage in stored red blood cells
The addition of a single dose of Vasceptor® to stored red blood cells also increases ATP (adenosine triphosphate) by about 40%. ATP is crucial because not only is it the molecule which produces energy in our cells, it also helps maintain the health of our vascular system.
Vasceptor® increases beneficial ATP in stored red blood cells
Statistical Significance levels: *P < 0.01 versus corresponding vehicle treated group
- The benefits shown by Vasceptor® in improving the health of red cells provided additional rationale for conducting human clinical testing of Vasceptor® in sickle cell disease.
Hypertension/High Blood Pressure
Disease Background
- Hypertension, also known as high blood pressure, is a chronic condition that occurs when the force of blood in your arteries is too high. It’s a common condition that can be serious if left untreated. Hypertension can increase your risk of heart disease and stroke, which are leading causes of death in the United States.
- According to the U.S. Centers for Disease Control (CDC), nearly 48% of U.S. adults had hypertension during August 2021–August 2023 (47.7%). The prevalence of hypertension increased with increasing age, with more than 70% of adults aged 60 and older having hypertension.
- The medical needs of many hypertension sufferers remain unsatisfied, since many common blood pressure medications can cause side effects like dizziness, drowsiness, headaches, nausea, fatigue, and sexual dysfunction, which can affect adherence to treatment plans.
Vascarta Hypertension Research – Key Findings to Date
- A key cause of hypertension is endothelial dysfunction, a condition where the inner lining of blood vessels (endothelium) does not function properly. Vascarta commissioned a study in which the blood vessel lining in rats was intentionally damaged causing leakage in the blood vessel. However, the application of Vasceptor® prevented the blood vessel damage and leakage:
Vasceptor® reduces blood vessel damage and leakage
Directly measuring the blood pressure of rats treated with Vasceptor® shows a substantial reduction in blood pressure lasting for hours. If this finding ultimately holds in human beings, there would be a large benefit to reducing blood pressure without unwanted side effects.
Vasceptor® reduces blood pressure in rats
Anti-aging
Disease Background
- There is an increasing gap between lifespan (how long you live) and healthspan (how long you remain healthy). According to recent research, there is a 9.6 year global gap between lifespan and healthspan, meaning people on average live 9.6 years in poor health. In the U.S., this gap is the largest in the world at 12.4 years.
- There is an urgent unmet need to improve our health, both physical and mental, as we age.
Vascarta Anti-Aging Research – Key Findings to Date
- Beginning at 20 months of age (equivalent of roughly 60-65 years old for human beings), male mice were treated only twice weekly with a topically applied placebo vehicle gel or Vasceptor® for 2-3 months.
- As shown below, Vasceptor® improves multiple aspects of health span in aged mice. Specifically, Vasceptor® tended to lead to a slight, albeit non-statistically different reduction in body weight (graph A). When assessing indicators of functional health and condition, Vasceptor® treated mice had improved coordination on balance beams of increasing difficulty, including fewer slips on easy, medium or hard difficulty beams (graph C). Vasceptor® significantly improved exercise capacity during an acute treadmill challenge (graph E) and led to a notably lower frailty index (graph F). If similar improvements in frailty, balance, and exercise capacity were to be experienced in human beings, this would have profound personal and societal benefits.
Vasceptor® improves multiple aspects of health span in aged mice, improving endurance and balance, while decreasing frailty
Statistical Significance levels: *p<0.05, **p<0.01
- Consistent with the findings on improved exercise capacity, there were improvements in key cardiac biomarkers. Specifically, the p16 protein which typically increases with aged hearts was significantly lower in Vasceptor®-treated mice (graph A). Similarly, the cytokine il-1b which signifies damaging inflammation in the heart was significantly lower in Vasceptor®-treated mice (graph B).
Vasceptor® improves key heart biomarkers – p16 and il-1b
Statistical Significance levels: *p<0.05, **p<0.01
- As a result of these and other findings, the authors of a recent anti-aging study write, “This effect of late-onset curcumin is somewhat reminiscent of rapamycin, which also has immune-modulating effects, and remains the most robust gerotherapeutic, with the most compelling evidence of efficacy among compounds initiated at middle to older ages.”
Brain Cancer/Glioblastoma
Disease Background
- Brain cancer is a malignant tumor that develops when abnormal cells in the brain grow out of control. It can be life-threatening because it can damage the brain’s vital structures. Glioblastoma is a fast-growing, aggressive, and deadly brain tumor that can affect people of any age. It is the most common primary brain cancer in adults.
