Treg Therapy Q&A
Detailed Information on Treatment Eligibility, Concomitant Medications, Safety, and Expected Benefits
Treg Therapy Q&A
QWhat are Tregs?
Tregs are formally known as Regulatory T Cells (Tregs).
Until relatively recently, it was not fully understood how the immune system regulates its "accelerator" and "brake" mechanisms. As a result, the causes of many autoimmune diseases remained unclear, and the mechanisms that prevent the immune system from attacking the body's own tissues were not yet fully understood.
This mystery was ultimately resolved when Professor Shimon Sakaguchi became the first scientist to identify a unique population of T cells capable of suppressing excessive immune responses. This groundbreaking discovery transformed our understanding of immune regulation and opened new possibilities for the treatment of a wide range of immune-mediated diseases. In recognition of this achievement, Professor Shimon Sakaguchi of Osaka University was awarded the Nobel Prize on October 6, 2025.
Our therapy is the first treatment in Japan to apply Treg-based therapy to patients with ALS in a clinical setting.
QWhich patients are more likely to respond?
Although research is ongoing, favorable signals have been observed in patients at earlier stages of disease and in those with lower cerebrospinal fluid phosphorylated neurofilament heavy chain (CSF-pNFH) levels. Levels of inflammation and oxidative stress may also influence treatment responsiveness.
QHow many treatments are administered?
The standard treatment protocol consists of eight administrations.
A treatment cycle is repeated at intervals of several weeks. The protocol combines multiple intravenous infusions of autologous Tregs with low-dose interleukin-2 (IL-2) and CTLA4-Ig.
If a patient becomes refractory to treatment, additional cell-based therapeutic options may be considered.
QDo Tregs reach the central nervous system?
In cross-disease studies, Tregs have been shown to cross the blood–brain barrier and may exert local anti-inflammatory effects and support tissue repair.
In ALS, intravenously administered cells are also expected to reach sites of neuroinflammation and exert anti-inflammatory effects.
QIs rapamycin used?
It is used as part of the manufacturing process to selectively expand Tregs ex vivo and helps maintain their purity and functional stability during production.
QIs the goal to improve symptoms?
The goals of treatment are to reduce inflammation and promote neuroprotection, with the aim of slowing disease progression. Treatment responses may vary among individuals and may depend on disease subtype.
QCan Treg therapy be used in combination with riluzole or edaravone?
Yes. Many studies have included riluzole as concomitant therapy, and at our clinic this treatment is generally administered on the condition that standard-of-care therapies are continued.
