Drugs and Interventions

Here we provide information on some of the treatments that have been given to patients in clinical trials. The information on this webpage provides an overview of the different drugs but is not a substitute for medical advice.

CancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More Research UK also have a useful list of different drugs and how they work. It can be accessed here.

ATR Inhibitors

Ataxia-telangectasia and Rad3 related (ATR) kinase is a proteinFound in cells and needed for the development, growth and function of our bodies. More involved in repairing DNAThe molecule that contains the genetic information that makes you the person you are. More and cell division. Some cancers rely on ATR to help them survive. ATR inhibitorsA drug that blocks or reduces the function of a particular protein. More block ATR and kill cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells.

ATR inhibitorsA drug that blocks or reduces the function of a particular protein. More are currently being tested in clinical trials and your trial team will be able to discuss the possible side effects of the drugs.

Drug examples:

Berzosertib (M6620), Elimusertib, Ceralasertib.

Bisphosphonates

Bisphosphonates are drugs that keep bones strong. Bone growth is regulated by cells called osteoclasts and osteoblasts, which work together to keep bones healthy. Osteoclasts remove old bone, while osteoblasts make new bone. Bisphosphonates prevent osteoclasts from removing old bone and therefore strengthen them.

Bisphosphonates can be given as a tablet or an injection into a vein. Side effects are usually mild and include flu like symptoms. Each bisphosphonate will also have its own specific side effects that your doctor will be able to explain.

Bisphosphonates are best known for their use in osteoporosis, a condition that causes weakening of bones. They are also used to control bone symptoms related to cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More and are now being investigated as treatments for cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More.

Drug examples:

Zoledronic acid (Zometa)

CAR T-Cell Therapy

CAR T-cell therapy is a type of personalised treatment that uses the body’s own immune systemA system in the body that fights infection. More to fight cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. As well as fighting infection, the immune systemA system in the body that fights infection. More is programmed to kill any abnormal cells such as cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. Many cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells learn to evade this. In CAR T-cell therapy, T-cells (a type of immune cell) are removed from the body and modified in a laboratory to recognise a certain marker expressed on a cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cell. The T-cells are then returned to the body with the ability to recognise and kill cells that express the marker. In this case, the CAR-T cell will target the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells.

CAR T-cell therapy is a very intensive treatment. Part of the regime includes chemotherapy, which is given to prepare the body for the CAR-T cells. Patients must be closely monitored for side effects including over-activation of the immune systemA system in the body that fights infection. More (cytokine release syndrome). Despite these side effects, CAR T-cell therapy has been approved for use in some blood cancers and is now being investigated in solid cancers.

Doctors can provide further information on the risks and benefits of CAR T-cells.

Drug examples:

B7-H3-CAR T cells, CD276 CAR T cells, EGFR806 CAR T Cells, GD2 CAR T cells, HER2 CAR T cells, iC9.GD2.CAR.IL-15 T-cells

Checkpoint Inhibitors

Checkpoint inhibitorsA drug that blocks or reduces the function of a particular protein. More (CI) activate the body’s immune systemA system in the body that fights infection. More to kill cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More. They achieve this by targeting T cells, a type of immune cell. CIs are part of a wider group of drugs that affect the immune systemA system in the body that fights infection. More known as immunotherapies.

T cells have markers on them which turn them on and off. These markers are known as checkpoint proteins and they are activated when they recognise markers on the surfaces of other cells. This ensures that the T cells are activated when needed, for example in the presence of an infection or cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells, and turned off when they are not. CancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells often express high levels of a marker which can turn off T cells preventing the immune systemA system in the body that fights infection. More from attacking the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells.

CIs block the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cell markers from turning off T cells. This turns the immune systemA system in the body that fights infection. More back on and it will attack the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells.

CIs are usually given as a drip directly into a vein. These drugs cause increased activation of the immune systemA system in the body that fights infection. More and can cause a range of side effects including tiredness, feeling sick, loss of appetite and diarrhoea. Doctors will discuss all possible side effects of the treatment,

CIs are approved to treat several cancers including skin and lung cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More. They are also being tested in clinical trials.

