Tuesday, April 6, 2010

ALS Clinical Trials Update

Back in January, as part of our coverage of the International ALS/MND Symposium in Berlin, Prize4Life blogged briefly about ALS clinical trials. We also featured clinical trials coverage here by Dr. Amber Dance, the science writer we sponsored through the ALS Forum, our collaboration with the Alzheimer Research Forum. And you all are wondering what has happened since then on the clinical trials front, right? Well, wonder no longer. Here’s the latest (in alphabetical order):

Ceftriaxone is an antibiotic used to treat Lyme disease, however it has also been shown to affect uptake of the neurotransmitter glutamate (which means, in English, that it may help protect brain cells which can be killed by too much glutamate) The Ceftriaxone Phase III study continues to enroll , and there is a potentially promising pre-clinical study looking at the drug’s effects for patients with spinal muscular atrophy (an infant-onset disease which shares many similarities with ALS). A 2005 study suggested that Ceftriaxone could increase lifespan, strength, and neuron survival in ALS model mice (for more information, see this review).

San Francisco, CA based Cytokinetics recently initiated a Phase IIa “Evidence of Effect” (EoE) clinical trial of their small molecule CK-2017357 for patients with ALS. The drug is reported to act on fast skeletal muscles to make them more responsive to signals from motor neurons. This means that the drug could, in essence, help muscles stay stronger and delay the loss of muscle control that accompanies ALS. In March 2010, Cytokinetics was granted orphan-drug designation for this compound by the United States Food and Drug Administration (FDA). The orphan-drug designation carries a number of benefits to help the drug reach the market sooner, including tax credits, and scientific and financial support.

Isis Pharmaceuticals in Carlsbad, California is using antisense technology to try to reduce the amount of a protein, superoxide dismutase (SOD1) that is mutated in some ALS patients. Antisense is a method by which scientists can reduce levels of a given protein and thus theoretically help in diseases thought to be related to mutant proteins, such as ALS. This approach was found to be effective in animal models (data will be presented at the upcoming American Academy of Neurology (AAN) Meeting next week) and the first patient was just enrolled in a Phase I clinical trial in March. When completed, this trial will enroll 32 subjects, and safety results will hopefully be completed in 2011.

Knopp Neurosciences
Knopp is working on a small molecule drug that is thought to protect the function of mitochondria, the energy source for all cells of the body (motor neurons are relatively huge cells so they are very sensitive to reduced energy supply). Nothing new has been reported by Knopp since the results presented at the Berlin Meeting, however, the company is scheduled to attend the AAN meeting next week. So stay tuned!


Neuralstem, Inc. of Rockville, Maryland has begun a Phase 1 safety trial utilizing stem cells derived from fetal tissue in ALS patients. The goal of the trial is to test a novel surgical method, developed by surgeon Nick Boulis, to safely deliver cells into the spinal cord. They have currently enrolled 2 patients and are planning on enrolling a dozen participants total (see ARF related news story).

TCA Cellular
TCA Cellular of Covington, Louisiana, has recently enrolled the first of 6 anticipated patients in a Phase I safety trial of its stem-cell based therapy.

While this progress on the clinical trial front looks promising, Prize4Life believes we still need other potential therapeutics in the pipeline. This is why we are excited about the impressive teams competing for the ALS Treatment Prize and why we are so focused on translational research (that is, the process of translating scientific discoveries into practical application, from the ‘bench’ to the ‘bedside’. See a full blog post on it here.) Those interested in the challenges and promise of translational neuroscience and the drug companies at the forefront of the field may be interested in an upcoming meeting happening in Boston in May:

The 2nd Annual Drug Development for Neurodegenerative Diseases conference will be held May 18-19. Convened by marcus evans, an internationally known conference organizer targeting senior industry decision-makers, the conference will feature a wide-ranging and high quality program covering innovations in pre-clinical studies and clinical trial design, biomarker discoveries, and target identification for therapeutic and drug discovery journey.

The conference will feature speakers from major drug companies, including Novartis, Merck, Pfizer, and BiogenIdec as well as some up-and-comers including Allon Therapeutics, Acorda Therapeutics, and EnVivo Pharmaceuticals (EnVivo is also one of the teams competing for the Avi Kremer ALS Treatment Prize). While the meeting will not focus exclusively on ALS, it will cover many topics and speakers in related fields that will hopefully “translate” into the ALS space. Prize4Life will be blogging live from this conference, so make sure to follow the blog for some hot-off-the-press info!

1 comment:

  1. Thank you for your posting.

    It's good to see results posted like this. As you know clinical trials are an important part of the medical community, and getting word out is imperative to the life of Clinical trials.

    Clinical Trials and research studies are what we work on here in DeLand, Florida. Some of our investigational treatments have shown promising results, but there is we need to do additional research studies to test investigational methods that may help us understand the condition.

    If you could pass on the word to your readership it would be incredibly helpful to many who suffer from various diseases and conditions.