FAQ
What is a cancer vaccine?
Are any cancer vaccines currently available in the United States?
How are therapeutic vaccines designed to treat cancer?
Why does the immune system need a vaccine to help fight cancer?
What strategies are used to design effective cancer treatment vaccines?
What types of treatment vaccines are currently under investigation?
Which antigens are commonly found in cancer vaccines?
What are adjuvants?
Why are some vaccines used to treat specific kinds of cancer?
Are there vaccines being developed to prevent cancer?
What is Melanoma?
Other than Melanoma, are there other types of skin cancers?
How many Americans are diagnosed each year with melanoma?
How many Americans will die from melanoma each year?
Do genetics and/or family history increase the risk of a person developing melanoma?
Does skin color increase the risk of developing melanoma?
Does where I live increase my lifetime risk of being diagnosed with melanoma?
What are the risk factors?
What are the warning signs of melanoma?
What are the most common symptoms of melanoma?
How is melanoma diagnosed?
What diagnostic tests need to be performed?
How do I distinguish the difference between a mole and melanoma?
What is Dysplastic Nevi Syndrome?
What are the stages of melanoma?
What are the treatment options for melanoma?
Should patients seek a second opinion after diagnosis?
Can melanoma be prevented? And if so, how?
Should a patient consult an oncologist rather than a dermatologist?
Does surgery remove all of the melanoma?
Does melanoma spread and/or metastasize?
What is metastatic melanoma?
What do I do if the melanoma has spread?
What is the risk of recurrence?
Is there a cure for melanoma?
Is chemotherapy the best treatment for melanoma?
Should a patient explore experimental treatment options, and if so, when?
Clinical Trials
Should a patient explore experimental treatment options, and if so, when?
What is a clinical trial?
What are the phases of clinical trials?
How are cancer vaccines different?
HCI001 (Melanoma Vaccine)
What is HCI001?
How is HCI001 different from other vaccines?
Who can be treated with HCI001?
What are the side effects?
Is HCI001 FDA-approved?
How was HCI001 developed?
How long does the treatment last? What is the course of treatment?
What is the fee to enroll in the clinical trial?
Will my insurance pay for treatment and necessary diagnostic testing?
What is a cancer vaccine?
A cancer vaccine is an immunization that either prevents the development of cancer or treats existing cancer in the body. A vaccine given for the treatment of existing cancer (a therapeutic vaccine) is administered to individuals who already have cancer. The purpose of a therapeutic vaccine is to stimulate or modulate the immune system so an immune response to the cancer can be generated.
A vaccine that is given for the prevention of cancer (a prophylactic vaccine) is administered to patients who do not have cancer but are at high risk for developing it.
Are any cancer vaccines currently available in the United States?
Yes. There are two prophylactic vaccines approved by the Food and Drug Administration (FDA). One prophylactic vaccine approved by the FDA for use in humans is a Hepatitis B antiviral vaccine that prevents the development of Hepatitis B viral infection. Hepatitis B infections are associated with the development of liver cancer. The other prophylactic vaccine currently approved by the FDA is Gardasil. It helps prevent the development of human papilloma virus (HPV) infection on the female cervix. HPV is an important causative agent for cervicl cancer. Gardasil immunizes against the most common HPV viruses, but not all HPV. No therapeutic vaccines have been approved so far, but several potential vaccines are in clinical trials.
How are therapeutic vaccines designed to treat cancer?
Therapeutic vaccines are designed to stimulate or modulate the immune system. Patients with cancer have, to a large extent, an intact immune system. Therapeutic vaccines retrain the immune system to recognize cancer cells as foreign so they can be destroyed. There are various components to the immune system and various strategies to modulate the immune system are being tested.
Why does the immune system need a vaccine to help fight cancer?
When you have cancer, the immune system fails to "recognize" tumors as dangerous or foreign, and is unable to mount a strong attack against them. In addition, cancer has developed ways to evade the immune system. Scientists are now beginning to understand some of the ways cancer escapes the immune system which include shedding tumor antigens, and reducing the number of molecules and receptors that the body normally relies on to activate the immune system. Reducing these molecules makes the tumor less "visible" to the immune system. This knowledge is currently being studied by researchers to design more effective vaccines.
