ISIS Announces Phase I trials For Myotonic Dystrophy Type 1 Drug

Happy Days! A clinical Trial is going to start on Phase one of Myotonic Dystrophy Drug. That means that a small number of patients in the US usually under 50 will get a chance to try the drug. The purpose of the Phase I trial is to

Try out the drug
Find an optimum dose
Assess Safety of Drug

More on what a phases of clinical testing mean  (From MD Anderson site)

What are Phase 1 Clinical Trials?

Developing new drugs involves four phases of clinical testing in cancer patients, ranging from Phase 1 to Phase 4. Phase 1 trials are the first stage of clinical testing and often involve drugs that have been tested extensively in the laboratory and on animals with encouraging results, but have not yet been given to humans. Patients in Phase 1 trials are sometimes the first to try new  drugs.

clinical trials phases graph

Phase 1 trials only enroll a small number of participants, usually 15 to 100 patients, most of whom have advanced cancer that has not responded to standard cancer treatments. Trial participants are divided into small groups, known as cohorts. The first cohort receives a low dose of the new drug. Doctors may collect blood or urine samples to measure drug levels in the patients.

If the first cohort does not have any severe side effects, then a new cohort receives a higher dose of the same drug. The dose increases until the trial investigators find the best dose for future testing. With each increasing dose, doctors test each patient to see if he or she is responding to the treatment. If the doctors find that the treatment is safe, then it will advance to a Phase 2 trial.

The objectives of  Phase 1 trials include: 

  • Understanding the side effects of new drugs
  • Determining how the drug affects  cells in patients
  • Observing patient response to the drug

At MD Anderson, drugs used in Phase 1 trials may have been developed by pharmaceutical or biotechnology companies. Some Phase 1 trials test new uses for drugs that have already been approved by the Food and Drug Administration, or test drugs given for the first time in combination. Drugs may also be administered in different ways, such as by regional therapy (infusing the directly into the liver for patients with liver metastases).

Some trials may be performed in collaboration with the National Cancer Institute (NCI) or the National Cancer Comprehensive Network (NCCN).  Many trials require frequent return visits to the Clinical Center for Targeted Therapy, mainly for safety monitoring.

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Published on June 9, 2014 at 2:34 PM · No Comments

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has initiated a Phase 1 study for ISIS-DMPKRx. Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement. ISIS-DMPKRx is designed to reduce the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells, including muscle cells, for the treatment of Myotonic Dystrophy Type 1 (DM1).

“ISIS-DMPKRx is an example of the broad applicability of our antisense technology to develop novel drugs to treat patients with severe and rare diseases. ISIS-DMPKRx is the first drug to enter our pipeline that is designed to target a toxic RNA, the first systemically administered drug to enter development from our Biogen Idec partnerships and the second generation 2.5 drug to enter clinical development,” said C. Frank Bennett, Ph.D., senior vice president of research at Isis. “Myotonic dystrophy represents an ideal opportunity for antisense as the disease-causing gene produces a toxic RNA, which is not accessible by traditional therapeutic approaches but is uniquely accessible with our antisense technology. We look forward to rapidly advancing the development of ISIS-DMPKRx.”

“Our collaboration with Biogen Idec has been very productive. ISIS-DMPKRx has rapidly advanced to the clinic, and we continue to make progress across the board in our drug discovery programs with Biogen Idec. All of these successes substantially advance our neuromuscular disease franchise and translate into the potential for significant revenue as our drugs and programs progress,” said B. Lynne Parshall, chief operating officer at Isis.

DM1 is a rare genetic neuromuscular disease characterized by progressive muscle atrophy, weakness and muscle spasms. DM1, the most common form of muscular dystrophy in adults, affects approximately 150,000 patients in the United States, Europe and Japan. Patients with DM1 have a genetic defect in their DMPK gene in which a sequence of three nucleotides repeats extensively, creating an abnormally long toxic RNA, which accumulates in the nucleus of cells and prevents the production of proteins needed for normal cellular function. The number of triplet repeats increases from one generation to the next, resulting in the possibility of more severe disease in each subsequent generation. There are currently no disease-modifying therapies that address more than one symptom of the disease. ISIS-DMPKRx is designed to improve the underlying genetic defect that causes DM1.

“Myotonic dystrophy is a progressive and debilitating disease that affects thousands of patients for whom there are no direct therapeutic options. The innovative science behind ISIS-DMPKRx is compelling and targets the underlying genetic defect that causes myotonic dystrophy,” said Molly White, executive director of the Myotonic Dystrophy Foundation. “ISIS- DMPKRx has a chance to fill the therapeutic void for DM1 patients and transform the hopes and future of thousands of patients and families.”

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2 thoughts on “ISIS Announces Phase I trials For Myotonic Dystrophy Type 1 Drug

  1. my 6yr. old grandson has myotonic dystrophy. he was born with it and got that gene from his mom, my daughter, who got it from her biological mom who recently died at the age of 60. we looked in to stem cell research and anything else that we could try while he’s still young and it is not full blown I’m now just trying to find a clinical trial with the Antisense that he could enroll in.

  2. Having a husband and 2 daughters with MD 1 I ‘m very interested to hear if there are any positive testresults to anounce.

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