Cancer: Twitching in the body can be a key warning sign for three major cancers
Childhood Leukemia
Childhood leukemia, the most common type of cancer in children and teens, is a cancer of the white blood cells. Abnormal white blood cells form in the bone marrow. They quickly travel through the bloodstream and crowd out healthy cells. This raises the body's chances of infection and other problems.
As tough as it is for a child to have cancer, it's good to know that most children and teens with childhood leukemia can be successfully treated.
Doctors don't know exactly what causes most cases of childhood leukemia. But certain things may raise the chances of getting it. Keep in mind, though, that having one of these things does not necessarily mean a child will get leukemia. In fact, most children with leukemia don't have any known risk factors.
The risk for childhood leukemia increases if your child has:
Although the risk is small, doctors say children who have things that make leukemia more likely should get regular checkups to spot any problems early.
Almost all cases of childhood leukemia are acute, which means they develop rapidly. A tiny number are chronic and develop slowly.
Types of childhood leukemia include:
Symptoms of leukemia often prompt a visit to the doctor. This is a good thing, because it means the disease may be found earlier than it otherwise would. Early diagnosis can lead to more successful treatment.
Many signs and symptoms of childhood leukemia happen when leukemia cells crowd out normal cells.
Common symptoms include:
Other symptoms may include:
To diagnose childhood leukemia, the doctor will take a thorough medical history and do a physical exam. Tests are used to diagnose childhood leukemia as well as classify its type.
Initial tests may include:
A pathologist examines cells from the blood tests under a microscope. This specialist also checks bone marrow samples for the number of blood-forming cells and fat cells.
Other tests may be done to help determine which type of leukemia your child may have. These tests also help the doctors know how likely the leukemia is to respond to treatment.
Certain tests may be repeated later to see how your child responds to treatment.
Have an honest talk with your child's doctor and other members of the cancer care team about the best options for your child. Treatment depends mainly upon the type of leukemia as well as other things.
The survival rates for most types of childhood leukemia have gone up over time. And treatment at special centers for children and teens has the advantages of specialized care. Childhood cancers tend to respond to treatment better than adult cancers do, and children's bodies often tolerate treatment better.
Before cancer treatment begins, sometimes a child needs treatment to address illness complications. For example, changes in blood cells can lead to infections or severe bleeding and may affect the amount of oxygen reaching the body's tissues. Treatment may involve antibiotics, blood transfusions, or other measures to fight infection.
Chemotherapy is the main treatment for childhood leukemia. Your child will get anticancer drugs by mouth, or into a vein or the spinal fluid. To keep leukemia from returning, there may be maintenance therapy in cycles over a period of 2 or 3 years.
Sometimes, targeted therapy is also used. This therapy targets specific parts of cancer cells, working differently than standard chemotherapy. Effective for certain types of childhood leukemia, targeted therapy often has less severe side effects.
Other types of treatment may include radiation therapy. This uses high-energy radiation to kill cancer cells and shrink tumors. It can also help prevent or treat the spread of leukemia to other parts of the body. Surgery is rarely an option to treat childhood leukemia.
If standard treatment is likely to be less effective, a stem cell transplant may be the best option. It involves a transplant of blood-forming stem cells after whole body radiation combined with high-dose chemotherapy happens first to destroy the child's bone marrow.
The FDA has approved a type of gene therapy for children and young adults up to age 25 whose B-cell ALL doesn't get better with other treatments. Scientists are working on a version of this treatment for people over 25 and for other kinds of cancer.
CAR T-cell therapy uses some of your own immune cells, known as T cells, to treat your cancer. Doctors take the cells out of your blood and change them by adding new genes. The new T cells can work better to find and kill cancer cells.
What Do Leukemia Blood Counts Mean?
The results of a blood count can help diagnose or manage leukemia. They can also show how well your current treatment is working.
Leukemia is a type of blood cancer that begins in the bone marrow, where many blood cells are made. If you have leukemia, you may not make enough of some kinds of blood cells.
This cancer can also cause abnormal blood cells to form, crowding out other healthy blood cells in the bone marrow and spilling into the bloodstream.
Getting tested to find out your specific blood counts is important for diagnosing and managing leukemia.
Leukemia mainly affects white blood cells called lymphocytes. These immune cells help your body fight off infections.
When someone has leukemia, their body makes larger, abnormal cells called leukemic cells instead of lymphocytes and other necessary cells like red blood cells (to carry oxygen) and platelets (to help blood clot). This is why leukemia can cause symptoms like fatigue or excessive bleeding and bruising.
A healthcare professional may order a complete blood count test as a part of your routine medical checkup. This is because blood counts can tell a lot about your health. For example, too many white blood cells and too few red blood cells or platelet cells may mean that you have leukemia or your leukemia is not well-controlled.
Typical blood cell counts vary by age and gender. They can also temporarily change depending on whether you recently had a bad flu, infection, or even injury. The healthy blood cell ranges are shown in the table below per microliter (μl):
What CBC results indicate leukemia?If you have leukemia, your blood cell count may show higher than usual levels of white blood cells, which include leukemic cells.
