Discussion Forums > Working Towards a Cure

HemaQuest Announces Initiation of Phase I Clinical Trial of HQK-1001

<< < (2/5) > >>

Andy Battaglia:
I believe the drug is similar to butyrate but does not suppress bone marrow production of RBC's. These are natural fatty acids that do not have the side effects of hydroxyurea.

Ever since I have been involved with thal, my intuition has told me that treatment is approaching thal from the wrong direction. Blood from someone else will always be rejected by the body. My feeling has always been that finding a way to get the body to produce its own blood was the direction that made the most sense. We may finally be on the verge of this and I plead with every single patient to comply with treatment fully, because things are happening so fast that treatment will undergo radical changes in the next 20 years. Have faith because this is for real. These doctors who have been working on hemoglobin induction and gene therapy are closer than ever to real success. The great thing about hemoglobin induction is that there is no iron overload caused when your own blood is produced. Fetal Hb may not be quite as efficient as adult Hb but it does the work needed and is an adequate substitute.

Sharmin:
Thank you Andy,
Wow, you are a wealth of information - with great intuition as well. 
I hope for the sake of everyone who can be potentially affected - that this oral drug will replace the current transfusion/chelation treatment.  What a lifestyle change that would be! 

Andy, if the trials are a success do you think that this treatment will be available to patients soon?  (within the next couple of years?) 

Thanks again,
Sharmin

Manal:
Hi

Last year i emailed Dr. Suzane Perrine to ask if i can enroll Ahmad in the butyrate studies as one of the patients here that i know. She answered me saying that the study was closed and there will be a new one in summer 2007 with an oral medicne. I guess that this is the one we are talking about. She asked me to send Ahmad's Blood in a tube  to Boston to try the medicne on his blood in the lab to see the effect. Actually, i searched every possible way to send the sample but couldn't because no shipping company would carry a blood sample without any kind of agreement or consent from the receiving country especially the States. I tried to email her to solve this, but she seems too busy and didn't answer. I will try again and hope she will answer my email

Manal

Andy Battaglia:
It's hard to say how long between the beginning of trials and eventual approval of a drug, but the average time is around 8 years. Prior to that, many patients normally get the chance to participate in phase 3 trials. If a drug is put on the fast track after early trials it can speed up the process, as it did with exjade. The potential  importance of this drug may allow for fast track status if early trials are successful and side effects, if any, are manageable.

Andy Battaglia:
I've had high hopes for the new fetal hemoglobin drug that Dr Susan Perrine has spent over 20 years developing and with the recently announced completion of stage 1 trials, HQK-1001 has performed as expected. In terms of therapies that have the possibility of helping the largest number of people, fetal hemoglobin inducers, led by HQK-1001 hold the most promise. To me, this is the best approach. If your own body makes your blood, you avoid the myriad of problems transfusions can lead to, basically making thalassemics normal. Such a simple idea but so long in coming.

http://www.medicalnewstoday.com/articles/132542.php


--- Quote ---  Medical News Today
HemaQuest Pharmaceuticals Announces Clinical Progress On New Sickle Cell Anemia Drug
10 Dec 2008   

HemaQuest Pharmaceuticals announced today the successful completion of Phase 1 clinical trials of HQK-1001, an orally-administered therapeutic which the company is developing to treat hemoglobin disorders, including sickle cell anemia and beta thalassemia. The company presented the results at the annual meeting of the American Society of Hematology in San Francisco.

HemaQuest performed two different trials. In the first placebo-controlled clinical study, 32 healthy volunteers were given single doses of HQK-1001, ranging from 2 to 20 mg/kg. This trial was followed by a second placebo-controlled study, in which 41 healthy volunteers received two weeks of daily doses of HQK-1001 ranging from 5 to 15 mg/kg.

In both studies, there were no clinically significant adverse effects, and the incidence of mild side effects was similar in subjects receiving placebo or HQK-1001, the company said. Pharmacokinetic studies showed that single doses at, or above, 10 mg/kg reached the targeted plasma drug levels. Significantly, subjects treated with HQK-1001 in the multiple dose Phase 1 clinical trial provided preliminary evidence of its therapeutic potential, as demonstrated by statistically significant increases in young red blood cells, known as reticulocytes.

HemaQuest Chief Scientific Officer and Vice President, Clinical Affairs, Susan Perrine, MD, said, "We are pleased to report these results demonstrating biological activity and favorable pharmacology of HQK-1001, and show that the drug was well-tolerated. We are very encouraged that a brief two-week treatment with HQK-1001 generated significant increases in reticulocytes, indicating that it may offer therapeutic benefit in hemoglobin disorders and other anemias."

HemaQuest President and CEO Ronald Berenson, MD, said, "These initial clinical studies provide the foundation for subsequent testing of HQK-1001 in patients with hemoglobin disorders, including sickle cell anemia and thalassemia in early 2009. If results of our Phase 1 clinical trials are confirmed in these upcoming studies, HQK-1001 may also be beneficial for treating other, common types of anemia characterized by reduced production of red blood cells."

Sickle cell anemia and beta thalassemia afflict millions of people, and are the most common genetic diseases. In the United States, there are nearly 80,000 patients suffering from one of these two inherited blood diseases. Few worldwide therapeutic alternatives exist for these serious and life-threatening diseases, which are associated with significant morbidity and reduced survival, creating a strong and pressing need for new treatments.

About Hemaquest Pharmaceuticals

HemaQuest Pharmaceuticals, Inc., established in late 2007, is a biopharmaceutical company focused on developing oral, small molecule therapeutics to treat hematological diseases including hemoglobin disorders. These therapeutics are based on short chain fatty acid derivative (SCFAD) technologies, which were discovered by Susan Perrine, MD, and her colleagues at Boston University. The company's first therapeutic, HQK-1001, has received orphan status drug designation in the U.S. for both sickle cell anemia and beta thalassemia. HemaQuest's investors include De Novo Ventures, based in Palo Alto, Calif.; Forward Ventures, based in San Diego; and Lilly Ventures of Indianapolis, Ind., the venture capital arm of Eli Lilly and Company.

Hemaquest Pharmaceuticals

Article URL: http://www.medicalnewstoday.com/articles/132542.php

--- End quote ---

This is excellent news and combined with the orphan drug status given this drug by the FDA, we can expect to see progress continue at a fast pace. Getting the body to make its own hemoglobin is the goal whether it's bone marrow transplant, gene therapy, or hemoglobin inducing drugs, but the latter holds the most promise for the most people and will result in much better lives for thalassemics and also make treatment far more simple and far less costly over the lifetime of the patients.This was a very short trial but the results were exactly what they hoped for and my prediction is that stage two trials will create the biggest news in the world of thalassemia since the introduction of desferal.

Navigation

[0] Message Index

[#] Next page

[*] Previous page