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This is in contrast to other blood cancers where novel treatments have turned fatal conditions into chronic diseases with long term remissions. Prominent examples are CML, which today has a low mortality rate and multiple myeloma where median survival is approaching 8 years and counting.
The recent launch of Imbruvica and Gazyva followed by the anticipated approval of ABT is expected to have a similar impact on CLL as well as on certain subtypes of B cell lymphoma.
Light at the end of the tunnel Prognosis for newly diagnosed AML patients is grim, especially in older patients, with bone marrow transplant as the only potentially curative option. AML is still treated with chemotherapy, which has significant toxicity issues as well as limited impact on survival if not followed by transplant.
All attempts to introduce novel targeted therapies for AML failed miserably. Today, for the first time, there is reason for cautious optimism thanks to a growing pipeline that employs a multitude of therapeutic approaches.
Consequently, researchers can tailor drugs for specific AML subsets on the one hand and introduce more potent antibody based treatments on the other.
Below is a recap of the most promising AML drugs in development.
Although most programs are still years from approval, many drugs should generate significant clinical data by YE Targeting driver mutations One of the striking differences between the treatment paradigm of solid and liquid tumors is the role of biomarkers for patient selection.
In contrast, breakthrough drugs for blood cancers Revlimid, Imbruvica, Velcade, Rituxan are given to the entire patient population without the need of a companion diagnostic.
In that sense, the current strategy in AML resembles the solid tumor approach, as the three most promising drugs are being studied in biomarker-defined subgroups of AML. Notably, most responses appear durable, which is unusual in AML as in most cases the effect is lost or treatment becomes too toxic for the patient.
Another way to get exposure to the Agios story is through Foundation Medicine, which will sell its diagnostic test as a companion diagnostic for both AG and AG Foundation Medicine is collaborating with other companies, which plan to use a test from FMI as companion diagnostic for their drugs.
Clear efficacy as monotherapy, a biomarker-defined population, good safety profile, regulatory visibility and a favorable phase III trial design.
Based on the large phase II experience, more than a third of the patients were bridged to transplant, which is the only option for achieving long term remission. Today, physicians use intensive chemotherapy as a bridge to transplant but success rates are lower and in many cases patients are too old and frail to tolerate side effects.
In this population, the ultimate goal is reaching transplant in eligible patients. The protocol also allows patients on the quizartinib arm to re-initiate quizartinib treatment after transplant.
The company expects to present data in the maintenance setting at ASH and management already hinted results are promising. For a company which would like to establish a presence in AML and is willing to fund a broad development program, quizartinib is the only high-quality late-stage asset available.
This makes quizartinib an attractive asset as biopharma companies are anxious to fill their advanced stage pipelines.
Epizyme — Waiting for updated data in Epizyme develops inhibitors for HMTs histone methyltransferasesa group of enzymes that play a role in regulation of gene expression.
The company is focusing on oncogenic HMTs that function as growth drivers in cancer. The drug has been licensed to Celgene in The company plans to present updated results later inwhich will hopefully show more robust activity at higher doses with more intensive dosing regimens.
Epizyme already disclosed 2 objective responses within one of the cohorts unclear whether responses were in AML as the trial enrolls patients with additional tumor types.
The company recently disclosed encouraging signs of efficacy early in phase I, including PRs in two patients with B cell lymphomas.
Antibody drug conjugates It is hard to predict what approaches will be the winners in AML but one can safely assume more chemotherapy is not the answer.Wrinkle Cream At Target Skin Beauty Clinic Top Korean Skin Care Brands Wrinkle Cream At Target Best Skin Care Products For Acne And Blackheads.
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