LEXINGTON, Mass., March 24, 2021 / PRNewswire / – Partner Therapeutics, Inc. (PTx), a commercial biotechnology company, announces publication of results from an investigator-initiated clinical trial (NCT01409915) evaluating the use of Leukine® (sargramostim, yeast-based rhuGM-CSF) in patients with moderate AD- moderately. Participants were evaluated for safety and tolerability, as well as cognitive function and AD pathology symptoms. Dr. Huntington Potter, with Jonathan Woodcock, Timothy Boyd and colleagues at the University of Colorado (CU) The Alzheimer’s and Cognition Center published their findings in Alzheimer’s & Dementia: Interventional Research and Clinical Interventions, an open access journal of the Alzheimer ‘s Society (“Safety and Efficacy of GM-CSF (Sargramostim) in the Treatment of Alzheimer’s Disease“(Potter, Woodcock, Boyd, et al.); DOI: 10.1002 / trc2.12158.
According to Dr. Potter, “The goal of the clinical trial was to investigate the effect of a natural human protein called granulocyte-macrophage colon stimulatory factor (GM-CSF) on people living with Alzheimer’s disease. We conducted a trial on Leukine because of people with rheumatoid arthritis is less likely to get Alzheimer’s disease and we had previously detected GM-CSF, which is increased in the blood of people with rheumatoid arthritis, reduced amyloid deposition in Alzheimer’s mice and returned the normal memory to normal after a few weeks of treatment. Thus, naturally higher levels of GM-CSF in people with rheumatoid arthritis may be one reason they are protected from Alzheimer’s disease. Human GM-CSF is the active fertilizer in the well-known human drug Leukine, and we are the first to study its effect on people with Alzheimer’s disease. “
The researchers conducted a randomized, double-blind, placebo-controlled trial to test the safety and efficacy of Leukine treatment in participants with moderate-to-severe Alzheimer’s disease. Participants who met eligibility criteria were randomized to receive an injection of both Leukine (20 patients received Leukine at a dose of 250 mcg / m2/ day subcutaneous injection for five days a week for three weeks) or placebo (20 patients took saline for five days a week for three weeks). Most of the study participants were recruited and treated at CU Anschutz by three from University of South Florida.
The researchers CU Anschutz studied and analyzed several neurological, neuropsychological, cell, cytokine, Alzheimer’s pathology biomarkers, and neuroimaging assessments. They found that short-term treatment with Leukine:
- it was safe and patient with participants and the most common adverse events were associated with dermatological Leukine (16 for Leukine vs. 5 for placebo), gastrointestinal (8 for Leukine vs. 5 for placebo), and headache ( 8 for Leukine vs. 2 for placebo), as would be expected for this medication; side effects were consistent with Leukine label and there were no adverse events associated with study drug;
- showed a significant change in cognitive loss, as measured by the MMSE (Minor State Mental Examination, AD assessment): cognitive memory measures improved by nearly 2 points in the 30 MMSE points compared to baseline (P = 0.0074); Patients with leukine treatment performed better than patients with placebo treatment (P = 0.0370) after three weeks of treatment; this improvement lasted up to 45 days after treatment (P = 0.0272);
- blood-based biomarkers with AD toward normal levels; Abeta40 amyloid plasma signal (decreased in AD) increased 10% (p = 0.0105), and plasma signals of neurodegeneration (Tau total and UCH-L1) decreased 24% (p = 0.0174) and 42% (p = 0.0019), individually, after treatment with Leukine versus placebo; and
- increased tissue and other immune cells and altered cytokine levels.
“These findings suggest that short-term treatment of Leukine leads to activation of the immune system, improvement of psychology and memory, and partial normalization of blood stages of amyloid and tau pathology and neuronal damage in com. partners with moderate-to-severe Alzheimer’s disease, “said Dr. Potter. He said, “Perhaps this remarkable discovery may have stimulated the immune system a new approach that led us to embark on a larger trial of Leukine in Alzheimer ‘s disease with more participants on the line. treatment over a longer period of time.This new trial is funded by the Alzheimer’s Society / Part The Cloud, the University of Colorado, the Global Down Syndrome Foundation and a recent major grant from the National Institute on Aging ”.
“Leukine has unique immunomodulatory properties and we commend Professor Potter and his team for taking their ideas in rheumatoid arthritis and“ chemo-brain ”and applying them to Alzheimer’s disease, “he said Roasowdhury Debasish, PTx Chief Technology Officer. “The study was conducted rigorously with several measures aimed at examining the impact on the clinical and pathology parameters while also seeing the underlying mechanism. We are excited to see that Leukine may , even in the short term, the transformation of disease to a growing research group reveals the potential benefits of the effects of Leukine on resident macrophages, such as microglial cells in AD or alveolar macrophages in hard aPAP or COVID-19. “
Leukine is not approved for the treatment of Alzheimer’s disease.
