In early January 2021, biopharmaceutical company BlueRock Therapeutics (“BlueRock”), in collaboration with the Memorial Sloan Kettering Cancer Center, announced via press release that its Investigational New Drug (IND) application for DA01 was cleared. Now, BlueRock, a subsidiary of Bayer AG, will be able to evaluate the safety, efficacy, and tolerability of DA01 for Parkinson’s disease. The IND clearance will allow BlueRock to run a Phase 1 clinical trial.
DA01 offers patients with Parkinson’s disease dopaminergic neurons derived from pluripotent stem cells. According to the Boston Children’s Hospital, pluripotent stem cells are master cells, which means that:
They’re able to make cells from all three basic body layers, so they can potentially produce any cell or tissue the body needs to repair itself. Like all stem cells, pluripotent stem cells are also able to self-renew, meaning they can perpetually create more copies of themselves.
In this case, DA01 sources dopaminergic (dopamine-producing) neurons from these pluripotent stem cells and delivers them to patients. According to an article published in Nature, which discusses the preclinical use of pluripotent stem cell-derived dopaminergic neurons in primates, the authors find that:
human iPS cell-derived dopaminergic progenitors are clinically applicable for the treatment of patients with PD.
Altogether, 10 patients will enroll in the trial. The primary objective will be to analyze DA01’s safety and tolerability. For this, patients will be followed for at least one year following the stem cell transplantation. For secondary endpoints, researchers will seek to evaluate how transplanted stem cells have survived and benefited motor skills, for both one and two years, and to determine how the safety and tolerability after one year relate to the same elements after two years.
Parkinson’s Disease (PD)
Parkinson’s disease is a progressive central nervous system (CNS) disorder that impacts movement. Typically, the condition develops slowly, starting only with a slurred tremor. This is usually felt in stage one of Parkinson’s disease, although there are five stages in total. By mid-stage of Parkinson’s disease, many patients begin to experience bradykinesia (slowed movement) and balance loss. By the fifth and final stage, patients are unable to walk, stand, or live independently, and may experience hallucinations. Generally, Parkinson’s disease symptoms appear after 50 years old.
Altogether, dopaminergic neuron death causes Parkinson’s disease. As dopamine-producing neurons die, there are reduced levels of dopamine in the body, impacting motor functions. While doctors are not sure exactly what causes this nerve death, factors are believed to include genetics, microscopic proteins, or environmental triggers. Symptoms include:
- Muscle rigidity
- Impacted posture and balance
- Changes in speech, such as slurring, stuttering, or speaking quickly
- Dementia and hallucinations
- Loss of ability to blink or smile
- Tremor in one or both hands
- Sleep disturbances
- Jaw stiffness