"Biopreparations" refers to any type of therapeutic therapy that is obtained using living organisms such as humans, animals or microorganisms. This is in contrast to traditional non-biological pharmaceuticals, which are synthesized in the laboratory by chemical processes without the use of parts of living things. Other terms that are also sometimes used include "biological therapy", "biological therapy", "biological drugs" and "biopharmaceuticals". You may also hear them referred to as over-the-counter names or as a specific sub-category of biological treatments (such as gene therapy).
The oldest forms of biologics have been around for many years, such as vaccines developed in the 19th century . Insulin was another relatively early biological treatment. However, since the 1990s, the number of biologics on the market has increased significantly. Many biological therapies are currently available to treat a wide variety of different diseases, such as various forms of cancer and autoimmune diseases. More are being developed and made available each year. All of these biological treatments have different potential benefits and risks .
What types of biological therapies are available?
Some of the common classes of biologics include :
- Blood or other blood products (such as platelets)
- Steroid hormones (eg, estrogen, testosterone)
- Vaccines (for example, for disease prevention)
- Antitoxins (for example, to treat a snake bite)
- Recombinant proteins (such as insulin or erythropoietin)
- Recombinant nucleic acids (eg, designed to treat genetic hypercholesterolemia)
- Interleukins (immune molecules that can be used to treat certain infections and cancers)
- Tendons, ligaments, or other materials used for transplantation.
- Monoclonal antibodies (such as those used to treat autoimmune diseases and cancer)
- Stem cell therapy (for example, for certain cancers or genetic diseases)
- Other cell therapies (such as specific T cells used to treat cancer)
- Gene therapy (for example, to treat genetic diseases)
How do biologics work?
Different biological treatments have different goals, objectives, and designs, and they all work in different ways. Feel free to ask your healthcare provider if you have questions about a specific biological therapy that is relevant to you.
For example, tocilizumab (brand name Actemra), a biologic drug used to treat rheumatoid arthritis, belongs to a class of biologic drugs called monoclonal antibodies. Technically, tocilizumab is a recombinant human IL-6 receptor IgG1 antibody. Unlike the antibodies produced naturally by your body, this type of antibody does not fight infection. Instead, it can be used to treat conditions like rheumatoid arthritis.
IL-6 is a cytokine (immune signaling molecule). It can join cells and send signals. When IL-6 binds, it tends to exacerbate inflammation and other processes that worsen rheumatoid arthritis. Tocilizumab partially blocks the IL-6 receptor, which can reduce symptoms.
These specific monoclonal antibodies are produced in several steps, beginning with the early production of antibodies in mice. Scientists then modify these antibodies by replacing most of them with portions of human antibodies. Inside the laboratory, many identical copies of these new antibodies are made. These hybrid antibodies can be administered to the patient to help block the body's response to IL-6.
If the letter "ab" appears at the end of the name of a biological product, this is a good indication that the product is a modified type of antibody.
How is biological therapy different from traditional pharmaceuticals?
In general, the manufacturing process for biologicals is more complex than the manufacturing process for low molecular weight medicines ('non-biological' medicines such as aspirin). This is one of the reasons why biological products are generally more expensive than non-biological alternatives. Because the manufacturing process is so complex, the structure of biological products cannot be fully understood. It may be difficult or impossible for another company to reproduce this accurately.
Biologics are larger and more complex molecules than traditional pharmaceuticals. Unlike traditional pharmaceuticals, their production requires some kind of component from a living organism.
In general, biological treatments are more specific than non-biological treatments. For example, methotrexate and sulfasalazine are two non-biological drugs for the treatment of rheumatoid arthritis. These drugs affect several different parts of the human immune system. In contrast, biologic treatments for rheumatoid arthritis have very specific goals (eg, blocking a specific receptor on a target immune molecule). This reduces the likelihood of some side effects, although others still pose potential risks .
Biologics are more sensitive to heat and light. They often cannot be taken orally and must be given by injection or infusion.
Conditions that can be treated with biological therapy.
