Alzheimer's Matters Blog

Drugs to “Fix” the APOE4 Gene May Be Safe

August 18, 2014

Category: Developing Drugs


Last week, new research revealed that a man without the APOE gene—which helps to carry cholesterol and, in certain forms, dramatically increases a person’s risk of developing Alzheimer’s disease—was able to function normally. The news has important implications for Alzheimer’s research, suggesting that if scientists could develop therapies to inhibit the potentially toxic effects of APOE, patients could take the drug without fear of neurological complications.

While this news is encouraging, it’s important to note that the road from understanding what a gene does to creating a therapy to fix it is long and often indirect. For more than 20 years, scientists have known that a specific form of APOE, called APOE4, substantially raises a person’s risk of Alzheimer’s disease, yet we still don't have a drug to treat this. (And there are many more diseases, such as Huntington’s, for which the genetic factor is well-known and a cure remains out of sight.)

But APOE drug research in Alzheimer’s is progressing. With your support, the Alzheimer’s Drug Discovery Foundation has provided more than $4.2 million in APOE research funding for 19 unique drug discovery programs.

We are currently funding Dr. Stephen Paul of Weill Cornell Medical College, whose research was highlighted in The New York Times. Dr. Paul is developing a gene therapy that involves implanting a virus containing APOE2 (a rare but possibly protective variation of the APOE gene) to "fix" the APOE4 defect. He has already shown that this approach is therapeutic in small mammals with Alzheimer's, and he is hoping to test the approach in human clinical trials.

Read more about what APOE means for your health on the ADDF's Cognitive Vitality website.