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Rare Cancers Australia

Treatment options

We understand it may be a little overwhelming at first when you hear about the treatment options available. We hope our quick guide helps ease the stress.


Chemotherapy, often called ‘chemo’, uses medicines to destroy cancer cells. Chemotherapy is used on its own or in combination with other types of treatment. Your doctor may recommend chemotherapy to shrink a tumour before surgery, to destroy remaining cancer cells after surgery, or to improve symptoms and prolong life, where it is not possible to cure the cancer. 

In combination chemotherapy a number of drugs may be given at the same time. Sometimes only one drug is used.

Chemotherapy can be given in a number of different ways including: 

  • directly into a vein (intravenously) 
  • by mouth as tablets or capsules 
  • as a cream 
  • directly injected into different parts of the body 

Chemotherapy can affect normal cells too and can cause side effects. Most people get some side effects. Different types of chemotherapy cause different side effects, but the most common ones are poor appetite, nausea and vomiting, hair loss, tired muscles, lethargy, infections, bruising, dry eyes and mouth ulcers. 

Most side effects are only temporary. However, sometimes chemotherapy can cause long term problems like damage to your heart, kidneys, liver, lungs or brain, or infertility. 

Your doctor may prescribe you medicine for the side-effects you are experiencing. These medicines can help with nausea, pain, and other issues. However, they can also have side effects of their own, such as diarrhoea and constipation. 

Sources: Cancer Council Victoria  (Chemotherapy),  Cancer Council Australia  (Chemotherapy),  Cancer Australia  (Types of chemotherapy)


Several types of surgery are helpful to people with cancer. Some surgeries are used in combination with other types of treatment.

Surgery is commonly used to: 

  • Confirm a cancer diagnosis. The doctor may remove all or part of a tumour in a procedure called a biopsy. You can read more about the different types of biopsies here
  • Investigate the size of the tumour and if the cancer has spread. This process is called staging and is often used to guide treatment decisions. Read more about the stages of cancer here
  • Treat small, early-stage cancers that haven't spread. This may be the only treatment, or it may be combined with other treatments. 
  • "Debulk" the tumour if it is not possible to remove the cancer without damaging nearby healthy organs. This involves removing as much of the tumour as possible to make other cancer treatments more effective. 

Surgery can also be used to prevent cancer (prophylactic surgery), repair tissues damaged by tumour removal (reconstructive surgery), insert a tube to allow the delivery of chemotherapy (supportive surgery) or to ease the symptoms of cancer and cancer treatment (palliative surgery). 

Possible side-effects from surgery include pain and discomfort, as well as side-effects from the general anaesthetic (nausea, vomiting, dizziness and agitation). 

Sometimes complications such as infection, bleeding and blood clots occur after surgery. Most complications are minor and can be treated easily. Talk to your surgeon about your actual risks. 

Sources: Cancer Council Australia; Cancer Council VictoriaCancer Institute NSW


Radiation therapy (also known as radiotherapy) uses radiation to destroy cancer cells. Although radiation also damages normal cells, cancer cells are especially sensitive to its effects. This makes radiation therapy an effective treatment for many cancer types.

Radiation therapy can be used: 

  • before surgery to shrink the tumour and make it easier to remove 
  • after surgery to destroy any remaining cancer cells 
  • as the main treatment, if surgery is not a good option 
  • in combination with surgery, chemotherapy or stem cell transplant[link to stem cell transplant] 
  • to relieve symptoms of the tumour.

Radiation therapy involves a total dose of radiation, which is divided into smaller doses or fractions. Typically, one fraction is given each day over several days until the total dose is reached. 

High doses of radiation can be aimed directly at the tumour, depending on where the tumour is and whether it has spread. This limits the amount of radiation that reaches healthy areas of the body. 

There are two main types of radiation: 

External radiation therapy

External radiation therapy (called external beam radiation) uses a machine that beams radiation onto the tumour. The area that receives radiation is therefore very precise, and this minimises the amount of radiation on surrounding healthy areas. 

External radiation therapy is usually given 5 times per week (with a 2-day rest), and can continue for several weeks. Each exposure will take a few minutes. 

The treatment itself is painless. However, you will need to keep very still during the procedure, and this can feel uncomfortable. The treatment team will make every effort to ensure that you are as comfortable as possible. External radiation therapy does not make you radioactive. 

Internal radiation

Internal radiation is called brachytherapy. It involves giving radiation via a needle, catheter or another specialised device. The device stays in place from a few minutes to a few days. Depending on the treatment and the dose of radiation, you may need to stay in hospital or make a daily visit for treatment. 