Recently developed biological therapies have revolutionized the treatment of many different diseases. They were especially important for treating autoimmune diseases, cancer, and certain genetic conditions .
For example, biological therapies have been developed to treat the following conditions:
- Rheumatoid arthritis
- Ankylosing spondyloarthritis
- Crohn's disease
- Multiple sclerosis
- Diabetic retinopathy
- Macular degeneration associated with age
- Stomach cancer
- Breast cancer
- Colon cancer
- Forms of leukemia and lymphoma
- Cystic fibrosis
- Sickle cell anemia
Biologics for autoimmune diseases
Some of the most widely used biologics are used for autoimmune diseases, diseases in which the body's immune system plays a role in an abnormal attack on its own tissues. These include conditions like rheumatoid arthritis, psoriasis, Crohn's disease, and others. Many of these treatments are approved by the FDA to treat more than one type of autoimmune disease. In some cases, healthcare professionals may prescribe these off-label treatments if they have not completed the full set of studies required for FDA approval, but there is still good reason to believe that they may be effective.
Because biologics are often expensive and more difficult to administer, they are often (but not always) prescribed after other non-biologic therapy has been tried.
One of the most common types of modern biologic treatments for autoimmune diseases is a TNF blocker . TNF blockers include the popular drugs etanercept (Enbrel), adalimumab (Humira), and infliximab (Remicade). All of these drugs block the inflammatory effects of an immune molecule called TNF-alpha. They are approved by the FDA for the treatment of several different autoimmune diseases .
Other biologics have been developed to block the receptors for various immune molecules. Others have been designed to target T cells, specific cells of the immune system. Some of these other important biologics in autoimmune diseases include:
- Ustekinumab (Stelara)
- Secukinumab (Cosentix)
- Abatacept (ORENCIA)
- Guselkumab (Tremfya)
Another important biological agent for autoimmune diseases is interferon beta-1a (Avonex), which is a key treatment for multiple sclerosis .
Biologics in the treatment of cancer
Biological therapies are also very important in the treatment of cancer and many of them continue to evolve. There are many different types of these treatments. They are sometimes used as a first-line treatment. In other cases, they are used after other treatments have failed or for advanced cancers. They are often used in addition to other treatments.
Some of these treatments are therapeutic antibodies. For example, the drug pembrolizumab (Keytruda) is a biologically engineered antibody approved for the treatment of many different types of solid tumors. It works by targeting a specific receptor found on specific immune cells. This makes it easier for immune cells to kill cancer cells. These biological therapies, which stimulate the body's immune system to better fight cancer, are called immunotherapy .
Other types of therapeutic antibodies, such as trastuzumab (herceptin), interfere with the signaling pathways that promote tumor growth. Or they can cause the destruction of cancer cells, such as rituximab (Rituxan). In other cases, they may be associated with a toxic substance that can help kill cancer cells. For example, the drug ado-trastuzumab emtansine (Kadsila) works in the same way. There are also other types of therapeutic antibodies.
Immunocellular therapy is another important area of emerging cancer biologic therapy. This involves harvesting some of the human immune cells, altering them in some way, and then reinjecting them. This allows the person's immune cells to better attack the tumor. Both tumor infiltrating lymphocyte therapy and CAR-T cell therapy fall into this category .
Another important class of biologics are proteins made in the laboratory. For example, several different immune molecules (different types of interferons and interleukins) are used in various types of cancer.
Biologics for rare genetic diseases
Biological therapy is also very important in the treatment of rare genetic diseases. This is likely to become even more important in the future as more and more gene therapies become available. For example, some biological treatments for rare diseases include enzyme replacement therapy for Gaucher disease, clotting factors for hemophilia, or immunoglobulins for people with certain genetic immune disorders.
Stem cell transplantation, which is used to treat many types of rare genetic diseases, including sickle cell anemia , is also a form of biological therapy. Researchers also continue to develop RNA therapies and gene therapies that could ultimately be used to treat many rare diseases .
Possible side effects.
Possible side effects of biologics vary depending on the specific biologic therapy. In some cases, these side effects are quite mild, like a rash. Some other common side effects can include respiratory infections, flu-like reactions, or redness at the injection site.
