ALS TDI to Test Multiple Sclerosis Drug in ALS

by Amy Madsen on Tue, 2012-02-14 11:30

The MDA-supported ALS Therapy Development Institute plans to launch a phase 2 clinical trial of TDI 132, a compound that modulates the immune system

Article Highlights:
  • The nonprofit biotech ALS Therapy Development Institute plans to launch a phase 2 trial to determine the safety and tolerability of TDI 132 — also known as fingolimod — in ALS.
  • Fingolimod (brand name Gilenya) is approved by the U.S. Food and Drug Administration for the treatment of multiple sclerosis. In humans, Gilenya inhibits certain immune system cells from entering the central nervous system, where they can result in damage to motor neurons.
  • ALS TDI reports that mice treated with TDI 132 showed positive outcomes on several disease measures.

A phase 2 clinical trial is set to begin of TDI 132, a compound that modulates the immune system, the ALS Therapy Development Institute (ALS TDI) announced today.

TDI 132, also known as fingolimod (brand name Gilenya), already is approved by the U.S. Food and Drug Administration (FDA) for the treatment of multiple sclerosis.

ALS TDI is an MDA-supported, nonprofit biotechnology organization dedicated to developing effective treatments for amyotrophic lateral sclerosis (ALS). Steve Perrin, ALS TDI CEO and Chief Scientific Officer, announced the upcoming trial at the 14th annual BIO CEO & Investor Conference in New York.

"The indications from preclinical testing of TDI 132 are encouraging, and MDA looks forward with great interest to the results that will come out of this phase 2 trial," said MDA Vice President of Research Sanjay Bidichandani. "The development of experimental therapies such as TD1 132 is exactly what our long-standing partnership with ALS TDI is meant to accomplish."

Significant funding from MDA supported ALS TDI's preclinical testing of TDI 132, paving the way for the experimental therapy to advance to human clinical trials.

TDI 132 works by dampening immune response

A growing body of evidence suggests that malfunction of the immune system is at least part of the complex ALS disease process. Previous ALS TDI studies conducted in the SOD1 research mouse have shown that blocking the activation of certain parts of the immune system slows disease progression and improves survival.

Preclinical testing by ALS TDI has demonstrated that TDI 132 is able to reduce the number of certain immune system cells circulating throughout the bloodstream. This, in turn, limits the number of these cells able to enter the central nervous system, where they are known to engage in interactions that result in damage to the motor neurons (muscle-controlling nerve cells).

Mice treated with TDI 132 showed positive outcomes on several disease measures.

Meaning for people with ALS

Although Gilenya is available by prescription for people with multiple sclerosis, it's not yet known what effect it has in people with ALS.

Therefore, it’s strongly recommended that individuals with ALS not seek an off-label prescription for the drug. ("Off label" refers to a physician’s use of a drug or therapy to treat conditions other than the ones for which it received FDA approval.)

"Although we are encouraged by these findings, we don’t know yet whether the drug is safe or effective in people with ALS," cautioned MDA Interim President and Medical DirectorValerie Cwik. "MDA does not recommend taking Gilenya for the treatment of any condition other than that for which it has FDA approval."

However, because the compound already is on the market and approved as a treatment for use in humans, a successful phase 2 trial could lead to rapid development and FDA approval of TDI 132 as a treatment for ALS.

MDA support of drug development in ALS

Through its Augie's Quest research initiative, MDA to date has awarded more than $24.5 million to ALS TDI. The two organizations joined forces in 2007 in the largest ALS drug discovery project in history, aimed at identifying biological components and pathways in ALS and finding drugs that target them.

The partnership has resulted in the achievement of several key milestones in ALS research, such as analysis of hundreds of blood samples from ALS patients and ALS mice to identify targets related to disease onset and progression; the screening of more than a dozen compounds against those targets; and the ongoing testing of two other immune system modulators, CDP7657 and CTLA4-FC, in the SOD1 ALS mouse.

"MDA support, through Augie's Quest, funded much of the preclinical work on TDI 132," Perrin said. "Being able to advance the experimental therapy to phase 2 trials is a fantastic achievement that is the end result of the dedication and drive, and the shared goals, of both MDA and ALS TDI to fund this clinical trial and others to end ALS."

Upcoming webinar on TDI 132

An informational webinar on the TDI 132 trial, hosted by Steve Perrin and one of the trial’s principal investigators, neurologist Merit Cudkowicz, is scheduled for March 12, 2012. (Cudkowicz has received MDA research funding and directs the MDA/ALS Center at Massachusetts General Hospital in Boston.)

Register online at

About Clinical Trials

About clinical trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more, see Understanding Clinical Trials and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to

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