The ALS Therapy Development Institute (ALS TDI) is a
non-profit
A nonprofit organization (NPO) or non-profit organisation, also known as a non-business entity, not-for-profit organization, or nonprofit institution, is a legal entity organized and operated for a collective, public or social benefit, in co ...
biotechnology
Biotechnology is the integration of natural sciences and engineering sciences in order to achieve the application of organisms, cells, parts thereof and molecular analogues for products and services. The term ''biotechnology'' was first used b ...
research organization focused on finding treatments for
amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND) or Lou Gehrig's disease, is a neurodegenerative disease that results in the progressive loss of motor neurons that control voluntary muscles. ALS is the most comm ...
(ALS). With a staff including more than 30 scientists, it operates a research and development program centered on ALS.
History
ALS TDI was founded as the ALS Therapy Development Foundation (ALS TDF) in 1999 by
James Heywood, Robert Bonazoli, and Melinda Marsh Heywood after James' brother,
Stephen Heywood
Stephen Heywood (April 13, 1969 – November 26, 2006) was an American builder and self-taught architect, specializing in the renovation of old houses.
He was diagnosed with ALS in 1998, at the age of 29. He was the subject of '' His Brother ...
, was diagnosed with the disease. Dr. Tennore Ramesh joined ALS-TDF when his sister in law was diagnosed with ALS and setup the research facility and served as Chief Scientific Officer from inception until 2003. The organization was initially funded through a donation from Stephen, as well as one from
Alex and Brit d'Arbeloff. The Foundation's first therapy concept was to replace
EAAT2
Excitatory amino acid transporter 2 (EAAT2) also known as solute carrier family 1 member 2 (SLC1A2) and glutamate transporter 1 (GLT-1) is a protein that in humans is encoded by the ''SLC1A2'' gene. Alternatively spliced transcript variants of thi ...
protein using gene therapy.
In 2004, the Foundation moved to a location in
Cambridge
Cambridge ( ) is a university city and the county town in Cambridgeshire, England. It is located on the River Cam approximately north of London. As of the 2021 United Kingdom census, the population of Cambridge was 145,700. Cambridge bec ...
, Massachusetts with an in-house lab. ALS TDF constructed a
biosafety level 2 lab in 2005, allowing for the expansion of "gene therapy and cell-based treatment pipelines."
In 2005, the Foundation started the Tri-State Trek, an annual 270-mile bike ride from
Boston, Massachusetts to
Greenwich, Connecticut
Greenwich (, ) is a New England town, town in southwestern Fairfield County, Connecticut, United States. At the 2020 United States Census, 2020 census, the town had a total population of 63,518. The largest town on Connecticut's Gold Coast (Conne ...
.
The Trek has since grown to include over 400 participants and has raised more than $7 million for research.
In 2006 the ALS patient
Augie Nieto became chairman of the board.
The next year, James Heywood resigned as president but joined the board and
Sean F. Scott, who ALS runs in his family, replaced him. Scott worked with
Augie Nieto as well as with the
Muscular Dystrophy Association
The Muscular Dystrophy Association (MDA) is an American 501(c)(3) umbrella organization that works to support people with neuromuscular diseases. Founded in 1950 by Paul Cohen, who lived with muscular dystrophy, it works to combat neuromuscular di ...
to bring together the two organizations in 2007. The collaboration allowed for MDA to match ALS TDI's annual budget for three years through Nieto's initiative,
Augie's Quest, though the partnership continued after that, with MDA cumulatively providing over $36 million. The same year, the organization replaced the "Foundation" part of its name with "Institute".
The Institute received a $1.1 million grant from the
U.S. Department of Defense
The United States Department of Defense (DoD, USDOD or DOD) is an executive branch department of the federal government charged with coordinating and supervising all agencies and functions of the government directly related to national secur ...
in 2008 and an additional $1.6 million grant in 2010.
Steven Perrin, previously only
chief scientific officer, was appointed CEO in 2009 following the death of Sean Scott. In 2011, the institute moved to a new facility, also in Cambridge, allowing for the hiring of more scientists and a bigger lab.
Two years later, in 2014, Augie's Quest officially transitioned from
MDA
MDA, mda, or ''variation'', may refer to:
Places
* Moldova, a country in Europe with the ISO 3166-1 country code MDA Politics
* Meghalaya Democratic Alliance (2018), ruling coalition government in the Indian State of Meghalaya led by National Pe ...
to ALS TDI. The same year, ALS TDI received over $3 million through the
Ice Bucket Challenge
The Ice Bucket Challenge, sometimes called the ALS Ice Bucket Challenge, is an activity involving the pouring of a bucket of ice water over a person's head, either by another person or self-administered, to promote awareness of the disease amyo ...
.
In 2016, the Institute announced the ALS ONE partnership with
Massachusetts General Hospital
Massachusetts General Hospital (Mass General or MGH) is the original and largest teaching hospital of Harvard Medical School located in the West End neighborhood of Boston, Massachusetts. It is the third oldest general hospital in the United Stat ...
,
Harvard Medical School
Harvard Medical School (HMS) is the graduate medical school of Harvard University and is located in the Longwood Medical Area of Boston, Massachusetts. Founded in 1782, HMS is one of the oldest medical schools in the United States and is consi ...
,
University of Massachusetts Medical School
The University of Massachusetts Chan Medical School is a public medical school in Worcester, Massachusetts. It is part of the University of Massachusetts system. It is home to three schools: the T.H. Chan School of Medicine, the Morningside Grad ...
, and Compassionate Care ALS to find a treatment for ALS within four years.