Drug examples:

Atezolizumab, Avelumab, Camrelizumab, Cemiplimab, Dostarlimab, Durvalumab, Nivolumab, Pembrolizumab (MK3475), sintilimab, Socazolimab (ZKAB001), Tremelimumab

Chemotherapy

Chemotherapy is a type of medication that kills cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. It acts throughout the whole body so it can target both the primary cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More site and any areas that it has spread to. There are many types of chemotherapy drugs, but they all act in a similar way by targeting cells that are dividing quickly, which is a key feature of cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells.

Chemotherapy is usually given as a course of several cycles over a few months. Between each cycle (dose of chemotherapy), there will be a rest period of a few weeks, giving time for the body to recover. Chemotherapy drugs can be given on their own or with other drugs. They can also be given in different ways including as a tablet or a drip directly into a vein. The course length, drug combination and delivery method of the drug are dependent on the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More type and stageA way to characterize the size and spread of cancer and help to guide treatment. More and will be decided by the medical team.

Chemotherapy can cause side effects. It is not possible to predict how a person will react to the treatment but doctors will monitor closely and provide support if needed. Side effects can include infection, hair loss, tiredness, nausea and vomiting, constipation, and diarrhoea. Doctors will discuss all the possible side effects before treatment starts.

Chemotherapy is the main treatment for osteosarcoma. The exact treatment regime varies between countries.

Drug examples:

Carboplatin, Cisplatin, Cyclophosphamide, Cytoxan, Dacarbazine, Decitabine, Docetaxel, Doxorubicin, Epirubicin, Eribulin mesylate, Etoposide, Fludarabine, Gemcitabine, Hydroxychloroquine, Ifosfamide, Irinotecan (MM-398), Ixabepilone, Lomustine, Melphalan, methotrexate, nab-paclitaxel, onivyde, oxaliplatin, paclitaxel, palifosfamide- tris, Pemetrexed, Tanespimycin, Temozolomide, Thiotepa, Topotecan, Trabectedin, Trimetrexate Glucuronate, Vincristine, Vinorelbine

Folinic Acid

Folinic acid (The active part of folic acid) has a number of uses in cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More treatment. It is sometimes given alongside methotrexate to reduce the risk of side effects. It is also given alongside the drug fluorouracil in the treatment of cancers of the digestive system.

Folinic acid can be given as an injection or a drip into a vein. It can also be given as a tablet.

Folinic acid doesn’t often cause side effects but some people will get a fever. Your doctor will discuss all possible side effects with you.

Drug examples:

Calcium Folinate, Leucovorin

G-CSF/GM-CSF

G-CSF (granulocyte-colony stimulating factor) and GM-CSF (Granulocyte-macrophage colony-stimulating factor) are a type of growth factor that increases the number of white (immune) cells in the body. They are often given to reduce the risk of infection while having chemotherapy. As it boosts the immune systemA system in the body that fights infection. More it also has some antitumor activity.

They are usually given as an injection under the skin. Common side effects include bone pain, headaches, bruising, feeling sick and fever. It can also cause other side effects including dizziness and build up of fluid in the body. Your doctor will discuss side effects with you before treatment.

Drug examples:

Filgrastim, Pegfilgrastin, Sargramostim

Haematopoietic stem cell transplantation (HSCT)

Haematopoietic stem cell transplantation (HSCT) is a procedure that replaces unhealthy blood cells with healthy ones. Before a person has the transplant their blood cells will be destroyed with chemotherapy drugs. They are then replaced with non-specialised cells, called stem cells, taken from bone marrow or the blood. Stem cell transplants are associated with a number of risks so your doctor will discuss them in detail before starting treatment.

Histone Deacetylase Inhibitor

Histone Deacetylases are enzymes which turn genes on and off. They are usually involved in cell growth and division which are key features of cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. Histone Deacetylases inhibitorsA drug that blocks or reduces the function of a particular protein. More (HDIs) stop their activity and prevent cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More growth.

HDIs are usually given as tablet. Side effects include tiredness, loss of appetite, diarrhoea. Each HDI will also have its own specific side effects that your doctor will be able to explain before you start on one.

