Tiziana's Experimental Therapy Shows Promising Results for Multiple Sclerosis Patient

At the Brigham and Women's Hospital (BWH) on the Harvard University Campus, a male patient with Secondary Progressive Multiple Sclerosis (SPMS) serves as a beacon of hope for those afflicted with the same neurodegenerative disease. The patient was given access to Tiziana’s immunotherapy, foralumab, through the U.S. Food and Drug Administration (FDA) Single-Patient Expanded Access Investigational New Drug (IND) program.

The FDA authorizes the use of the Single-Patient Expanded Access IND program under special circumstances, allowing some patients to avoid the long process a drug takes from research to FDA approval. 

Foralumab offers the convenience of being a “take-home immunotherapy”, as worded by Tiziana’s CEO and CSO, Kunwar Shailubhai. The monoclonal antibody treatment has overcome the complex task of crossing a patient’s blood-brain barrier to deliver an effective drug substance to neurons experiencing degeneration. The patient did not experience any adverse reactions following administration. Rather, the disease progression halted, and the patient’s symptoms began to improve.  

The treatment regimen was relatively simple: on Mondays, Wednesdays and Fridays, foralumab would be administered to the patient intranasally. Treatment would go on for two weeks, then allow for one week of recovery before beginning again. The cycle went on for six months with an intermittent evaluation of the patient’s response at three months. The results showed such success at these timepoints that the FDA has agreed to extend this patient’s study for an additional six months.  

Activation of microglial cells, or activated microglial cells (AMCs), can cause damage to the central nervous system because activation triggers toxic molecule release. One of these toxic molecules is pro-inflammatory cytokines. Of course, the naked eye is not able to see these changes, thus requiring complex imaging. 

To measure the status of the SPMS progression and the efficacy of foralumab, positron emission tomography (PET) scans were conducted at the three-month and six-month timepoints. Shown in the PET scans, inhibition of AMCs was measured in comparison to the patient’s baseline. In the whole brain, as well as specialized areas of concern, including the cerebral cortex, cerebellum, thalamus and white matter, AMCs continued to be inhibited. The patient’s entire brain measurement showed 23% improvement after three months of treatment, and 38% after six months of treatment. 

Additionally, pro-inflammation cytokines were seen to be downregulated during the therapeutic cycles of foralumab administration. These cytokines, including interleukin and interferon-gamma, are measure of the damage being caused to the CNS. To evaluate the patient’s clinical status, the G-hole Peg test, Timed 25-Foot Walk Test and Symbol Digit Modality Test were conducted and showed improvement during the treatment. These are measures of dexterity, fine motor function and cognitive impairment.

In response to the positive results announced by Tiziana, the FDA has agreed to authorize the use of foralumab through the Single-Patient Expanded Access IND program for a second patient. Tiziana’s NASDAQ stock prices have also responded accordingly, increasing in anticipation of the marketability of a breakthrough treatment.