Medicinal Signaling Cell Therapy Fast Tracked for Mild Alzheimer Disease

The Food and Drug Administration (FDA) has granted Fast Track designation to Lomecel-B for the treatment of mild Alzheimer disease.

Lomecel-B^™ is derived from culture-expanded medicinal signaling cells that are sourced from the bone marrow of young healthy adult donors. The investigational allogeneic cellular therapy is believed to impart anti-inflammatory, pro-vascular, and pro-regenerative effects that can apply to a number of aging related diseases, including Alzheimer disease.  

The Fast Track designation was based on data from the phase 2 CLEAR MIND study (ClinicalTrials.gov Identifier: NCT05233774), which evaluated the safety and efficacy of Lomecel-B in patients 60 to 85 years old with mild Alzheimer disease. Patients (N=50) were randomly assigned to receive 1 of 3 dosing regimens of Lomecel-B or placebo. 

The primary endpoint of the trial was safety. The secondary outcome measure was the change from baseline to week 39 in the Composite Alzheimer Disease Score (CADS), which combines information across cognitive, functional capacity, and brain MRI domains.

Findings showed the study met its primary endpoint of safety. Moreover, a statistically significant improvement in CADS was observed in the low-dose Lomecel-B group (P =.091) and for the pooled Lomecel-B group (P =.099) compared with placebo. 

Additional analysis from the trial showed Lomecel-B improved cognitive function, based on Montreal Cognitive Assessment and Mini-Mental State Examination scores, compared with placebo. The investigational therapy also reduced neuroinflammation, improved cerebral brain flow, and reduced brain volume loss in areas associated with Alzheimer disease. With regard to quality of life, improvements were observed based on caregiver reports using the Alzheimer Disease Related Quality of Life scale.

“Fast Track designation is another important milestone for Longeveron and Lomecel-B, which, along with the recent granting of Regenerative Medicine Advanced Therapy designation, recognizes the critical need to quickly advance novel, safe and effective investigational treatments for Alzheimer disease, which has a devastating impact on patients and their families,” said Wa’el Hashad, Chief Executive Officer of Longeveron.

Lomecel-B is also being investigated in patients with hypoplastic left heart syndrome and in those with aging frailty.

This article originally appeared on MPR