Carlsson Research is a small biotech company that is developing a promising drug for Huntington's, called ACR16. Dr. LaVonne Veatch Goodman tells the history of this drug and her hopes for more good news to come next.

Carlsson Research is a small biotech company based in Sweden that has recently reported Phase II clinical trial results for its very promising drug for Huntington's. This company deserves our applause, and not just for developing a drug that is looking good. They have unselfishly done what most small pharmaceutical companies won't: they've likely taken a financial hit for our orphan disease. For this they deserve a standing ovation.

The Drug Their drug ACR16 stabilizes dopamine systems which are disrupted in Huntington's. In May 2006, the company reported results from a clinical trial performed in Sweden and Norway (press release): fifty eight people received either the drug (ACR16) or placebo, and were tested over a 6 month period. Those who received treatment had improved motor function, particularly chorea, walking ability, and speech. The drug also reduced bradykinesia (slowness of movement) symptoms. There was also a strong trend in improving depression and anxiety. Treated participants also had "marked" improvement in (trail-making) tests of cognitive function. Further, there were no adverse side effects reported.

In short, this drug improved motor, cognitive and psychiatric components of Huntington's. All this and no side effects.

Company History Carlsson Research is a model for orphan disease, and a star for Huntington's; at least the 2nd time around. In 1998, Avrid Carlsson, a Nobel prize-winning neuroscientist developed and tested a drug (-)-OSU6162 developed in his laboratory in a single Huntington's patient. A single dose had dramatic effects on chorea for several weeks, and allowed this bedridden person to walk and talk again [Tedroff J 1999]. Then after what seemed like a drug too good to be true, it was sold to a large pharmaceutical company (Pharmacia) who, in what is common big pharma practice, stopped its development for Huntington's.

In a move fortunate for HD people, this wasn't the end of the story. Carlsson Research developed another drug (ACR16) and held onto it through phase I trials in 2001. This time when the drug was sold to a big company (Fuzisawa Pharmaceutical), Carlsson retained the rights to develop it for Huntington's. And more good news: they completed their own phase II trial and are moving towards phase III trials in Europe and the US. And even though the company was recently sold to Neurosearch (news story in Forbes), clinical trials for Huntington's will go forward in both Europe and the US, with US trials slated to begin in mid 2007

Carlsson, founder of Carlsson Research is a shining altruistic star. No doubt his company took a financial hit by "holding out" for Huntington's. And we're certainly glad he did.


Tedroff J, Ekesbo A, Sonesson C, Waters N, Carlsson A. Long-lasting improvement following (-)-OSU6162 in a patient with Huntington's disease. Neurology. 1999 Oct 22;53(7):1605-6. PubMed abstract