However, you may also have a lower reduced number of white cells (leukopenia), red blood cells (anemia), or platelets (thrombocytopenia). If all three types are low, this is known as pancytopenia or cytopenia as follows per liter (L) or in grams per deciliter (g/dL).
That said, any value for these markers that's outside of the healthy range can be considered leukemia if your doctor cannot find a different cause for it, such as infection, for example. Your exact values also depend on the type of leukemia you have.
If you have higher than healthy levels of white blood cells and low counts of red blood cells and platelets, the doctor will order additional tests to get more information.
If you're having symptoms, this might reinforce the doctor's suspicion. These may may include:
If you have any or all of these symptoms, you may need certain imaging tests, including:
Your leukemia treatment plan will take several factors into consideration, such as your:
Your doctor will let you know how often you'll need to return for more blood tests. If you are diagnosed with leukemia, you may need regular physical exams and blood tests, so your doctor can watch for signs of disease progression or remission.
How well treatment is workingRegular leukemia blood count tests, such as complete blood count, are very important in the treatment process because they can help show:
Leukemia blood cell counts also help to show whether you need additional treatments. For example:
Leukemia is a type of blood and bone marrow cancer. You may not notice symptoms for a long time, if at all. This is why a complete blood count test can help show imbalances in your blood cells.
Different types of leukemia can cause your blood cell counts to be higher or lower than expected. If you have been diagnosed with leukemia, blood cell count tests can also indicate how well your treatment is working and if adjustments are needed.
European Medicines Agency (EMA) Grants Orphan Drug Designation For Moleculin's Treatment Of Acute Myeloid Leukemia (AML)
– Next-generation non-cardiotoxic anthracycline Annamycin with notable signs of clinical efficacy in AML in combination with Cytarabine achieves preliminary CRc rate of 60% in 2nd line AML subjects in a European clinical trial
– There are approximately 160,000 people with AML worldwide
– Annamycin continues to show no signs of cardiotoxicity (N=82 across multiple studies); Lower toxicity profile than traditional intensive therapy
– Annamycin is advancing towards pivotal AML study in 2024 and may qualify for an accelerated approval pathway
HOUSTON, April 18, 2024 /PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to Annamycin for the treatment of AML.
Moleculin Biotech, Inc. Is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)
Annamycin is the Company's next-generation anthracycline that has been designed to be non-cardiotoxic (unlike currently prescribed anthracyclines) and has shown to be non-cardiotoxic in the 82 subjects treated in multiple studies in the U.S. And in Europe. Furthermore, Annamycin has recently shown in Moleculin's European clinical study for the treatment of AML using Annamycin in combination with Cytarabine (MB-106) a preliminary 60% composite complete response (CRc) rate in 2nd line subjects (N=10) and an overall interim CRc of 39% in all subjects (N=18), regardless of the prior number of therapies, in the European trial. Durability of the CRc's is developing. One subject has surpassed the one-year mark with a durable complete response. Recruitment in MB-106 has ended for 2nd line subjects while recruitment for 1st line and 3rd line subjects continue. Annamycin is currently in development for the treatment for AML and STS lung mets, and the Company believes the drug may have the potential to treat additional indications.
"We are pleased to receive Orphan Drug Designation from the EMA for Annamycin, which further supports our ongoing efforts to advance this compelling next-generation anthracycline for the treatment of hard-to-treat cancers," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Combined with the Orphan Drug Designation we already have in the US and with the new composition of matter patent just awarded by the US Patent and Trademark Office with coverage through 2040, we believe the commercial exclusivity of Annamycin is now very well protected. We continue to be encouraged by the growing body of promising interim clinical data demonstrated by Annamycin. We remain focused on advancing our clinical and regulatory strategies toward our next phase of development, including the planned commencement of a pivotal registration study as a 2nd line therapy in AML before year end."
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The EMA grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and up to 10 years of potential market exclusivity in the European Union if approved.
Annamycin currently has Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of relapsed or refractory AML, in addition to Orphan Drug Designation for the treatment of STS lung mets.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. Is a clinical stage pharmaceutical company with a growing pipeline, including Phase 2 clinical programs, for hard-to-treat tumors and viruses. The Company's lead program, Annamycin is a next-generation anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. All interim and preliminary data discussed above is subject to change.
Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers, and WP1220, an analog to WP1066, for the topical treatment of cutaneous T-cell lymphoma. Moleculin is also engaged in the development of a portfolio of antimetabolites, including WP1122 for the potential treatment of viruses, as well as cancer indications including brain tumors, pancreatic and other cancers.
For more information about the Company, please visit www.Moleculin.Com and connect on Twitter, LinkedIn and Facebook.
Forward-Looking Statements
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the timing of the commencement of a pivotal registration study of Annamycin as a 2nd line therapy in AML before year end. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward-looking statements by terminology including 'believes,' 'estimates,' 'anticipates,' 'expects,' 'plans,' 'projects,' 'intends,' 'potential,' 'may,' 'could,' 'might,' 'will,' 'should,' 'approximately' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
Investor Contact:JTC Team, LLCJenene Thomas(833) 475-8247MBRX@jtcir.Com
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SOURCE Moleculin Biotech, Inc.
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