In 2018, ALS TDI was the original beneficiary of the ALS Pepper Challenge, where participants eat a
chili pepper
Chili peppers (also chile, chile pepper, chilli pepper, or chilli), from Nahuatl '' chīlli'' (), are varieties of the berry-fruit of plants from the genus ''Capsicum'', which are members of the nightshade family Solanaceae, cultivated for ...
. Various public figures, including
Kelly Clarkson
Kelly Brianne Clarkson (born April 24, 1982) is an American singer, songwriter, author, and television personality. She rose to fame after winning the first season of ''American Idol'' in 2002, which earned her a record deal with RCA. Her debu ...
,
Jimmy Kimmel
James Christian Kimmel (born November 13, 1967) is an American television host, comedian, writer, and producer. He is the host and executive producer of ''Jimmy Kimmel Live!'', a late-night talk show that premiered on ABC on January 26, 2003, ...
,
Shaquille O'Neal
Shaquille Rashaun O'Neal ( ; born March 6, 1972), known commonly as "Shaq" ( ), is an American former professional basketball player who is a sports analyst on the television program ''Inside the NBA''. O'Neal is regarded as one of the greates ...
,
Nancy O'Dell
Nancy O'Dell (born Nancy Evelyn Humphries; 1966) is an American television host and entertainment journalist. She served as co-anchor of the syndicated entertainment news show ''Entertainment Tonight'' from January 3, 2011, to August 2, 2019.
E ...
,
Wolf Blitzer
Wolf Isaac Blitzer (born March 22, 1948) is an American journalist, television news anchor, and author who has been a CNN reporter since 1990, and who currently serves as one of the principal anchors at the network. He is the host of ''The Situat ...
, the
Miami Heat
The Miami Heat are an American professional basketball team based in Miami. The Heat compete in the National Basketball Association (NBA) as a member of the league's Eastern Conference (NBA), Eastern Conference Southeast Division (NBA), Southe ...
and
Andy Cohen
Andrew Joseph Cohen (born June 2, 1968) is an American radio and television talk show host, producer, and writer.
Cohen is the host and executive producer of Bravo's late night talk show, '' Watch What Happens Live!'' He also has a pop culture ...
have participated in the challenge.
In February 2021, the ALS Therapy Development Institute moved to Watertown, MA.
In April 2021, Fernando Vieira, M.D., was appointed the chief executive officer of the ALS Therapy Development Institute by the board of directors.
Research
The institute has raised and spent more than $100 million on research into effective treatments for ALS and practices
open-source science. After the discovery that the
multiple sclerosis
Multiple (cerebral) sclerosis (MS), also known as encephalomyelitis disseminata or disseminated sclerosis, is the most common demyelinating disease, in which the insulating covers of nerve cells in the brain and spinal cord are damaged. This d ...
drug
Gilenya
Fingolimod, sold under the brand name Gilenya, is an immunomodulating medication, mostly used for treating multiple sclerosis (MS). Fingolimod is a sphingosine-1-phosphate receptor modulator, which sequesters lymphocytes in lymph nodes, preven ...
might also be a treatment for ALS, the Institute enrolled 30 people in a Phase 2A clinical trial the drug in 2013, though it did not progress further.
ALS TDI launched the Precision Medicine Program in partnership with Denali Therapeutics in 2013 "to identify subgroups of ALS, potential treatments for them using patient data,
genomics
Genomics is an interdisciplinary field of biology focusing on the structure, function, evolution, mapping, and editing of genomes. A genome is an organism's complete set of DNA, including all of its genes as well as its hierarchical, three-dim ...
and
iPS cell
Induced pluripotent stem cells (also known as iPS cells or iPSCs) are a type of pluripotent stem cell that can be generated directly from a somatic cell. The iPSC technology was pioneered by Shinya Yamanaka's lab in Kyoto, Japan, who showed ...
technology". By 2015, over 300 people had been registered and pre-screened in the program, significantly funded by money raised in the
Ice Bucket Challenge
The Ice Bucket Challenge, sometimes called the ALS Ice Bucket Challenge, is an activity involving the pouring of a bucket of ice water over a person's head, either by another person or self-administered, to promote awareness of the disease amyo ...
.
In 2018, the Institute entered Phase I clinical trials for AT-1501, a potential treatment for ALS and
Alzheimer's
Alzheimer's disease (AD) is a neurodegenerative disease that usually starts slowly and progressively worsens. It is the cause of 60–70% of cases of dementia. The most common early symptom is difficulty in remembering recent events. As t ...
that blocks the activation of certain immune cells in order to protect nerves from ALS. The development of the drug allows ALS TDI to be viewed as a successful drug development organization. ALS TDI received funding from the ALS ONE partnership to develop the drug.
In 2020 the Institute Published "Type I PRMT Inhibition Protects Against C9ORF72 Arginine-Rich Dipeptide Repeat Toxicity" in
Frontiers in Pharmacology' with a follow-up paper in 2021 called "Hypothesis and Theory: Roles of Arginine Methylation in C9orf72-Mediated ALS and FTD" in
Frontiers in Cellular Neuroscience'.
See also
*
PatientsLikeMe
PatientsLikeMe is the world’s largest integrated community, health management, and real-world data platform. Through PatientsLikeMe, a growing community of more than 830,000 people with over 2,900 conditions share personal stories and informat ...
References
External links
*
{{Authority control
Research institutes established in 1999
Medical and health organizations based in Massachusetts
Biotechnology companies of the United States
Organizations based in Cambridge, Massachusetts
Amyotrophic lateral sclerosis
1999 establishments in Massachusetts
1999 in biotechnology