The Generative Brain

Treatments being researched

No treatment has yet been proven to slow or stop CJD or other prion diseases. Care teams can ease symptoms and help a person stay comfortable, and researchers are testing possible treatments in clinical trials.

Where treatments in development try to step in
  1. The PRNP geneSwitch off or edit the gene that makes the protein.
    • Base editing
    • Epigenetic editing
    • Zinc finger repressors
  2. The RNA messageLower how much protein the cell makes.
    • ION717
    • PrP siRNA
  3. The prion proteinBind the protein or change how cells handle it.
    • PRN100 antibody
    • Small molecules
  4. The brain's responseSupport the cells reacting to the damage.
    • Early ideas

A simplified view: none of these is a proven treatment. ALN-6457 also targets PRNP, but the trial registry does not state its drug type, so it is not placed here.

Where approaches stand
  1. Early research
  2. First human studies
  3. Clinical trials
  4. Approved
Base editing
Epigenetic editing
Zinc finger repressors
Small molecules
PRN100 antibody
ION717 (PrProfile)
PrP siRNA (PRiSM)
ALN-6457

A simplified view. No treatment is proven yet. See the Atlas for each record and its date.

What can help now?

Care focuses on comfort and on easing symptoms, and there is a lot a care team can do. Medicines can ease anxiety and low mood, reduce muscle jerks and tremors, and relieve pain. As needs change, nurses, therapists and social workers can help with feeding, washing, moving and swallowing.

Many people record their wishes early, including where they would like to be cared for and which treatments they would or would not want. The care team can help with this. The pages on what to expect, palliative and hospice care and support groups may be useful next.

What are researchers testing?

Several current studies test medicines aimed at the prion protein, the protein that folds abnormally in these diseases. One large study is testing a medicine called efavirenz against a placebo. These details come from trial registries and can change, so the current trials page and the registry records are the places to check.

StudyBeing testedStageRegistry status on 31 October 2026
PrProfile (NCT06153966), IonisION717Phase 1/2Active, not recruiting (updated 18 September 2026)
PRiSM (NCT07444580), Broad InstitutePrP-siRNA, a medicine aimed at prion proteinPhase 1Recruiting at US sites (updated 16 October 2026)
ALN-6457 trial (NCT07832370), RegeneronALN-6457, designed to reduce prion protein made in the brainPhase 1/2Not yet recruiting (updated 22 September 2026)
Efavirenz trial (NCT07482085), Xuanwu Hospital, BeijingEfavirenz compared with placeboPhase 3Recruiting in China (updated 3 September 2026)

No results from these four studies are posted in the registry records we read. We make no claim that any of them will work. The therapies page has more on each approach.

Have earlier treatments worked?

Not so far. Two medicines that looked possible earlier, quinacrine and doxycycline, were studied in people and did not change the course of the disease.

In a small UK program, six people with CJD received an antibody called PRN100. It was well tolerated and reached the brain, but everyone’s condition continued to get worse. The researchers said that formal trials would be needed to learn whether it works.

Would joining a trial help the person taking part?

Nobody can know that in advance. Phase 1 and 2 studies are mainly designed to learn about safety, dosing and what a medicine does in the body. A trial can also mean travel, tests and lumbar punctures, and a caregiver is usually needed to take part. Choosing not to take part is a valid choice, and good care continues either way.

Who can join is set by each sponsor and changes over time. As stated in the registry records on 31 October 2026, the studies above are for adults with symptoms, and several limit how advanced those symptoms can be. PRiSM also lists adults without symptoms who carry a specific genetic change. If this raises questions about inheritance, genetic counselling comes first, and not testing is a valid choice.

Who can I ask?

The neurologist or specialist centre looking after the person can say whether any study might be relevant. In the UK, NHS care links people with the National Prion Clinic in London and the National CJD Research and Surveillance Unit in Edinburgh. In the US, the National Prion Disease Pathology Surveillance Center keeps a list of treatment trials.

If anyone offers a treatment outside a registered trial, the care team can help you look at it before you decide.

Sources for this page