Let’s start this article about POlycythemia vera with Tuesday’s news about approval of a Stage 3 trial design for a new drug, INCB18424:
WILMINGTON, Del., Sep 13, 2010 (BUSINESS WIRE) — Incyte Corporation announced today that it has reached agreement with the U.S. Food and Drug Administration (FDA) regarding a Special Protocol Assessment (SPA) for the design of a pivotal Phase III trial for its JAK1 and JAK2 inhibitor, INCB18424 in patients with polycythemia vera (PV), a blood cancer that belongs to a group of diseases known as myeloproliferative neoplasms (MPNs). Two Phase III trials of INCB18424 in myelofibrosis, also an MPN, COMFORT-I and COMFORT-II, are already fully enrolled and are expected to be completed later this year.
RESPONSE (Randomized, open label, multicenter phase III study of Efficacy and Safety in POlycythemia vera subjects who are resistant to or intolerant of hydroxyurea: JAK iNhibitor INC424 tablets verSus bEst available care) is a global study conducted by Incyte in the US and Novartis in rest of world and is expected to enroll approximately 300 patients with PV who are resistant to or intolerant of hydroxyurea (HU). RESPONSE will compare the efficacy and safety of INCB18424 to the physician’s choice of best available therapy. Patient enrollment in the US is expected to begin in October (www.responsetrial.com).
“Securing the SPA for INCB18424 in PV establishes the requirements we must meet to obtain approval in this second indication and supports our objective to expand beyond myelofibrosis,” stated Paul A. Friedman, M.D., Incyte’s President and CEO. “The encouraging results from the ongoing Phase II PV trial, combined with the data we anticipate achieving from RESPONSE, have the potential to clearly establish the long-term safety and efficacy of INCB18424 and optimally position the compound for use in these underserved patients.”
Srdan Verstovsek, M.D., Ph.D., Associate Professor, Leukemia Department, Myeloproliferative Disorders Program Leader, University of Texas M.D. Anderson Cancer Center, and the US principal investigator for RESPONSE, stated, “Patients with advanced PV represent a particularly high-risk group with few therapeutic options for long-term care. These patients would benefit from new chronic therapies that safely and effectively address the aberrant hematological measures, enlarged spleens and significant disease-related symptoms that so negatively impact their lives. In previous trials, INCB18424 has been well tolerated in patients with advanced PV, and much sicker patients with myelofibrosis for as long as 30 months. In the Phase II trial involving 34 PV patients who were HU resistant or HU intolerant, INCB18424 provided rapid and durable activity in these patients. We look forward to evaluating INCB18424 in this Phase III trial designed in agreement with FDA.”
After reading this news release I stopped and asked myself: What the heck is POlycythemia vera? Here is a short primer I found on Google Health:
Polycythemia vera is an abnormal increase in the number of blood cells (primarily red blood cells) produced by the bone marrow.
Breathing difficulty when lying down
Fullness in the left upper abdomen
Itchiness, especially after a warm bath
Red coloring, especially of the face
Shortness of breath
Symptoms of phlebitis
Note: Symptoms are due to increased blood thickness and clotting.
Other symptoms that may occur with this disease:
Bluish skin discoloration
Red skin spots
The goal of treatment is to reduce the thickness of the blood and prevent bleeding and clotting.
A method called phlebotomy is used to decrease blood thickness. One pint of blood is removed weekly until the hematocrit level is less than 45, then therapy is continued as needed.
Occasionally, chemotherapy (specifically hydroxyurea) may be given to suppress the bone marrow. Interferon may also be given in an attempt to lower blood counts. A medicine called anegrelide may be given to lower platelet counts.
The use of blood thinners (such as aspirin) is controversial because it may cause stomach bleeding. However, it does prevent blood clots.
Polycythemia vera is a disorder of the bone marrow. It causes too much production of white blood cells, red blood cells, and platelets.
It is a rare disease that occurs more often in men than women, and is rare in patients under age 40. The exact cause is unknown.
Tests & diagnosis
The health care provider will perform a physical exam. Tests that may be done include:
Bone marrow biopsy
Complete blood count with differential
Vitamin B12 level
This disease may also affect the results of the following tests:
Leukocyte alkaline phosphatase
Platelet aggregation test
Serum uric acid
The disease usually develops slowly. Most patients do not experience any problems related to the disease after being diagnosed.
The following complications occur in a small number of patients:
The abnormal bone marrow cells may begin to grow uncontrollably in some patients, leading to the development of acute myelogenous leukemia (AML).
The bone marrow may develop a scarring condition called myelofibrosis.This condition may lead to dangerously low levels of white blood cells, red blood cells, and platelets.
Patients with polycythemia vera are also more likely to form blood clots that can cause strokes or heart attacks. Some patients may experience abnormal bleeding because their platelets are abnormal.
Bleeding from the stomach or other parts of the intestinal tract
Peptic ulcer disease
As a multiple myeloma patient/survivor, I think I understand how it feels to be living with a incurable, chronic cancer like POlycythemia vera. I hope this drug proves helpful as it is developed in the future.
Feel good and keep smiling! Pat