$1.5 Million Cinque Foundation Grant Goes To ALS Drug Research At Johns Hopkins Packard Center

The Robert Packard Center for ALS Research at Johns Hopkins announced today that it has received a three-year, $1.5 million grant from the Cinque Foundation. The money will support the screening of thousands of drugs already approved by the U.S. Food and Drug Administration and on the market for their potential value in treating people with amyotrophic lateral sclerosis, or ALS.

ALS, also known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disease marked by gradual and crippling paralysis that affects about 30,000 people in the United States. No cure exists, so treatment is currently limited to palliative care.

A team of researchers at Johns Hopkins, led by Packard Center Director Jeffrey Rothstein, M.D., says the value of hunting among already approved drugs is that they have already undergone extensive safety testing and can be quickly used in clinical trials of ALS patients.

That strategy previously worked to identify ceftriaxone, an FDA-approved antibiotic commonly prescribed to treat pneumonia, bacterial meningitis and Lyme disease. Rothstein and his team demonstrated the drug’s effectiveness on cell cultures or slices of spinal cord from rats that have an ALS-like disease.

The new experiments will add two additional twists to the search for drugs to treat ALS. First, rather than screening single drugs, the researchers will be working with millions of combinations of drugs to learn whether some pharmaceuticals are more potent against ALS when combined. Second, the team will be testing these drugs for the first time on human stem cell-derived brain cells called astrocytes, which are now known to be defective in ALS.

“What we hope is that our search will reveal drugs that already exist in the vast reservoir of FDA-approved drugs and are just waiting to be unleashed on ALS,” notes Rothstein.

About the Robert Packard Center for ALS Research. Headquartered in Baltimore, the Robert Packard Center for ALS Research at Johns Hopkins is a collaboration of scientists worldwide who are working to develop new treatments and a cure for ALS. The Center’s goal is to speed translational research from the laboratory bench to the clinic. It was named after a San Francisco investment banker whose foundation supports the Center. Robert Packard died of ALS in 2000.

About the Cinque Foundation. Established in 2007, the Cinque Foundation is dedicated to eliminating suffering and death caused by ALS. The Foundation funds medical research that contributes to understanding this complex disease, with a particular emphasis on expediting the translation of promising results to benefit ALS patients.