What strategies are used to design effective cancer treatment vaccines?
Researchers have developed several strategies to stimulate the immune system to fight cancer. One strategy is to identify unusual or unique cancer cell antigens that are rarely present on normal cells. Another strategy involves making the tumor-associated antigen more immunogenic by altering its amino acid structure. Yet another strategy places the gene for the tumor antigen into a harmless virus that can be used as a vehicle to deliver the vaccine to the tumor. There are other techniques being used to trick the immune system into attacking a patient's tumor.
What types of treatment vaccines are currently under investigation?
Listed below are some of the types of vaccines devised for presenting cancer antigens to the body's immune system.
Antigen/Adjuvant Vaccines:
Antigen vaccines were among the first cancer vaccines to be investigated. These vaccines commonly use specific protein fragments or peptides to stimulate the immune system to fight tumor cells. With these vaccines, one or more cancer cell antigens are combined with a substance that causes an immune response, known as an adjuvant. When the cancer patient is treated with this vaccine, it is expected that the immune system will respond to the antigen and adjuvant. It is expected that tumor cells will be killed in the process.
Whole Cell Tumor Vaccines:
This technique involves extracting tumor cells from the patient's own tumor, or sometimes from other patients. These whole cell vaccines contain cancer antigens that are used to stimulate an immune response. Tumor cells usually have to be “killed” with radiation therapy before administration to patients.
Dendritic Cell (DC) Vaccines:
Specialized white blood cells called dendritic cells (DCs) are taken from a patient's blood through a process called leukapheresis. These DCs are stimulated with the patient's own cancer antigens, grown in petri dishes, and are reinjected into the patient. Once injected, DC vaccines activate the immune system's T-cells to multiply and attack tumor cells expressing cancer antigens.
Viral Vectors and DNA Vaccines:
These types of vaccines are attractive because they are easier to manufacture than some of the other types of vaccines. Viral vectors and DNA vaccines use the nucleic acid sequence of the tumor antigen to produce the cancer antigen or protein. The DNA containing the gene for a specific cancer antigen is manipulated in the laboratory so that it will be taken up and processed by immune cells called antigen-presenting cells (APCs). The APC cells then display part of the antigen together with another molecule on the cell surface. The hope is that when the APC cells are injected into a patient, the immune system will respond by attacking tumor cells containing the same antigen.
Idiotype Vaccines:
Because antibodies are molecules containing proteins and carbohydrates, they can act as antigens and induce an antibody response. Antibodies produced by certain cancer cells (such as B-cell lymphomas and myelomas) are called idiotype antibodies. These are unique to each patient and can be used to trigger an immune response in a manner similar to antigen vaccines.
Which antigens are commonly found in cancer vaccines?
Cancer cell antigens may be unique to individual tumors, shared by several tumor types, or expressed by the normal tissue from which a tumor grows. In 1991, the first human cancer antigen was discovered in the cells of a patient with metastatic melanoma, the most deadly form of skin cancer. This discovery led to a flurry of research to identify antigens for other cancers.
Sialyl Tn (STn):
A small, synthetic carbohydrate that mimics the mucin molecules (the primary molecule present in mucus) found on certain cancer cells.
Heat Shock Proteins (HSPs):
HSP’s are produced in cells in response to heat, low blood sugar levels and other stress signals. Besides protecting against stress, these molecules are also involved in the proper processing, folding, and assembling of proteins within cells. In laboratory experiments, HSPs from mouse tumors, in combination with small peptides, protected mice from developing cancer. The human vaccine consists of heat shock protein and associated peptide complexes isolated from a patient's tumor. HSPs are under investigation for treatment of liver, skin, colon, lung, lymphoma and prostate cancers.
Ganglioside Molecules (e.g., GM2, GD2, and GD3):
These are complex molecules containing carbohydrates and fats. When these molecules are incorporated into the outside membrane of a cell, they make the cell more easily recognized by antibodies.
Tyrosinase:
Tyrosinase is a key enzyme involved in the initial stages of melanin production. Studies have shown that tyrosinase is a specific marker for melanoma and more abundant on melanomas than on normal cells.
