Happy New Year - 2022 a Year of Hope

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Research Update

By Dr. Robert H. Brown, Jr.

Prospects for ALS -

The View at Year's End - 2021

Remarkably, today we say farewell to 2021; this is an appropriate time to look back on recent developments in ALS research. It can truly be said that there has been meaningful progress in the quest to find treatments for ALS. Much has been accomplished in understanding the basic biology of this disease.

In the Angel Fund ALS research program, new methods of gene analysis are defining critical events that trigger motor neuron pathology, both in mouse models of ALS and in humans. At multiple laboratories, including those at UMass Chan Medical School (UMCMS), fresh insights into the role of non-neuronal cells have emerged, implicating specific pathways of inflammation in the brain and spinal cord.

A consortium of investigators, including the UMCMS team, has conducted a study of nearly 30,000 ALS cases and more then 120,000 controls that identified multiple new genetic risk factors for ALS. The search for biomarkers that indicate ALS disease activity has also advanced across multiple centers this year.

At UMCMS, investigators funded by the Angel Fund, have developed a new type of physiological biomarker for motor neurons that we anticipate could have wide application

across many motor neurons diseases. And therapeutic advances have continued. In the last three years, the UMCMS team has done 3 first-in-human trials of biological therapies for familial ALS.

One, as recently reported, relied on extensive Angel Fund support to document that it is possible to suppress the C9orf72 gene in a human (see article below). An expanded trial to silence the C9orf72 gene is being planned.

More generally, the UMCMS clinical trials team has participated in multicenter trials. One study sponsored by the Cambridge company Amylyx showed benefit in slowing disease course and increasing survival in ALS; hopefully, this will be approved by the FDA for widespread use. And there are numerous exciting studies either in clinical trials or in the preclinical pipeline.

As we welcome 2022, we anticipate even more momentum in ALS investigations, confident that there will be sustained progress in the development of meaningful ALS therapies.

We are proud to say, that of funds expended, The Angel Fund donates 92% to ALS research.

Pilot human study shows novel treatment

suppresses mutant ALS gene

The Angel Fund for ALS Research provides major funding

A clinical trial funded by The Angel Fund for ALS Research has shown significant progress in suppressing expression of the most commonly mutated ALS gene, C9ORF72 (C9). The C9 gene mutation, a lengthy expansion of a repeated segment of six molecules of DNA, causes both familial ALS and frontotemporal dementia. The results of the human trial, initiated and led by Drs. Robert H. Brown and Jonathan Watts at UMass Chan Medical School, were published in Nature Medicine.

Welcome Dr. Daryl Bosco

Co-Chief Scientific Advisor

The Angel Fund for ALS Research welcomes Dr. Daryl Bosco as co-Chief Scientific Advisor. She will be joining Dr. Robert Brown in this position.

Dr. Bosco is a professor of Neurology at UMass Chan Medical School. She earned her Ph.D. (bio-organic chemistry) in 2003 from Brandeis University, where she used NMR spectroscopy to study enzyme dynamics,

Prior to joining the faculty at UMMS in 2008, Dr. Bosco was an Instructor of Neurology at Harvard Medical School working with Dr. Robert H. Brown, Jr. on various aspects of ALS.

Currently, the Bosco laboratory at UMass Chan Medical School is investigating the mechanisms underlying ALS using a multidisciplinary approach involving protein biochemistry, structural biology, human stem cell technology (to generate human motor neurons and human microglia) and ALS mouse models. The ultimate goal of this research is to understand the factors that trigger and perpetuate ALS, and to translate these findings into therapies for ALS.

2022 Events

April 9th - Annual Gala

April 18th - Boston Marathon

May 22nd - Harpoon 5 Miler

June 11th - Squirrel Run

June 18th - Sharon Timlin Memorial Race to Cure ALS

September 10th - Walk of Hope for ALS

September 26th - Annual Golf Tournament

October 8th & 9th - 100 Innings of Baseball

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