Currently, the HDI Panobinostat is used alongside other drugs to treat myeloma (a type of blood cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More). They are also being tested in clinical trials.

Drug examples:

Depsipeptide (Romidepsin), Entinostat Panobinostat, Vorinostat

Metformin

Metformin is a medication used to prevent and control diabetes. It lowers blood sugars by helping the body respond to insulin (the hormone that regulates blood sugars).

It is now thought that metformin may also have anti-cancer properties and it is being used in clinical trials for some cancers.

Metformin is usually given as a table. Side effects can include feeling sick, loss of appetite, stomach pains and diarrhoea. Your doctor will discuss the side effects before starting treatment.

Mifamurtide

Mifamurtide (also known by the brand name Mepact) is a type of treatment that activates the body’s own immune systemA system in the body that fights infection. More to kill cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. Mifamurtide increases the number of two types of cells in the body called monocytes and macrophages.

Mifamurtide is given as a drip directly into a vein. General side effects include tiredness, diarrhoea, and feeling sick. It can also cause your red cells to drop leading to feeling tired and looking pale. Your doctor will discuss the full list of side effects with you before you agree to the treatment.

Mifamurtide has been approved to treat osteosarcoma in some countries. Research suggests that it may reduce the risk of osteosarcoma coming back if given alongside chemotherapy. Find out more here.

Monoclonal Antibodies and Antibody-Drug Conjugates

Antibodies are part of the immune systemA system in the body that fights infection. More and are found naturally in the body. They recognise markers on the surface of foreign cells and bind to them. Monoclonal antibodies (MABs) work in a similar way to naturally occurring antibodies however they have a more targeted approach. Antibodies are modified in the lab to target one specific cell marker. For example a marker on cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. Once in the body, they will bind to cells expressing that marker and lead to its destruction. Researchers are able to identify what markers are expressed on cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells and modify MABs to target them.

Some MABs are combined with other drugs. These are called antibody-drug conjugates (ADCs). They behave similarly to standard MABs but when they bind to cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells they also deliver a drug that helps to kill them.

MABs/ADCs are usually given as a drip directly into a vein. General side effects include tiredness, diarrhoea, and flu-like symptoms. However, each type of MAB has its own specific side effects which your doctors will discuss before you agree to treatment.

MABs/ ADCs are approved for use in a range of different cancers. They are also being tested in clinical trials.

MAB Drug examples:

A1G4, ALMB-0168, Atezoluzumab, Bevacizumab, Cixutumumab, Dinutuximab, Denosumab, Enoblituzumab, HGS-ETR2, hu14.18K322A, Humanized 3F8 bispecific antibodyPart of the immune system which recognises things that do not belong in the body and sticks to them including cancer cells. More, IBI-188, I-3F8, Ipilimumab, Magrolimab, Nivolumab, Oleclumab, PEN-866, R1507, Robatumumab, Trastuzumab deruxtecan.

ADC Drug examples:

CAB-AXL-ADC, Glembatumumab Vedotin, hu14.18-IL2, Trastuzumab deruxtecan,

mTOR Inhibitors

mTOR is an enzymeMolecules in the body that speed up chemical reactions. More that leads to cell growth. In cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells, mTOR may be permanently active resulting in cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More growth. mTOR inhibitorsA drug that blocks or reduces the function of a particular protein. More block its activity and stop cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells from growing and dividing.

mTOR inhibitorsA drug that blocks or reduces the function of a particular protein. More are usually given as tablets. They can also be given as an injection or a drip through a vein. Side effects include tiredness, loss of appetite and diarrhoea. Each mTOR inhibitor will also have its own specific side effects that your doctor will be able to explain before starting treatment.

mTOR inhibitorsA drug that blocks or reduces the function of a particular protein. More have been approved for use in several cancers including breast cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More, lung cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More and kidney cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More. They are currently being tested in clinical trials for several cancers.