Prevention Vaccines:
Viral proteins on the outside coat of cancer-causing viruses are commonly used as antigens to stimulate the immune system for prevention vaccines.
What are adjuvants?
An adjuvant is a decoy substance, which researchers usually attach to tumor antigens to trick the immune system into recognizing tumors as foreign. Adjuvants are foreign proteins that make the immune system mount an attack on tumor cells. Several adjuvants are described below:
Bacillus Calmette Guerin (BCG):
BCG is an inactivated form of the tuberculosis bacterium routinely used for decades to vaccinate against TB. BCG is added to some cancer vaccines in an effort to boost an immune response to the vaccine antigen.
Interleukin-2 (IL-2):
Interleukin-2 is a protein made by the body's immune system that may boost the cancer killing abilities of certain specialized immune system cells called natural killer cells. Although it can activate the immune system, many researchers believe IL-2 alone will not be enough to prevent cancer relapse.
Granulocyte Monocyte-Colony Stimulating Factor (GM-CSF):
GM-CSF is a protein that stimulates the proliferation of antigen-presenting cells.
QS-21:
QS-21 is a plant extract that, when added to some vaccines, may improve the immune response.
Montanide ISA-51:
Montanide is an oil-based liquid intended to boost an immune response.
Why are some vaccines used to treat specific kinds of cancer?
Many cancer vaccines treat only specific types of cancers because they target antigens found on specific cancers. For example, a prostate cancer vaccine may be able to attack cancer cells within the prostate itself or cancer cells that have spread to other parts of the body. It would not effect cancers originating from other organs and tissues.
Are there vaccines being developed to prevent cancer?
Yes. Some vaccines currently being developed have the potential to reduce the risk of cancer. These vaccines target infectious agents that cause cancer and are similar to traditional prophylactic vaccines, such as the polio or measles vaccines. Gardasil, a vaccine for the prevention of cervical cancer, is now widely used.
What is Melanoma?
Melanoma is a type of skin cancer and though it is not the most common type, it is the most serious. Unlike other skin cancers, melanomas generally appear as pigmented spots on the skin and often look similar to moles or freckles. Melanomas, however, will usually have undefined boundaries and can be irregularly shaped. Because of its tendency to spread to other parts of the body, melanoma is a very serious disease. If not detected and treated early, melanomas can and will metastasize (spread to distant organs) and can lead to death. Some melanomas called amelanotic melanomas do not have pigment, so it is important to monitor all abnormal lesions on the skin. This is especially important if you use tanning beds, are regularly exposed to direct sunlight, live in the Sunbelt states, or have a family history of melanoma.
Other than Melanoma, are there other types of skin cancers?
In general, there are two other very common types of skin cancers. One type is called squamous cell carcinoma and develops just below the surface of the skin. The epithelium, or outer layer of the skin, does not have any live cells, but underneath the exterior lining are cells that look like they are diminished, and in time, will form the epithelial layer of the skin. Sometimes these cells can become cancerous, often appearing as a non-healing wound on the skin. Patients often mistake squamous cell carcinomas as bug bites or other irritations, but the hallmark of squamous cell carcinoma is that these lesions will not heal and will continue to evolve, grow and become more irritated.
The other common type of skin cancer, called basal cell carcinoma, has a similar presentation to squamous cell carcinoma. They too develop just below the surface of the skin and tend to look like a bug bite. Both squamous cell and basal cell carcinomas need to be treated, usually with surgery, but unlike melanoma, these cancers are almost always localized and do not spread to internal organs.
There are other types of skin cancer but these are very rare and are not seen very often.
How many Americans are diagnosed each year with melanoma?
The National Cancer Institute estimates that in 2008 there were in excess of 62,000 cases of melanoma. The incidence of melanoma is continuing to increase and has increased every 10 years by 50%. This has been due, in part, to increased exposure to the sun and the use of UV tanning beds. As our population ages, the extent of sun exposure also increases. Additionally, the older population is moving to Sunbelt states like Arizona and Florida and the Southwest including Texas. As a consequence, this aging population is experiencing increased sun exposure which is a direct risk factor for melanoma.
