DEERFIELD, Ill.—Two leaders in Huntington's diseaseresearch, global pharmaceutical company H. Lundbeck AS and not-for-profitbiomedical research organization CHDI Foundation Inc., have joined forces toconduct preclinical studies on a targeted therapy for the neurodegenerativecondition.
The partners announced this endeavor in May, coinciding withHD Awareness Month, and it is part of Lundbeck's HD Research Initiative, acommitment announced in 2010 to identify and ultimately commercialize therapiesthat may slow or halt the progression of HD. The initiative is driven bycollaborations with like-minded academic institutions, research organizationsand companies.
Although the collaboration is a first for the two parties,CHDI is no stranger to partnerships in HD research. The organization activelyenables HD research by collaborating with research organizations andpharmaceutical companies conducting promising research, often providingfinancial support. CHDI's activities include exploratory biology, clinicalstudies and trials and educational workshops.
In Lundbeck, CHDI gains a partner that is well establishedin central nervous system drug research and development, said theorganization'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 newtherapies to market, but also working to support the needs of their patientcommunities," Munoz-Sanjuan added. "We hope this research collaborationprovides a stepping stone for future therapies that slow the progression ofHD."
But Lundbeck "can't do research all by ourselves," saysStevin Zorn, executive vice president for Lundbeck Research USA. "That is onlypart of how we do business. In a number of interactions with CHDI in the pastfew years, we developed a deep respect for each other. CHDI is very closelyfocused on biomedical research, and since we don't have much experience in thatarea, we thought getting together would benefit us both by bringing somethingthey could help us advance."
As part of the collaboration, CHDI will conduct preclinicalstudies on a Lundbeck investigative compound. The research will focus on thecompound's effect on P2X receptors that may be involved in HD. P2X receptorsare membrane ion channels that open in response to the binding of extracellularadenosine triphosphate (ATP), the molecule that cells use for energy to powerall their processes.
In 2011, Lundbeck and the University of MassachusettsMedical School began to investigate RNAi-based therapies to suppress theproduction of mutant huntingtin (mHtt), the abnormal protein that causes HD.
"We worked with scientists to study RNAi-based therapy thatshould provide a way of suppressing the protein mHtt, which could potentiallychange the course of this illness," says Zorin.
According to the partners, the study results will influencefuture research into this and other compounds for HD. Currently availabletherapies for HD treat only its symptoms.
HD is a neurodegenerative genetic disorder that affectsmuscle coordination and leads to cognitive decline and psychiatric problems.These symptoms can vary from patient to patient. The survival time after theonset of symptoms can range from 10 to 30 years.
Complications such as pneumonia, heart disease and physicalinjury contribute to that figure. There is no cure for HD, and full-time careis usually required in the later stages of the disease.
"There are about 20,000 people who suffer from HD. There isno cure, and the disease can be devastating. Coming up with an effectivetreatment for this illness could have a fairly robust impact on society," saysZorin.