- The overall 5-year survival rate for brain cancer is around 36%, meaning that roughly 36% of people diagnosed with a brain tumor will be alive 5 years after diagnosis; however, this rate varies significantly depending on the type of brain tumor and the patient’s age, with more aggressive tumors like glioblastoma having a much lower survival rate (around 5%).
- There is unmet medical need to improve the survivability of brain cancers like glioblastoma.
Anti-Cancer Linker Chemistry Background
- City University of New York’s (CUNY) novel, patent-pending technology exclusively licensed by Vascarta involves linking proven anti-cancer therapeutics such as paclitaxel (Taxol®), gemcitabine (Gemzar®), doxorubicin (Adriamycin®), and methotrexate (Jylamvo®) with curcumin. Linking the anti-cancer agents to curcumin optimizes the effectiveness of both agents. The linker breaks once the compound enters the cells within the tumor microenvironment.
- The curcumin-linked compounds can overcome many of the limitations of conventional chemotherapies, including by:
- Activating the beneficial immune system within the tumor environment without triggering systemic autoimmune responses
- Delivering anti-oxidant and anti-inflammatory properties that reduce typical negative side effects such as nausea, fatigue, nerve damage, appetite loss, brain fog, etc.
- Crossing the blood-brain barrier that prevents most chemotherapeutics from being effective against brain tumors such as glioblastomas.
- Offering in many cases a more convenient topical application usage experience versus intravenous infusion.
Vascarta-licensed Brain Cancer/Glioblastoma Technology – Key Finding To Date
- Initial results from testing in typically incurable glioblastoma brain tumors have been extremely positive. Mice were implanted with glioblastoma tumors. Some of the mice were injected with the novel compound STO-1 which links the proven anti-cancer therapeutic Taxol® (Paclitaxel or “PAC”) with curcumin while other mice were injected with a placebo.
- As shown below, all of the mice injected with placebo died in less than 45 days. However, 2/3rds of the mice receiving STO-1 remained alive through 85 days when the experiment ended, and these mice were sacrificed.
- This is a remarkably encouraging result in glioblastoma, where survivability is typically minimal.
Novel Compound STO-1 Increases Glioblastoma Survivability
Pancreatic Cancer
Disease Background
- Pancreatic cancer is a type of cancer that forms in the pancreas, a gland that produces digestive enzymes and hormones. It is often difficult to diagnose early, and most people are diagnosed when the cancer has spread to other organs.
- According to the American Cancer Society the relative 5-year survival rate for pancreatic cancer is around 13%, although this rate varies depending on the degree to which the cancer has spread when diagnosed.
- There is unmet medical need to improve the survivability of pancreatic cancer.
Anti-Cancer Linker Chemistry Background
- City University of New York’s (CUNY) novel, patent-pending technology exclusively licensed by Vascarta involves linking proven anti-cancer therapeutics such as paclitaxel (Taxol®), gemcitabine (Gemzar®), doxorubicin (Adriamycin®), and methotrexate (Jylamvo®) with curcumin. Linking the anti-cancer agents to curcumin optimizes the effectiveness of both agents. The linker breaks once the compound enters the cells within the tumor microenvironment.
- The curcumin-linked compounds can overcome many of the limitations of conventional chemotherapies, including by:
- Activating the beneficial immune system within the tumor environment without triggering systemic autoimmune responses
- Delivering anti-oxidant and anti-inflammatory properties that reduce typical negative side effects such as nausea, fatigue, nerve damage, appetite loss, brain fog, etc.
- Crossing the blood-brain barrier that prevents most chemotherapeutics from being effective against brain tumors such as glioblastomas.
- Offering in many cases a more convenient topical application usage experience versus intravenous infusion.
Vascarta-licensed Pancreatic Cancer Technology – Key Finding To Date
- Initial results from testing in typically poorly survivable pancreatic cancer tumors have been extremely positive. Mice were implanted with pancreatic cancer tumor cells. Then the mice were split into 3 groups:
- The red color group only received placebo treatment injected into their tumor cells
- The tumor cells of the blue color group were injected with the novel compound STO-2 which links the proven anti-cancer therapeutic Gemzar® (Gemcitabine) with curcumin
- The tumor cells of the orange color group received STO-2 delivered topically using Vascarta’s patented transdermal gel system, Vasporta™
- As shown below, the tumor cells in the placebo treated mice grew much larger while the tumors of the STO-2 treated mice were much smaller.
- These tumor size reductions are an extremely encouraging result in pancreatic cancer which is poorly survivable.
- Moreover, the strong results for STO-2 delivered topically using Vascarta’s Vasporta™ system provides hope that cancer treatment could be much more conveniently administered topically than via the typical injections/infusions.
Pancreatic Cancer Tumors Treated with STO-2 are Much Smaller – Even Delivered Topically