How many Americans will die from melanoma each year?
The National Cancer Institute estimates that in 2008 there were in excess of 8,000 deaths from metastatic melanoma. When melanoma occurs on the skin, immediate and judicious surgical treatment is indicated. However, if the diagnosis is missed, the melanoma can spread to other organs, leading to morbidity and mortality associated with metastatic melanoma.
Do genetics and/or family history increase the risk of a person developing melanoma?
There are a small group of individuals who develop melanoma because of a family history. Certain genes have been described in these families, and they are usually clustered in family members. We are learning more about the family history of melanoma, but this type is very rare.
Most cases of melanoma are directly due to sun exposure and UV tanning. Those individuals with a family history will need to be screened, and as we learn more about the genetics of this familial type of melanoma, additional screening and intervention may be indicated.
Does skin color increase the risk of developing melanoma?
Skin color does have an impact on the development of melanoma. Individuals with fairer skin have an increased risk of UV damage to melanocytes (cells just below the surface of the skin that contain melanin, the pigment responsible for skin coloration) in the skin and, as a consequence, fairer individuals have a higher risk of developing melanoma. However, melanoma can develop in all individuals, including persons of Hispanic, African, or Asian decent. The presentation of melanoma in these individuals, however, is somewhat different. In Caucasians, melanomas will very often occur in the extremities like the arms and legs, and sometimes in the truncal areas such as the neck, back and face. However, in non-Caucasian individuals, especially persons of African decent, melanomas can occur in various mucocutaneous junction areas such as the inner rectal area, the palms of the hands and the soles of the feet. These are common sites for a melanoma in these individuals. Additionally, melanoma can occur in the eye.
Does where I live increase my lifetime risk of being diagnosed with melanoma?
Yes, where you live does have an impact on the incidence of melanoma. The incidence of melanoma increases with the risk of sun exposure. Therefore, those individuals who live in sunny areas like the Southwest, where there is very little cloud cover and very little rain, have a higher risk of melanoma because of their increased exposure to the sun. More specifically, the UV radiation emitted from the sun has a direct impact on the development of melanoma. As a consequence, we see a higher incidence of melanoma in those populations closest to the equator because of the more direct UV radiation these individuals are exposed to. Those individuals who use sunscreen and protect their skin from the sun with clothing and shade reduce their risk of melanoma, even if they live in tropical areas.
What are the risk factors?
The risk factors for melanoma primarily include UV exposure, mostly from the sun. Individuals who use tanning booths also have an increased risk of developing melanoma. Some individuals who have been treated for certain skin conditions such as cutaneous T-cell lymphoma also run an increased risk of developing melanoma. A family history of melanoma is also a risk factor.
What are the warning signs of melanoma?
Melanomas usually occur on the skin, although they can occur in other areas like the eye and in mucocutaneous areas like the oral and anorectal areas. The warning signs include a skin lesion that is often pigmented that continues to grow, often in an irregular fashion. Many times they can have multiple colorations, vertical and/or rapid growth, irregular borders, and sometimes there can be bleeding. Clinicians use the concept of ABCDE to help them gauge a skin lesion and predict the risk of melanoma in a lesion.
Asymmetry of lesion
Border irregularity of the lesion
Color variegation of the lesion
Diameter greater than 6mm. of the lesion
Evolving lesion
What are the most common symptoms of melanoma?
The symptoms of melanoma primarily related to a skin lesion that will often continue to grow and be present. Sometimes there can be irritation. Sometimes there can be changes in coloration with bleeding and irritation. Often times, however, the patient may not readily see the lesion. Skin lesions can occur in places that are not readily accessible like in the scalp/hair for example. Other lesions can occur in the back of the leg such as the knee joint where personal visual inspection is difficult. Lesions in the back will often be missed because inspection of the back is difficult without multiple mirrors or a friend or family member who can look at these areas. Finally, lesions that occur in areas such mucocutaneous junctions are easily missed, because inspection of the anorectal area, for example, is not frequently done.
How is melanoma diagnosed?
