New Myasthenia Drug Under Study
A drug known as Monarsen (formerly EN101), developed by Ester Neurosciences in Herzelia, Israel, is being studied in the UK under the direction of Jon Sussman at Hope Hospital in Greater Manchester. If all goes well, testing may be expanded to the United States and Europe.

In myasthenia gravis (MG), Lambert-Eaton myasthenic syndrome and some congenital myasthenic syndromes, patients benefit from drugs that prolong the action of acetylcholine at the junction of nerve and muscle fibers.

In MG, the immune system mistakenly attacks receptors for acetylcholine on muscle cells. In Lambert-Eaton syndrome, the immune system inhibits acetylcholine’s release from nerve fibers. And in some congenital myasthenic syndromes, the action of acetylcholine is insufficient because of a genetic mutation that affects the neuromuscular junction.

A common treatment for myasthenia is pyridostigmine (Mestinon), which interferes with the breakdown of acetylcholine by the enzyme AchE. But pyridostigmine’s actions are limited and, at high doses, it can have unwanted side effects.

Monarsen targets AchE before it’s synthesized, while pyridostigmine targets the finished protein. According to Ester Neurosciences, attacking the finished protein stimulates the body to produce more AchE, triggering a battle between the drug and the nervous system. In contrast, the company says, Monarsen, which inhibits AchE synthesis, doesn’t cause this vicious cycle.

The company says that Monarsen could also have applications for amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease), a disease in which muscle-controlling nerve cells die.

lisag - Can you post the link to the above article please? The statement "The company says that Monarsen could also have applications for amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) . . . ". hmmm-should post it on ALSTDF too and get Dr. McCarty's take on it.

Hi Terrafirma...here's the link...I did post at TDF earlier this morning to ask Dr. McCarty if TDF has tested it..no reply as of yet..but I'm sure he stays pretty busy...Lisa


http://www.mdausa.org/research/06101..._monarsen.html

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Ester Neurosciences Ltd. today presented preclinical data showing the therapeutic potential of Monarsen (EN101) in ALS. The data was presented at the 2nd Annual Israeli ALS Research Association meeting. ALS, Lou Gehrig ’s disease, the most common motor neuron disease, results from progressive degeneration of both upper and lower motor neurons. There is no cure for ALS. Treatment is based on individual therapy and the continual adaptation of medications. Riluzole (Rilutek®) of Novartis is the only approved drug for ALS and may prevent progression and prolong life for a few months or so. Monarsen (EN101), a novel antisense oligonucleotide drug for neuromuscular impairments, has already been shown safe and effective in patients with myasthenia gravis. The drug based on the research of Prof. Mona Soreq of the Hebrew University, binds to a novel splice variant of acetylcholinersterase (AChE) mRNA, resulting in a marked decrease in AChE levels in many tissues including muscle and spinal cord neurons. Monarsen is currently completing its Phase II clinical studies. For ALS, Monarsen was tested in a dedicated transgenic mouse model (G93A mice) using a very low daily administration (200-500 ug/kg), covering both the disease prevention and the therapeutic protocols. The onset of the disease, the appearance of disease symptoms and disease related mortality were all significantly delayed or diminished when compared to the control group. “These results show that Monarsen delays the disease process in a mouse model of ALS and supports the hypothesis that acetylcholinesterase are involved in the pathogenesis of ALS –We plan to test the safety and efficacy of Monarsen in ALS in a double-blind study in patients with definite or probable ALS, said Dr. Lior Carmon, General Manger at Ester Neurosciences. The primary endpoint of the study will be the change of Revised ALS Functional Rating Scale, with survival, manual muscle strength, forced vital capacity and a single item quality of life scale as secondary endpoints.

About ALS
Amyotrophic lateral sclerosis (ALS), also called Lou Gehrig’s disease, is a progressive neuromuscular disease that weakens and eventually destroys motor neurons (components of the nervous system that connect the brain with the skeletal muscles). According to the ALS Survival Guide, 50% of ALS patients die within 18 months of diagnosis and 80% die within five years. In the U.S. an estimated 30,000 people are living with ALS and nearly 6,000 new cases are diagnosed each year, according to the ALS Association. There are more than 120,000 people living with ALS worldwide.

About Ester Neurosciences Ltd.& Monarsen™
Ester Neurosciences Ltd. is a phase II, clinical stage biopharmaceutical company committed to the development of novel therapeutic products for the treatment of neurological disorders with a focus on niche neuromuscular indications.


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Ester Neurosciences Ltd. is a development stage biopharmaceutical company committed to the discovery and development of novel therapeutic products for the treatment of neurological disorders such as myasthenia gravis, Alzheimer's disease, multiple sclerosis and acute stress reactions. Ester's unique platform technology is based on the company's breakthrough discoveries relating to cholinergic neuromodulation and its involvement in the diseased state.


Address: Ester Neurosciences Ltd., Ackerstein Towers, Bldg. B. 10th Flr.
11 Hamanofim St., Herzlia Pituach 46725
POBox 2206, Herzlia Pituach 46120
Israel
Telephone: +972-9-960 1919
Fax: +972-9-954 2266
E-mail: info@esterneuro

MORE INFO FOR YOU, HOPE OF HELP TO YOU. SORRY WAS CONFUSED WITH BT 1, 2 ETC.

Hi Gumby ..nice to meet you...thanks for posting the addditional information here...
I'm curious to see if Dr. MCCarty at TDF has any thoughts about this drug or if TDF has tested it..
The one thing I have learned though is sometimes press releases can be very misleading..often they give the best spin possible...but hopefully it has some promise..Lisa

Hi, nice to meet you too. I have read of all of your struggles and strength over at the other bt, with great respect for you all. I hope this may be of some promise and help for you. I know the M.G. community is keeping an eye out. I know I am. If I find anything else, I will post. God keep you all.

Hi thanks so much for the post and your kind words ..but unfortunately this drug does not seem that promising in regards to ALS ....Dr. MCCarty just responded on the thread at TDF...Lisa
http://www.als.net/forum/topic.asp?TOPIC_ID=960

Sorry that there is not much promise for use in ALS. I thought it would be of some kind of use. I hope that something will become of hope for you guys soon, real soon. I wish all of you the best, God keep you all.

Hi Gumby...thank -you...many of us too hoped it would help..but hopefully there will be somerhing else found one day soon...Lisa