The American Academy of Neurology will honor two scientists for their contributions to research on amyotrophic lateral sclerosis at its annual meeting in Seattle, Washington, at the end of the month. The pair study opposite ends of the ALS disease process but both will receive the Sheila Essey Award—An Award for ALS Research. Orla Hardiman of Beaumont Hospital in Dublin, Ireland, will be recognized for her work in identifying human gene variations associated with ALS; Merit Cudkowicz of Massachusetts General Hospital, Boston, will be honored for her research focused on drug development.

Hardiman’s work is focused on what causes ALS. She and colleagues identified angiogenin as a gene mutated in both sporadic and familial ALS (see ARF related news story and Greenway et al., 2006). Angiogenin promotes blood vessel growth and also participates in neurite extension (Subramanian et al., 2008). In another paper, Hardiman and others performed a genomewide association screen to pick out DP66, which encodes a protein that helps make up potassium channels, as a gene associated with risk for developing ALS (see ARF related news story and Cronin et al., 2008).

In contrast, Cudkowicz focuses not on the source of ALS but on how to treat it. She has led clinical trials of potential ALS therapeutics, such as recent evaluations confirming the safety of the drugs sodium phenylbutyrate (Cudkowicz et al., 2008) and arimoclomol (Cudkowicz et al., 2008). Currently, there is only one FDA-approved treatment for ALS, riluzole, and more effective drugs are urgently needed. However, scientists have struggled to find drugs that work as well in people as they do in mice (see ARF Live Discussion).

Hardiman and Cudkowicz will each receive $25,000 to be put toward further ALS research during the AAN’s 61st Annual Meeting in Seattle, to be held April 25 through May 2, 2009.—Amber Dance.

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References

News Citations

  1. ALS—Study Strengthens VEGF Connection, Potential Biomarkers Proffered
  2. Sporadic ALS Linked to Potassium Channel

Webinar Citations

  1. Mice on Trial? Issues in the Design of Drug Studies

Paper Citations

  1. . ANG mutations segregate with familial and 'sporadic' amyotrophic lateral sclerosis. Nat Genet. 2006 Apr;38(4):411-3. PubMed.
  2. . Human angiogenin is a neuroprotective factor and amyotrophic lateral sclerosis associated angiogenin variants affect neurite extension/pathfinding and survival of motor neurons. Hum Mol Genet. 2008 Jan 1;17(1):130-49. PubMed.
  3. . A genome-wide association study of sporadic ALS in a homogenous Irish population. Hum Mol Genet. 2008 Mar 1;17(5):768-74. PubMed.
  4. . Phase 2 study of sodium phenylbutyrate in ALS. Amyotroph Lateral Scler. 2009 Apr;10(2):99-106. PubMed.
  5. . Arimoclomol at dosages up to 300 mg/day is well tolerated and safe in amyotrophic lateral sclerosis. Muscle Nerve. 2008 Jul;38(1):837-44. PubMed.

Further Reading

Papers

  1. . Screening for replication of genome-wide SNP associations in sporadic ALS. Eur J Hum Genet. 2009 Feb;17(2):213-8. PubMed.
  2. . Therapy development for ALS: lessons learned and path forward. Amyotroph Lateral Scler. 2008 Jun;9(3):131-40. PubMed.
  3. . Combined riluzole and sodium phenylbutyrate therapy in transgenic amyotrophic lateral sclerosis mice. Amyotroph Lateral Scler. 2009 Apr;10(2):85-94. PubMed.
  4. . Screening of hypoxia-inducible genes in sporadic ALS. Amyotroph Lateral Scler. 2008 Oct;9(5):299-305. PubMed.
  5. . Minocycline for patients with ALS. Lancet Neurol. 2008 Feb;7(2):119-20; author reply 120-1. PubMed.
  6. . Amyotrophic lateral sclerosis: an emerging era of collaborative gene discovery. PLoS One. 2007;2(12):e1254. PubMed.
  7. . Arimoclomol at dosages up to 300 mg/day is well tolerated and safe in amyotrophic lateral sclerosis. Muscle Nerve. 2008 Jul;38(1):837-44. PubMed.
  8. . Trial of celecoxib in amyotrophic lateral sclerosis. Ann Neurol. 2006 Jul;60(1):22-31. PubMed.
  9. . ANG mutations segregate with familial and 'sporadic' amyotrophic lateral sclerosis. Nat Genet. 2006 Apr;38(4):411-3. PubMed.
  10. . A novel candidate region for ALS on chromosome 14q11.2. Neurology. 2004 Nov 23;63(10):1936-8. PubMed.