Melanoma is diagnosed when a suspicious lesion, usually on the skin, is excised or biopsied. Biopsies can be done by a dermatologist, a surgeon or a primary care physician. After the suspicious lesion is excised, pathology review must be completed. If a diagnosis of melanoma is established by biopsy and pathology review, then additional testing and therapies need to be instituted to determine the stage of the melanoma and the appropriate treatment for that stage of disease.
What diagnostic tests need to be performed?
When a diagnosis of melanoma is established, comprehensive pathology review is important because the stage of the melanoma depends on the depth of the primary lesion. Additionally, there is always the question of whether there is any lymph node involvement in the local regional draining lymph nodes. If the melanoma is deeply invasive, there can be risk of internal organ involvement. These are determinations that the clinician will have to make and establish the appropriate mode of treatment. In general, the goal of treatment of a localized melanoma that occurs in the skin is to make sure that there is adequate surgical resection of the primary lesion. Whether or not there is any lymph node involvement is important because establishing lymph node involvement determines the stage of disease. Additional treatment in the form of adjuvant therapy may be indicated in those individuals who have lymph node involvement.
Additional diagnostic testing can include such things as CT scans and sentinel node biopsy of a lymph node that drains that particular limb (if the melanoma occurs on a limb). Often a second operation that involves wide excision of the primary melanoma site is required. This is to ensure that all the melanoma cells have been removed.
How do I distinguish the difference between a mole and melanoma?
Distinguishing a mole from a melanoma can be very difficult. Very often, patients need to consult their doctor, either their primary care physician or dermatologist. Because it is so difficult to distinguish a benign mole from a melanoma, patients and doctors often use the ABCDE criteria to help make the distinction. These criteria cannot always make a definite distinction, but a lesion that is suspicious for melanoma should be biopsied. If it turns out to be a benign mole, then no further intervention is necessary. Skin lesions that appear to be benign have fooled clinicians and patients and, after biopsied, have turned out to be melanoma. If there is any index of suspicion, a biopsy is indicated.
What is Dysplastic Nevi Syndrome?
Dysplastic Nevi Syndrome is a clinical term that is used for a mole that looks suspicious for melanoma. On subsequent biopsy of this mole, there are microscopic features that suggest that the melanoma cells are abnormal, but do not meet the criteria for melanoma. Dysplastic nevi are pre-malignant lesions. The diagnosis is made on a histological basis, which means that the biopsy is done and microscopic review suggests that the skin lesion is a pre-malignant lesion. The problem with pre-malignant lesions is that they are at increased risk for transforming into a melanoma at a later time. In individuals who have dysplastic nevi, there are increased risks for developing melanoma and these individuals need to have increased surveillance of their skin for early detection of a melanoma. Once again, the ABCDE criteria is used to help determine when to biopsy a new lesion that may be a dysplastic nevi and could transform into a melanoma.
What are the stages of melanoma?
Melanoma is staged like most other cancers. There are four stages (I, II, III and IV), with stage I being the earliest form of melanoma and stage IV representing widespread metastasis of the disease. Usually a clinician will need to determine the stage of the melanoma. In order to stage melanoma a clinician may need to obtain such things as additional testing like CT scans and possibly a sentinel node biopsy to determine whether the melanoma has spread to the lymph nodes. Microscopic examination of the tumor with measurement of the depth of melanoma invasion is an integral aspect of staging. Staging is important because it determines further treatment as well as prognosis. The stage of melanoma can help the clinician determine additional treatment, including wide excision of the primary melanoma and adjuvant therapy, to prevent the recurrence of metastatic melanoma.
What are the treatment options for melanoma?
The treatment options for melanoma will often depend on the stage of disease. They can include surgery, chemotherapy, biologic therapy and occasionally radiation therapy. Melanoma is a very aggressive type of cancer and its staging will determine the best treatment options. When it occurs in a local area (Stage I), then surgical intervention is very important. If melanoma has spread to distant organs (Stage IV), chemotherapy and biologic therapy can be offered, but unfortunately the prognosis for these individuals is not very encouraging. In melanoma that has metastasized to local regional areas such as lymph node areas (Stage II or III), then additional surgery with excision of lesions in those areas can be helpful. Occasionally, if there is a solitary metastasis to a lung or liver area, then surgical resection of these sites may also be indicated. Biologic therapy and immunotherapy are becoming popular alternative treatment options for melanoma, but they are still in testing stages.
