Thrombopoetin (and Myelodysplastic syndromes)

In <9406211618.AA07574@artist>, fla…@cli.com (Arthur D. Flatau) asks:

>The main proposed use of thrombopoetin is supposed to be in cancer
>patients, presumably ones that are receiving chemotherapy.  However I
>wonder if in fact patients on chemotherapy are deficient in
>thrombopoetin.  No doubt such patients are deficient in bone marrow
>stem cells.  You can not get platelets from a dead stem cell.  Once
>the stem cells recover the platelets come back rather quickly.  I have
>to wonder whether additional thrombopoetin would be useful.  It seems
>to me that most patients on chemotherapy do not require platelet
>transfusions.

Myelodysplastic Syndromes (MDS, aka Myelodysplasia, aka pre-leukemia)
are conditions in which the stem cells do not properly differentiate
into platelets (and/or red cells, white cells). Thrombopoetin holds
much promise of saving the lives of about 10,000 patients from MDS,
which under today’s state-of-the-art has a prognosis of about 2-5 year
lifespan for the most mild forms. MDS is also a secondary condition
of chemotheraphy and other cancers.

Regards,
Craig


Craig Hansen, Chief Architect                   Tel: (408) 734-8100
MicroUnity Systems Engineering, Inc.            Fax: (408) 734-8136
255 Caspian Drive, Sunnyvale, CA 94089-1015     Email: cr…@microunity.com
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6 Responses to “Thrombopoetin (and Myelodysplastic syndromes)”

  1. admin says:

    >>> On Tue, 21 Jun 1994 21:29:59 GMT, cr…@microunity.com (Craig Hansen) said:
    > Myelodysplastic Syndromes (MDS, aka Myelodysplasia, aka
    > pre-leukemia) are conditions in which the stem cells do not
    > properly differentiate into platelets (and/or red cells, white
    > cells). Thrombopoetin holds much promise of saving the lives of
    > about 10,000 patients from MDS, which under today’s
    > state-of-the-art has a prognosis of about 2-5 year lifespan for the
    > most mild forms. MDS is also a secondary condition of chemotheraphy
    > and other cancers.

    I do not know a whole lot about MDS.  I did read the statement in
    cancernet about it.  According to that statement most patients
    actually have too much Erythropoetin (Epo).  Seems likely they also
    have too much thrombopoetin as well.  On the other hand some patients
    have too little Epo and may benefit from the use of Epo.  So some may
    also benefit from Tpo as well.  I wonder how much of an increase in
    average lifespan this represents, though.

    Art

    fla…@cli.com
    Computational Logic, Inc.
    Austin, Texas

  2. admin says:

    fla…@cli.com (Arthur D. Flatau) writes:

    > >>> On Tue, 21 Jun 1994 21:29:59 GMT, cr…@microunity.com (Craig Hansen) sai

    > > Myelodysplastic Syndromes (MDS, aka Myelodysplasia, aka
    > > pre-leukemia) are conditions in which the stem cells do not
    > > properly differentiate into platelets (and/or red cells, white
    > > cells). Thrombopoetin holds much promise of saving the lives of

                                    ^^^^^^^^^^^^^^^^^^^^^^^^^^^^^^^^
    > > about 10,000 patients from MDS, which under today’s
    > > state-of-the-art has a prognosis of about 2-5 year lifespan for the
    > > most mild forms. MDS is also a secondary condition of chemotheraphy
    > > and other cancers.

       While there is no conclusive evidence that growth factors (particularly
    myeloid growth factors like G-CSF) accelerate the leukemic transformation of
    MDS, there is no evidence that the differentiating effect retards this
    tendency to transform to leukemia. I believe the same will hold true with
    thrombopoetin. It is premature to be too optimistic at this stage to say that
    the drug holds promise of "saving lives" in patients with MDS.

    —————————————————————————-
    Dr. Alan Teh                                           fax:    603 7562253
    Kuala Lumpur               M A L A Y S I A           phone:    603 7502867
    —————————————————————————-

  3. admin says:

    In article <9406211618.AA07574@artist>,
    Arthur D. Flatau <fla…@cli.com> wrote:

    >First a technical one, does anyone know where in the body
    >thrombopoetin is produced?

    Liver, spleen, muscles, and kidneys.  They don’t know why.  This is from a
    long article on thrombopoetin in today’s New York Times.

    Ken Shirriff                    shirr…@cs.Berkeley.EDU

  4. admin says:

    We don’t yet know if patients receiving chemotherapy are relatively
    deficient in thrombopoietin (Tpo).  This is one of the things that
    will have to be measured.  Recall that we didn’t know that patients
    getting chemotherapy were deficient (sometimes) in erythropoietin (Epo),
    and now, Ortho-Biotech has FDA approval for use of Epo in cancer/chemotherapy
    related anemia.
    A second point to be raised: oncologic advances in the area of high-dose
    chemotherapy or dose-intensification have been hampered by the fact that
    we had no way of boosting platelet counts as we can with neutrophils.
    Platelet transfusions work up to a point, but patients can become refractory
    and there is risk of transfusion-borne infection.

