Inflammatory and Immune
Inflammatory and immune disorders represent a significant burden to society. There are over 80 recognized autoimmune disorders, which are conditions caused by an acute or chronic imbalance in the immune system. In these conditions, cells of the immune system begin to attack certain tissues or organs in the body, resulting in tissue damage and loss of function.
Some inflammatory and immune conditions are associated with age-related conditions (e.g., rheumatoid arthritis), but some are due to other causes that may be genetic, environmental or a combination of both (e.g., Acute Respiratory Distress, Type 1 diabetes, IBD). Still other conditions may reflect complications associated with the treatment of other conditions (e.g., GvHD, a frequent complication associated with transplant procedures used to treat leukemia or related blood-borne cancers). Each of these conditions shares certain biological characteristics, in that an immune system imbalance results from the inappropriate activation of certain populations of immune cells that subsequently results in significant tissue damage and destruction. This immune imbalance may result in a complex cascade of inflammation that can result in pain, progressive tissue deterioration and loss of function. While currently available immunomodulatory drugs have proven to be effective for many patients, they have failed to adequately address the needs of many other patients that suffer from inflammatory and immune disorders.
In preclinical studies, MultiStem has shown potent immunomodulatory properties, including the ability to reduce active inflammation through various modes of action, stimulate tissue repair and restore immune system balance. Accordingly, we believe that MultiStem could have broad application in the area of treating immune system disorders, such as Acute Respiratory Distress Syndrome (ARDS), as well as other indications.
Acute Respiratory Distress Syndrome (ARDS)
In January, 2019 we announced positive results for an exploratory clinical study evaluating MultiStem cell therapy for treatment of Acute Respiratory Distress Syndrome (ARDS). ARDS is a serious immunological and inflammatory condition characterized by widespread inflammation in the lungs. ARDS can be triggered by pneumonia, sepsis, or trauma and represents a major cause of morbidity and mortality in the critical care setting. It has significant implications, as it prolongs intensive care unit (ICU) and hospital stays, and requires convalescence in the hospital and rehabilitation.
Patients in the exploratory study were evaluated through 28 days for the primary clinical assessment and will be further assessed through a one-year follow-up period. Data highlights from the initial evaluation include the following results from the double-blind, randomized, placebo-controlled portion of the study:
- Lower mortality of 25% in the MultiStem treatment group vs. 40% in the placebo group;
- 40.2% higher ventilator-free (VF) days, (12.9 VF days in the MultiStem treatment group vs. 9.2 VF days for the placebo group);
- 27.2% higher ICU-free days, (10.3 days in MultiStem subjects vs. 8.1 days for subjects receiving placebo);
- In more severe ARDS patients (as evident in a prospectively defined analysis), the difference between MultiStem treatment and placebo was greater – 25% mortality in MultiStem group vs. 50% in placebo group, 14.6 VF days in MultiStem group vs. 8.0 VF days in placebo group, and 11.4 ICU-free days in MultiStem group versus 5.9 ICU-free days in placebo group; and
- MultiStem treatment was well tolerated in this very sick ARDS patient population, with no serious adverse events related to administration.