However, more serious side effects such as a severe allergic reaction are possible. There are some specific potential side effects of biological therapies that target different parts of the immune system. In particular, many of these treatments are associated with the risk of immunosuppression. This means that part of your immune system cannot fight infections as usual. This can make you more susceptible to certain types of infections. In some cases, they may be at risk of reactivating latent infections that might not otherwise necessarily cause you problems, such as tuberculosis.
Certain biological therapies that target the immune system can also increase the risk of certain types of cancer. However, this does not apply to all biological treatments. Also, the risk may be negligible or absent with a drug that would otherwise offer many potential benefits. Discuss this with your doctor to make sure you make the best decision for you. In general, risks are better understood when using biological therapies that have been around for some time, compared to newer treatments. Your healthcare professional can give you a better idea of the possible side effects of a particular biological therapy in your situation.
Are biological products safe during pregnancy and breastfeeding?
Most biological therapies have not been studied in pregnant or lactating women, but we do know that specific biological therapies can be dangerous to the fetus or nursing baby. However, it can also be associated with the risk of stopping biological treatment if you are already using it. Talk to your doctor about your specific situation and the general risks and benefits. If you are taking biological therapy and discover that you are pregnant, do not stop taking it immediately. Instead, call your doctor's office and tell him about the situation.
As always, your healthcare provider will want to obtain a complete medical history and clinical examination before prescribing biological therapy for you. This will help your doctor make sure that the possible benefits of the treatment outweigh the possible risks. In some cases, your healthcare provider will need to make sure you don't have certain risk factors before taking a biologic. People with certain medical conditions may not receive biological treatment. Or you may need a TB test or a hepatitis test. But this is not necessary for all biological therapies. Your healthcare provider will tell you which screening tests can help.
Generally speaking, you should not give certain types of vaccines (those that contain some live viral component) while taking biologics that affect your immune system and may increase the chances of infection. Therefore, you may need to receive some of these vaccines before starting therapy.
Can they be taken biological with other non-biological treatments?
Yes, generally. Biological treatments are often used in conjunction with older non-biological treatments. For example, a person with rheumatoid arthritis can continue to take methotrexate by adding additional biologic treatments. In other cases, biological treatments will replace previous non-biological treatments. This will depend on your specific situation.
How are biologics administered?
It depends on the specific biological product. Currently, most biologics cannot be taken orally, although pharmaceutical companies are working to develop oral medications.
Biologics are typically administered by injection or infusion. It may be injected under the skin, or you may need help from a family member.
Biological treatments are sensitive to heat and light, so carefully follow the preparation instructions given to you by your doctor.
Intravenous infusions are given through a vein. This usually takes longer, maybe a couple of hours. They are usually done in a doctor's office.
In some cases, only one treatment is required. In other cases, it will be necessary to administer biological treatment at regular intervals.
How fast do biological products work?
How quickly a biological agent works depends on the specific therapy. For example, an insulin injection takes effect almost immediately. But you may not notice something like biological therapy for rheumatoid arthritis or psoriasis for a couple of weeks or even a month or more. Ask your healthcare provider what to expect in your specific situation.
What are biosimilars?
Due to the way biological products are manufactured, it is difficult for competing companies to produce products that are exactly equivalent to newly developed biological products. Unlike older drugs based on chemical compounds, most biologics do not have strictly defined analogues . The FDA defines generics as those with the same active ingredient as the brand name drug. In addition, they are also bioequivalent, that is, they have the same pharmaceutical form, concentration, quality and efficacy.
Instead, biologics have so-called "biosimilars" that were defined by law in 2009. These treatments have been approved by the FDA because they are not clinically relevant to the original brand name drug. They must function as the reference product and be equally safe and effective. Biosimilars are often cheaper than the original product, but pharmaceutical companies have lobbied against their use, claiming there is no evidence of safety or equivalence.
If there is a biosimilar medicine available, be sure to talk to your doctor about whether it is the best option for you. It also makes sense to ensure that the FDA has identified the biosimilar as interchangeable with the original product.