April 2009

ALS Association and American Academy of Neurology Give
Top Scientists Sheila Essey Award for ALS Research

The ALS Association joins the American Academy of Neurology in presenting The 2009 Sheila Essey Award for ALS Research to two clinician scientists who have significantly impacted clinical trials, epidemiology and genetics in ALS.

Dr. Merit Cudkowicz

Dr. Merit Cudkowicz, Associate Professor of Neurology at Massachusetts General Hospital, Boston, Mass., is an international leader of clinical therapeutics in ALS. She is co-founder and co-director of the Northeast ALS Consortium (NEALS) a clinical trials network of 76 clinical sites throughout the U.S. and Canada dedicated to performing academic led clinical trials. The trials network has completed six trials: three phase III efficacy studies (topiramate, creatine and celecoxib) and three phase II studies (coenzyme Q10, sodium phenylbutyrate, arimoclomol).

Dr. Cudkowicz is currently playing a leadership role in three clinical trials through this network: ceftriaxone, lithium and arimoclomol in patients with SOD1 mutations. 

In 2007 through The Association’s Translational Research Advancing Therapies of ALS (TREAT ALS) program, The Association partnered with NEALS to establish the TREAT ALS/ NEALS clinical network facilitating broader participation in ALS clinical trials. In addition, Dr. Cudkowicz has been advisor to The Association’s pilot clinical trial program, which is currently funded by The Association for the SOD1 antisense trial in familial ALS and has established a repository for patient samples.

Ongoing studies in her group, facilitated by this resource, attempt to find biomarkers or signatures for the disease to allow for earlier diagnosis and improved clinical trials. Furthermore, Dr. Cudkowicz recognizes the importance of encouraging young clinician scientistsin ALS and has mentored, among others, Dr. Aggarawal, recipient of this year’s AAN/ALS Association Clinician Scientist Development Award. Her group has focused on adaptive clinical trial design to identify promising treatments for ALS more rapidly.

“It is an honor to receive this award for my research team at the Massachusetts General Hospital Neurology Clinical Trial Unit and our collaborators in the Northeast ALS consortium. The Sheila Essey award will be used to support a novel approach to more quicklydevelop treatments for people with ALS. The award will help continue our mission to find new treatments for people with ALS,” commented Dr. Cudkowicz.

Dr. Orla Hardiman

Dr. Orla Hardiman, Professor of Neurology, Trinity College Institute of Neuroscience, Dublin, Ireland, developed the longest running population based register of ALS in the world. The register is now part of the European ALS group, founded by Dr. Hardiman.

Many of her studies in clinical trials, outcome measures and patient care have led to important changes in how people with ALS are cared for. Her group undertook a systematic review of the impact of ethnicity on ALS epidemiology and demonstrated that the frequency of ALS is not uniform across the world. In addition, capitalizing on the relative homogeneity of the Irish population, her group identified a series of novel mutations in angiogenin(involved in formation of blood vessels and also thought to play a role in the protection of motor neurons) linked to some cases of ALS.

Dr. Hardiman is currently part of an international consortium using genome wide technology to identify potential genes linked to sporadic cases of ALS. Dr. Hardiman, funded by The ALS Association, is undertaking a detailed population-based longitudinal survey of cognition in ALS. Her group’s data suggest that cognitive impairment in ALS occurs in about 40% of patients and occurs early in the disease. This work has generated an important resource of DNA from patients followed longitudinally with detailed neuropsychological profiling. “This is a great honor for my research team, our Irish and international collaborators and the Irish ALS community. Finding the causative genes in small homogeneous populations and looking for protective genes in ethnically mixed populations can help to identify new pathways that lead to neurodegeneration. This award will be used to help to develop our research ideas, with the overall aim of finding pathways in ALS that can be harnessed to develop new treatments,” said Dr. Hardiman. “We are grateful to The ALS Association for their continued support of our work.”

In 1996 The ALS Association in partnership with the American Academy of Neurology inaugurated the Sheila Essey Award for ALS Research to acknowledge and honor an individual actively engaged in ALS research who is making significant contributions in research for the cause, treatment, prevention or cure for amyotrophic lateral sclerosis (ALS). The recipient receives a $25,000 prize to be used specifically for continuing his/her ALS research. Funding of the award is made possible through The Essey Family Fund and The ALS Association.