Good News The Phase II results for ACR16, a new HD drug being developed by Carlsson Research, is good news for Huntington's people. Here are the results as reported by the company and discussed on HDDW.
The company's founder, Arvid Carlsson, is a Nobel Prize
winner who has spent his career studying dopamine, a critically
important brain protein whose function is disrupted in HD and other
neurologic diseases. His Nobel Prize lecture, available from the official Nobel Foundation
site, is heavy going for the non-scientist but offers a
fascinating look at the research that led up to ACR16. Page 16 of
the lecture discusses an early version of the drug, (-)-OSU6162,
which Carlsson tested in a single HD patient with dramatic
But How Much Longer? The timeline of events may be of interest to HDDW readers. Carlsson Research was founded in 1998, the OSU6162 result was published in 1999, Carlsson won his Nobel Prize in 2000, the Phase I trial for ACR16 began in November 2001 and was successfully completed in May 2002, and now more than four years later, the Phase II study has been completed. Phase III trials will begin soon in Europe and in mid-2007 in the US. I believe the company is planning limited trials that can be completed quickly with relatively few patients - probably a couple of hundred patients for 6-12 months. Still, under the best case scenario, it will be 3-4 years before the whole process wraps up and the company gets approval to start selling the drug in the US. Another year or two may pass while the company ramps up manufacturing and provides the drug to pharmacies.
Adding it up, we may expect to see this drug on the US market somewhere in the 2010-2012 range, a dozen years after OSU6162 was first tested in an HD person.
Yes, it's exciting to see new HD drugs like ACR16 getting closer to reality, but for people who need treatment now, the 4-6 years that it will take to finish the job on ACR16 will be years lost forever. At present, drugs like ACR16 are only available to the relatively few patients enrolled in clinical trials and only for the relatively short duration of the trials themselves.
Of course, we can't be certain that ACR16 will prove to be terrific drug. Once the drug is approved, additional trials will be needed to better understand how well it works, what symptoms it treats, what are the side effects, and whether it provides long term neuroprotection. The good news is that while these post-approval trials are underway, the drug will be available to everyone, not just people participating in trials, so no more time will be lost.
Treatment NowWe need to find ways to speed up the endgame and get promising treatments more quickly to Huntington's people who want this choice.
One approach is to squirm through various loopholes in the US Food and Drug Administration (FDA) regulations and obtain drugs for compassionate use or for investigational use. This is a difficult course but is doable. We need to get better at this, since these loopholes are the best short term option we have.
Another approach is to change the law and provide an easier path
for seriously ill patients to obtain drugs like ACR16 that have
passed their Phase I safety tests and have shown benefit in Phase
II trials. Legislation to this effect (Bill S.1956, The Access
Act) has been filed in the US Senate by Sens. Brownback (R-KS)
and Inhofe (R-OK). This bill has little chance of passing in its
current form, but the idea may be included in upcoming FDA reform
legislation and may offer help to Huntington's people who need
The Access Act
S.1956, The Access, Compassion, Care and Ethics for Seriously Ill Patients Act, short name The Access Act, filed by Sens. Brownback (R-KS) and Inhofe (R-OK) with cosponsors Allen (R-VA), Isakson (R-GA), and Sessions (R-AL). Full text from U.S. Government Printing Office