Drug examples:

Everolimus, Ridaforolimus, Sirolimus, Temsirolimus

Oncolytic Virus Therapy

Oncolytic virus (OV) therapy is a type of cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More treatment which uses the body’s own immune systemA system in the body that fights infection. More to kill cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. When viruses enter the body they infect cells, hijack their mechanisms and either kill, damage or change them. This is what makes you feel unwell. OVs are viruses that are modified in a laboratory to target cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. Therefore, when they enter the body, they will hijack cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells and kill them. Additionally, as the body thinks that you have an infection it will lead to an immune response that will also help to kill cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells.

OV treatments are given as an injection directly into the tumourA mass of cells that are growing where they shouldn’t be. Tumours can be benign or malignant. Benign tumours do not spread to other parts of the body whereas malignant tumours are cancerous and have the potential to spread. More or into a vein. Side effects will vary between individuals but in general they include flu-like symptoms, headaches, and nausea.

The OV talimogene laherparepvec (T-VEC) has been approved for use in melanoma (a type of skin cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More) that cannot be surgically removed. OVs are also being tested in clinical trials for a range of cancers.

Drug examples:

HSV-1 (G207), HSV1716, JX-594, Reolysin

PARP Inhibitors

PARP is an enzymeMolecules in the body that speed up chemical reactions. More in the body that helps cells to repair themselves. If cells can’t repair themselves, they will die. Most of the time we don’t want this to happen but in cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells, the repair mechanism actually keeps the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells alive. PARP inhibitorsA drug that blocks or reduces the function of a particular protein. More prevent PARP from repairing cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells, causing the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells to die.

Our cells have multiple different repair systems. PARP inhibitorsA drug that blocks or reduces the function of a particular protein. More are particularly effective in cancers where other repair systems are already damaged, making it even easier to kill the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. For example, in cancers where there are changes in a gene called BRCA.

PARP inhibitorsA drug that blocks or reduces the function of a particular protein. More are usually given as a capsule or tablet to be swallowed. General side effects include tiredness, diarrhoea, and feeling sick. Your doctor will discuss the full list of side effects with you before you agree to the treatment.

PARP inhibitorsA drug that blocks or reduces the function of a particular protein. More are currently approved for use in breast, ovarian, pancreatic and prostate cancers. They are being tested in clinical trials on a range of different cancers.

Drug examples:

Niraparib, Olaparib, Talazoparib

PI3K Inhibitors

PI3K, also known as phosphoinositide 3-kinases, are a group of enzymes that have multiple roles in cells. They can cause cells to grow and divide and play a role in cell migration, the process by which cells move from one location to another. This is a normal process in our body and PI3K enzymes are only active when needed. In cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells, PI3K can become permanently active leading to cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More growth and the potential to spread. PI3K inhibitorsA drug that blocks or reduces the function of a particular protein. More block the activity of PI3K causing the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More to stop growing and shrink.

PI3K inhibitorsA drug that blocks or reduces the function of a particular protein. More are usually given as tablets, although they can also be given as an injection or a drip through a vein. General side effects include tiredness, loss of appetite and diarrhoea. Each PI3K inhibitor will also have specific side effects that your doctor will be able to explain before starting treatment.

PI3K inhibitorsA drug that blocks or reduces the function of a particular protein. More have been approved for use in some blood cancers including chronic lymphocytic leukaemia (CLL). They are also being tested in clinical trials.

Drug examples:

Copanlisib, Samotolisib

Proteasome inhibitors

Proteasomes break down proteins in cells that are not needed. Proteasome inhibitorsA drug that blocks or reduces the function of a particular protein. More block this process. Proteins build up in the cells and the cells die. Proteasome inhibitorsA drug that blocks or reduces the function of a particular protein. More are used to kill cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells.

Proteasome inhibitorsA drug that blocks or reduces the function of a particular protein. More are usually given as an injection or a drip into a vein. Side effects include infection, breathlessness, tiredness, loss of appetite, and diarrhoea. Each proteasome inhibitor will also have its own specific side effects that a doctor will explain before you start on one.

Proteasome inhibitorsA drug that blocks or reduces the function of a particular protein. More are currently licenced for use in some blood cancers and are being tested in clinical trials for a range of cancers.