Should patients seek a second opinion after diagnosis?
Patients who have questions about their diagnosis and want a second opinion can and should obtain a second opinion. Melanomas are often treated by a variety of physicians including dermatologists, plastic surgeons, general surgeons, medical oncologists and occasionally, radiation therapists. Patients who have particularly advanced melanomas, and who are looking for treatment options, should discuss available options with their primary treating oncologist. Patients with advanced melanoma often do not have good options for treatment, and these individuals should seek second opinions at comprehensive cancer centers, especially if they are interested in participating in a clinical trial.
Can melanoma be prevented? And if so, how?
Melanoma can be prevented. The primary risk factor for melanoma is sun exposure, specifically, the UV radiation emitted by the sun which is extremely damaging to the skin. Simply put, prevention of melanoma can take place by avoiding sun exposure. Additional prevention can take place in the form of careful screening of any skin lesions. Early diagnosis and treatment can be helpful and if treated early enough, can be curative. Dysplastic Nevi (abnormal moles) can have pre-malignant characteristics and excision of these lesions can prevent the development of melanoma. Individuals who live in the Sunbelt areas can certainly wear protective clothing when out in the sun. Additionally, avoiding UV tanning booths can help prevent melanoma. Those individuals who have a family history of melanoma should be screened comprehensively. Suspicious lesions should be biopsied for early detection of melanoma.
Should a patient consult an oncologist rather than a dermatologist?
Individuals who have a very localized melanoma can and should be treated by a dermatologist or plastic surgeon. Excision of melanoma including wide excision of the primary lesion is the first step. After pathology review, if it is determined that the patient has advanced disease (either locally advanced or metastatic disease) then additional consultations, especially with an oncologist and a surgeon familiar with melanoma surgery, should be considered.
Does surgery remove all of the melanoma?
If a lesion on the skin is amenable to surgical excision, then surgery is certainly helpful. One of the important concepts in the treatment of melanoma, particularly surgical treatment of melanoma, requires that wide margins (area around visible lesion) be available. Initially, when there is a skin lesion, the surgeon or dermatologist will do an excisional biopsy in order to determine whether or not the lesion is in fact a melanoma. Once the diagnosis of melanoma is established, then a second operation where a wide excision is made to ensure total removal of residual malignant melanoma cells in the skin may be necessary.
In general, wide excision requires a margin of 1 to 2 cm around the primary lesion. Sometimes in cosmetically sensitive areas, such as the face, this can be problematic. Plastic surgeons will often work with patients to ensure a good cosmetic outcome while maintaining adequate surgical margins at the same time. When lesions occur in areas like the mucocutaneous junction, such as anorectal melanomas, surgery can be complicated and patients will need to seek consultations with surgeons who have experience in doing those operations. When melanoma occurs in the eye, there are many different types of treatments that can be offered. Some ophthalmologists specialize in the treatment of ocular melanoma and laser therapies can be effective for very early melanomas. More advanced melanoma of the eye requires enucleation (removal of the eye).
Does melanoma spread and/or metastasize?
Unfortunately, melanoma does spread and metastasize to all parts of the body. That is the distinguishing feature of melanoma from other skin lesions. The likelihood of a skin melanoma spreading to internal organs depends on how deeply invasive the melanoma has become. Clinicians use the depth of invasion to determine whether or not the melanoma may have spread or metastasized. Very often, if the lesion is deeply invasive in the skin, then the clinicians will recommend a sentinel node biopsy to determine whether the melanoma has spread to the lymph nodes. If this has occurred, the risk of widespread metastases is high, and additional treatment in the form of adjuvant chemotherapy or biologic therapy may be indicated.
What is metastatic melanoma?
Metastatic melanoma is a condition where a local melanoma which may have occurred in the skin has spread to distant organs. Melanoma can and will spread through the blood system to distant organs like the brain and bone, and to internal organs like the lungs and liver. Melanoma is very dangerous because it can spread to nearly every organ in the body from a single skin site. When it has spread to these distant organs it becomes life-threatening.
