CASE STUDIES

Brain Chemistry Labs

Brain Chemistry Labs is a research-focused nonprofit seeking to uncover innovative treatments for life-threatening neurological diseases—specifically diseases such as Alzheimer’s and ALS. It operates with a lean staff, a network of partners, and a single goal of improving outcomes for patients, which allows it to be creative, nimble, and cost-effective.

METHODS

We do a lot of things that make us look like a small pharmaceutical firm,” says Executive Director Paul Alan Cox, PhD, “with one main difference: We’re not interested in profits; we’re interested in patients.” The organization’s nonprofit model provides useful flexibility and allows it to avoid some pitfalls encountered by for-profit companies, such as sunk costs, says Cox. This means it can shift gears quickly if a line of inquiry isn’t working. Its six-person staff is also very efficient. “Of every dollar we receive, 87 percent goes straight to the research,” Cox says. Additionally, the team takes an interdisciplinary approach, tapping into a consortium of 50 other scientists from around the world with various specialties, thus enabling the organization to tackle the problem from multiple angles. Brain Chemistry Labs’ current research is focused on the potential of L-serine, a naturally occurring amino acid, to slow the progression of diseases characterized by misfolded proteins, or tangles, in the brain, including Alzheimer’s, ALS, and Parkinson’s.

IMPACT

The organization’s ultimate goal is to change the trajectory of these diseases for patients. It has already had some success on that front: It has developed a biomarker derived from a patient’s blood draw to detect ALS, a significant advancement in a disease that has been difficult to diagnose. It has also recently completed a phase I clinical trial whose results showed that L-serine significantly slowed disease progression, and it is now conducting two FDA-approved phase II trials—one for Alzheimer’s and one for ALS. “It appears that people with ALS taking this investigational therapy, instead of dying in two and a half or three years, are living five to six years from diagnosis, but this needs to be confirmed through the clinical trials,” says Cox. The lab is seeking to find therapies that slow disease progression so that ALS can one day become more of a manageable chronic condition and so that the onset of Alzheimer’s might be delayed until people are in their late 90s instead of their 60s and 70s. Additionally, the nonprofit helps connect people to these trials and provides support through its partners at Dartmouth Medical School to cover their airfare and hotel stays.

HOW THE SATTER FOUNDATION HAS HELPED

In addition to providing significant financial support—including an investment that gave the organization the confidence to move forward with a clinical trial on mild cognitive impairment—the Satters have provided mentorship and moral support. “We’re doing high-risk, long-shot research, so to have somebody as sophisticated as the Satter Foundation want to get behind it meant a great deal,” Cox says. The Satter Foundation’s resources were instrumental in funding the discovery of the ALS biomarker, he adds.

WHAT'S NEXT

If the phase II trials are successful, the organization will need a pharmaceutical partner to help execute a phase III trial, which could happen within the next year or two, leading to relief and hope for those struggling with neurological diseases.

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