You may give off a small amount of radiation for a short time after internal radiation therapy. If this will only be for a few minutes, you will stay in a separate room for that time to prevent others being exposed to the radiation. If the radioactivity will last for longer, and you are allowed to leave the hospital, you will need to take some safety precautions. Certain people (e.g. pregnant women, children, adolescents) should not get too close to you until the radiation weakens. Your doctors will discuss this with you in more detail.

Side effects

Side-effects vary and will depend on which area of your body is being treated. Possible side-effects include 

  • fatigue (tiredness) 
  • dry, red or itchy skin 
  • loss of appetite 
  • nausea (feeling sick) 
  • digestive problems 
  • hair loss 
  • dry or sore throat or mouth 
  • cough or shortness of breath. 

Most side-effects can be managed and will gradually disappear once your treatment has finished. However, some may be permanent, and others may not appear until after treatment has finished. 

If the side effects are severe, the radiation oncologist may change the treatment or prescribe a break. If the doctor thinks pausing treatment could affect how well the treatment is working then a break may not be possible.

Sources: Cancer Australia; Cancer Council Australia

Targeted Therapy

This is a type of drug treatment that attacks specific features of cancer cells, known as molecular targets, to stop the cancer growing and spreading. Other names for targeted therapy include biological therapies and molecular targeted therapy.

Cancer is caused by abnormal changes in a person’s genes that can cause cancer cells to multiply and grow. These gene abnormalities are known as molecular targets. They may be acquired or inherited. To find out if the cancer contains a molecular that may respond to a particular targeted therapy drug, your doctor will take a tissue sample from the cancer and send it to a laboratory for molecular testing. It may take from a few days to a few weeks before you receive the results.

Targeted therapy drugs circulate throughout the body. Each drug acts on a specific molecular target within or on the surface of cancer cells (for example, a gene or protein). These molecular targets are involved in the growth and survival of cancer cells. Blocking them can kill cancer cells or slow their growth, while minimising damage to healthy cells. 

Targeted therapy drugs work in a different way to chemotherapy drugs. Chemotherapy drugs also circulate throughout the body, but they particularly affect cells that divide rapidly. They kill cancer cells, but can also damage other rapidly dividing cells, such as the healthy cells in a person’s mouth, stomach, skin or hair. 

Targeted therapy drugs are used to control cancer growth. They often cause the signs and symptoms of cancer to reduce or disappear. This means many people can return to their usual activities. The drugs may need to be taken long-term, and you will need to have regular tests to monitor the cancer. 

Most targeted treatments are given orally as a pill or capsule, or intravenously (directly into a vein). Oral treatments can usually be taken at home. You will need to go to a clinic or hospital for IV treatment. Like other cancer treatments, targeted therapies may be given in cycles. This is a period of treatment followed by a period of rest. 

Sources: Cancer Council NSW; Cancer AustraliaCancer Council Australia

Understanding Personalised Medicine


Immunotherapy, sometimes called biological therapy, is a type of cancer treatment that works by boosting a person’s own immune system to fight the cancer. Immunotherapy is currently approved in Australia for some types of cancers and is also being trialled for other cancers.

The immune system’s ability to detect and destroy abnormal cells usually prevents cancers from developing. However, some cancer cells find ways to stop the immune system destroying them. The natural immune response to cancer cells may not be strong enough to fight them off. Also, cancer cells can change over time (mutate) and then escape from the immune response.

Types of immunotherapy include Checkpoint Inhibitors and Immune Stimulants.  

Drugs called checkpoint inhibitors block certain proteins so the T-cells can recognise and destroy cancer cells. The checkpoint inhibitors that are currently available can block the following proteins: 

  • Programmed death-1 (PD-1) 
  • Programmed death-ligand 1 (PD-L1) 
  • Cytotoxic T-lymphocyte-associated antigen (CTLA-4). 

Checkpoint inhibitors are now the most widely used form of immunotherapy. The types currently subsidised by the Australian Government through the Pharmaceutical Benefits Scheme (PBS) include pembrolizumab (Keytruda), nivolumab (Opdivo) and ipilimumab (Yervoy). These drugs are approved and reimbursed for people with certain cancers and they are also being tested for other cancers. 

Immune Stimulants are used to stimulate the immune system so it reactivates and attacks the cancer. For example, in non-muscle-invasive bladder cancer, the vaccine Bacillus Calmette-Guérin (BCG) may be used as an immune stimulant. It is given into the bladder through a catheter. The BCG stimulates the immune system to stop or delay bladder cancer coming back or becoming invasive.  

