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CHDI: 4th Annual Therapeutics Conference (Part 1)

By Dr. LaVonne Goodman

CHDI held its 4th Huntington's Disease Therapeutics Conference on April 27-30 in Cannes, France. This energetic and truly international conference included almost two hundred scientists from academic and pharmaceutical institutions -- all working to develop drugs for Huntington's.

An enormous dose of drug development information - a larger portion of it coming from pharmaceutical collaborators than previous years -- was presented in lectures and posters. Of equal importance, outside the lecture halls there were lively discussions, sharing of additional information, and the beginnings of new collaborative efforts.

But unique to this year -- from the initial keynote speaker all the way to the closing bell--was the spotlight on serious, and sometimes controversial discussions about clinical trials. This highlights that CHDI is getting ready for clinical trials.

Julie's Awesome Beginning: As in past years a Huntington family advocate opened the conference. In the tradition begun by James Tretheway and followed by Charles Sabine, keynote speaker Julie S. -- whose husband is gene positive -- took the stage. And like a lightening bolt she set the energetic tone for this meeting.

Drawing from her film industry background -- both she and her husband are California producers and directors -- Julie spoke of the power of film as a tool to reach millions of people that can both educate and increase public awareness of HD. She presented an edited series of poignant film portrayals of Huntington Disease dating from the 1960's to the present. Then in a documentary film produced by Julie and her husband, she captured the essence of present day suffering of Huntington's in extended families.

More Lightening and Thunder: Julie alluded to discouragement they encountered when they requested gene testing. After this experience she and her husband have become vocal advocates for simplifying the gene testing experience and making it more easily assessable -- by allowing patient control over initiating gene testing.

HD Ready: Also an advocate for early testing, Julie has coined this great and catchy new phrase "HD Ready" for the Huntington's community. She defined "HD ready" as those HD individuals who test early, who actively self-educate, and who are proactive in healthy lifestyles that may potentially slow disease progression: exercise, available supplements, stress reduction and adequate hours of sleep.

She also stressed that "HD Ready" means ready for efficient entry into clinical trials that can potentially bring treatments for this generation of Huntington's. - even before symptoms develop.

We enthusiastically welcome Julie and her husband to the HD family community. They are talented additions to the HD advocacy effort. In addition to films included in the presentation, Julie and her husband have produced several other film spots films that feature Olivia Wilde (Thirteen) and Peter Jacobson (Dr. Taub) from House M.D. They are being used in a TV and print campaign for HDSA and Huntington's May awareness month and on HDTrials.org. And with more good things coming, they are presently working on behind the scenes interviews with Olivia Wilde and Peter Jacobson.

Dr. Pacifici's CHDI Update
Robert Pacifici is Chief Scientific Officer of CHDI, a non-profit organization whose sole purpose is to seek treatments that delay or slow Huntington's disease. Since its beginning 5 years ago, CHDI now employs more than 50 individuals, coordinates many internal drug development projects and is collaborating with more than 500 scientists from academic and pharmaceutical institutions around the world.

In a positive signal marking the preparation and approach of CHDI clinical trials, Dr. Dan van Kammen was brought on board as Chief Medical Officer in 2007.

In addition to its internal work, the scope of CHDI effort for drug development and clinical research is broad, ranging from basic research all the way to clinical trials. In addition to its own drug discovery programs, their work includes:

• Providing financial support for preclinical and clinical research

• Organizing workshops that bring experts together for sharing of ideas
• Establishing cooperative partnerships and projects with academic

and pharmaceutical scientists

• Generating and sharing research tools

CHDI enables the research of many others. As example, they fund the operation of biologic repository centers, one in Europe and one in North America. These are the centers that receive, process, maintain and distribute biologic samples used for research. As one example specific cells from blood samples obtained from participants in COHORT are grown in cultures that are maintained long term. This means that one blood sample can provide enough DNA or other molecules for any number of experiments now and in the future. Because this service is available, investigators can request and obtain the hundreds, even thousands of different samples needed in the search for biomarkers or other research.

CHDI Research Crossroads is a new effort rolled out at this meeting. This project utilizes a confidential Web Portal that can be used to increase communication - the Wikipedia of the HD research community. Though its use is restricted to enrolled individuals, it allows researchers to stay informed and to efficiently communicate updates on promising compounds.
CHDI directs internal programs for drug development: Dr. Pacifici reported that greatest effort is ongoing in 11 focused programs that include

HDAC inhibitors
• Transglutaminase-2 inhibitors
• Caspase (2 and 6) inhibitors
• JNK3 inhibitors (Neuroinflammation)
• Phosphodiesterase inhibition
• Antisense Oligo's (ISIS)
• KMO
• Trophic factors
• Adenosine A2a inhibitors
• Toxic fragments
• Protein function

In Part 2 of this CHDI report, there will be further discussion of those programs that were highlighted during the conference.

View the Keynote Address from the CHDI 2009 Conference