ALS Clinical Trials Briefs: Cytokinetics, Neuralstem Advance

by Amy Madsen on Thu, 2011-11-10 15:43

CK-2017357 is being tested in combination with riluzole; Neuralstem is testing its stem cells in the cervical region of the spinal cord

Article Highlights:
  • Two human clinical trials to test experimental therapies in ALS are moving forward.
  • After having tested its experimental therapy CK-2017357 in people with ALS who weren't taking riluzole, biotechnology company Cytokinetics has now begun the second part of its phase 2 trial, in which the two drugs will be tested in combination.
  • Biotherapeutics company Neuralstem has begun testing the safety of transplanting its neural stem cells into the cervical (neck) region of people with ALS.

Editor's note 3/19/12: This story has been updated to reflect that Neuralstem has begun testing its stem cells in the cervical (neck) region of the spinal cord.

Two human clinical trials to test therapies in amyotrophic lateral sclerosis (ALS) are moving forward.

CK-2017357 now being tested in combination with riluzole

South San Francisco biotechnology company Cytokinetics announced Nov. 1, 2011, that it has begun testing its experimental ALS therapy CK-2017357 in people with ALS who are taking riluzole (brand name Rilutek). Riluzole is the only drug approved by the U.S. Food and Drug Administration (FDA) for treatment of ALS.

This marks Part B of a phase 2 trial of CK-2017357 in ALS which is testing safety and tolerability of the drug, and assessing any effects on motor function, respiratory function, muscle strength or fatigue. CK-2017357 is designed to enhance muscle contraction. It was granted "orphan drug" status March 10, 2010, by the FDA. (Orphan drug status provides financial incentives for the development of drugs for rare diseases.)

For more, read Cytokinetics' Nov. 1, 2011, press release: Cytokinetics Announces Initiation of Second Cohort of Ongoing Phase 2 Clinical Trial Evaluating CK-2017357 in Patients with ALS.

In Part A of the trial, people with ALS who were not taking riluzole were randomly assigned to one of four groups in which they received daily oral doses of placebo (fake treatment), or CK-2017357 at a dose of 125, 250 or 375 milligrams, for two weeks.

Part B will follow the same treatment schedule; in addition, each participant also will take 50 milligrams of riluzole.

In the first group, clinical assessments took place at scheduled intervals during the two-week treatment phase, and again one week after the final dose. The same assessment schedule is planned for the B group.

For details on participating in the trial, and for contact information, see A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients with ALS; or enter NCT01378676 into the search box at

Neuralstem will begin stem cell injections into cervical spinal cord

Editor's note 3/19/12: Neuralstem issued a press release on March 7, 2012, announcing that the 14th patient in the trial has now received stem cells into the cervical (neck) region of the spinal cord. She is the second participant to receive cells in the cervical region and is the first woman in this trial.

Maryland biotherapeutics company Neuralstem announced Oct. 24, 2011, that it has received approval from the U.S. Food and Drug Administration (FDA) to begin transplanting stem cells into the cervical (neck) region of people with ALS.

This first U.S.-based trial of neural stem cells in ALS opened at Emory University in January 2010. It's designed to assess the safety of Neuralstem's spinal cord neural stem cells and intraspinal transplantation method in people with ALS.

Testing has progressed through increasing levels of risk. After reporting "smooth progress" in June 2011 for the first 12 trial participants, who received transplantations into the lumbar (lower back) region of the spinal cord, Neuralstem sought FDA approval to begin transplantation in the cervical spinal cord region.

Now, three people with ALS will receive injections on one side of the cervical region of the spinal cord. If results are favorable, the final three trial participants will receive injections on both sides of the cervical region.

"By moving the cell delivery to cervical spinal cord — the first time the FDA has approved intraspinal injections in this region — we will demonstrate that we can deliver our cells safely and routinely to all parts of the spinal cord," Karl Johe, Neuralstem Chairman and Chief Scientific Officer, said in an Oct. 24, 2011, Neuralstem press release.

Although the study is a safety trial, it is hoped that cervical injections may help with breathing and swallowing in ALS. Study investigators plan to evaluate any effects on motor and respiratory function.

Enrollment for this trial is still open; however, in addition to other criteria, only those who live in close proximity to Emory University (located in Atlanta, Ga.) will be considered for the trial. For details and contact information, see Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of ALS; or enter NCT01348451 into the search box at

Further details can be found at the Emory ALS Center website.

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