In some types of skin cancers, a cream is applied directly to the affected area to stimulate a local immune response. 

Common side effects 

  • eye inflammation, causing dry, irritated eyes 
  • joint inflammation, causing joint pain 
  • bowel inflammation, causing stomach pain, blood in the faeces (poo), bloating and diarrhoea 
  • dermatitis, causing skin rashes 
  • tiredness

Less common side effects 

  • headache and changes in eyesight 
  • thyroid problems, leading to weight loss or weight gain 
  • lung inflammation, causing coughing and shortness of breath 
  • liver inflammation, causing yellow skin, dark urine and abdominal pain 

Sources: Cancer Council Victoria; Cancer Council AustraliaHealth Direct


CAR T-cell therapy is a form of immunotherapy that uses specially altered T cells — a part of the immune system — to fight cancer. A sample of a patient's T cells are collected from the blood, then modified to produce special structures called chimeric antigen receptors (CARs) on their surface.

When these CAR T cells are reinfused into the patient, the new receptors enable them to latch onto a specific antigen on the patient's tumour cells and kill them.

Watch this video from Moffitt Cancer Centre to learn more.

CAR T-cell therapy has been shown to be effective in B-cell acute lymphoblastic leukaemia (ALL) and adult diffuse large B-cell lymphoma (DLBCL). There are no approved CAR-T treatments worldwide for solid cancers however there is research being conducted in this area. 

CAR T-cell therapy can be dramatically effective, but as currently practiced, it has important limitations. According to Caron Jacobson MD, medical director of the Immune Effector Cell program at Dana-Farber:  

  1. For most currently available therapies, immune cells must be removed from the patient who is being treated (autologous cells) and fitted in a specialized lab with an engineered molecule, then returned to the patient to fight a particular type of cancer. This typically takes 17 to 22 days. 
  2. Also, the patients may have been heavily treated previously with other anti-cancer agents, which affects the health of their T cells that are the starting material for CAR manufacturing,” Jacobson says. “And each time you manufacture autologous CAR T cells, it’s one dose, at a significant cost.” 
Regulatory approval in Australia: 

The Therapeutic Goods Administration (TGA) has approved Novartis’ CAR-T product Kymriah® (tisagenlecleucel, formerly CTL019). The approved indications are for the treatment of paediatric and young adult patients up to 25 years of age with B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant, or in second or later relapse; and for the treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy.  

Funding approval: 

CAR T-cell therapy is currently funded for the following indications: 

Paediatric and young adult (up to 25 yrs) with Acute Lymphoblastic Leukaemia (ALL) who relapse or do not respond to initial therapy 

Under consideration: 

CAR T for DLBCL is currently being considered by MSAC, however we have a number of active clinical trials for lymphoma and other cancers, see below. See more about the consideration here 

Sources: Peter MacDana Faber

Hormone Therapies

Hormone therapy (also called endocrine or hormone blocking therapy) aims to stop or slow the growth of certain types of cancer that use hormones to grow. Hormone therapy is typically given in combination with other treatments. It may be used to make a tumour smaller before surgery (neo-adjuvant therapy), reduce the risk of cancer returning after surgery (adjuvant therapy), and to control or slow the growth of cancer cells that have spread around the body (metastasised) and cannot be cured (palliative therapy).

There are 2 main categories of hormone therapy – therapies that stop the production of hormones that cause cancer growth, and therapies that change how these hormones behave.

Hormone therapy can: 

  • be taken orally as a pill or tablet 
  • be injected into a large muscle, such as the thigh or arm (intramuscularly) 
  • be injected or implanted under the skin, usually in the thigh or belly (subcutaneously) 
  • involve surgically removing hormone-producing organs or glands. 

Hormone therapy is a systemic treatment, which means it can affect cells throughout the body. As each hormone in the body has a specific function, altering the balance of hormones in the body can cause side effects. Whether or not you experience side effects, and how severe they are, depends on the type of hormone you are taking, the dose, and how long you take the treatment. Talk to your doctor about the risks and benefits of taking hormone therapy. 

Common side effects include tiredness, hot flushes, mood changes, weight gain and sweating. Hormone therapy can also affect the fertility of both women and men, bring on menopause, and have an impact on your sexuality. Hormone therapy may also cause bones to weaken and break more easily (osteoporosis). Talk to your doctor about having a bone density test or taking medicine to prevent your bones from becoming weak. Regular exercise, eating calcium-rich foods (e.g. yoghurt, milk, tofu, green vegetables) and getting enough vitamin D will also help keep your bones strong. 

