Recruiting

Stem cell transplant therapy for ALS patients

Plain-English translation of NCT06765564 on ClinicalTrials.gov ↗ · Source last updated January 2025 · Translation generated June 2026 · How we translate trials

This study doesn't follow the usual testing phases — it may be an observational study or a different type of research.

What is this trial?

This is an early-stage study testing cells—nerve cells grown from a person's own reprogrammed cells in the laboratory—as a treatment for ALS. Researchers will transplant these cells directly into the brain of people with ALS to see if they can help nerve cells survive and function better. The goal is to learn whether this treatment is safe and tolerable in a small group of patients.

ALS is a disease where nerve cells gradually die, causing loss of muscle control and strength. Most current medicines offer only modest benefits, and many patients continue to get worse. This trial is exploring whether stem cell therapy might offer a new way to protect or repair damaged nerve cells and slow disease progression.

Do you qualify?

You likely qualify if…

you are between 18 and 60 years old
you have been diagnosed with ALS within the past 6 to 24 months
your ALS has continued to get worse despite standard treatment
your lung function (measured as forced vital capacity) is at least 50%
you are in good overall health, with a body mass index of at least 18.5
you are willing to take immunosuppressant medications for 3 months to prevent your body from rejecting the transplanted cells

You likely don't qualify if…

you have severe cognitive impairment, dementia, or serious mental health conditions like schizophrenia or severe depression
you currently use a breathing machine or have had a tracheostomy
you have a history of cancer or malignant tumor
you have received any other stem cell or organ transplant in the past
you have severe liver or kidney disease, as shown by blood tests

What participation looks like

If you enroll, you will spend about 7 months in the study. First, you'll complete baseline tests and exams over one week, then begin taking immunosuppressant medications. Next, you'll have surgery under general anesthesia to place a special pouch in your brain that will deliver the stem cells. Three days later, you'll receive the first stem cell transplant through this pouch. You'll be monitored closely for the next 45 days, with regular check-ups to watch for side effects. Then you'll return for follow-up visits over the next 6 months to see how the treatment affects your symptoms and overall condition.

Stem cell transplant therapy for ALS patients

What is this trial?

Do you qualify?

What participation looks like

Trial locations(1 site)