Moleculin Biotech, Inc. is a pre-clinical and clinical-stage pharmaceutical company focused on the development of game-changing anti-cancer drug candidates, many of which are based on discoveries made at M.D. Anderson Cancer Center. Each of our projects represents a breakthrough discovery and a highly disruptive technology.

About Childhood Leukemia

child-cancerPROBLEM: Cardiotoxicity and multidrug resistance severely limit pediatric oncologists’ ability to effectively treat children suffering from acute leukemia.

SOLUTION: We believe Annamycin’s ability to avoid both cardiotoxicity and multidrug resistance has the potential to eliminate this critical problem.


One of the most exciting scientific discoveries here at Moleculin, which we believe will have a profound impact on the treatment of children suffering from Acute Leukemia, is our lead drug Annamycin’s ability to be non-cardiotoxic.

We believe that this characteristic alone has the ability to change the way pediatric oncologists’ treat children suffering from acute leukemia for the foreseeable future.

Cardiotoxicity can be a killer, causing severe, permanent and sometimes fatal damage to the heart of both adults and children and cardiotoxicity is highly present in the currently approved drugs used to treat all acute leukemia patients. The drugs that are used to treat acute leukemia patients are called “anthracyclines.”

Oncologists (and especially pediatric oncologists) are limited in how they can treat their patients, due to the harmful effects of cardiotoxicity.

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Here are some quotes from other publications:

“The effectiveness of anthracyclines and related compounds (doxorubicin, daunorubicin, idarubicin, epirubicin, and the anthraquinone mitoxantrone) is hindered by their cardiotoxicity.” – Cardiotoxicity of anthracycline-like chemotherapy agents

“Anthracyclines remain an important class of drugs in the treatment of cancer, but also remains a problematic chemotherapeutic agent given their cardiotoxic effects.” – Anthracycline Cardiotoxicity: Prevalence, Pathogenesis and Treatment

To better understand how cardiotoxicity impacts children suffering from acute leukemia, consider that acute leukemia is the most common form of cancer in children, comprising approximately 30 percent of all childhood malignancies. Of the acute leukemias, acute lymphoblastic leukemia (ALL) occurs more commonly than acute myeloid leukemia (AML), however the treatment of both typically involves the same induction therapy (the preparation for a bone marrow transplant) used in adults to reduce the presence of leukemic cells in the patient’s bone marrow and bloodstream in preparation for a curative bone marrow transplant.

Most unfortunately for children, however, the standard drug used in such induction therapies is significantly cardiotoxic.

This often means that even though the chances are often good that current treatments can cure a child’s leukemia, it is very likely that the child’s lifespan will be shortened and their quality of life diminished as a result of permanent heart damage caused by current therapies.

This is why we believe Annamycin’s lack of cardiotoxicity has the ability to change the way children are treated for the foreseeable future.

Annamycin has been shown to be non-cardiotoxic in animal models, when compared with one of the leading anthracyclines (doxorubicin).

To make matters worse, the leading induction therapy drugs lose efficacy due to something called “multidrug resistance” requiring more and more drug to try and prepare patients for a successful bone marrow transplant, but because of the harmful effects of cardiotoxicity, patients simply cannot tolerate an increase in dosage.  At the same time that Annamycin avoids cardiotoxicity, it also avoids multidrug resistance, and as a result, Annamycin has demonstrated in early clinical trials the ability to deliver efficacy in patients who have failed repeated attempts with the existing induction therapy drugs.

Oncologists, and especially Pediatric Oncologists, have been clear that there is a tremendous unmet need here and we believe Annamycin has the potential to fill that need and change forever how children with leukemia are treated.  The severity of this unmet need may allow us to position Annamycin for accelerated approval under FDA guidelines and we will be working hard this year to secure an accelerated approval pathway. We look forward to moving quickly to translate this important breakthrough into new hope for those children who have run out of options.

Here are some interesting articles on the effects of cardiotoxicity:

Risk of heart damage follows some childhood cancer survivors

Woman battling breast cancer diagnosed with heart failure

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