Alkahest, a California-based biotech start-up, has just revealed some compelling early results from an ongoing Phase 2 trial into the efficacy of its novel formulation of plasma proteins derived from young blood, developed to slow, or even stop, the cognitive decline associated with Alzheimer's disease.

The idea of young blood holding a magical kind of rejuvenating power has been anecdotally shared for centuries. More recently, a company called Ambrosia thrust the idea into mainstream controversy, charging thousands of dollars for young blood transfusions at several clinics around the US before swiftly ceasing operations after the FDA set its sights on the unregulated young blood market.

Alkahest, while essentially working from the same belief in the therapeutic properties of young blood, has trod a more cautious path over the last couple of years, using classical evidence-based science to establish the efficacy of its products. Instead of simply using whole blood, or even whole plasma, Alkahest focuses on highly specific proteins found in young blood plasma.

The company calls these plasma proteins chronokines, and suggests these molecules can be either beneficial or detrimental as certain combinations of them increase or decrease with age. Several proprietary formulations of chronokines, called plasma fractions, are being tested by Alkahest for a variety of conditions, including Parkinson's disease and other undisclosed inflammatory diseases.

The most recent announcement from the company concerns a plasma fraction it calls GRF6019. Currently in the late stages of a Phase 2 human trial targeting mild-to-moderate Alzheimer's disease dementia, Alkahest has revealed some initial top-line data suggesting the experimental treatment is indeed working as intended.

The trial has been designed to investigate whether GRF6019 can slow, or even stop, cognitive decline associated with Alzheimer's disease. What is currently being reported by the company is that subjects receiving GRF6019 are displaying no decline in cognition across a six-month study period. However, it is important to note that these results are reported ahead of any formally published or peer-reviewed study, and there are many unanswered questions hovering around this particular announcement from the company.

The trial frustratingly does not seem to contain a placebo arm, instead it is just offering a randomized, double-blind dose comparison between low and high doses of GRF6019. So while it may be impressive to report a halt in cognitive decline across the six-month study period, it is unclear exactly how much cognitive decline could be expected over that timeframe for similar patients not receiving the treatment.

Until more data from the study is revealed it is difficult to clearly ascertain how significant these results are. Nevertheless, CEO of Alkahest Karoly Nikolich, says these results do support the company's ongoing work.

"These top-line results indicate a potential benefit of this plasma protein fraction in slowing the progression of cognitive decline in patients with mild to moderate Alzheimer's disease," says Nikolich. "Developing impactful treatments for this disease and other neurodegenerative diseases of aging has proven unsuccessful for decades. GRF6019 represents an innovative approach to effectively treating Alzheimer's disease by targeting multiple underlying mechanisms of disease, and these data support the continued development of GRF6019 and other plasma protein fractions in Alzheimer's disease."

While at this stage all of Alkahest's plasma fraction formulations do come from young blood donations, it is hypothesized that once specific chronokines are identified and shown to be effective, they may be able to be synthesized at some point in the future. In the short term, if Alkahest is successful at moving these new young blood products to market over the coming years, they will initially be derived from young human plasma.

Source: Alkahest