What do I do if the melanoma has spread?
If the melanoma has spread, the patient needs to look for different treatment options including seeking a second opinion, appropriate clinical trials and chemotherapy and biologic therapies. Depending on specific organ involvement, these options should be tailored to the specific involvement of the metastatic disease.
What is the risk of recurrence?
The risk of recurrence of melanoma depends on the original stage of the disease. If an individual had melanoma and the primary lesion was deeply invasive, or had lymph node involvement, the risk of recurrence is high. If the melanoma was superficial, and adequate surgery was performed, the risk of recurrence is much lower. Individuals who have had a melanoma are always at risk for a new melanoma developing on their skin. Surveillance of the skin with monthly examinations is important. Individuals who have had a deeply invasive primary skin melanoma are at risk for metastatic melanoma. The risk of recurrence is high and can occur many years after the primary diagnosis.
Is there a cure for melanoma?
When melanoma is diagnosed at an early stage where there is only a localized skin lesion, it can be cured by surgery. That is why surveillance of the skin is very important. When melanoma has spread, particularly to internal organs, a cure is generally not likely. There are many new treatments being investigated including biologic therapies, immune therapies and vaccine therapies, but these still remain experimental.
Is chemotherapy the best treatment for melanoma?
For metastatic melanoma, chemotherapy remains a standard of treatment. Unfortunately, most patients who undergo chemotherapy treatments are not greatly benefited by the treatment. This has sparked much interest in developing additional types of therapies for patients with metastatic melanoma including biologic and immune therapies.
Should a patient explore experimental treatment options, and if so, when?
Patients should consider experimental treatment options if they have developed metastatic melanoma. Because standard therapies, such as chemotherapies, are not particularly effective, patients may have a better chance of benefit through new types of treatments such as immune therapies, vaccine therapies and biologic therapies. If they fail these experimental treatments, patients can always go back to the standard chemotherapy treatments. Individuals who want to explore experimental treatments should be encouraged to do so. Experimental treatments are available at comprehensive cancer centers across the country. Through internet searches, one should be able to find experimental treatments that have the potential for benefit.
Are experimental treatment options safe?
Guidelines set forth by the Food & Drug Administration and the Human Subjects Committees provide a certain measure of protection to patients. In addition, the consent form will often explain the experimental treatment, its potential risks and what is known about these risks. Each patient needs to carefully assess and evaluate experimental treatments for side effects, complications and potential benefit before participating.
What is a clinical trial?
A clinical trial is really an experiment. There are many different types of clinical trials, but in general they investigate a particular aspect of treatment for the patient.
What are the phases of clinical trials?
In general, there are four phases to clinical trials. A Phase I clinical trial evaluates an experimental drug or treatment that has never been used in humans. The purpose of Phase I clinical trials is to determine if there are any serious side effects from the treatment and to obtain vital information on how the drug is metabolized and excreted in order to determine an appropriate dose for use in humans.
In general, Phase II clinical trials establish the level of benefit to patients. For example the percentage of patients who respond to the drug is established.
In Phase III clinical trials, there is randomization of the participants with one group of individuals being treated with a standard form of treatment for that stage of disease, and another group of individuals being treated with an experimental treatment or drug. Here, the purpose is to determine efficacy compared to standard treatment. In other words, the trial is to determine whether the standard treatment is better or worse than the experimental treatment.
Phase IV trials are done after a drug has been shown to work and has been granted a license. These trials look at drugs that are already available for doctors to prescribe, rather than new drugs that are still in development. The main reasons pharmaceutical companies run Phase IV trials are to find out more about the side effects and safety of the drug, what the long term risks and benefits are as well as the drug’s efficacy when it is used more widely than in clinical trials.
Who monitors clinical trials?
Clinical trials are monitored by various safety boards. The Human Subjects Committee monitors clinical trials, while other independent safety boards look at the results of the clinical trials. Clinical trials many times may be allowed to go to completion, or may be terminated prematurely for a variety of reasons including safety and lack of benefit.