Sources: Cancer AustraliaCancer Council Australia

Proton Beam Therapy

Proton beam therapy is radiation therapy that uses heavier particles (protons) instead of the X-rays used in conventional radiotherapy. These particles can more accurately target tumours closer to vital organs, which can be especially beneficial to patients with brain cancer and children whose organs are still developing and are more vulnerable to damage.

Proton therapy is used as a treatment for cancer and some noncancerous tumours. Proton therapy may be used as the only treatment for your condition. Or it may be used in conjunction with other treatments, such as surgery and chemotherapy. Proton beam therapy is not currently available in Australia. 

Sources: The University of SydneyMayo Clinic

Stereotactic Radiosurgery

Stereotactic radiosurgery (SRS) is a specialised type of radiation therapy, not a type of surgery. SRS is sometimes called Gamma Knife® or CyberKnife® treatment, after the machines used to give it. 

It is a non-invasive treatment that uses high doses of precisely targeted radiation to treat a brain tumour. Treatment is so accurate that surrounding areas of healthy brain tissue are unlikely to be affected.

SRS and SRT employ the latest in treatment technology coupled with the use of sophisticated x-ray imaging to enable sub-millimetre accuracy. To help you keep very still during treatment, a special head frame is used to maintain your position exactly as required. SRS is delivered in one single session and generally lasts less than an hour. SRT is delivered in multiple treatments and takes approximately 30 minutes each time. You will usually be able to return home afterwards. 

Radiosurgery may be offered when traditional surgery is not suitable or as an alternative to open surgery. It is most commonly used for some meningiomas and pituitary tumours, and acoustic neuroma. It is also used for metastatic cancers that have spread from another part of the body. 

Radiosurgery is not usually used for gliomas, as they require a larger treatment area. 

Stereotactic radiotherapy (SRT) may also be used to deliver a long course of radiation, particularly for benign brain tumours. The same precise system is used, but several small treatments are given. 

Sources: Cancer Council SAPeter Mac

Stem Cell Transplants

(also known as Bone Marrow Transplant)

Bone marrow and blood stem cell transplantation (commonly referred to as simply stem cell transplantation) is used to treat a range of diseases. These include haematological (blood) disease such as leukaemia, as well as non-haematological diseases.

The following list gives you some examples of conditions which can be treated with stem cell transplantation:

  • leukaemia
  • lymphoma 
  • myeloma 
  • amyloidosis 
  • aplastic anaemia 
  • some solid tumours (for example testicular cancer, breast cancer) 
  • some immune system disorders (for example scleroderma). 

There are two main types of transplants: autologous transplants and allogeneic transplants: 

An autologous transplant uses the patient’s own stem cells, collected in advance and returned to them after they receive high doses of chemotherapy. More than 1,000 stem cell transplants are carried out in Australia each year. The majority of these (more than two-thirds) are autologous transplants. 

In an allogeneic transplant the stem cells are donated from another person, who is a genetically-matched stem cell donor. While all transplants are serious procedures, allogeneic transplants are more complicated and therefore carry more short and long-term risks than autologous transplants. 

Your doctor will discuss the advantages and disadvantages of both types with you. This can be a complex decision that will depend on several factors, such as your age, the type of cancer you have, the health of your existing bone marrow and your overall health, and any previous chemotherapy treatments. 

Side effects of stem cell transplants can include: 

  • increased risk of infection – you will be prone to getting other illnesses because the treatment initially destroys blood-forming cells, and therefore weakens your immune system 
  • GVHD – if new stem cells are donated by another person, they may see your body as ‘foreign’ and attack your existing cells. In severe cases, this can be life-threatening. However, in a mild or moderate form, it actually helps to fight the cancer. Doctors can manage GVHD with antirejection medicines 
  • those related to the side effects chemotherapy or radiation therapy. 

Sources: Leukaemia FoundationCancer Australia

Clinical Trials

A clinical trial is a study that compares responses to different interventions in real settings, to test the effectiveness of medicines or other health measures. Clinical trials are voluntary and are governed by strict rules and ethics. They are often undertaken in a clinical setting, such as a hospital or outpatient clinic.

Clinical trials are an essential step that show whether new approaches work better than those currently used, and whether they are safe. There are several types of clinical trials designed to answer different research questions. Trials might investigate new ways of giving existing treatments, or new combinations of treatments such as drugs, radiation therapy, surgery, nutrition, physiotherapy and complementary therapies. Most clinical trials in Australia are treatment trials.

Clinical trials explained

Learn moreFind a trial

Sources: Cancer Council Australia