Today, we welcome a guest scientist blogger, Dr. Sandrine Da Cruz from University of California San Diego (UCSD). She, along with her colleagues, just published an important paper that looks into how SOD1 misfolding, the second most common inherited cause of ALS, impacts sporadic ALS (SALS). A huge percentage of ALS cases – approximately 90% are sporadic – meaning we do not know the cause. It is extremely important to understand more about this population of people living with ALS. The ALS Association is proud to support Dr. Da Cruz’s work. She is a past awardee of our Milton Safenowitz Postdoctoral Fellowship and is now an Assistant Investigator the Ludwig Institute at UCSD.
Last night, WCVB, the Boston ABC affiliate, featured a half-hour show called “Unlocking ALS,” highlighting all the great work coming out of the Boston area in the fight against ALS. The show did a wonderful job covering all the progress that has occurred since the ALS Ice Bucket Challenge (ALS IBC), an organic online movement that catapulted ALS into the spotlight while raising $220 million worldwide. From the ALS IBC, The ALS Association received $115 million dollars, of which over $77 million has been dedicated to research. Peter Frates and his family were and continue to be instrumental in advancing this important movement.
Last week, AB Science announced that the Masitinib phase 2/3 clinical trial reached its primary endpoint of ALSFRS-R in people living with ALS. Currently, the company has applied for conditional marketing approval to the European Medicines Agency (EMA) in Europe and is sharing results with officials at the Federal Drug Administration (FDA) to decide next steps in the U.S. The ALS Association is encouraged by the limited results released by AB Science and looks forward to hearing more later this year. Here is a brief overview of the phase 2/3 Masitinib study.
The Association is pleased to continue on the tradition of supporting bright, young scientists in ALS research through the Milton Safenowitz Postdoctoral Fellowship Program. This year, we are supporting six new postdoctoral fellows out of a highly competitive applicant pool. In this series, we highlight the dedication and unique contribution each fellow makes to ALS research. Today, we feature Dr. Amanda Gleixner from the University of Pittsburgh.
The Milton Safenowitz Postdoctoral Research Program, falls under our TREAT ALSTM Global Research Program and was founded by the Safenowitz family through the Greater New York Chapter of The Association. Mr. Safenowitz died of ALS in 1998 and the family and chapter continues its support to this day. Each award is for $100,000 over a two year period.
These awards are designed to encourage and facilitate promising young scientists to enter the ALS field and most importantly, to remain. Lending to the success of our program, over 90 percent of our postdoctoral fellows go on to start their own ALS research laboratories and then mentor their own young scientists.
Here we highlight each of our postdoctoral fellows awarded in 2016 in a series to learn more about their exciting studies and get to know the person behind the bench. Our final featured fellow is Dr. Amanda Gleixner who is exploring how trafficking of molecules between the cell’s nucleus and surrounding cytoplasm contributes to C9orf72-associated ALS.
Amanda Gleixner, Ph.D.
Mentor: Christopher Donnelly, Ph.D., University of Pittsburgh, Pittsburgh, Pa.
Project: Linking impaired nucleocytoplasmic trafficking in C9orf72 ALS to altered nuclear pore complex O-linked N-acetylglucosamine (O-GlcNAc) posttranslational modifications
What is the impact of your research?
Intracellular transport has been shown to be compromised in the most common ALS genetic type. My studies seek to identify what drives the transport deficits in this and perhaps other types of ALS. By further characterizing what is impaired in ALS cells, we hope that new drugs can be designed to halt or reverse disease progression.
Why do you love working in ALS research?
I have found that those impacted by ALS are often highly engaged in the ongoing research efforts. This, in turn, motivates me to better our understanding of the disease so that one day we see ALS as a curable diagnosis.
Tell me something unique about yourself.
While growing up, I enjoyed swimming and dancing competitively.
Who are your heroes?
To me, heroes are people, such as parents, caregivers and police officers, who selflessly put the well-being and safety of others before their own needs.
Amanda’s project was generously supported by the Greater Philadelphia Chapter. Tune in to our blog next month for an in depth interview with Dr. Gleixner.
Read more about her project here.
Read more about this year’s postdoctoral class here in our press release.
The Association is pleased to continue on the tradition of supporting bright, young scientists in ALS research through the Milton Safenowitz Postdoctoral Fellowship Program. This year, we are supporting six new postdoctoral fellows out of a highly competitive applicant pool. In this series, we highlight the dedication and unique contribution each fellow makes to ALS research. Today, we feature Dr. Vicente Valenzuela from University of Chile.
This week, former San Francisco 49ers receiver Dwight Clark announced he has ALS. He joins other NFL football greats including Steve Gleason, Tim Shaw, Kevin Turner, O.J. Brigance, and others, who have been diagnosed with ALS following their pro football careers. These announcements have brought much attention to the connection between football and traumatic brain injury (TBI) and the question of whether such injuries from football can lead to ALS or chronic traumatic encephalopathy (CTE). Here, we highlight some research behind the possible connection.
In late-February 2017, The ALS Association announced the release of 11 new Living with ALS resource guides on our website, www.alsa.org. The guides were designed to inform and educate people about ALS in a comprehensive and easily understood format, addressing many of the common concerns and issues that face people living with ALS. They can be found in electronic format HERE.
We are also pleased to announce that several new educational resources are now available to view and download from our website, including: Families and ALS: A Guide for Talking with and Supporting Children and Youths and the Medical Information Packet and Key Medical Information Card.