How are cancer vaccines different?
Cancer vaccines are treatments that use the immune system to treat cancer. Interestingly, patients with cancer often have an intact immune system. We believe that if the immune system can be harnessed to attack cancer cells there can be potential benefit. Many types of cancer vaccines are currently being investigated. Some cancer vaccines are called whole cell vaccines. This is where cells in the body are removed and trained to recognize the cancer by exposure to cancer proteins. Then they are re-infused into the body. There are other types of vaccines that are non-specific. These vaccines enhance immune surveillance but do not target a particular cancer and can have variable effects on the body. However, these non-specific vaccines frequently have limited benefit. A type of vaccine exists that is directed against specific tumor proteins. However, cancer-specific vaccines have not been very successful.
What is HCI001?
HCI001, developed by Raj Sadasivan, M.D., Ph.D., is a vaccine that Vaxco Pharmaceuticals is preparing to administer in upcoming clinical trials. It is for patients with metastatic melanoma. This vaccine was developed to elicit a direct immune response against melanoma. By using antigens from a melanoma-common protein, the vaccine can potentially stimulate the immune response to destroy cancer cells. In pre-clinical trials using mice, the response of the vaccine was tremendous. In 19 out of 20 mice injected with melanoma, the vaccine destroyed the disease. The purpose of the clinical trial is to determine if this vaccine can potentially benefit individuals.
How is HCI001 different from other vaccines?
HCI001 is different from other vaccines because it utilizes a more fundamental understanding of the immune system. Many immune responses are dependent on the human HLA system. The HLA system is a set of proteins on each of our cells in the body. It is what makes us individually unique. This is why we cannot randomly transplant an organ such as a kidney from one individual to another without crossmatching. This particular vaccine bypasses that system, and has the potential for a rapid response against the melanoma cells. In general, our immune system has two basic forms of functioning. When the body’s cells become cancerous then the HLA system has to be matched for an immune response. This may be the reason why traditional vaccines have not been effective. HCI001 allows the immune system to detect cancer in the same way it detects bacteria, parasites and fungi. It is hopeful that HCI001 will be able to stimulate an immune response that is independent of the HLA system, allowing the body to recognize this vaccine as a completely foreign protein and attack it independently of HLA matching. When there is an immune response against the vaccine there is a cross reaction, and the melanoma cells are destroyed.
Who can be treated with HCI001?
Patients who have metastatic melanoma and meet eligibility criteria can potentially participate in this clinical trial. In general, patients must have failed at least one standard chemotherapy or biologic therapy for metastatic melanoma. In addition, patients must be at least 18 years of age, have no brain metastases, and have adequate heart, lung, kidney and liver function.
What are the side effects?
The side effects of the vaccine are not completely known. Vaccines of this type can, however, have side effects such as irritations of the skin and joint symptoms.
Is HCI001 FDA-approved?
HCI001 is pending FDA approval for a clinical trial. We expect the FDA to give its approval in the near future and at that time we will open a clinical trial. In the interim, however, a patient may request he or she be treated on a Schedule C, compassionate use basis. Such requests will be reviewed and determined on a case-by-case basis.
How was HCI001 developed?
HCI001 was developed through careful research using pre-clinical models including mice. The proteins used in developing the vaccine were developed through computer modeling and a fundamental understanding of the binding of proteins to immune cells.
How long does the treatment last? What is the course of treatment?
In general, the treatment for participants in the clinical trial will last approximately six months. Patients will receive two weeks of intensive therapy repeated by a second course of two weeks of intensive therapy. Thereafter they will go on maintenance therapy for up to six months.
What is the fee to enroll in the clinical trial?
There is no fee to enroll in the clinical trial. The drug is offered free of charge to participants. However, ancillary testing such as CT scans, blood work and other diagnostic tests and some physician and nurses services are billed to the patient’s insurance company.
Will my insurance pay for treatment and necessary diagnostic testing?
Because the participants have advanced disease and need continued monitoring, insurance companies will generally pay for the diagnostic testing and some physician and nursing services. However, this is not guaranteed, as every insurance plan has its own rules and regulations.