FINALLY LEUKINE
LEUKINE® (sargramostim) is an FDA-approved granulocyte-macrophage human colonization factor (rhu GM-CSF). GM-CSF is a naturally occurring protein called cytokine that plays an important role in myeloid hematopoiesis, immunomodulation, and cell remodeling. Leukine is designated as an Essential Medicine by the FDA and is held by the U.S. Government in the National Strategic Store. Leukine is available outside United States through a Designated Patient Program administered by Tanner Pharma Group.
Leukine has identified:
- To shorten time to neutrophil regeneration and reduce the incidence of life-threatening and life-threatening diseases after induction chemotherapy in adult patients 55 years and older with acute myeloid leukemia (AML).
- For transfusion of hematopoietic progenitor cells into peripheral blood for accumulation by leukapheresis and autologous transplantation in adult patients.
- To accelerate myeloid regeneration after autologous bone marrow or peripheral blood progenitor cell transplantation in adult and pediatric patients 2 years of age and older.
- To accelerate myeloid regeneration after allogeneic bone marrow transplantation in adult and pediatric patients 2 years of age and older.
- For the treatment of delayed neutrophil or graft failure after autologous or allogeneic bone marrow transplantation in adult and pediatric patients 2 years of age and older.
- To increase survival in adult and pediatric patients from birth to 17 years of age exposed to doses of myelosuppressive radiation (Hematopoietic Syndrome of Acute Radiation Syndrome [H-ARS]).
Important safety information for Leukine (sargramostim)
Contraindications
- LEUKINE is contraindicated in patients with sensitization to human granulocyte-macrophage colony as a stimulatory factor such as sargramostim (GM-CSF), yeast-derived products, or any component of LEUKINE.
Warnings and warnings
- Adverse hypersensitivity reactions, including anaphylactic reactions, have been reported with LEUKINE. If a severe sensory or anaphylactic reaction occurs, discontinue LEUKINE therapy immediately and initiate medical management. Discontinue LEUKINE permanently in patients with severe allergic reactions.
- LEUKINE can cause infusion-related reactions, including respiratory distress, hypoxia, water flow, hypotension, syncope and / or tachycardia. Watch closely during irrigation, especially in patients with preexisting lung disease, as dose adjustment or discontinuation may be required.
- Do not administer LEUKINE concurrently with or within 24 hours before cytotoxic chemotherapy or radiotherapy or within 24 hours after chemotherapy.
- Edema, capillary leak syndrome, pleural effusion and / or pericardial effusion have been reported in patients after LEUKINE administration. LEUKINE should be used with caution and monitored in patients with preexisting fluid retention, lung infiltrates, or congestive heart failure.
- Supraventricular arrhythmia has been reported in uncontrolled studies during LEUKINE administration, especially in patients with a previous history of cardiac arrhythmia. Use LEUKINE with caution in patients with preexisting heart disease.
- If ANC is> 20,000 cells / mm3 or if WBC counts> 50,000 / mm3, The administration of LEUKINE should be discontinued or the dose reduced by half. CBC should be checked twice a week with a difference.
- LEUKINE treatment should be discontinued if disease progression is detected during treatment.
- Treatment with LEUKINE may neutralize anti-drug antibodies. Use LEUKINE for the shortest time required.
- Liquid solutions containing benzyl alcohol (including LEUKINE injection) or LEUKINE for injection should not be reconstituted with bacteriostatic water for injection, USP (0.9% benzyl alcohol) should not be given to infants and low birth weight babies.
- Concomitant use of drugs that may induce myeloproliferative effects of LEUKINE should be avoided.
Harmful responses
Adverse events occurring in> 10% of patients receiving LEUKINE in controlled clinical trials and reported at a higher frequency than placebo are:
- In patients with autologous bone marrow transplantation (BMT) – asthenia, malaise, diarrhea, broth, peripheral edema, urinary tract disorders
- In BMT Allogeneic patients – abdominal pain, chills, chest pain, diarrhea, nausea, vomiting, hematemesis, dysphagia, GI hemorrhage, pruritus, bone pain, arthralgia, eye hemorrhage, hip tension, tachycardia, bilirubinemia, hyperglycemia, more creatinine, hypomagnesemia, edema, pharyngitis, epistaxis, dyspnea, insomnia, anxiety, high glucose, low albumin
- In AML patients – fever, weight loss, nausea, vomiting, anorexia, skin reactions, metabolic disorders, edema
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SOURCE Partner Therapeutics, Inc.
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