What is amyotrophic lateral sclerosis?

Learn about ALS and
what Biogen is doing to
help

What is amyotrophic lateral sclerosis?

Amyotrophic lateral sclerosis (ALS) is a rare, progressive (worsens over time), and fatal disease that causes loss of nerve cells (motor neurons). Motor neurons of the brain and spinal cord send messages to the muscles throughout the body. ALS affects the ability to move, speak, swallow, and breathe.

What are the different types of ALS?

Sporadic ALS:

The most common type, affecting around 90-95% of people with ALS. ‘Sporadic’ means ALS without a known family history. It can affect anyone and there is no clear cause.

Familial ALS:

A small percentage of patients (5-10%) have this form of ALS, which typically is inherited from a family member. ‘Familial’ simply means ALS with a family history.

Genetic ALS:

In some cases, genetic mutations are known to play a part in the development of both familial and sporadic ALS. These are also referred to as ‘genetic’ ALS. Recent scientific discoveries have identified more than 25 genes that have been linked to ALS. The most common are:

  • C9orf72
  • SOD1
  • TARDBP
  • FUS

What are some of the signs and symptoms of ALS?

Early symptoms can vary from person to person, making ALS difficult to diagnose. However, a common symptom is gradual muscle weakness.

Other early symptoms can include:

Our ALS research

At Biogen, we’re looking to develop treatments that could potentially slow the progression of ALS.

Our studies are focused on both genetic forms of ALS as well as ALS with no known genetic association. We are conducting trials in people who have been diagnosed with ALS as well as with people who have certain mutations associated with ALS, but do not currently display any signs or symptoms of disease.

What could a Biogen ALS trial involve?

Before you join a trial, we first confirm your eligibility with some health assessments. Then, if you’re eligible and choose to take part in an ALS trial, you’ll need to attend several appointments so that the trial team can closely monitor your health and condition with health assessments, such as:

Blood tests

These can tell us about many things, such as how your blood is clotting, whether you have any illnesses that we need to know about, to confirm you have a genetic mutation related to ALS, and to see how certain levels may be affected by the investigational drug.

Lumbar puncture

Lumbar punctures involve inserting a needle between the bones of the spine in your lower back, in order to take a small amount of cerebrospinal fluid (fluid surrounding the brain and spinal cord). The trial doctor will discuss any risks associated with this procedure with you, including, but not limited to: headache and back discomfort/pain.

Muscle tests

Various tests may be performed to check your muscle health (for example, by placing sensors on the skin), how your muscles are working (e.g, by passing an electrical current through them), and how strong they are (e.g., by pressing a device against your arms and legs to see if you can resist the pressure).

Other assessments will be involved. A full list of these, and all other requirements around taking part in a trial, would be fully discussed with you before you made a decision about whether to join a trial or not.

ALS trials are designed to take up as little of your time as possible, and there may be assistance available (e.g., reimbursement for travel costs) to help you get to and from the trial site to make it as easy as possible for you to participate.

And remember, if you join a clinical trial, there are many rules and regulations in place to protect your rights, safety, welfare, and personal data.

What is the role of a caregiver in an ALS clinical trial?

Someone who cares for you, for example a partner, sibling, other family member, close friend, or professional caregiver, may need to help you in order for you to take part in the trial. This may be to help you get to and from your appointments and support you throughout the trial. Or, they might be asked to help monitor any changes in your condition and report them to us. Visit our section on caregivers for more information about this role.

Why might many ALS trials include a placebo group?

When researching potential new treatments for diseases, trials must compare the investigational drug with something else in order to prove its effect. That’s why many ALS trials will have one or more groups of people taking the investigational drug and another group taking a placebo.

In some trials, participants – assigned to receive either the investigational drug or the placebo – may continue with their standard of care therapies

How can I get involved?

To find new treatments options for ALS, we must first test investigational drugs in clinical trials. But in the same way that diseases like ALS can impact groups of people in different ways, how medicine works in the body can also vary for different groups of people. That’s why it’s so important that a diverse range of people take part in clinical trials.

You can learn more about the importance of diversity in clinical trials, and Biogen’s efforts to make an impact, here.

Interested in participating in a trial?

If you’re interested in learning more, please use our trial finder to see if there may be a clinical trial that is right for you.

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Help and resources

There are organizations that offer information and support for people living with ALS and their families. Below are some groups where you can find out more about AD, connect with others, or learn about the latest research. Remember, this list of organizations does not imply an endorsement from Biogen or the listed organization.