Karina's Cancer Blog
Finding Clinical Trials for Cancer
Here's some of our lessons learned from researching clinical trials for our daughter. There are many clinical trials and none are equal. Most oncologists are overworked and cannot know every clinical trial - but no doubt about it, though clinical trials have saved lives (look at childhood leukemia, GIST, etc.). Although clinical trials can save, they can also cause a lot of toxicity and inadvertently cause more harm than benefit, so it pays to do your research!
1. Learn what you can about the biology of your type of cancer. If you have a little science background you may be able to find out which drugs may work better or worse. For alveolar soft part sarcoma, we know that it is a vascular tumor (possible candidate for anti-angiogenesis agents), has relatively slow cell cycle dividing time (so agents dependent on rapid cell division or cycling less likely to help). There may also be different biologies early in the course of disease or with involvement of certain organ systems.
2. Do no harm. If your particular cancer type has patient forums or registries, it may be able to possible to find out what side effects others have had with the drug or class or drugs. In some cases therapies can worsen cancer progression or cause such serious side effects and not extend life that it isn't worth trying. I visited renal cancer forums before our daughter started Sutent, and often visit places like sarcomaalliance.com or cancercompass.com.
3. Look for successes. The best information will be if a particular drug has had success in another patient with your type of cancer, but there will be variables regarding that person's age, stage of cancer, involvement of different organs, prior treatment, and individual molecular make-up, that you can't control for or know, so nothing is ever 100%. What works for someone else may not work for you, and what works for you may not work for someone else. But regardless, with those caveats, you can find a promising drug early through research this way, and most cancer families who post on forums are happy to be contacted about their positive experiences and share information.
Even if you don't have enough successes in your type of cancer, there are often families of similar cancers that may provide helpful information about our own. Searching for review papers and your cancer might help you learn related cancers (and cancer trials that you can search for more information.
Sites for Clinical Trials Searches:
Be aware that you may be limiting your options by only putting in the name of your type of cancer. For instance, there are very few options that may come up with a search for alveolar soft part sarcoma, so I also look under soft tissue sarcoma and even solid tumor studies. In some cases a drug you want may be offered under a general category like recurrent or refractory malignancies or metastatic cancer. If you are dealing with a rare cancer, many clinical trials descriptions won't list your particular type, but it still may be a good drug and effective.
1. ClinicalTrials.gov : http://clinicaltrial.gov/ct2/home
2. National Cancer Institute Clinical Trials: http://www.cancer.gov/CLINICALTRIALS
3. Individual Cancer Therapy Centers - Most cancer trial sites will have their own list of clinical trials that they are enrolling. There has been a trend for more cancer trials to be conducted in buildings away from big hospitals and universities. For instance, in the Los Angeles area for sarcoma, I might check the trials page at Premiere Oncology (http://www.premiereoncology.com) and Sarcoma Oncology (http://sarcomaoncology.com/). UCLA is better known for its surgery for sarcoma, but some clinical trials are listed at its Jonsson Cancer and Children Hospital of LA pages.
Next Level of Investigation
1. After I find possible drugs I want to learn more from, I will often search for information about the success of the drug and side effect profile at a large scientific cancer meeting. For sarcoma, good sites are ASCO (http://www.asco.org/, American Society of Clinical Oncology) and CTOS (http://www.ctos.org/) because they often put the slides online after the meeting is over. Because we are dealing with a rare cancer, I look for news about ASPS, but also search related sarcomas and unrelated but similar cancers (e.g. vascular cancers like renal, melanoma, lung, breast) to look for promising agents with low toxicity.
2. I look through patient forums for others who may have tried the drug. It may tell me about the day-to-day experiences of the drug and possible severe side effects. Quality of life is important.
3. Finally, I go to the drug company's site to find out more information about the drug. I know some of this is advertising, but usually more information can be find under "Investors" links. Early information about preliminary data is often listed, and webcasts are often provided (for investors) that can provide access to data that won't be published for months or a year. Sometimes the SEC report will be there (or somewhere else on the Internet) and that also may trigger alerts for side effects or promising benefits. Because I have a scientific background, I often read all I can on a drug before our daughter tries it. That includes basic science data. Cancers are nasty tenacious things - and more often than not they will require multiple molecular hits to be killed. As a result, growth or appearance of a new tumor is not always an indicator that a drug hasn't worked at all - it may just mean the cancer has mutated to overcome its latest challenge, and so something new needs to be used. Often even if you look at very promising basic science data, the tumor kill is not 100% - but reducing tumor burden is not a trival thing...it can make other therapies more effective or give the body's immune system a better chance of fighting against the cancer.
When looking at a clinical trial, you may want to know that it's possible to ask for exceptions. It is much easier to ask to amend an existing protocol than to create an entirely new one. Also it's easier to ask for an exception once you're in the trial than when you're on the outside looking in.
The exceptions we were able to obtain at entry:
- age of entry (our daughter was only 10 years old; the youngest age on the protocol was 13)
- measurable disease (measurable disease is 1 cm, our daughter was below the cut-off, but we were able to appeal and gain access to the medication as a "non-evaluable case")
Exceptions we were able to obtain after entry:
- waiver of pharmacokinetics study which require multiple blood draws (she had terrible IV access)
- waiver of PET scans for monitoring (our initial PET was so low in activity, unreliable to monitor...we didn't want to expose her more to radiation than we needed)
- substitution of MRIs for CTs for abdomen and pelvis (reduce radiation exposure)
- petition to re-enter study after left to have surgery on one lung (we were very worried about them not letting us back in because we voluntarily left the study when some of her lung nodules increased in size; 2 nodules showed evidence of chemotherapeutic effect though, and this likely helped our request to resume the drug0
- petition to stay on medication after trial ended (this is common if the medication seems to help)
** We were told that we had received an extraordinary number of exceptions and we are grateful for this. But wanted to share this information. Chief medical officers and clinical trial coordinators are people and they want to help patients with cancer, but also have to abide by rules and regulations, the needs of their company, etc. There is no harm in asking.
FYI Compassionate Use: Companies are very reluctant to give medication out on a compassionate use basis. They prefer to collect data in a research trial. Companies will only give out drug on a compassionate use protocol if it is at Phase III trial stage - that is there is sufficent proof that it is effective. If you want to request compassionate use, be aware that companies are besieged by people all the time - and their standard answer is no. More effective appeals (from what people have told us) are from your physician, build a case for the drug in a succinct way (not emotional), and convey the idea that the patient has serious enough disease to warrant the medication, but is not so ill that he or she is terminal or likely to die very soon. It is a delicate dance, but that is the advice we have heard. Companies do not want to give their drugs out for free and have people die soon after. It could give their drug a bad name - and make it less like to make it to approval.