PDSA E-News: March 27, 2017




ITP Treatment PRTX-100 Granted Orphan Drug Designation

Protalex logoThe U.S. Food and Drug Administration (FDA) has granted pharmaceutical company Protalex, Inc. orphan drug designation for its new ITP therapy “PRTX-100.” Drug companies receive the special designation to exclusively develop therapies for a specific disease, especially rare disorders. PRTX-100 is a purified form of a protein that alters the immune system by moderating the immune response by B-lymphocytes and macrophages, which secrete antibodies and destroy pathogens, respectively.

PRTX-100 has already successfully completed two initial dose escalation trials, demonstrating that the drug is safe and effective in ITP patients; both clinical trials will continue to enroll patients at a higher dosage. The therapy is also being tested in another dose escalation study in chronic ITP patients who have been previously treated with a TPO-agent, such as Promacta® or Nplate®, and at least one other ITP therapy; patients will receive PRTX-100 intravenously once per week for four weeks. They will be monitored for 48 weeks for platelet count response, number of days until response, durability of response, and potential adverse events. If the therapy proves successful, ITP patients can rejoice in knowing that another ITP treatment will be available for public use soon.

More information about the trial can be found here: http://bit.ly/2mWh0KL

Black, Andrew. “Immune Thrombocytopenia Treatment Granted Orphan Drug Designation.” Rare Disease Report. 2/21/17.

Low-dose IL-2 Increases Regulatory T Cells and Elevates Platelets in an ITP Patient


Past research has found that patients with immune thrombocytopenia (ITP) have fewer regulatory T cells (Tregs) than patients without the disease. Also, people who are in remission from ITP have increased Tregs. In addition, recent research studies found that low-dose IL-2, a synthetic form of interleukin-2 (a protein that the body produces naturally) increased Tregs. IL-2 has been used to treat other autoimmune diseases and after organ transplants. Knowing these facts, researchers decided to treat a 36-year-old woman with ITP by giving her a low dose of interleukin-2 (IL-2) for five consecutive days per week for four weeks. They wanted to see if her ITP improved after receiving the IL-2. The results showed that low-dose IL-2 expanded Tregs significantly, as her platelet counts rose from 36,000 to 85,000. No side effects of the low-dose IL-2 were observed.

The researchers concluded that possibly IL-2, by raising Treg levels in ITP patients may be another approach for treatment of ITP. This is a very preliminary result with only one patient involved so further studies will be needed to confirm these results. The same results cannot be guaranteed in other studies. However, the simplicity and lack of side effects from the treatment are encouraging.

Zhang J, Ruan Y, Shen Y, et al. “Low dose IL-2 increase regulatory T-cells and elevate platelets in a patient with immune thrombocytopenia.” Cytometry B. Clin. Cytom., 2016 Nov. 9. Doi: 10.1002/cyto.b.21494. [Epub ahead of print].


HHS and NIH Issue New Registration and Results Dissemination Rules for Clinical Trials

clinical trial1The U.S. Department of Health and Human Services (HHS) now operates under a new policy for clinical trials. This makes participation and study results more accessible to the public. The new rule specifically defines new legal requirements for registering clinical trials for drug, biological, and device products and publishing analyses on ClinicalTrials.gov.

The National Institutes of Health (NIH) Director Francis Collins, MD, PhD, expressed strong support for the new policies, explaining that “access to more information about clinical trials is good for patients, the public and science. The final rule and NIH policy… “will help maximize the value of clinical trials, whether publicly or privately supported, and help us honor our commitments to trial participants, who do so much to help society advance knowledge and improve health.” The NIH has issued a complementary policy to HHS, stipulating that NIH-funded trials and their results must be catalogued using the newly updated ClinicalTrials.gov as well.

These changes will not only impose legal consequences for incorrectly reporting results, adverse events, and non-compliance to studies, but will also enhance public accessibility to discover information about trials in which patients might be eligible to participate. These new regulations can only assist in further educating patients and healthcare providers to make more informed decisions about their health.
National Institutes of Health. “HHS Takes Steps to Provide More Information about Clinical Trials to the Public.” The National Institutes of Health. 9/16/16.



Vitamin D Reduced Incidence of Colds and Flu Better than Flu Vaccine

Vitamin D pillWe know from research that many cases of ITP started after the person experienced a cold or flu. To avoid the flu most people are advised to get annual flu vaccines. Now maybe there’s something else that can help. A recent scientific review of 25 randomized controlled trials has confirmed that vitamin D supplementation boosts immunity and cuts rates of flu and colds. The meta-analysis included almost 11,000 individuals (ages 1 – 95) from more than 12 countries.

Acute respiratory infections have led to millions of emergency room visits each year in the U.S. according to Carlos Camargo, Professor of Epidemiology at Harvard University and senior author of the study. The findings stated, “People who took daily or weekly vitamin D supplements were less likely to report acute respiratory infections, like influenza or the common cold, than those who did not.” People with the most vitamin D deficiencies (D blood levels below 10 ng/mL) who took a supplement cut their risk of respiratory infection in half. Average daily doses of vitamin D used in the studies ranged from 800 IU to 2,000 IU.

Researchers believe vitamin D offers protection by increasing antimicrobial peptides in the lungs and that may be the reason colds and flus are more common in the winter, when sunlight exposure (thus the body’s natural production of vitamin D) is lowest. The researchers estimated that vitamin D supplementation could prevent more than 3.25 million cases of cold and flu in the UK alone. Another statistic showed that vitamin D supplementation is a more effective strategy than the flu vaccine, based on the concept of “number needed to treat” (NNT). Researchers said one person would be spared from influenza for every 33 people taking a D supplement (NNT = 33), whereas 40 people have to receive the flu vaccine in order to prevent one case of the flu (NNT + 40). For patients with severe vitamin D deficiency at baseline, the NNT was 4. So, for patients who were vitamin D-deficient to begin with taking a vitamin D supplement was 10 times more effective than getting the flu vaccine.

You can increase your vitamin D levels by including fish like tuna, mackerel, and salmon, eggs, and more vitamin D fortified foods (such as cereal, milk and orange juice) in your diet; and by sensible sun exposure whenever possible. However, because of geographic location and working indoors many people do not get enough sun exposure and most likely won’t get enough D through foods. Taking a D supplement may be the best way to raise their D levels. Past research found vitamin D deficiency has been linked with autoimmune diseases as well as other chronic illnesses. Diagnostic testing for vitamin D levels indicates these results:
<20 ng/mL -- Deficiency; 20-29 ng/mL – Insufficiency, and ≥30 ng/mL – Optimal.

Martineau A, Jolliffe D, Hooper R, et al. “Vitamin D Supplementation to Prevent Acute Respiratory Tract Infections: Systematic Review and Meta-analysis of Individual Participant Data.” BMJ 2017; 356: i6583.

Aubrey, Allison. “A Bit More Vitamin D Might Help Prevent Colds and Flu.” Morning Edition, NPR, Feb. 16, 2017.



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