The ALS Therapy Development Institute to Launch Phase II Clinical Trial of TDI 132 in ALS Patients

Via Scoop.it – ALS Lou Gehrig’s Disease

The ALS Therapy Development Institute (ALS TDI) will launch a Phase II clinical trial on TDI 132 (aka: fingolimod/Gilenya®) as a potential treatment for ALS (Lou Gehrig’s disease). Fingolimod is currently being marketed by Novartis AG as Gilenya® as a treatment for some forms of multiple sclerosis. “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. We are excited to announce this trial and will be working with the ALS community to ensure the funding is there to move as quickly as possible from starting enrollment to reporting results,” said Steve Perrin, Ph.D., CEO & CSO of ALS TDI. Perrin will make the official announcement during a company presentation as part of the 14th Annual BIO CEO Investor Conference taking place in New York City (www.bio.org) later this morning. ALS TDI researchers first began preclinical testing TDI 132 in 2011 for its ability to block certain immune cells from entering the central nervous system (CNS) where they can cause activities that result in damage to motor neurons. The Institute has confirmed that TDI 132 significantly alters the trafficking of these immune cells through the bloodstream, resulting in fewer of them infiltrating into and damaging the nervous system. Further experiments at ALS TDI showed treatment with TDI 132 resulted in positive outcomes on several disease measures in preclinical studies in the SOD1 mouse model. A key partner of ALS TDI, the Muscular Dystrophy Association, provided significant funding for some of that preclinical work, as did numerous other private donors to ALS TDI.
Via www.prnewswire.com

Dilemma: When 1 Drug Treats 2 Diseases

Via Scoop.it – ALS Lou Gehrig’s Disease

A drug used to treat multiple sclerosis has also been shown to slow the progression of Lou Gehrig’s disease in mice, a nonprofit biotechnology company plans to announce Tuesday. Now, scientists face a dilemma.Many patients with Lou Gehrig’s disease, a fatal disorder formally known as amyotrophic lateral sclerosis, may want to try the drug on their own—even though there’s no evidence that it is safe or effective in patients with the disease. But scientists want patients to enroll in a clinical trial, one that specifically examines how the drug, Gilenya, works in people with ALS. They hope to launch that trial later this year. We want to make sure we are not doing any harm. We want to do the trials correctly and quickly,” said Steven Perrin, president and chief executive officer of the ALS Therapy Development Institute, a Cambridge, Mass.-based group that is releasing preliminary findings on Gilenya. Still, Dr. Perrin acknowledged the difficulty in asking ALS patients to hold off while a clinical trial is conducted on a drug that could potentially ease their condition. When it comes to developing therapies for the disease, “for ALS patients, yesterday is not fast enough,” Dr. Perrin said.
Via online.wsj.com

Knopp Neurosciences – Who says you can’t grow a drug company in Pittsburgh?

Via Scoop.it – ALS Lou Gehrig’s Disease

Knopp Neurosciences is a Pittsburgh story. The drug development company is a startup gem – from founding, raising money, and executing on its business plan. But wait; Pittsburgh is supposedly not a good place to start a drug company. I hear statements like, “You can’t raise money in Pittsburgh for a drug company,” and “You can’t recruit to Pittsburgh the talent necessary for a drug company.” Oh yeah? Knopp proves that we can. First, a diagnostic. At Pitt, Bob Bowser, PhD, had discovered a potential ALS diagnostic. In the spring of 2004, LaunchCyte formed a company around the technology. Leading the charge were Tom Petzinger, LaunchCyte CEO, and Greg Hebrank, MD, a LaunchCyte investor who had been helping us triage opportunities. The company was named after Walter Knopp, a regional grocery store entrepreneur who died from ALS. Second, a drug. Like many startups, Knopp had to shift its business model as it morphed from a diagnostics to a drug company. Over the next three years, Knopp took dexpramipexole through Phase 1 and Phase II clinical trials. In August 2010, Knopp licensed the drug for ALS to Biogen Idec, a $4B-a-year pharmaceutical company that focuses on neurological diseases, for $345M in potential payments, plus royalties. With an upfront payment of $20M, a Knopp stock purchase of $60M, and future milestone and royalty payments if the drug is approved, that’s a lot of millions flowing into Pittsburgh.
Via www.popcitymedia.com

Congressman Promotes Legislation To Speed Up Drug Development for Rare Diseases

Via Scoop.it – ALS Lou Gehrig’s Disease
Congressman Leonard Lance stood proudly alongside cancer survivors and patients of rare diseases on Thursday as he promoted a new piece of legislation designed to speed the process of getting drugs and treatments for rare diseases from research labs into the hands of the public. The hopeful sentiments surrounding this proposed bill was shared by many at the event and was also echoed by Dorine Gordon, President and CEO of The ALS Association Greater New York Chapter. “We are highly encouraged by this bill and what it will mean to so many people impacted with ALS. Those diagnosed with Lou Gehrig’s Disease on average die within two to five years. The MODDERN Cures Act provides hope that an effective treatment and ultimately a cure for ALS is even closer than before. This bill will help break down barriers to scientific research and harness greater innovations in the lab. The ALS Association Greater New York Chapter is grateful to Congressman Lance for his dedication to the health and well-being his constituents and to the American people.”
Via www.asbestos.com