Skip to Content. Use the menu to see other pages. Doctors and scientists are always looking for better ways to care for children with ALL. To make scientific advances, doctors create research studies involving volunteers, called clinical trials. In fact, every drug that is now approved by the U. Clinical trials are used for all types and stages of childhood ALL. Many focus on new treatments to learn if a new treatment is safe, effective, and possibly better than the existing treatments.
These types of studies evaluate new drugs, different combinations of treatments, new approaches to radiation therapy or surgery, and new methods of treatment.
Children who participate in clinical trials are often among the first to get a treatment before it is available to the public. However, there are some risks with a clinical trial, including possible side effects and the chance that the new treatment may not work.
Some clinical trials study new ways to relieve symptoms and side effects during treatment. Others study ways to manage the late effects that may happen a long time after treatment. People decide to participate in clinical trials for many reasons.
For some people, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients and their families are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Other people volunteer for clinical trials because they know that these studies are a way to contribute to the progress in treating children with ALL.
Even if they do not benefit directly from the clinical trial, their participation may benefit future children with ALL. Insurance coverage and the costs of clinical trials differ by location and by study. In some programs, some of the expenses from participating in the clinical trial are reimbursed.
In others, they are not. It is important to talk with the research team and your insurance company first to learn if and how your treatment in a clinical trial will be covered. Learn more about health insurance coverage of clinical trials. Find out more about placebos in cancer clinical trials.
To join a clinical trial, parents and children must participate in a process known as informed consent. During informed consent, the doctor should:. Describe all of the treatment options so that the person understands how the new treatment differs from the standard treatment. List all of the risks of the new treatment, which may or may not be different from the risks of standard treatment. Explain what will be required of each person in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment. New understanding about the biology of CLL has paved the way to develop drugs for additional targets in relapsed or refractory disease.
This remains an essential area for research, as the currently approved therapies are generally viewed as noncurative, experts said. Results showed longer median PFS in the duvelisib group than the ofatumumab group by both blinded independent review committee Venetoclax-rituximab conferred a higher rate of PFS at 2 years This benefit persisted across patient subgroups, including for those with 17p deletion 2-year PFS, In addition to developing new treatments for resistant disease, researchers aim to determine why disease becomes resistant in the first place.
Characterization of a recurrent mutation in the BCL2 protein offered new insights into the pathobiology of venetoclax resistance among patients with progressive CLL. Moreover, the mutation conferred a selective advantage when continuously exposed to sublethal concentrations of venetoclax in 3-week cultures, and in primary patient mutant cells in both short-term survival assays and when cultured.
This discovery will allow drug developers to create novel strategies to tweak the chemical structure of venetoclax and develop second-generation BCL2 inhibitors to overcome this particular mutation. Although these data need to be confirmed in more patients before they can be applied to the clinic, experts say this discovery shows the potential role of testing for BCL2 mutations in patients treated with venetoclax indefinitely. But, it was very rare that they found a mutation before the 2 years, so I do not know how helpful it will be to detect for the mutation if we are only giving the drug for 2 years.
As insights into targeted treatments pay dividends for patients with relapsed and refractory disease, researchers are exploring the potential benefits of moving these combinations into earlier lines of treatment. The FDA based the expanded approval on results of the phase 3 iLLUMINATE trial, in which patients were randomly assigned to mg ibrutinib continuously in combination with 1, mg IV obinutuzumab over six cycles, or chlorambucil on days 1 and 15 of each cycle plus 1, mg IV obinutuzumab over six cycles.
At median follow-up of 31 months, patients who received ibrutinib-obinutuzumab achieved significantly longer median PFS not evaluable vs. The randomized phase 3 CLL14 trial is comparing that combination with the standard regimen of obinutuzumab plus chlorambucil in patients with treatment-naive CLL and coexisting medical conditions.
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Preliminary results reported by the manufacturer showed the trial met its primary endpoint of PFS, and in March the combination received breakthrough therapy designation for previously untreated CLL. Most would have predicted this, but the important part will be the details of the investigational arm and the depth of the remissions achieved. I assume this will hold up in the large phase 3 trial.
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The ultimate hope, according to Shanafelt, is that some of these newer combinations will allow for use of nonchemotherapy-based treatment in a time-limited way to achieve deep remissions. For instance, although some patients have demonstrated no evidence of CLL recurrence 8 years out from CAR T-cell therapy, experts agree that there is a need for better understanding of what makes a CAR T-cell therapy active and effective to reach more patients.
Gauthier said what will get the field closer to the concept of cure is achieving deeper responses in patients with high-risk CLL. This is what we are striving for. This will be the way forward. Researchers also aim to define the length of time patients can remain off therapy without relapse. Researchers seek to also show how using the drugs together affects the progression and lifespan of leukemia cells. For younger patients and those with high-risk molecular characteristics, however, the newer agents provide benefit but do not typically provide durable control of CLL long enough, Shanafelt said.
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For this population of patients, long-term disease control with low toxicity may be the right long-term goal, rather than a cure. N, Nashville, TN ; email: iflinn tnonc. N, Seattle, WA ; email: jgauthier fredhutch.
Major breakthrough in the treatment of leukemia
Brad S. Louis, MO ; email: bkahl wustl. David L. Jennifer A. Tell us what you think about Healio. Login Register My Saved. Cover Story. HemOnc Today, April 10, Please provide your email address to receive an email when new articles are posted on this topic. Receive an email when new articles are posted on this topic. You have already added this topic to your email alerts.
Click here to manage your alerts. Ibrutinib dominates Ibrutinib — a Bruton tyrosine kinase inhibitor first approved for previously treated CLL in — now is approved for use in all stages of the disease, offering the potential for chemotherapy-free treatment.