Drug examples:

Bortezomib, Ixazomib

Radiotherapy

Radiotherapy is a treatment where radiation (usually x-ray) is used to kill cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells by damaging the DNAThe molecule that contains the genetic information that makes you the person you are. More in the cell. This can be given inside or outside the body and is usually focused on a specific area. Treatment is designed to give a high dose of radiation to the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More but a low dose to healthy cells around the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More, reducing side effects.

Radiotherapy can be used to cure specific cancers, reduce the chances of it returning or reduce pain in areas affected by cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More. It is usually given in a series of sessions and can be administered daily over a period of weeks, or a set number of days per week. The length of the course and dosage is determined by your medical team and is based on your specific cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More.

Radiotherapy can be given alone or as part of a treatment plan.

Radiotherapy can give side effects, usually confined to the area of treatment. It is common to experience tiredness (fatigue), skin irritation and redness in the treated area. Side effects usually last a few weeks and should be discussed with your doctor.

Radioisotopes/ Radiotracers

Radioisotopes/ Radiotracers are molecules that have had a small amount of radioactive material added to them as a “label”. These molecules move to tumours or areas of inflammation. They can also bind to specific proteins in the body.

Some radioisotopes are used to help doctors to visualise areas of active cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More. They can also help with pain caused by cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More or be used to deliver radiation directly to cancers to kill them.

Radiotracers can be given as an injection or drip into a vein. They can also be swallowed or inhaled. They do not normally cause side effects but they can cause an allergic reaction in a small number of people. Your medical team will discuss this prior to starting treatment.

Drug examples:

153Sm-DOTMP, 177Lu-3BP-227, 18F-sodium Fluoride, 18-F FTC 146, Methionine, ra-223 dichloride, Sm-EDTMP

TIL Therapy

Lymphocytes are white cells involved in the immune systemA system in the body that fights infection. More. They patrol the body and eliminate cells that should not be there including cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. Tumour-infiltrating lymphocytes (TIL) therapy involves removing lymphocytes from the body that target and kill cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells. In a laboratory the cell number is increased. They are then put back into the body where they should kill the cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells.

TIL therapy is normally only given one of two times. Part of the regime includes chemotherapy, which is given before the lymphocytes to prepare the body. Side effects can include fever and shortness of breath. Each type of TIL Therapy has its own specific side effects which your doctors will discuss before you agree to start treatment.

TIL therapy is currently being tested in clinical trials.

Drug examples:

LN-145/LN-145-S1

Tyrosine Kinase Inhibitors

Tyrosine kinase inhibitorsA drug that blocks or reduces the function of a particular protein. More (TKIs) work by blocking the activation of enzymes known as tyrosine kinases. Tyrosine kinases are involved in multiple processes in the body and are also involved in cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More growth. By blocking their activity TKIs can prevent cancerA disease where cells divide and grow uncontrollably and can spread to other areas of the body. More cells from growing and dividing.

There are many different types of TKIs.

TKIs are usually given as tablets although they can also be given as an injection or a drip through a vein. Side effects include tiredness, loss of appetite, and diarrhoea. Each TKI will also have its own specific side effects that a doctor will explain before you start on one.

TKIs are currently licenced for use in some cancers and are being tested in clinical trials to treat osteosarcoma.

Drug examples:

Apatinib, Abemaciclib, Alisertib, Alvocidib, Cabozantinib, Dasatinib, Ensartinib, Entrectinib, Erdafitinib, Erlotinib, Famitinib, Gefitinib (ZD1839), Ibrutinib, Imatinib, Lenvatinib, Pazopanib, Pexidartinib ( PLX3397) ,Regorafenib, Selumetinib, Selpercatinib, Sorafenib, Sunitinib, surufatinib, TQB3525, Vemurafenib

“It’s that connection between the patient and the team and myself and also the interplay between looking after a teenager and their parents and the rest of the family I found really rewarding”

Dr Sandra Strauss, UCL

Hello Twitter! We are a medical research charity driven by our ambition to cure #raredisease - currently focusing on Birt-Hogg-Dubé Syndrome #BHDsyndrome and #osteosarcoma. Please RT and get in touch if you'd like to find out more! pic.twitter.com/iplhnoMonF
— Myrovlytis Trust (@myrovlytis) June 8, 2021

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