Tuesday, February 14, 2012

ALS Therapy nonprofit launches first Phase 2 drug trial

By Lori Valigra

The ALS Therapy Development Institute (ALS TDI), a Cambridge-based nonprofit formed in 1999,  said it is starting a Phase 2 trial of TDI 132, a candidate to treat Lou Gehrig’s disease (amyotrophic lateral sclerosis  or ALS).

Institute CEO Steve Perrin announced the trial Tuesday at the BIO CEO Investor Conference in New York. The drug TDI 132, also known as fingolimod/Gilenya, is currently being sold by Novartis AG to treat some forms of multiple sclerosis. This is the first Phase 2 trial for the nonprofit ALS TDI, and may be one of the only later-stage clinical trials enrolling ALS patients this year, according to the institute.
 
The trial will involve repurposing Novartis’ FDA-approved compound, which lets the drug move immediately into a later-stage clinical trial for people with ALS, the institute said, noting that some 35,000 Americans with ALS could potentially benefit from the drug.

Researchers at ALS TDI started preclinical tests of TDI 132 last year, looking at the drug candidate’s ability to block certain immune cells from entering the central nervous system, where they can cause motor neuron damage. The institute said it has confirmed that TDI 132 significantly alters the movement of these immune cells through the bloodstream, thus resulting in fewer of them getting into the nervous system. ALS TDI partner the Muscular Dystrophy Association (MDA) and other private donors provided significant funding for the preclinical work, according to the institute.

“Getting to this point was only possible because ALS patients, families and the community stood up and took ownership over funding the search for potential treatments for ALS,” Perrin said in a statement. MDA’s interim President Dr. Valerie Cwick said she is encouraged by the early findings.

The primary goal of the Phase 2 clinical trial will be to determine the safety and tolerability of TDI 132 in people suffering from ALS. ALS TDI will work with the Northeast ALS Consortium to help design and run the trial.

Dr. Merit Cudkowicz, director of the Neurology Clinical Trials Unit at Massachusetts General Hospital and one of the principal investigators of the trial, said participants will be evaluated over a month-long exposure to the drug. “This initial study will provide important information on dosing and safety for a subsequent efficacy study,” Cudkowicz said.

In February 2010, ALS TDI extended and expanded its collaboration with California Stem Cell Inc. (CSC) in an effort to develop a potential stem cell treatment for ALS. The previous year, the institute named then-CSO Perrin as its CEO, following the February 2009 death of Sean Scott, who had presided over the institute as president from December 2007 until his passing.