Sha said her interest in testing the plasma-infusion method came about after a Stanford neurology colleague observed promising results in mice. “This [trial] was really based upon the basic science work of Tony Wyss-Coray,” she said. “After his experiments demonstrated improvements in memory and ‘rejuvenation’ in the brain in old mice with young blood, we thought to try a small, phase one trial, in patients.”
The trial took place at Stanford Hospital and was supported by funding from the private biotechnology company Alkahest, which holds intellectual property rights associated with the treatment plan. Wyss-Coray is a co-founder of Alkahest, but was not involved in the latest study.
In the first phase of the trial, nine Alzheimer’s patients were given either four weekly infusions of plasma from donors aged 18 to 30 years old or a placebo of saline solution. The patient and the person administering the infusion were both kept in the dark about which type of infusion the patient was receiving. After six weeks, the groups were reversed: The patients who received plasma were administered saline and vice versa.
Patients completed questioares prior to treatment in order to gauge their mood, cognition, and functional ability and then again after the first round plasma-placebo distribution. The process was repeated before and after the second four-week treatment period.
In phase two of the study, an entirely new group of nine participants received plasma infusions from young donors. None of the subjects received a placebo. Both the patients and their caregivers were informed that the patient was receiving plasma.
“The first double blind crossover cohort was getting ‘study fatigue’ and required participation for almost 6 months,” Sha commented. “We had a couple of patients drop out of the study due to this, so we switched the protocol to the open-label plasma only, which allowed for less than 3 months of participation.”
The second group received the same mood, cognition, and functional ability tests as the first group, both before and after infusions.
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There were no significant changes in mood, the ability to memorize lists, or recall recent events observed in any of the participants, which Sha did not find surprising. For these changes to take place, clinical trials typically must exceed one year, she noted. However, Sha was surprised to see an improvement in functional ability in the patients who received the plasma infusions because the study was not designed to show the treatment’s efficacy, but rather evaluate its safety.
At first, Sha and her team suspected these results may have come from the group who knew they were getting the plasma treatment, because caregivers would be more likely to report positive results. But they found the opposite to be true. The group that didn’t know they were receiving plasma showed the most improvement in their functional ability. The placebo group showed no significant change in functional ability.
While the findings are encouraging, Sha cautioned that they’re based on reports from patient caregivers, not from clinicians. “This is very exciting and promising, but needs to be replicated with a larger sample size and with enough time to detect changes in measure of memory as well,” she said.
Based on these results, Alkahest, who funded the trial, is moving forward with the development of a plasma-derived treatment for mild to moderate Alzheimer’s disease.
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