First Parkinson’s patient dosed in Unixell stem cell therapy study
Dosed participant reports 'remarkable improvement in my sleep quality'
Unixell Biotechnology has dosed its first Parkinson’s disease patient in a clinical study of its stem cell therapy candidate, UX-DA001.
According to a press release from Unixell, a surgeon at Ruijin Hospital in Shanghai successfully injected the experimental medication directly into a patient’s brain. UX-DA001 is derived from patients’ own induced pluripotent stem cells (iPSCs) and is designed to replace damaged brain cells and alleviate symptoms.
Preliminary data suggest the treatment was safe and demonstrated signs of efficacy.
The progressive neural degeneration seen in Parkinson’s primarily affects the substantia nigra, a brain region involved in regulating voluntary movement. Nerve cells in this area that produce the signaling molecule dopamine are especially vulnerable and their loss disrupts communication within the basal ganglia, leading to the motor symptoms that mark the disease. For this reason, many therapies for Parkinson’s focus on increasing the amount of dopamine in the brain.
These typically don’t directly address the nonmotor symptoms of Parkinson’s, which can include sleep disturbances and cognitive changes. The therapies, such as levodopa and its derivatives, can also lose efficacy over time.
“Current standard treatment for Parkinson’s disease primarily relies on medications to alleviate symptoms,” said Liu Jun, MD, PhD, the trial’s principal investigator. “However, after prolonged oral drug treatment, patients often experience fluctuations in efficacy and disease progression continues.”
An alternative approach to treating Parkinson’s
Stem cell therapies like UX-DA001 offer a potential alternative. The experimental treatment is designed to introduce new dopamine-producing nerve cells, or neurons, into the brain, replacing those lost to disease and potentially reducing the need for ongoing treatments. “Cell therapy offers the prospect of ‘one-time treatment with long-term benefits,’” Jun said.
UX-DA001 begins with a patient’s own peripheral blood cells, which are then reprogrammed into iPSCs, that is, stem cells derived from adult cells that have been reprogrammed back into a pluripotent state, meaning they can develop into nearly any cell type.
In the laboratory, the cells can then be differentiated into neurons that produce dopamine. When surgically injected into the putamen — a key region of the basal ganglia — the new neurons have the potential to integrate into the brain’s neural network. The procedure is minimally invasive, according to Unixell.
This first patient is part of Unixell’s Phase 1 clinical trial (NCT06778265) testing the treatment’s safety and tolerability in people with Parkinson’s. By using the patient’s own cells, rather than donor cells, the risk of the treatment being rejected by their immune system is minimized, according to the company. People treated with UX-DA001 also don’t require immunosuppressants, which come with side effects.
Secondary endpoints for the trial include initial measures of how the transplanted cells integrate into brain circuits. Investigators will also test if it affects motor functioning.
In the month after surgery, the dosed participant reported no serious side effects, according to Unixell. The participant said, that, after the stem cell treatment, “I experienced a remarkable improvement in my sleep quality. I fall asleep quickly and notice gradual progress every day. Although recovery takes time, the ‘day-by-day improvement’ fills me with hope for the future.”
UX-DA001 has been approved for clinical trials by both China’s National Medical Products Administration and the U.S. Food and Drug Administration. These approvals let the treatment be tested in patients with Parkinson’s not only in China, but around the world.
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