Frustrated for decades with failure after failure in efforts to find effective medications to treat Alzheimer’s and other cognitive disorders, researchers are now increasingly exploring modifying the genes that control every aspect of our bodies in hopes of finding a cure.
In yet another sign of the exploding possibilities of gene modification, biotechnology giant Biogen has put down a $350 million marker in the field, partnering with a genetic medicine company to develop experimental treatments for neurological diseases, including Alzheimer’s and Parkinson’s disease.
The Biogen partnership with Sangamo Therapeutics will focus on DNA, the material that provides operating instructions for each of our cells. In each cell, DNA is packed tightly into chromosomes, which in turn have thousands of segments known as genes.
Genes, of course, carry information that defines our appearance such as our hair color or our height. But, according to the National Institute on Aging (NIA), genes also play a role in keeping the body’s cells happy. Even the smallest problems with genes can cause diseases like Alzheimer’s.
“As a pioneer in neuroscience, Biogen will collaborate with Sangamo on a new gene regulation therapy approach, working at the DNA level, with the potential to treat challenging neurological diseases of global significance,” said Alfred Sandrock, executive vice president at Biogen.
In a joint news release, Sangamo CEO Sandy Macrae noted that there are currently no approved disease-modifying treatments for people with neurodegenerative diseases such as Alzheimer’s and Parkinson’s.
“We believe that the promise of genomic medicine in neuroscience is to provide a one-time treatment for patients to alter their disease natural history by addressing the underlying cause at the genetic level,” Macrae said.
Sangamo has developed a gene editing technology called zinc finger that delivers inactivated viruses to cells, where they can edit DNA to manipulate the activity of specific genes.
One of Sangamo’s preclinical treatments — known as ST-501 — may be able to repress tau protein, which scientists believe is involved in the development of Alzheimer’s. Another preclinical treatment called ST-502 has shown promise against alpha synuclein, which is involved with Parkinson’s.
Analysts noted that the technology is far from ready for the public. The companies face years of additional development work and clinical trials before determining if the treatments could prove effective.
But the field of genetic modification is definitely hot. One new study found that DNA changes in a gene called Presenilin1 or PSEN1 are associated with Alzheimer’s. And in February, scientists in London identified a type of genetic mutation that appears to protect against late onset Alzheimer’s disease.