Notes from ACARM7

We just came back from the 7th All California Ataxia Research Meeting (ACARM7) held at the San Jose Doubletree Hotel today. This year's meeting was almost canceled due to budget issues stemming from lower than expected attendance. At the eleventh hour, it was saved by a generous anonymous underwriter. The other unsung heroes are Mike Fernandes, the organizer of the venue, the doctors and presenters of the program and the numerous volunteers who helped to bring the event into fruition.

The first speaker of the morning was Dr. Richard Ivry who talked about "The Effect of Ataxia on Cognition". Specifically, he spoke about Dr. Jeremy Schmahmann's work on the "Cerebellar Cognitive Affective Syndrome". Dr. Ivry concluded that ataxics have difficulty with non-motor tasks but their cognitive problems might be secondary to their motor problems.

Dr. Susan Perlman was next. Her topic was "An Update of Clinical Trials for the SCAs and HD". According to Dr. Perlman, if one were to do a web search on the key words "clinical trials for Ataxia", one would find 42 trial listings of which only 35 actually dealt with Ataxia. 13 of these trials have been completed, 5 are active but no longer recruiting for subjects while the remaining 17 are still recruiting. Of these 17, one is available in California ---- Phase III Idebenone and Extension Study for FA. (Friedreich's Ataxia) Also, the pilot study of Lithium for patients with SCA1 has now started in Maryland and is still recruiting.

Dr. Lisa Ellerby followed Dr. Perlman with "Target Validation in HD: siRNA screens in HD cell culture models". After conducting studies to screen through possible druggable targets using siRNA technology, her team has found that HDAC4 inhibitors to be effective against Huntington's Disease.

Dr. Elizabeth Thomas was the last speaker of the morning. She further elaborated on the HDACs with "Novel Histone Deacetylase Inhibitors as Therapeutics for Triplet Repeat Disorders". While conventional HDAC inhibitors (used in treating cancers) are very toxic, novel HDAC inhibitors have been shown in (non-human) trials to be low in toxicity. Experiments on transgenic mice using HDACi4b have shown it to be therapeutic and non toxic for triplet repeat disorders such as HD, SCA1, 2 and 3. Repligen Corp. in Waltham, Massachusetts has licensed HDAC inhibitors in development.

The three presenters in the afternoon were Richard Devylder, (Emergency Disasters for the Disabled) Colleen Campbell (Falling Prevention) and Arya Pathria. (Laughing: Laughter Yoga Workshop)

Finding your inner Tigger

Just in case you missed "The Last Lecture", I highly recommend it on those "why me" days.

All California Ataxia Research Meeting 2008 (ACARM7)

ACARM7 will be held on Sunday, September 14, 2008 at the Doubletree Hotel by the San Jose Airport. This year's meeting will concentrate on the SCAs. Richard and I are looking forward to the event. As usual, kudos to Mike Fernandes for putting this together.

Sad news

Our friend and fellow Nanshan patient, Nadia, succumbed to ALS in late June. We will miss her terribly but we take comfort in our fond memories of the time that we spent with her in Shenzhen last year. Our deepest sympathy to Fabio and his family.

Please help in finding a cure for this terrible disease by donating to the ALS Association.

Point/Counterpoint

Remember the NPR article about China stem cells that Richard mentioned in this post? Here's Jake Young's opinion of the article from his blog, Pure Pedantry. He also briefly mentioned and quoted from the Jane Qiu's Lancet Neurology article.

The Lancet Neurology February 2008

Freelance journalist and neuroscientist, Jane Qiu, interviewed Richard last year about his experience at Nanshan. Her article, "Injection of hope through China's stem cell therapies", was published in the February 2008 issue of the Lancet Neurology.

Thank you, Jane, for making your article available to us.

Download the Lancet article through here

A Spoonful of Sugar

We just returned from a visit with Dr. Perlman last Friday at UCLA. Richard's SARA score stayed pretty much the same which is encouraging.

Dr. Perlman wanted Richard to start taking a new supplement called Trehalose which is a sugar derivative. Trehalose has been shown to help with Huntington's Disease in preventing the clumping of proteins in the cells. One of the hallmarks of MJD is also clumping of the proteins so Trehalose should theoretically help stop this. Moreover, Trehalose is fairly safe with few side effects and is very cost-effective and readily available.

Here's a link to the website of Neurocoat which is a 100% Trehalose supplement. This article has details on the recommended dosage. We have already placed our order.