The ALS Therapy Development Institute (ALS TDI)
Author: Thomas C. Weiss : Contact: Disabled World
Published: 2015-07-20 : (Rev. 2017-06-25)
Information regarding The ALS Therapy Development Institute (ALS TDI), a non-profit biotech that focuses on ALS research.
The ALS Therapy Development Institute (ALS TDI) and its scientists actively discover and develop types of treatment for Amyotrophic Lateral Sclerosis (ALS). The organization is the world's first and largest nonprofit biotech that focuses on ALS research. ALS TDI is led by people who experience ALS themselves, as well as their family members. The organization understands the urgent need to slow and stop this form of disease.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease and Charcot disease, is a specific disorder that involves the death of neurons. The disease belongs to a group of disorders known as motor neuron diseases, which are characterized by the gradual degeneration and death of motor neurons. ALS is characterized by stiff muscles, muscle twitching, and gradually worsening weakness due to muscle wasting. This results in difficulty speaking, swallowing, and eventually breathing.
ALS TDI's unique and comprehensive approach leverages more than fifteen years of treatment-focused ALS research while applying it on an industrial scale in order to receive therapeutics to today's people with ALS as efficiently, quickly and cost-effectively as possible. The translational research environment brings together more than thirty full-time scientists whose expertise includes:
- Molecular biology
- Assay development
- Protein biochemistry
- Computational biology
Dr. Steve Perrin, Chief Scientific Officer and CEO of ALS TDI often talks about the organization's research plan to people, their family members, academics and industry executives around the world.
The ALS TDI Drug Pipeline
ALS is a complex form of disease with several different parts of a person's body and biology having a role in either its onset or its progression. ALS TDI believes that this requires them to explore a vast pipeline of possible treatments at the same time in their laboratories. The pipeline provides a glimpse into the current possible treatments they are working on, why ALS TDI is examining them, as well as where they are in the process.
"We have met the people who work at ALS TDI and their passion shines through, their personal connections to this disease drive them. We believe they are one of the most progressive and effective institutes around, and they will be part of the cure."- Jeremy Truman
Every hour and a half, someone is diagnosed with Amyotrophic Lateral Sclerosis (ALS). ALS is a form of progressive neurodegenerative disease that causes paralysis, muscle weakness and in the end - respiratory failure. ALS is also known as Charcot's disease, Lou Gehrig's disease and Motor Neuron Disease. ALS attacks certain cells in a person's spinal cord and brain which are needed in order to keep the person's muscles moving. Early signs and symptoms of ALS may include the following:
- Muscle cramps
- Muscle twitching
- Difficult with speaking
- Difficulty with swallowing
- Weakness in the feet, ankles, legs or hands
The senses, to include sight, hearing, taste, smell and touch, are not affected by ALS. There is no single diagnostic test for ALS; however, experts in the disease usually neurologists who specialize in neuromuscular diseases, are highly capable of diagnosing ALS. In some instances, they may order additional tests if the diagnosis is not clear. Additional tests for ALS may include the following:
- A spinal tap
- Genetic testing
- A muscle biopsy
- Urine and blood testing
- Magnetic resonance imaging (MRI)
- Electromyography and nerve conduction
The majority of people with ALS live two to five years after their first signs of the disease. Around 10% of people with ALS survive at least ten years. The variable rate of disease progression makes achieving a prognosis hard to predict and therapies challenging to develop. At this time there is only one medicine for specifically treating ALS, 'Riluzole.' The medication extends a person's survival by a mere two to three months. The urgent and unmet medical need for effective treatments for this fatal and devastating form of disease is the basis for the research and drug development pursued at ALS TDI.
ALS TDI's Precision Medicine Program
ALS TDI's Precision Medicine Program is the world's first fully implemented effort to identify subgroups of ALS and possible forms of treatment for those affected by using people's data, genomic and iPS cell technology. The 100th person was enrolled in the Precision Medicine Program as of April, 2015. ALS TDI is funded to enroll up to 300 people. The organization's program is unique in several ways and is the only one of its kind currently operating in ALS.
ALS TDI provides all data produced back to the individual person through a secure, online portal which is accessible after the person's enrollment. Their program combines individual disease progression measured with accelerometers, family medical history, speech recordings and, 'ALSFRS-R,' scores as well as full genome sequencing and the creation of iPS cells from each person enrolled. Well-characterized representative cell lines of subgroups identified may then be differentiated into relevant cell types such as motor neurons and later used in high-content drug screening with the goal of identifying effective forms of treatments for ALS.
ALS Therapy Development Institute (ALSTDI) - www.alstdi.org
- One or two of 100,000 people develop ALS each year.
- People of all races and ethnic backgrounds are affected.
- In Europe, ALS affects about 2.2 people per 100,000 per year.
- In the United States, more than 5,600 are diagnosed every year, and up to 30,000 Americans are currently affected. ALS is responsible for two deaths per 100,000 people per year. ALS cases are estimated at 1.2 to 4.0 per 100,000 individuals in Caucasian populations with a lower rate in other ethnic populations.
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