    As I understand it, Tpo is a megakaryocyte maturation factor, and not
    a megakaryocyte colony-stimulating factor, as is GM-CSF and IL-3.  Perhaps
    using the two cytokines in combination will help to boost the number of
    stem cells with Tpo-receptors, such that the relative lack of megakaryocytes
    can be overcome.
    –Gary


    Gary Takahashi            |  
    Hematology Clinic         |  
    9155 SW Barnes Road #530  |  
    Portland, OR  97225       |  

  5. admin says:

    Subsequent to posting my article I read the NY Times article (June 21,
    page B8 at least in the National Edition).  As Ken Shirriff
    (shirr…@cs.Berkeley.EDU) points out this answers the question about
    where Tpo is produced, namely the liver, spleen, muscles, and kidneys.

    >>> On 22 Jun 1994 06:32:21 GMT, takah…@ohsu.edu (Gary W. Takahashi) said:
    > We don’t yet know if patients receiving chemotherapy are relatively
    > deficient in thrombopoietin (Tpo).  This is one of the things that
    > will have to be measured.  Recall that we didn’t know that patients
    > getting chemotherapy were deficient (sometimes) in erythropoietin
    > (Epo), and now, Ortho-Biotech has FDA approval for use of Epo in
    > cancer/chemotherapy related anemia.

    It seems to me that it was quite reasonable to suspect that patients
    getting chemo were sometimes deficient in Epo.  Many chemotherapy
    agents are toxic to the kidneys (where Epo is produced).  Most
    patients getting chemotherapy are getting tons of fluids IV and the
    kidneys are working overtime to try and flush these fluids out.  It
    seems reasonable (to me) that under such stress they are less able to
    produce Epo.  Apparently Tpo is produced in many places in the body
    and seems to me less likely than Epo to be deficient.

    According to the NY Times article, Tpo was discovered using pigs.  The
    pigs were irradiated, presumably to kill (some of?) the bone marrow.
    This would subsequently produce a shortage of platelets and increase
    the amount of Tpo produced.  This was then filtered from the pig’s
    blood (in a process that I do not completely understand).  This is not
    exactly like patients undergoing chemo, but is pretty similar.  So it
    seems that most patients on chemo would have more than enough Tpo.

    >  A second point to be raised:
    > oncologic advances in the area of high-dose chemotherapy or
    > dose-intensification have been hampered by the fact that we had no
    > way of boosting platelet counts as we can with neutrophils.
    > Platelet transfusions work up to a point, but patients can become
    > refractory and there is risk of transfusion-borne infection.

    I understand this.  As I pointed out in my original article it may be
    of use to post bone marrow transplant patients some of whom have a
    somewhat chronic need of platelets.

    My principal questions about Tpo were what was the market (that is
    should someone buy Genetech stock, based on this announcement :-) ?
    According to the newspapers this is a $1 billion/ year drug.  Even at
    $200/dose (my guess at the price based on the price of Epo, G-CSF and
    GM-CSF) this is 5,000,000 doses.  I do not see that many people
    needing Tpo.

    My second (much more important) question is, is this going to be a
    ‘miracle’ drug for cancer patients.  I think it will be useful for a
    few patients, but most will not need it.

    Art

    fla…@cli.com
    Computational Logic, Inc.
    Austin, Texas

  6. admin says:

    Well, your view of Epo and chemotherapy is a touch simplistic, but not
    too far off.  First of all, in practice, only a handful of chemo agents
    are significantly toxic to the kidneys at the usual doses to factor it
    into the equation.  And most chemo patients are NOT given tons of IV fluids.
    In clinical trials, Epo has only been shown to lower transfusion requirements
    in patients receiving platinum-containing regimens, but not significantly
    in other regimens.  Epo is produced by the juxtaglomerular cells of the kidney
    and are not involved in the excretion of toxins, which are more the job of
    the renal glomeruli and tubules.  But in truth, the liver is probably the
    most important organ in detoxifying chemo drugs.

    And to your other point, the only way to know how Tpo will impact clinical
    practice is to conduct clinical trials.  Just because Tpo is produced in
    many organs doesn’t really tell you that much.  Leukine (GM-CSF) is produced
    by endothelial cells of the vasculature, which are everywhere. Did that
    prevent Leukine from being used clinically?  Nope. (Of course, GM-CSF is
    produced by other cells, too.) What may be most important is how growth
    factors interact in the bone marrow microenvironment, a model which
    still defies easy analysis. Pharmacologic doses of a cytokine may make up
    for deficiencies in the local milieu of the marrow. This seems to be the
    case for Tpo, where preclinical murine experiments were very impressive.
    In fact, so much, that I myself worry about lineage steal, something we’ve
    not yet seen with the existing clinical cytokines.

    Finally, not having read the NY Times article, is Genentech involved?
    I thought it was Zymogenetics!  E-mail me back if it’s really Genentech.
    –Gary


    Gary Takahashi            |  
    Hematology Clinic         |  
    9155 SW Barnes Road #530  |  
    Portland, OR  97225       |  

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