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On the hunt for Huntington’s disease therapy
DEERFIELD, Ill.—Two leaders in Huntington's disease research, global pharmaceutical company H. Lundbeck AS and not-for-profit biomedical research organization CHDI Foundation Inc., have joined forces to conduct preclinical studies on a targeted therapy for the neurodegenerative condition.
The partners announced this endeavor in May, coinciding with HD Awareness Month, and it is part of Lundbeck's HD Research Initiative, a commitment announced in 2010 to identify and ultimately commercialize therapies that may slow or halt the progression of HD. The initiative is driven by collaborations with like-minded academic institutions, research organizations and companies.
Although the collaboration is a first for the two parties, CHDI is no stranger to partnerships in HD research. The organization actively enables HD research by collaborating with research organizations and pharmaceutical companies conducting promising research, often providing financial support. CHDI's activities include exploratory biology, clinical studies and trials and educational workshops.
In Lundbeck, CHDI gains a partner that is well established in central nervous system drug research and development, said the organization's vice president of translational biology, Ignacio Munoz-Sanjuan, in a press release announcing the deal.
"Lundbeck has a proven track record of not only bringing new therapies to market, but also working to support the needs of their patient communities," Munoz-Sanjuan added. "We hope this research collaboration provides a stepping stone for future therapies that slow the progression of HD."
But Lundbeck "can't do research all by ourselves," says Stevin Zorn, executive vice president for Lundbeck Research USA. "That is only part of how we do business. In a number of interactions with CHDI in the past few years, we developed a deep respect for each other. CHDI is very closely focused on biomedical research, and since we don't have much experience in that area, we thought getting together would benefit us both by bringing something they could help us advance."
As part of the collaboration, CHDI will conduct preclinical studies on a Lundbeck investigative compound. The research will focus on the compound's effect on P2X receptors that may be involved in HD. P2X receptors are membrane ion channels that open in response to the binding of extracellular adenosine triphosphate (ATP), the molecule that cells use for energy to power all their processes.
In 2011, Lundbeck and the University of Massachusetts Medical School began to investigate RNAi-based therapies to suppress the production of mutant huntingtin (mHtt), the abnormal protein that causes HD.
"We worked with scientists to study RNAi-based therapy that should provide a way of suppressing the protein mHtt, which could potentially change the course of this illness," says Zorin.
According to the partners, the study results will influence future research into this and other compounds for HD. Currently available therapies for HD treat only its symptoms.
HD is a neurodegenerative genetic disorder that affects muscle coordination and leads to cognitive decline and psychiatric problems. These symptoms can vary from patient to patient. The survival time after the onset of symptoms can range from 10 to 30 years.
Complications such as pneumonia, heart disease and physical injury contribute to that figure. There is no cure for HD, and full-time care is usually required in the later stages of the disease.
"There are about 20,000 people who suffer from HD. There is no cure, and the disease can be devastating. Coming up with an effective treatment for this illness could have a fairly robust impact on society," says Zorin.